Novartis AG
Investor Relations
Q4 and FY 2019 Results
Investor Presentation
January 29, 2020
Disclaimer
This presentation contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, that can generally be identified by words such as "potential," "expected," "will," "planned," "pipeline," "outlook," "may," "could," "would," "anticipate," "seek," or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, potential product launches, or regarding potential future revenues from any such products; or regarding the development or adoption of potentially transformational technologies, treatment modalities and business models; or regarding potential future or pending transactions, including the potential outcome, or financial or other impact on Novartis, of the proposed divestiture of certain portions of our Sandoz Division business in the US; or regarding the potential impact of share buybacks; or regarding potential future sales or earnings of the Group or any of its divisions or potential shareholder returns; or by discussions of strategy, plans, expectations or intentions. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. You should not place undue reliance on these statements. In particular, our expectations could be affected by, among other things: global trends toward healthcare cost containment, including ongoing government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; uncertainties regarding potential significant breaches of data security or data privacy, or disruptions of our information technology systems; regulatory actions or delays or government regulation generally, including potential regulatory actions or delays with respect to the proposed transactions or the development of the products described in this presentation; the potential that the proposed divestiture of certain portions of our Sandoz Division business in the US or the planned acquisition of the Japanese operations and associated assets of Aspen Global Incorporated, may not be completed in the expected time frame, or at all; the potential that the strategic benefits, synergies or opportunities expected from the acquisition of The Medicines Company, the proposed divestiture of certain portions of our Sandoz Division business in the US, or the planned acquisition of the Japanese operations and associated assets of Aspen Global Incorporated, and other transactions described, may not be realized or may be more difficult or take longer to realize than expected; the successful integration of The Medicines Company into the Novartis Group and the timing of such integration; potential adverse reactions to the transaction by customers, suppliers or strategic partners; dependence on key personnel of The Medicines Company; dependence on third parties to fulfill manufacturing and supply obligations; the uncertainties involved in predicting shareholder returns; the uncertainties in the research and development of new healthcare products, including clinical trial results and additional analysis of existing clinical data; our ability to obtain or maintain proprietary intellectual property protection, including the ultimate extent of the impact on Novartis of the loss of patent protection and exclusivity on key products that commenced in prior years and is expected to continue this year; safety, quality, data integrity, or manufacturing issues; uncertainties involved in the development or adoption of potentially transformational technologies and business models; uncertainties regarding actual or potential legal proceedings, including, among others, product liability litigation, disputes and litigation with business partners or business collaborators, government investigations generally, litigation and investigations regarding sales and marketing practices, and intellectual property disputes; our performance on environmental, social and governance measures; general political, economic and trade conditions, including uncertainties regarding the effects of ongoing instability in various parts of the world; uncertainties regarding future global exchange rates; uncertainties regarding future demand for our products; and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this presentation as of this date and does not undertake any obligation to update any forward- looking statements as a result of new information, future events or otherwise.
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Participants
Vas Narasimhan | John Tsai |
Chief Executive Officer | Head of Global Drug Development and CMO |
Harry Kirsch | Richard Saynor |
Chief Financial Officer | CEO, Sandoz |
Marie-France Tschudin | Shannon Thyme Klinger |
President, Novartis Pharmaceuticals | Group General Counsel |
Susanne Schaffert | |
President, Novartis Oncology |
3 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
4 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
In 2019, we kept executing on our strategy
Focus Novartis as a leading medicines company powered by advanced therapy platforms and data science
Our focus | Our priorities |
Focus our company | Strengthen our core | Deliver transformative | Embrace operational | Go big on data and |
and capital | innovation | excellence every day | digital |
Accelerate | Unleash the power of | Build trust with society |
key geographies | our people |
5 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
We have focused Novartis as a medicines company
Medicinal chemistry | Diversified | Portfolio | Focused medicines company powered by |
and industrials | healthcare group | transformation | advanced therapy platforms and data science |
1920 - 1996 | 1996 - 2009 | 2009 - 2017 | 2018 - 2019 |
Acquired | Divested OTC1 | Acquired | Acquired | Spun off | Acquired | Acquired | |||
USD 3.9bn | USD 13bn | USD 8.7bn | USD 2.1bn | USD 28bn2 | USD 3.4bn3 | USD 9.7bn | |
1. OTC - Consumer Healthcare | 2. Alcon market capitalization on close of 1stday of trading | 3. USD 3.4bn upfront + potential milestone payments of up to USD 1.9bn |
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Transformative innovation
Leading pipeline with strong replacement power
Scale | Value | Innovation |
# of projects1
114PHASE 1 / 2
37PHASE 3
13REGISTRATION
Estimated 2024 sales from
products launched between 2019-242
#1Replacement power
Company A
Company B
Company C
Company D
Company E
Company F
Company G
Company H
Company I
16 | Advanced platform | |
therapies | ||
in clinical development | ||
Pipeline potentially | |
first-in-class / | |
~90%first-in-indication | |
~80% | Target areas of high |
unmet need | |
1. Including Global Health, excluding Sandoz. 2. Innovative medicine product sales excl. Vaccines and LCM products (e.g. new formulations, combinations with off patent molecules); compound-based analysis (Phase 2 and 3) with additional indications allocated to 1stlaunch. Inclisiran included. Source: Novartis peer group analysis based on data from Evaluate Pharma (download from November 27, 2019)
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Transformative innovation
2019 was a breakthrough year for innovation
5NME approvals of potential blockbusters | 30+major submissions | 30+clinical data readouts | ||||
aSPMS | Select examples | Select examples | ||||
| Entresto®(JP) | | Zolgensma® | |||
SMA | | Cosentyx®nr-AxSpA(US/EU) | | Cosentyx® | ||
| Ofatumumab (US) | | Ofatumumab | |||
Breast cancer | | Adakveo®(US/EU) | | Entresto® | ||
| Beovu®(US/EU/JP) | | Fevipiprant | |||
Wet AMD | | INC280 (US) | | Kisqali® | ||
| QVM149 / QMF149 (EU/JP) | | INC280 | |||
Sickle cell disease | | Inclisiran (US)1 | | Inclisiran1 | ||
aSPMS - Active secondary progressive multiple sclerosis SMA - Spinal muscular atrophy | AMD - age-related macular degeneration 1. Readout / submission by The Medicines Company |
8 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Transformative innovation
2020 catalysts maintaining long-term momentum
Potential catalysts
Major approvals1
Major submissions2
Major readouts3(Phase 3)
Phase 3 starts
Select examples
Ofatumumab (OMB157) | Capmatinib (INC280) | Cosentyx® |
Relapsing MS | NSCLC | nr-axSpA |
QVM / QMF 149 | Entresto® | Inclisiran (KJX839) |
Asthma | HFpEF (US) | Hyperlipidemia (US) |
Inclisiran (KJX839) | AVXS-101 IT4 | Alpelisib (BYL719) |
Hyperlipidemia (EU) | SMA | PROS |
177Lu-PSMA-617 | Spartalizumab (PDR001) combo | Entresto® |
mCRPC | Metastatic melanoma | HFpEF (US) |
177Lu-PSMA-617 | Beovu® | Entresto® |
mCRPC | DME | Post-acute MI (IA5) |
Asciminib (ABL001) | Kisqali® | Jakavi® |
Chronic Myeloid Leukemia | Breast cancer(MONALEESA-2 OS) | Chronic GvHD |
TQJ230 | LNP023 | MBG453 |
CVRR | PNH | MDS |
Tropifexor (LJN452) | Alpelisib (BYL719) | Beovu® |
NASH | Multiple indications6 | PDR |
1. First approval in any market. 2. First submission in any market 3. Readouts enabling submission, label change or pivotal trial initiation 4. FDA placed a partial clinical hold based on findings in a small preclinical animal study 5. Planned interim analysis expected Q1 2020 (full readout 2021) 6. HER2+ aBC, TNBC, ovarian cancer, head and neck cancer, PROS
9 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Transformative innovation
Innovation driving growth in China
Number of NDA approvals1
50+
x2
25
2015-2019 | 2020-2024 |
Actual | Expected |
- Growth driven by regulatory approvals from innovative pipeline, market access and optimizing resources
- 13 NME approvals over the past 5 years, and 22 NRDL listings since 2017
- Average # of NDAs expected to double in the next 5 years
- 5 average NDA approvals / year2015-2019
- 10 average NDA approvals expected / year2020-2024
- 50+ NDA approvals planned over the next 5 years
- Goal to deliver >90% of 2024+ China submissions simultaneously with global submission
1. NDA approvals of new compounds and new indications
10 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Operational excellence
Delivered strong performance in 2019
Continuing operations1, FY 2019, TSR as of YE 2019
47.4 bn | +9% |
NET SALES (USD) | vs. 2018(cc2) |
14.1 bn | +17% |
CORE OPERATINGINCOME2(USD) | vs. 2018(cc2) |
12.9 bn | +15% |
FREE CASH FLOW2(USD) | vs. 2018(USD) |
33.5% | +1.8% pts |
IM CORE MARGIN2(%) | vs. 2018(cc2) |
5.28 | +17% |
CORE EPS2(USD) | vs. 2018(cc2) |
22.3% | Top tier |
1-YEAR TSR3(%) | RANKING 3 |
1. Continuing operations as defined on page 45 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well as the continuing corporate functions. 2. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the Condensed Financial Report. 3. TSR in USD from Jan 1st2019, using 1 day average price at start and 3 month average price at end; ranking when compared to the global HC peer group as defined in the Novartis 2019 Annual Report
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Operational excellence
Strong operational performance from growth drivers
Selected growth driver sales
Sales | Growth vs. PY | Growth vs. PY | ||
USD Million | USD Million | cc | ||
3,551 | 714 | 28% | ||
1,726 | 698 | 71% | ||
361 | 361 | nm | ||
Lutathera® | 441 | 274 | 160% | |
480 | 245 | 111% | ||
1,416 | 242 | 23% | ||
278 | 202 | nm | ||
192 | 192 | nm | ||
1,338 | 183 | 20% | ||
1,114 | 137 | 20% | ||
1,173 | 134 | 19% | ||
671 | 117 | 25% | ||
116 | 116 | nm | ||
nm - not meaningful |
Growth drivers and recent launches as % of Innovative Medicines sales
35% | Adakveo® | ||||||||
Luxturna® | |||||||||
Mayzent® | |||||||||
Beovu® | |||||||||
27% | Aimovig® | ||||||||
Piqray® | |||||||||
Xiidra® | |||||||||
20% | Kymriah® | ||||||||
Zolgensma® | |||||||||
Lutathera® | |||||||||
14% | Kisqali® | ||||||||
Jakavi® | |||||||||
Ilaris® | |||||||||
Xolair® | |||||||||
Tafinlar®+Mekinist® | |||||||||
Promacta® | |||||||||
Entresto® | |||||||||
2016 | 2017 | 2018 | 2019 | Cosentyx® | |||||
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Operational excellence
Focused on launch excellence for 15 ongoing and upcoming major launches
Inclisiran (KJX839) | ||||
Hyperlipidemia | ||||
Mayzent® | Entresto® | |||
aSPMS | HFpEF | |||
Zolgensma®IV | Ofatumumab (OMB157) | AVXS-101 IT | ||
SMA | Relapsing MS | SMA | ||
Piqray® | Capmatinib (INC280) | Alpelisib (BYL719) | ||
Breast Cancer | NSCLC | PROS | ||
Beovu® | Cosentyx® | 177Lu-PSMA-617 | ||
Wet AMD | nr-axSpA | mCRPC | ||
Adakveo® | QVM / QMF 149 | PDR001 combo | ||
Sickle cell disease | Asthma | Metastatic melanoma | ||
2019 | 2020 | 2021 |
Ongoing Upcoming
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Operational excellence
Zolgensma®strong launch (June) with FY 2019 sales of USD 361m
Broad access, strong patient demand
Q3 end (~) | YE (~) | |
Patients treated commercially | 100 | 200 |
Commercial lives covered | 90% | 97% |
Medicaid lives covered | 30% | >50% |
Newborns screened1 | 30% | 39% |
Approval rate for on-label patients | >99% |
Global Managed Access Program initiated
Next steps
Intrathecal formulation on partial clinical | ||
hold - regulatory discussions ongoing | ||
CHMP opinion anticipated Q1 2020 | ||
Approval anticipated in H1 2020 | ||
Decisions anticipated late 2020 or early | ||
Others | ||
2021 in Switzerland, Canada, Australia | ||
1. Expected to reach 70% by end of 2020
14 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Operational excellence
Committed to driving consistent margin expansion
Innovative Medicines
Core margin (% rounded)
Mid to high
Mid 30s
34 30s
31 32
2017 | 2018 | 2019 near term medium |
term |
- Sales momentum of key growth drivers and operational excellence on upcoming launches
- Productivity programs in Novartis Technical Operations and Novartis Business Services
- Resource allocation in commercial units
- Generic erosion
- Launch investments for potential future blockbusters, including inclisiran
15 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Operational excellence
Advancing deep transformations in NTO and NBS
Strategic levers
Organization
Footprint
Procurement
Data & Digital
NTO | NBS |
Reduced ~1,800FTEs across global functions and | On track to reduce ~600FTEs by 2022 |
site operations (net of site exits) | Associates in NGSCs from <40% to >50% |
10sites exited | Footprint reduction through sale, lease-back, and |
9additional exits announced | Activity-Based Working (20+offices) |
102out of 210 warehouses consolidated | Moved to singleservice provider for REFS |
28%reduction in suppliers for finished product, API | NewChief Procurement Officer |
45%reduction in suppliers for indirect materials | Optimizing terms with top 100suppliers |
Delivering advanced analytics solutions on asset, | Continued investments in tech enablers |
material andinventory management | Re-designofkey enterprise processes |
On track for ~USD 2bn savings by end of 2020; new efforts expected to deliver additional ~USD 1.5bn savings medium term
NTO - Novartis Technical Operations; Baseline EOY 2016 NBS - Novartis Business Services; Baseline EOY 2018 NGSCs - Novartis Global Service Centers API - Active pharmaceutical ingredients REFS - Real estate and facility services
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Operational excellence
Sandoz delivered accretive growth in 2019 by implementing refined strategy
Sales growth (vs. PY, cc)
Refined strategy
+2%GLOBAL
+7%EX-US
+16%BIOPHARMACEUTICALS
Core operating income growth (vs. PY, cc)
+10%
Geographic priorities
Increasing autonomy
Portfolio update
- EU: Solidifying #1 position
- JP: Closing Aspen acquisition and investing
-
US: Stabilizing the business
In the process of concluding oral solids business divestment
Launching pegfilgrastim - Creating Sandoz TechOps organization
- Building biosimilar pipeline further - trastuzumab / natalizumab deals
- Appealing US Erelzi®decision
- Gx Advair®discontinued further development
17 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Go big on data and digital
Four core elements to our digital transformation
Scale 12 digital | Make Novartis digital | Become the #1 partner | Pursue bolder moves | |||
lighthouses | in tech ecosystem | |||||
Spanning the entire value chain, from development to commercial operations
In full flight with 2-3yearimplementation horizon
Investing in technology platforms, including CRM,
MDM, API
>1,500 associates mobilized | Scale novel partnership |
Rapidly build Data Science | accelerator: the Novartis |
and AIcapabilities | Biome |
Move to One Digitalglobal | Complementinternal skills |
and capabilities | |
collaboration platform | |
Dedicated leadership | Closely linked to business |
priorities | |
capability program | |
Getting ready for disruptive healthcare scenarios through large-scale alliances, e.g.:
- Microsoft: AI Innovation Lab
- AWS1: TechOps optimization
- Tencent: Heart Failure patient solution in China
1. AWS: Amazon Web Services
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Unleash the power of our people
Broad set of initiatives to drive culture change
Inspired | Curious | Unbossed | ||
Connect to our purpose | Go big on learning | Build leadership | ||
and provide an inspiring | self-awareness and | |||
working environment | capabilities | |||
| Spark live to 83,000 associates, | | Coursera: ~3,500 courses completed | 18 countries visited by CEO in 2019 | |
230,000 recognitions given in 2019 | by 7,000+ users (~85,000 hours)1 | | Unbossed Leadership Experience | ||
| Minimum 14 weeks paid leave for all | | LinkedIn Learning: ~14,000 courses | (ULE) to be completed in 2020 for the | |
parents, regardless of gender | available, 12,500+ users1 | top 300 leaders in the company |
1. As of YE 2019
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Build trust with society
In 2019, we made important progress on our efforts to build lasting trust with society
Ethical Standards | Pricing & Access | Global Health | Corporate Citizenship |
Rolled out globally the new Third | BroughtLIC & LMIC prices in | Signedpartnership for SCD | Joined theUN Equal Pay |
Party Risk Management system | line with EU5 average | with the Government of Ghana | International Coalition |
Drafting the new Code of Ethics, | Outlined new access strategy | JoinedGlobal Chagas Disease | Achieved44% female |
representation in management | |||
co-created with our associates | forSub-Saharan Africa | Coalition | |
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Build trust with society
Introducing ambitious 2020 ESG targets which are deeply embedded in our operating model
Holistic set of ESG targets for 2020...
Pillar | Target |
Ethical | Transparency on clinical trials |
Standards | Strengthen Third Party Risk Management |
Fully integrate Human Rights into TPRM | |
Pricing | Increase patient reach |
& Access | Enhance access |
Implement pricing principles | |
Global | Malaria: Advance development of new drugs |
Health | Sickle cell disease: Expand coverage |
Chagas: Progress on clinical trial | |
Corporate | Reduce energy & carbon |
Citizenship | Reduce waste |
Reduce water |
... deeply embedded in our operating model
Systematically reviewed
Tracked bi-monthly at the Trust & Reputation Committee, a sub-committee of the Executive Committee of Novartis (ECN) chaired by the CEO
Linked to compensation
Cascaded into ECN personal objectives, and directly impacting compensation
Transparently disclosed
To be included in 2020 Annual Report, providing disclosure on our goals and progress
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Build trust with society
Bold long-term aspirations across the ESG spectrum
Select examples
Reducelaunch time lag | Achieve carbon | ||||
neutrality in own | |||||
to 3 monthsin LMICs | |||||
operations by 2025 | |||||
Implement access | Deliver on UN EPIC | ||||
strategy foradvanced | andLGBTI equity | ||||
therapies in LMICs | pledges | ||||
Transform treatment of | Holistically address | ||||
malaria withUSD 100m | sickle cell disease in | ||||
committed in R&D1 | Ghana | ||||
www.novartis.com/nisreport2019 | |||||
1. Commonwealth Heads of Government Meeting April 2018, commitment over the next 5 years | LMICs - low and middle income countries |
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In-market growth drivers, major launches and a rich pipeline expected to sustain our long-term growth
- 15ongoing / upcoming major launches
- 80+major submissions planned to 2022
- 50+late stage programs1
Major launches
In-market growth drivers
Novel assets
Inclisiran | MBG453 |
TQJ230 | Asciminib |
LNP023 | Canakinumab |
Iscalimab | Capmatinib |
Ligelizumab | Spartalizumab |
AD portfolio2 | 177Lu-PSMA-617 |
LNA043 | |
Tropifexor | |
Ofatumumab | |
UNR844 | |
QVM / QMF149 |
New indications
Cosentyx®HS | Alpelisib PROS |
Cosentyx®GCA | Piqray®TNBC |
Cosentyx®LP | Piqray®HER2+ aBC |
Cosentyx®JIA | Piqray®ovarian cancer |
Cosentyx®LN | Piqray®HNSCC |
Entresto®post-AMI | Kisqali®HR+/HER2- BC (adj) |
Beovu®DME | Kymriah®FL |
Beovu®RVO | |
Beovu®DR | |
Beovu®PDR | |
Ofatumumab pediatric | |
AVXS-101 IT |
S E L E C T E X A M P L ES
1. Ph3 / in registration 2. AD portfolio - atopic dermatitis portfolio incl. ZPL389, CEE321
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Strong Pharmaceuticals growth driven by Cosentyx®, Entresto®and recent launches
Pharmaceuticals net sales
USD billion, growth in % cc
+7% | +12% | |||||||
23.3 | ||||||||
0.7 | ||||||||
21.5 | ||||||||
20.0 | ||||||||
3.9 | 5.5 | 7.1 | ||||||
16.1 | 16.0 | 15.5 |
201720182019
Key growth drivers1Recent launches2Established products3
Strong sales momentum driven by growth drivers
- Mainly Cosentyx®and Entresto®
- Maintaining solid performance of established products
Focus on launches to deliver next phase of growth
- Launched Zolgensma®, Beovu®and Mayzent®in US
- Xiidra®acquired and integrated into Ophtha franchise
- Added inclisiran to CRM pipeline
CRM - Cardiovascular, Renal and Metabolism 1. Cosentyx®, Entresto®, Xolair®, Ilaris®2. Zolgensma®, Xiidra ®, Aimovig®, Luxturna®, Mayzent®and Beovu®3. All other brands
25 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®Q4 sales driven by strong demand and broad access across indications and regions
Cosentyx®sales evolution | Strong performance, above market | |||||||||
USD million | ||||||||||
Q4 sales up +21% cc | ||||||||||
Ex-US | 3.6bn | |||||||||
| PsO TRx outperform market (+27% YoY vs. +12%)1 | |||||||||
US | ||||||||||
2.8bn | 937 | 965 | SpA TRx growing >2x faster than market2 | |||||||
858 | ||||||||||
806 | 791 | Maintain momentum | ||||||||
701 | 750 | |||||||||
| nr-axSpA submitted to FDA in Dec | |||||||||
580 | ||||||||||
| Pediatric PsO submitted to EMA in Nov | |||||||||
| Expected to maintain broad access in 2020 | |||||||||
Upgrading expected peak sales >USD 5bn | ||||||||||
Q1 | Q2 | Q3 | Q4 | Q1 | Q2 | Q3 | Q4 | |||
2018 | 2019 | |||||||||
1. IQVIA US National Prescription Audit for Dermatology WE 12/20/2019; market includes Enbrel®, Humira®, Siliq®, Skyrizi®, Stelara®, Taltz®, Tremfya®2. IQVIA US National Prescription Audit for Rheumatology WE 12/20/2019; SpA market includes Cimzia®, Enbrel®, Humira®, Simponi®, Stelara®, Taltz®All trademarks are the property of their respective owners
26 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Entresto®solidifying leadership position in 2019 as an essential first-choice treatment in heart failure
Entresto®sales evolution
USD million, cc
Ex-US | 1.7bn | |||||
US | ||||||
1.0bn | 518 | |||||
421 | 430 | |||||
318 | 357 | |||||
271 | ||||||
239 | ||||||
200 | ||||||
Q1 | Q2 | Q3 | Q4 | Q1 | Q2 | Q3 | Q4 |
20182019
Strong momentum
- Q4 sales +65% driven by increased demand in hospital and ambulatory settings
- US TRx growth (+48% vs. PY)
Poised for further growth acceleration
- New data on reverse cardiac remodeling,in-hospital use and QoL
- Inclusion on China NRDL, supporting expanded use
- US regulatory submission for HFpEF on track for Q1
- Japan launch expected in H2
AHA - American Heart Association; QoL - Quality of Life; NRDL - National Reimbursement Drug List; HFpEF - Heart Failure with preserved Ejection Fraction
27 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Strong start to Beovu®US launch with excellent customer feedback and broad access
Strong uptake in 3 months since launch1 | Broad access and reimbursement | ||
84% | of US retina specialists have | Jan 1 | permanent J-Code received, |
Beovu®available in their office | providing greater reimbursement | ||
confidence | |||
90% | of retina specialists who don't | 95% | positive benefits verification |
currently use Beovu®plan to | outcomes to date2 |
use it in next 6 months
EU approval expected Q1 2020, JP approval expected Q2 2020
1. Novartis commissioned Retina Specialist Panel Online Survey, Dec 2019. 2. Covered or covered with restrictions
28 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Ofatumumab (OMB157) is poised to set a new standard for simple, broad and early B-cell therapy adoption
Unsurpassed efficacy and favorable safety supporting broad and early use in RMS
45.9%risk reduction (p<0.001) in 24-week CDW post-hoc analyses with revised definition used in OPERA trials1
[%] | 14 | ||||||||||
rate | |||||||||||
12 | |||||||||||
event | 10 | TER 10.5% | |||||||||
8 | |||||||||||
cumulative | |||||||||||
6 | HR (95% CI): 0.541 | ||||||||||
4 | |||||||||||
OMB 5.6% | (0.381; 0.768) | ||||||||||
of | 2 | ||||||||||
K-M estimate | |||||||||||
0 | |||||||||||
0 | 3 | 6 | 9 | 12 | 15 | 18 | 21 | 24 | 27 |
- Regulatory file submitted in US and EU
- Easy-to-useautoinjector available upon launch, enabling simple at-homeself-administration and improved patient experience
- Strong scientific presence at major congresses and in markets through 2020
- Engaging with payers on rapid, broad early
Study month
access
CDW - Confirmed Disability Worsening Source: ASCLEPIOS I & II data presentation available here 1. Adapted from the OPERA trials, Hauser et al. 2017. Different to the ASCLEPIOS study, a disability "progression" was defined as an increase from the baseline EDSS score of at least 1.0 point (or 0.5 points if the baseline EDSS score was >5.5) that was sustained for at least 12 (24) weeks, used in OPERA trials
29 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Inclisiran (KJX839), preparing to launch potentially transformative cholesterol-lowering therapy
Differentiated asset
Efficacy | Potent, durable, consistent LDL-C |
reduction >50% | |
Safety | Profile similar to placebo (no liver, |
muscle, renal nor platelet signals) | |
in entire clinical program | |
Convenience | Durable efficacy with only 2 |
subcutaneous injections per year, | |
less patient abandonment | |
Adherence | Payers confidence reinforced by |
physician administration dosing | |
regimen | |
Preparing for launch
Organizational | | MDCO now subsidiary of Novartis, expected to |
be fully integrated end March 2020 | ||
Regulatory | | US and EU files submitted1 |
JP bridging program in progress | ||
CN local development aligned with CFDA | ||
Commercial | | Finalizing hiring and training of US teams |
Value-based pricing and flexible access strategy | ||
Engaging with payers and health systems to | ||
enable broad and affordable access, including | ||
population models (e.g. UK NHS) | ||
| Scaling up supply |
1. Regulatory submissions filed by The Medicines Company
30 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Focus in 2020 will be launches and preparing for next big bets, building on the strong foundation
Maximize growth drivers
Cosentyx®: nr-axSpA launch doubles addressable axSpA population
Entresto®: NRDL-driven expansion in CN, JP approval expected
Deliver on new launches
Beovu®: Global rollout and DME / RVO expansion
Ofatumumab: Broad and early access in CoEs and community
Inclisiran: Broad & affordable access
Mayzent®: Urgency to treat and patient services optimization
Xiidra®:Return to growth
Prepare for next big bets
TQJ230: Lp(a) awareness and testing
Iscalimab: Educate on unmet need, leveraging transplant legacy
Ligelizumab: Build US infrastructure
Tropifexor: Drive disease awareness given asymptomatic nature of NASH
LNP023: Educate physicians on complement-driven renal & hematologic diseases
31 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
32 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Strong Oncology growth driven by recent launches and growth drivers
Oncology net sales
USD billion, growth in cc
+9% | +10% | |||||||||
14.4 | ||||||||||
13.4 | ||||||||||
1.3 | ||||||||||
12.3 | ||||||||||
0.5 | ||||||||||
0.1 | 3.3 | 3.9 | ||||||||
2.5 | ||||||||||
9.7 | 9.6 | 9.2 |
201720182019
Key growth drivers1Recent launches2Established products3
Potential future growth
- Strong uptake of recent launches
- Growth drivers deliverdouble-digit performance
- Resource allocation/productivity to fuel strategic investment (i.e. launches, China)
- Generic impact4
- Healthcare cost containment / pricing
1. Including Promacta®/Revolade®, Jakavi®(marketed by Novartis ex-USA), Tafinlar®+ Mekinist® | 2. Including Kisqali®, Kymriah®, Lutathera®, Piqray®, Adakveo®3. All other brands, including those with generic competition in the market |
4. Afinitor®, Exjade®, Glivec®and Sandostatin®LAR in EU |
33 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Accelerated sales and innovation reinforce the long-term growth of our breast cancer portfolio
2019 sales in USD m
43
6
67
~40% of HR+/HER2- breast cancer patients have a PIK3CA mutation, |
associated with poor prognosis |
NCCN guidelines updated, PIK3CA testing rate ~25% FY 2019 |
Foundation Medicine PIK3CA CDx tissue approved Q4 2019, plasma |
anticipated Q2 2020 |
"EPIK" development program for 5 new indications1with potential |
Q2 | Q3 | Q4 |
155
91111123
Q1 | Q2 | Q3 | Q4 |
opportunity to serve an additional ~100k patients |
Only CDK 4/6 inhibitor that consistently demonstrated superior OS in 2 |
pivotal Phase 3 studies in 2019 |
Early signs of OS accelerating US growth momentum in Q4 |
Strong growth across key geographies ex-US in Q4 2019 |
Potential registration for high and intermediate adjuvant BC as early as 2022 |
based pre-planned interim analysis (NATALEE, 3-year treatment duration) |
1. TNBC, HER2+ aBC, ovarian cancer, HNSCC; alpelisib PROS
34 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Adakveo®is off to a solid start in US (approved Nov)
SCD Patients in US
60% are 16yr old +
90% have >1 VOC +
100K
60K
54K
- The only approved therapy for reduction in frequency of VOCs in SCD
- Commercial product available within 2 days of approval
- Payer engagement ongoing:
- Community centers driving initial uptake
- Permanent J CODE anticipated July 2020
- Medicaid coverage criteria approved in Florida, Kentucky, Maryland, Washington, Delaware and Alabama
VOCs - Vaso-occlusive crises SCD - Sickle cell disease
35 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Maximizing momentum for our growth drivers and new launches, while preparing for next big bets
Maximize growth drivers
Kisqali®: Continued strong growth driven by M3 and M7 OS data
Lutathera®: Leverage potential to grow in earlier lines of treatment
Promacta®/Revolade®: Growth in ITP and
uptake in 1L SAA in the US and Japan
Deliver on new launches
Piqray®: Maximize US launch momentum and expand further to EU markets
Adakveo®: Enable access in larger US accounts and continue education and patient activation
Kymriah®: Drive further capacity increase to meet strong demand in pALL and
DLBCL
Prepare for next big bets
Capmatinib:Establish awareness of
MET's role in NSCLC among HCPs /
patient community
Spartalizumab (PDR001) combo: Build
on Taf/Mek leadership position in metastatic melanoma
177Lu-PSMA: Further evolve commercial infrastructure for best in class launch
Canakinumab: Focus on medical education ontumor-promotinginflammation
36 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
37 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
2019 financial results in line with upgraded guidance
Group full year guidance(as revised in October 2019) | FY 2019 vs. PY | |
in cc | in cc | |
"Salesexpected to grow high single digit" | 9% |
"Core operating incomeexpected to grow mid to high teens" | 17% |
38 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Strong sales growth drove double digit increases in core operating income and free cash flow
Continuing operations1 | Q4 | Change vs. PY | |
USD million | 2019 | % USD | % cc2 |
Net Sales | 12,403 | 8 | 9 |
Core Operating income 2 | 3,462 | 11 | 13 |
Operating income | 1,823 | 34 | 37 |
Net Income | 1,129 | -7 | -6 |
Core EPS (USD)2 | 1.32 | 14 | 15 |
EPS (USD) | 0.50 | -6 | -4 |
Free Cash Flow 2 | 3,488 | 20 | |
FY | Change vs. PY | |
2019 | % USD | % cc2 |
47,445 | 6 | 9 |
14,112 | 12 | 17 |
9,086 | 8 | 14 |
7,147 | -44 | -41 |
5.28 | 12 | 17 |
3.12 | -43 | -40 |
12,937 | 15 | |
- Continuing operations excludes Alcon and includes the Sandoz US dermatology and oral solids portfolio business
- Constant currencies (cc), core results and free cash flow arenon-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the Condensed Financial Report
39 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
2019 free cash flow increased to USD 12.9bn driven by higher operating income
Continuing operations1free cash flow2
USD billion
+15% | Key drivers vs. PY: | |||
12.9 | +Higher operating income | |||
11.3 | (adjusted for non-cash items) | |||
Offsetting one-time effects:
+
−
Real estate divestment proceeds
Prior year OTC JV dividend and GSK milestone income
20182019
1. Continuing operations excludes Alcon and includes the Sandoz US dermatology and oral solids portfolio business. 2. Free cash flow is a non-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the Condensed Financial Report
40 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Novartis proposes 23rdconsecutive dividend increase to the AGM: 2.95 CHF / share1
2019 dividend yield 3.2% | |||||||||
2019 dividend growth 3.5%3 | |||||||||
3.0 | CHF | 2.85CHF 2.84USD | 2.95CHF | 3.04USD | |||||
2.5 | USD | ||||||||
2.0 |
1.5
1.0
0.5
0.0
1996 1997 1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019
1. Proposal to shareholders at the 2020 Annual General Meeting, taking place on February 28, 2020 2. Converted at historic exchange rates at the dividend payment dates as per Bloomberg; assumes an exchange rate of USD/CHF of 0.9690 as of December 31, 2019 for 2019 3. In CHF
41 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Core margin expansion of +1.9%pts
Continuing operations
Q4 2019 | FY 2019 | ||||||
Core operating | |||||||
Core margin 2 | Net sales | income 2 | Core margin 2 | ||||
Core margin 2 | change vs. PY | change vs. PY | change vs. PY | Core margin2 | change vs. PY | ||
(%) | (%pts cc)2 | (in % cc) | (in % cc) 2 | (%) | (%pts cc)2 | ||
Innovative Medicines | 31.5 | 0.7 | 11 | 18 | 33.5 | 1.8 | |
Sandoz | 20.8 | 1.5 | 2 | 10 | 21.5 | 1.5 | |
Continuing Operations1 | 27.9 | 0.8 | 9 | 17 | 29.7 | 1.9 | |
1. Continuing operations excludes Alcon and includes the Sandoz US dermatology and oral solids portfolio business. 2. Core results, constant currencies and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the Condensed Financial Report
42 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
2020 Novartis full year guidance
Barring unforeseen events; growth vs. PY in cc
Focused medicines company | full year guidance
Excl. Sandoz US oral solids & dermatology businesses1
Sales expected to grow mid to high single digit
- IM Division expected to growmid to high single digit
- Sandoz expected to growlow single digit
Core operating income expected to grow high single to low double digit
Key assumption: Guidance above includes the forecast assumption that no Gilenya®or Sandostatin®LAR generics enter in 2020 in US
1. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during Q1 2020. 2019 FY sales and core operating of the Sandoz US oral solids and dermatology businesses were approximately USD 1.1bn and 0.3bn, respectively.
43 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
FY 2020 Guidance on other financial KPIs
Barring unforeseen events (in cc)
Focused medicines company | full year guidance
Excl. Sandoz proposed US portfolio sale to Aurobindo1from both 2018 and 2019
Core net | Expenses expected to increase by around 0.2bn vs. 2019 reflecting |
financial result | additional financing costs to acquire The Medicines Company |
Core tax rate | FY core tax rate expected to be broadly in line with 2019 |
1. The announced sale of Sandoz US dermatology and oral solids portfolio to Aurobindo, expected to close during Q1 2020. 2019 FY sales and core operating of the Sandoz US oral solids and dermatology businesses were approximately USD 1.1bn and 0.3bn, respectively.
44 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
45 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Delivered strong performance in 2019
Continuing operations1, FY 2019, TSR as of YE 2019
47.4 bn | +9% |
NET SALES (USD) | vs. 2018(cc2) |
14.1 bn | +17% |
CORE OPERATINGINCOME2(USD) | vs. 2018(cc2) |
12.9 bn | +15% |
FREE CASH FLOW2(USD) | vs. 2018(USD) |
33.5% | +1.8% pts |
IM CORE MARGIN2(%) | vs. 2018(cc2) |
5.28 | +17% |
CORE EPS2(USD) | vs. 2018(cc2) |
22.3% | Top tier |
1-YEAR TSR3(%) | RANKING 3 |
1. Continuing operations as defined on page 45 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well as the continuing corporate functions. 2. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 58 of the Condensed Financial Report. 3. TSR in USD from Jan 1st2019, using 1 day average price at start and 3 month average price at end; ranking when compared to the global HC peer group as defined in the Novartis 2019 Annual Report
46 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
In-market growth drivers, major launches and a rich pipeline expected to sustain our long-term growth
- 15ongoing / upcoming major launches
- 80+major submissions planned to 2022
- 50+late stage programs1
Major launches
In-market growth drivers
Novel assets
Inclisiran | MBG453 |
TQJ230 | Asciminib |
LNP023 | Canakinumab |
Iscalimab | Capmatinib |
Ligelizumab | Spartalizumab |
AD portfolio2 | 177Lu-PSMA-617 |
LNA043 | |
Tropifexor | |
Ofatumumab | |
UNR844 | |
QVM / QMF149 |
New indications
Cosentyx®HS | Alpelisib PROS |
Cosentyx®GCA | Piqray®TNBC |
Cosentyx®LP | Piqray®HER2+ aBC |
Cosentyx®JIA | Piqray®ovarian cancer |
Cosentyx®LN | Piqray®HNSCC |
Entresto®post-AMI | Kisqali®HR+/HER2- BC (adj) |
Beovu®DME | Kymriah®FL |
Beovu®RVO | |
Beovu®DR | |
Beovu®PDR | |
Ofatumumab pediatric | |
AVXS-101 IT |
S E L E C T E X A M P L ES
1. Ph3 / in registration 2. AD portfolio - atopic dermatitis portfolio incl. ZPL389, CEE321
47 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Q&A session
Vas Narasimhan | John Tsai |
Chief Executive Officer | Head of Global Drug Development and CMO |
Harry Kirsch | Richard Saynor |
Chief Financial Officer | CEO, Sandoz |
Marie-France Tschudin | Shannon Thyme Klinger |
President, Novartis Pharmaceuticals | Group General Counsel |
Susanne Schaffert | |
President, Novartis Oncology |
48 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Appendix
Sales performance driven by Innovative Medicines
Key growth drivers Q4
Sales | Growth vs. PY | Growth vs. PY | |
USD Million | USD Million | cc | |
518 | 200 | 65% | |
186 | 186 | nm | |
965 | 159 | 21% | |
155 | 95 | 166% | |
90 | 90 | nm | |
96 | 68 | nm | |
67 | 67 | nm | |
380 | 50 | 16% | |
356 | 43 | 15% | |
293 | 37 | 17% | |
303 | 35 | 16% | |
Lutathera® | 107 | 26 | 31% |
178 | 23 | 16% | |
nm - not meaningful |
50 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Expected currency impact for full year 2020
Currency impact vs. PY
%pts, assuming late-January exchange rates prevail in 2020
FX impact on Net sales
-1 | 0 to -1 | ||||||
-3 | -1 to -2 | ||||||
Q4 | FY | Q1 | FY | ||||
2019 | 2020 | ||||||
FX impact on Core operating income
-2 | -1 to -2 | |||||
-3 | ||||||
-5 | ||||||
Q4 | FY | Q1 | FY | |||
2019 | 2020 |
Actual Simulation
51 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Net debt slightly reduced by USD 0.3bn in 2019 mainly driven by strong FCF
+0.3
-16.2 | 0.2 | -15.9 | ||||||||
2.9 | ||||||||||
-6.6 | ||||||||||
-3.8 | ||||||||||
-5.3 | 12.9 | |||||||||
Dec 31, 2018 | Dividends | M&A transactions Treasury share | Free Cash Flow1Net debt Alcon2 | Others | Dec 31, 2019 |
transactions, net
1. Continuing operations excludes Alcon, includes the businesses of Innovative Medicines and Sandoz (including the US generic oral solids and dermatology portfolio), as well as the continuing corporate functions. 2. Includes the net de- recognition of USD 0.6bn cash and cash equivalents and USD 3.5bn of financial debts related to the Alcon spin-off.
52 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
2019 pipeline milestones
H1 2019 | H2 2019 | ✓Achieved* | ✕Missed* | |||||||
Regulatory | Mayzent®1 | SPMS (US) | ✓ | BYL719 (Piqray®) | HR+ Breast Cancer (US) | ✓1 | ||||
Kymriah® | Ped / Young Adult r/r ALL (JP) | ✓ | Beovu® | nAMD (US) | ✓ | |||||
decisions and | ||||||||||
Kymriah® | r/r DLBCL (JP) | ✓ | Lucentis® | RoP (EU / JP) | ✓ | |||||
opinions | ||||||||||
Promacta® | Severe aplastic anaemia (EU) | ✕ | Lucentis® | Diabetic Retinopathy (EU) | ✓ | |||||
Zolgensma®IV | SMA (US) | ✓ | Mayzent® | SPMS (EU / JP) | ✓2 | |||||
Zolgensma®IV | SMA (EU / JP) | EU Q1 2020 | Xolair® | Pollinosis (JP) | ✓ | |||||
JP H1 2020 | ||||||||||
Major | Brolucizumab (RTH258) | nAMD (US / EU / JP) | ✓ | Cosentyx® | nr-axSpA (US / EU / JP) | ✓ | ||||
Crizanlizumab (SEG101) | Sickle Cell Disease (US / EU) | ✓ | Entresto® | HF-rEF (JP) | ✓ | |||||
expected | ||||||||||
Mayzent®1 | SPMS (JP) | ✓ | Entresto® | HF-pEF (US / EU) | Q1 2020 | |||||
submissions | ||||||||||
Capmatinib (INC280) | NSCLC (US / JP) | ✓ | ||||||||
Ofatumumab (OMB157) | Relapsing MS (US / EU) | ✓3 | ||||||||
PDR001 (combination | Metastatic Melanoma (US / EU) | H2 2020 | ||||||||
with Tafinlar®+Mekinist®) | ||||||||||
QVM / QMF 149 | Asthma (EU / JP) | ✓ | ||||||||
Major | AVXS-101 IT | SMA | ✓ | Cosentyx® | nr-axSpA | ✓ | ||||
Zolgensma®IV | SMA presymptomatic | ✓ | Entresto® | HF-pEF | (✓)4 | |||||
expected trial | ||||||||||
Fevipiprant (QAW039) | Asthma | (✓)5 | ||||||||
readouts | ||||||||||
Ofatumumab (OMB157) | Relapsing MS | ✓ | ||||||||
PDR001 (combination | Metastatic melanoma | H2 2020 | ||||||||
with Tafinlar®+ Mekinist®) | ||||||||||
*Represents achieving on-time readout of the data, irrespective of trial outcome 1. Piqray®FDA approval achieved in H1 2019. | 2. Positive CHMP opinion Nov 2019, EMA approval Jan 2020 | 3. EU submission early January 2020 4. Study |
narrowly missed primary endpoint, but showed benefit in pre-specified large subgroups including women and patients with lower ejection fraction 5. LUSTER 1&2 readouts completed and did not meet the clinically relevant threshold for reduction in rate of moderate-to-severe exacerbation.
53 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
2020 expected pipeline milestones
H1 2020 | H2 2020 | ✓Achieved | ✕Missed |
Regulatory decisions and opinions
Major expected submissions
Major expected trial readouts*
Beovu® | nAMD (EU/JP) | Adakveo® | Sickle cell disease (EU) | ||
Cosentyx® | nr-axSpA (EU/US) | Capmatinib (INC280) | NSCLC (US/JP) | ||
Cosentyx® | AS (CN) | Cosentyx® | Pediatric psoriasis (EU) | ||
Ofatumumab (OMB157) | Relapsing MS (US) | Cosentyx® | nr-axSpA (JP) | ||
Piqray® | HR+/HER2- aBC with PIK3CA | Entresto® | HFpEF (US) | ||
mutation (EU) | |||||
QVM149 | Asthma (EU/JP) | Inclisiran (KJX839) | Hyperlipidemia (US) | ||
Tafinlar®& Mekinist® | Adjuvant melanoma (CN) | Xolair® | Nasal Polyposis (US/EU) | ||
Xiidra® | DED (EU) | ||||
Zolgensma®IV | SMA (JP/EU) | ||||
Entresto® | HFpEF (US) | Alpelisib (BYL719) | PROS (US) | ||
Inclisiran (KJX839) | Hyperlipidemia (EU) | ✓ | AVXS-101 IT | SMA (US) | |
Cosentyx® | Juvenile PsA / enthesitis-related arthritis | ||||
(US/EU) | |||||
Spartalizumab (PDR001) | Metastatic melanoma (US/EU) | ||||
and Tafinlar®& Mekinist® | |||||
177Lu-PSMA-617 | mCRPC (US) | ||||
Entresto® | Post-acute MI1 | Asciminib (ABL001) | CML 3L | ||
Tropifexor (LJN452) | NASH | Beovu® | DME | ||
UNR844 | Presbyopia | Jakavi® | chronic GVHD | ||
Kisqali® | aBC (MONALEESA-2 OS) | ||||
177Lu-PSMA-617 | mCRPC |
*Achieved = on-time readout of data, irrespective of trial outcome. 1. Interim analysis readout
54 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Capitalizing on the rich pipeline, faster and broader access in China
NDA Approval
NRDL Access
FY 2019 sales: | 2019 | 2020 | |||||||||||||
USD 2.2bn | Achieved | Planned | |||||||||||||
Cosentyx® | Tasigna® | Cosentyx® | Pataday® | Zykadia® | |||||||||||
PsO | Pediatric CML | AS | Allergic Conj. | ALK+NSCLC 1stline | |||||||||||
Gilenya® | Tafinlar®+Mekinist® | Mayzent® | Tafinlar®+Mekinist® | ||||||||||||
MS | BRAF+ mM | RMS | Adj. Melanoma | ||||||||||||
Afinitor ® | Exelon®Patch | Lucentis® | Vigamox® | Cosentyx® | Tafinlar®+Mekinist® | Votrient® | |||||||||
TSC-SEGA and | AD | DME/RVO/CNV | Bacterial Conj. | PsO | BRAF+ mM | aRCC | |||||||||
GI/LUNG NET | |||||||||||||||
Entresto® | Exjade® | Revolade® | Xolair® | Gilenya® | Tasigna® | Zykadia® | |||||||||
Chronic HF | IOL | ITP | Asthma | MS | Adult CML | ALK+NSCLC 2ndline | |||||||||
Eucreas® | Jakavi® | Ultibro® | Sandostatin®LAR | Tasigna® | Zykadia® | ||||||||||
T2D | Myelofibrosis | COPD | Acromegaly and | Pediatric CML | ALK+NSCLC 1stline | ||||||||||
GEP NET | |||||||||||||||
55 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Building depth across our core therapeutic areas…
O N C O L O GY
Select | ||
commercial | ||
assets | ||
Spartalizumab combo | ||
Metastatic Melanoma | ||
177Lu-PSMA-617 | ||
mCRPC | ||
Select | Canakinumab (ACZ885) | |
Lung | ||
pipeline | ||
Asciminib (ABL001) | ||
assets | CML | |
MBG453 | ||
MDS, AML | ||
P H A R M A C E UTI C A LS
CRM | IHD | Neuroscience | Ophthalmology | Respiratory | |||||
1 | |||||||||
2 | |||||||||
LNP023 | LNA043 | Ofatumumab (OMB157) | UNR844 | QVM149 | |||||
Renal diseases | Primary Osteoarthritis | MS | Presbyopia | Asthma | |||||
TQJ230 | Iscalimab (CFZ533) | LMI070 | ECF843 | CSJ117 | |||||
CVRR | Transplant / Sjögren's | SMA | Dry Eye | Asthma | |||||
Inclisiran (KJX839) | Ligelizumab (QGE031) | SAF312 | QBW251 | ||||||
Hyperlipidemia | CSU / CIU | Chronic Ocular Pain | COPD | ||||||
Adriforant (ZPL389)
AD
Tropifexor (LJN452)
NASH
LOU064
CSU
CRM - Cardiovascular, Renal & Metabolism IHD - Immunology, Hepatology & Dermatology 1. Aimovig® is developed in collaboration with Amgen 2. Luxturna®marketed ex-US
56 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
...while strengthening our innovative platforms
G E NE THE RA P Y | CE L L THE RA P Y | RA DI O - L IG AND THE RA P Y | |||||||||||||
Select | 1 | ||||||||||||||
commercial | |||||||||||||||
assets | |||||||||||||||
Zolgensma® | CPK850 | CD19 CAR-T | CD19 CAR-T | 177Lu-PSMA-617 | 177Lu-PSMA-R2 | ||||||||||
SMA | Retinis Pigmentosa | DLBCL in 1st relapse | r/r Follicular Lymphoma | mCRPC | Prostate Cancer | ||||||||||
AVXS-101 IT | CD19 CAR-T | CD19 CAR-T | 177Lu-NeoB | ||||||||||||
SMA | r/r DLBCL in combo with pembro | Adult r/r ALL | Various cancers | ||||||||||||
AVXS-201 | CD19 CAR-T | CD19 CAR-T | |||||||||||||
Select | Rett Syndrome | r/r CLL in combo with ibrutinib | Pediatric NHL | ||||||||||||
AVXS-301 | CD19 CAR-T | CD19 CAR-T | |||||||||||||
pipeline | |||||||||||||||
1st line high risk pediatric and | |||||||||||||||
ALS | r/r DLBCL in combo with ibrutinib | ||||||||||||||
assets | young adult ALL | ||||||||||||||
AVXS-401 | CD19 CAR-T | CD19 CAR-T | |||||||||||||
Friedrieich's Ataxia | BCMA&CD19 | CD22&CD19 | |||||||||||||
AVXS-501 | CD19 CAR-T | CD19 CAR-T | |||||||||||||
Undisclosed | CD123 | EGFRv3 | |||||||||||||
AVXS-601 | |||||||||||||||
Undisclosed | |||||||||||||||
1. Luxturna® marketed ex-US
Includes preclinical and launched programs
57 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Our pipeline projects at a glance
Phase 1/2 | Phase 3 | Registration | Total | |
O N C O LO G Y | 57 | 20 | 3 | 80 |
P H A R MA C E U T IC A LS | 57 | 17 | 10 | 84 |
Cardiovascular, Renal, Metabolism | 9 | 5 | 1 | 15 |
Immunology, Hepatology, Dermatology | 22 | 6 | 2 | 30 |
Neuroscience | 9 | 0 | 2 | 11 |
Ophthalmology | 5 | 3 | 1 | 9 |
Respiratory | 7 | 3 | 3 | 13 |
Global Health | 5 | 0 | 1 | 6 |
B IO S IMILA R S | 0 | 1 | 0 | 1 |
Total | 114 | 38 | 13 | 165 |
58 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Novartis submission schedule
New molecular entity: lead and new indications
2020 | 2021 | 2022 | 2023 | ≥2024 | ||||||||||||||||||
TQJ230 | ||||||||||||||||||||||
spartalizumab | Lead | asciminib | Lead | ECF843 | Lead | LOU064 | Lead | 177Lu-NeoB | Lead | LNA043 | Lead | CPK850 | Lead | |||||||||
PDR001 | ABL001 | Dry eye | Chronic spontaneous urticaria | 177Lu-NeoB | Osteoarthritis | RP | CVRR-Lp(a) | |||||||||||||||
m BRAF V600+ melanoma | (+Taf/Mek) | CML 3L | Multiple Solid Tumors | |||||||||||||||||||
ganaplacide | ||||||||||||||||||||||
177Lu-PSMA-617 | Lead | MBG453 | Lead | iscalimab | Lead | VPM087 | Lead | tropifexor | Lead | AVXS-201 | Lead | |||||||||||
INDICATIONS | 177Lu-PSMA-617 | HR-MDS | CFZ533 | 1st line CRC / 1st line RCC | LJN452 | OAV201 | KAF156 | |||||||||||||||
mCRPC | Renal/Liver Tx | NASH | Rett syndrome | Malaria | ||||||||||||||||||
ligelizumab | Lead | LNP023 | Lead | adriforant | Lead | tropifexor&cenicriviroc | Lead | LMI070 | Lead | cipargamin | ||||||||||||
QGE031 | PNH | ZPL389 | LJC242 | SMA | KAE609 | |||||||||||||||||
Chronic urticaria | Atopic dermatitis | NASH | Malaria | |||||||||||||||||||
CSJ117 | Lead | CEE321 | Lead | SAF312 | Lead | MIJ821 | Lead | LXE408 | ||||||||||||||
Severe asthma | Atopic Dermatitis | COSP | Depression | Visceral leishmaniasis | ||||||||||||||||||
LEAD | denosumab | BioS | ianalumab | Lead | UNR844 | Lead | QBW251 | Lead | ||||||||||||||
GP2411 | VAY736 | Presbyopia | COPD | |||||||||||||||||||
anti RANKL mAb | AIH | |||||||||||||||||||||
canakinumab | LCM | canakinumab | LCM | spartalizumab | LCM | capmatinib | LCM | MBG453 | LCM | LOU064 | LCM | LNP023 | |||||||
INDICATIONS | ACZ885 | ACZ885 | PDR001 | INC280 | LCM | Unfit AML | SjS | iMN | |||||||||||
MBG453 | |||||||||||||||||||
NSCLC 2L | Adjuvant NSCLC | Malignant melanoma (combo) | Solid tumors | ||||||||||||||||
canakinumab | LCM | LNP023 | LCM | crizanlizumab | LCM | iscalimab | LCM | tropifexor | LCM | inclisiran | |||||||||
ACZ885 | C3G | SEG101 | CFZ533 | LJN452 | LNP023 | ||||||||||||||
NSCLC 1L | Sickle cell anaemia w ith crisis | SjS | NASH (combos) | Hyperlipidemia | |||||||||||||||
crizanlizumab | LCM | LNP023 | LCM | MBG453 | LCM | ianalumab | LCM | ofatumumab | LCM | cipargamin | |||||||||
SEG101 | IgAN | Fit AML | VAY736 | OMB157 | KAE609 | ||||||||||||||
Sickle cell anaemia new formulations | pSjS | Ped MS | Malaria | ||||||||||||||||
NEW | AML | ||||||||||||||||||
Compared to past reports, we have categorized submission schedules into NMEs (lead & new indications) and supplementary indications for existing brands
59 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Lead
Lead
Lead
Lead
LCM
LCM
LCM
Novartis submission schedule
Supplementary indications for existing brands
2020 | 2021 | ||
alpelisib, BYL719 | LCM | Kymriah | |
PROS | tisagenlecleucel-T, CTL019 | ||
r/r DLBCL 1st relapse | |||
Cosentyx | LCM | Kymriah | |
secukinumab, AIN457 | tisagenlecleucel-T, CTL019 | ||
Psoriasis 300mg AI | r/r Follicular lymphoma | ||
Cosentyx | LCM | Tafinlar | |
secukinumab, AIN457 | dabrafenib, DRB436 | ||
PsA H2H | Neoplasm Pedia | ||
Cosentyx US | LCM | Promacta | |
secukinumab, AIN457 | eltrombopag, | ETB115 | |
Ped Psoriasis | Food effect free formulation | ||
AVXS-101 | LCM | Jakavi | |
onasemno-geneabepar-vovec, OAV101 | ruxolitinib, INC424 | ||
SMA IT | Chronic GVHD | ||
Xolair | LCM | Jakavi | |
omalizumab, IGE025 | ruxolitinib, INC424 | ||
CSU (for CN) | Acute GVHD | ||
Entresto | LCM | Beovu | |
valsartan+sacubitril, LCZ696 | brolucizumab, RTH258 | ||
HFpEF | DME | ||
Xolair | |||
omalizumab, | IGE025 | ||
Food allergy | |||
Xolair | |||
omalizumab, | IGE025 | ||
Auto-injector |
Entresto
valsartan+sacubitril, LCZ696 Post-AMI
Lamprene
clofazimine, LAM320 Tuberculosis
2022
LCMKisqali
ribociclib, LEE011 HR+/HER2- BC (adj)
LCMPromacta
eltrombopag, ETB115 Radiation sickness syndrome
LCMCosentyx
secukinumab, AIN457 SpA IVIV
LCMCosentyx
secukinumab, AIN457 Hidradenitis suppurativa
LCMCosentyx
secukinumab, AIN457 AS H2H
LCMEntresto EUa
valsartan+sacubitril, LCZ696 Pediatric HF
LCM
LCM
LCM
LCM
LCM
2023
LCMPiqray
alpelisib, BYL719
TNBC
LCMPiqray
alpelisib, BYL719 HER2+ adv BC
LCMPiqray
alpelisib, BYL719 Ovarian cancer
LCMKymriah
tisagenlecleucel-T, CTL019 Adult r/r ALL
LCMTafinlar
dabrafenib, DRB436 Tyroid cancer
LCMBeovu
brolucizumab, RTH258 Diabetic retinopathy
Beovu
brolucizumab, RTH258
RVO
LCMPiqray
alpelisib, BYL719 HNSCC 2/3L
LCMJakavi
ruxolitinib, INC424 Pediatrics Acute GVHD
LCM177Lu-PSMA-R2
177Lu-PSMA-R2 Prostate cancer
LCM
LCM
LCM
LCM
≥2024 | |||||
Jakavi | |||||
LCM | Kymriah | LCM | |||
tisagenlecleucel-T, CTL019 | ruxolitinib, INC424 | ||||
1L high risk ALL, pediatrics & young adults | Pediatrics Chronic GVHD | ||||
Cosentyx | |||||
LCM | Kymriah | LCM | |||
tisagenlecleucel-T, CTL019 | secukinumab, AIN457 | ||||
r/r DLBCL (+ pembro) | GCA | ||||
LCM | Jakavi | LCM | Cosentyx | ||
ruxolitinib, INC424 | secukinumab, AIN457 | ||||
Myelofibrosis (combination) | Lichen Planus |
LCM | Cosentyx | LCM | |
secukinumab, AIN457 | |||
Lupus Nephritis | |||
LCM | Cosentyx | LCM | |
secukinumab, AIN457 | |||
Ankylosing spondylitis | |||
LCM | Mayzent | LCM | |
siponimod, BAF312 | |||
Ped MS |
a) Approved in US
Compared to past reports, we have categorized submission schedules into NMEs (lead & new indications) and supplementary indications for existing brands
60 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Novartis pipeline in registration
6 lead indications
Lead indication
Oncology
Code | Name | M echanism | Indication(s) | |||
BYL719 | Piqray | PI3Kα inhibitor | PIK3CA mutant HR+, HER2 (-) postmenopausal adv BC5 2nd line | |||
(+fulv) | ||||||
INC280 | capmatinib | Met Inhibitor | NSCLC | |||
SEG101 | Adakveo® | P-selectin Inhibitor | Sickle cell disease |
Immunology, Hepatology, Dermatology
Code | Name | M echanism | Indication(s) | |||
AIN457 | Cosentyx | IL17A Inhibitor | Ped Psoriasis | nr-axSpA |
Ophthalmology
Code | Name | Mechanism | Indication(s) | |||
RTH258 | Beovu | VEGF Inhibitor | nAMD |
Neuroscience
Code | Name | M echanism | Indication(s) | ||
OAV101 | Zolgensma® | Gene therapy | SMA IV | ||
OMB157 | ofatumumab | CD20 Antagonist | r MS |
Respiratory Disease
Code | Name | M echanism | Indication(s) | |||
IGE025 | Xolair | IgE Inhibitor | Nasal polyps | |||
QMF149 | Indacaterol acetate | Long acting β2-adrenergic | Asthma | |||
+mometasone furoate | agonist + inhaled corticosteroid | |||||
QVM149 | Indacaterol acetate | Long acting β2-adrenergic | Asthma | |||
+mometasone fuorate | agonist + long-acting muscarinic | |||||
+glycopyrrnium bromide | antagonist + inhaled | |||||
corticosteroid |
Cardiovascular, Renal, Metabolism
Code | Name | Mechanism | Indication(s) | ||
KJX839 | inclisiran | siRNA (PCSK9) | Hyperlipidemia |
Global Health
Code | Name | Mechanism | Indication(s) | |||
LAM320 | Lamprene® | SMPD1 Inhibitor | Tuberculosis |
61 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Novartis pipeline in Phase 3
5 lead indications
Lead indication
Oncology
Code | Name | Mechanism | Indication(s) | ||
177Lu-PSMA-617 | 177Lu-PSMA-617 | Targeted Radioligand Therapy | mCRPC | ||
ABL001 | asciminib | BCR-ABL Inhibitor | CML 3L | ||
ACZ885 | canakinumab | IL-1b Inhibitor | NSCLC 1L | NSCLC 2L | Adjuvant |
NSCLC | |||||
BYL719 | Piqray® | PI3Kα inhibitor | HER2+ adv BC | TNBC | HNSCC 2/3L | Ovarian cancer |
CTL019 | Kymriah | CD19 CART | r/r Follicular | 1L high risk | r/r DLBCL 1st | Adult r/r ALL |
lymphoma | ALL, pediatrics | relapse | ||||
and young | ||||||
adults | ||||||
ETB115 | Promacta® | Thrombopoietin receptor (TPO-R) Radiation sickness syndrome | Food effect free formulation | |||
Agonist | ||||||
INC424 | Jakavi | JAK1/2 Inhibitor | Acute GVHD | Chronic GVHD | ||
LEE011 | Kisqali® | CDK4 Inhibitor | HR+/HER2- BC (adj) | |||
PDR001 | Spartalizumab | PD1 Inhibitor | m BRAF V600+ melanoma (+Taf/Mek) | |||
SEG101 | crizanlizumab | P-selectin Inhibitor | Sickle cell anemia new formulation |
Respiratory Disease
Code | Name | Mechanism | Indication(s) | |||
IGE025 | Xolair® | IgE Inhibitor | CSU (for CN) | Auto-injector | Food allergy |
Cardiovascular, Renal, Metabolism
Code | Name | Mechanism | Indication(s) | |||
KJX839 | inclisiran | siRNA (PCSK9) | Hyperlipidemia | |||
LCZ696 | Entresto® | AT-II / NEP,NEP,AGTR1,AGTR2 Inhibitor | Post-AMI | HFpEF | Pediatric HFa | |
TQJ230 | TQJ230 | Anti-Apo(a) ASO targeting Lp(a) | CVRR-Lp(a) |
Biosimilars
Code | Name | Mechanism | Indication(s) | ||
GP2411 | denosumab | anti RANKL mAb | Denosumab BioS |
Immunology, Hepatology, Dermatology
Code | Name | Mechanism | Indication(s) | ||||
AIN457 | Cosentyx | IL17A Inhibitor | Lupus | Psoriasis | Hidradenitis | AS H2H | PsA H2H |
Nephritis | 300mg AI | suppurativa | |||||
QGE031 | ligelizumab | IgE Inhibitor | Chronic spontaneous urticaria |
Ophthalmology
Code | Name | Mechanism | Indication(s) | ||
RTH258 | Beovu® | VEGF Inhibitor | Diabetic retinopathy | RVO | DME |
a) Approved in US
62 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Novartis pipeline in Phase 2
27 lead indications
Lead indication
Oncology | Neuroscience | |
Code | Name | M echanism | Indication(s) | |||
ACZ885 | canakinumab | IL-1b Inhibitor | Sickle cell anaemia | |||
BYL719 | alpelisib | PI3Kα inhibitor | PROS | |||
CTL019 | Kymriah | CD19 CART | r/r DLBCL (+ pembro) | |||
EGF816 | nazartinib+capmatinib Opdivo EGFR Inhibitor | NSCLC | ||||
INC280 | capmatinib | Met Inhibitor | Solid tumors | |||
Met Inhibitor + spartalizumab | HCC | NSCLC | ||||
INC424 | Jakavi® | JAK1/2 Inhibitor | Myelofibrosis (combination) | |||
LAG525 | LAG525 | LAG3 Inhibitor | Solid Tumors | |||
MBG453 | MBG453 | TIM3 Antagonist | HR-MDS | Unfit AML | AML | Fit AML |
NIR178 | NIR178, spartalizumab | Ad2AR Inhibitor, PD1 Inhibitor | Cancers | |||
PDR001 | spartalizumab | PD1 Inhibitor | Nasopharyngeal cancer | Metastatic melanoma (combo) | ||
SEG101 | crizanlizumab | P-selectin Inhibitor | Ped sickle cell anaemia with | |||
crisis |
Immunology, Hepatology, Dermatology
Code | Name | Mechanism | Indication(s) | ||||
AIN457 | Cosentyx® | IL17A Inhibitor | SpA IVIV | GCA | Lichen Planus | ||
CFZ533 | iscalimab | CD40 Inhibitor | Renal/Liver Tx | SjS | HS | T1DM | |
LJC242 | tropifexor&cenicriviroc | CCR2 Inhibitor, FXR agonist | NASH | ||||
LJN452 | tropifexor | FXR agonist | NASH | Nash (combos) | |||
LNA043 | LNA043 | ANGPTL3 Agonist | Osteoarthritis | ||||
LOU064 | LOU064 | BTK Inhibitor | Chronic spontaneous urticaria | SjS | |||
LYS006 | LYS006 | Anti-inflammatory | Acne | ||||
VAY736 | ianalumab | BAFF-R Inhibitor | AIH | pSjS | SLE | ||
ZPL389 | adriforant | HRH4 Antagonist | Atopic dermatitis |
Ophthalmology
Code | Name | Mechanism | Indication(s) | |||
CPK850 | CPK850 | RLBP1 AAV | RP | |||
ECF843 | ECF843 | rh-Lubricin | Dry eye | |||
LKA651 | LKA651 | EPO Inhibitor | DME | |||
SAF312 | SAF312 | TRPV1 Antagonist | COSP | |||
UNR844 | UNR844 | disulfide bonds Modulator | Presbyopia |
Code | Name | Mechanism | Indication(s) | |||
AFQ056 | AFQ056 | mGluR5 Antagonist | Addiction | |||
BAF312 | Mayzent® | S1P1 Modulator | Ped MS | Stroke | ||
BLZ945 | BLZ945 | CSF-1 Inhibitor | ALS | |||
LMI070 | branaplam | Survival motor neuron protein | SMA | |||
MIJ821 | MIJ821 | NR2B Inhibitor | Depression | |||
OMB157 | ofatumumab | CD20 Antagonist | Ped MS |
Respiratory Disease
Code | Name | Mechanism | Indication(s) | |||
ACZ885 | canakinumab | IL-1b Inhibitor | Sarcoidosis | |||
CJM112 | CJM112 | IL-17A Inhibitor | Asthma | |||
CSJ117 | CSJ117 | TSLP Inhibitor | Severe asthma | |||
LOU064 | LOU064 | BTK Inhibitor | Asthma | |||
QBW251 | QBW251 | CFTR Potentiator | COPD | |||
VAY736 | ianalumab | BAFF-R Inhibitor | IPF |
Cardiovascular, Renal, Metabolism
Code | Name | Mechanism | Indication(s) | |||
CFZ533 | iscalimab | CD40 Inhibitor | Lupus Nephritis | |||
LMB763 | nidufexor | FXR Agonist | Diabetic Nephropathy | |||
LNP023 | LNP023 | CFB Inhibitor | PNH | IgAN | C3G | iMN |
Global Health
Code | Name | Mechanism | Indication(s) | |||
KAE609 | cipargamin | PfATP4 inhibitor | Malaria | Malaria severe | ||
KAF156 | ganaplacide | - | Malaria | |||
LXE408 | LXE408 | Protozoan Inhibitor | Visceral leishmaniasis |
63 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Novartis pipeline in Phase 1 (1 of 2)
33 lead indications
Lead indication
Oncology
Code | Name | Mechanism | Indication(s) | |||
177Lu-NeoB | 177Lu-NeoB | Radioligand therapy target GRPR | Multiple solid tumors | |||
177Lu-PSMA-R2 | 177Lu-PSMA-R2 | Radioligand therapy target PSMA | Prostate cancer | |||
ADPT01 | NIR178, LAG525, spartalizumab, canakinumab, capmatinib | LAG3 Inhibitor,PD1 Inhibitor | TNBC | |||
BLZ945 | BLZ945 + spartalizumab | CSF-1 Inhibitor + PD1 Inhibitor | Solid tumors | |||
CSJ137 | CSJ137 | Growth Factor Inhibitor | Anaemia | |||
CTL019 | Kymriah® | CD19 CART | Lymphoma | |||
DKY709 | DKY709 + spartalizumab | - | Cancers | |||
EGF816 | nazartinib + LXH254, ribociclib, capmatinib, Opdivo, Mekinist | EGFR Inhibitor | NSCLC | |||
HDM201 | HDM201 + MBG453, venetoclax | MDM2 Inhibitor | Haematological malignancy | |||
JEZ567 | JEZ567 | CD123 CART | AML | |||
JJO686 | JJO686 | CD22 CART | ALL | |||
LHC165 | LHC165 + spartalizumab | TLR7 Agonist | Solid tumors | |||
LSZ102 | LSZ102, ribociclib, alpelisib | SERD | BC | |||
LXF821 | LXF821 | EGFR CART, PD1 Inhibitor | Glioblastoma multiforme | |||
LXH254 | LXH254 (combos) | cRAF Inhibitor | Solid tumors | Solid tumors | ||
MAK683 | MAK683 | EED Inhibitor | Cancers | |||
MAS825 | MAS825 | - | Inflammatory diseases | |||
MBG453 | MBG453 (combos) | TIM3 Antagonist | Cancers | |||
MCM998 | MCM998, LXG250 | BCMA CART, CD19 CART | Multiple myeloma | |||
MIK665 | MIK665 | MCL1 Inhibitor | AML | Haematological malignancy | AML (combo) | |
NIS793 | NIS793, spartalizumab | TGFB1 Inhibitor, PD1 Inhibitor | Solid tumors | |||
NIZ985 | NIZ985, spartalizumab | IL-15 Agonist | Solid tumors | |||
NJH395 | NJH395 | - | Solid tumors | |||
NZV930 | NZV930, spartalizumab, NIR178 | CD73 Antagonist | Solid tumors | |||
PDR001 | spartalizumab, CJM112, LCL161 | PD1 Inhibitor, TIM3 Antagonist | AML | Solid tumors (combo) | Solid tumors (combo) | Solid tumors (combo) |
SQZ622 | SQZ622 | CD123xCD3 Modulator | AML | |||
TNO155 | TNO155 | SHP2 Inhibitor | Solid tumors (single agent) | Solid tumors (combo) | ||
VAY736 | ianalumab + ibrutinib | BAFF-R Inhibitor,BTK Inhibitor | Haematological malignancy | |||
VOB560 | VOB560 | - | Cancers | |||
VPM087 | VPM087 | IL1B Antagonist | 1st line CRC / 1st line RCC | |||
WNT974 | WNT974 + spartalizumab | Porcupine Inhibitor | Solid tumors | |||
WVT078 | WVT078 | - | Multiple myeloma | |||
YTB323 | YTB323 + ibrutinib | CD19 CART | Haematological malignancy |
64 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Novartis pipeline in Phase 1 (2 of 2)
34 lead indications
Lead indication
Immunology, Hepatology, Dermatology
Code | Name | M echanism | Indication(s) | |||
DFV890 | DFV890 | - | Multiple Indications | |||
LRX712 | LRX712 | - | Osteoarthritis | |||
MHS552 | MHS552 | - | Autoimmune Indications | |||
MHV370 | MHV370 | - | SLE |
Neuroscience
Code | Name | M echanism | Indication(s) | |||
OAV101 | AVXS-101 | Survival motor neuron protein | SMA IT1 | |||
gene therapy | ||||||
OAV201 | AVXS-201 | MECP2 gene therapy | Rett syndrome |
Respiratory Disease
Code | Name | M echanism | Indication(s) | |||
CMK389 | CMK389 | IL-18 Inhibitor | Sarcoidosis |
Cardiovascular, Renal, Metabolism
Code | Name | M echanism | Indication(s) | |||
HSY244 | HSY244 | - | Atrial fibrillation | |||
LTW980 | LTW980 | - | Hypertriglyceridemia | |||
MBL949 | MBL949 | - | Diabetes |
Global Health
Code | Name | Mechanism | Indication(s) | ||
KAF156 | ganaplacide | - | Malaria prophylaxis |
1. FDA placed a partial hold on AVXS-101 intrathecal clinical trials for SMA patients based on findings in a small pre-clinical animal study
65 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Abbreviations
AIH | Autoimmune hepatitis | IT | Intrathecal formulation |
ALL | Acute lymphoblastic leukemia | IV | Intravenous formulation |
ALS | Amyotrophic lateral sclerosis | JIA | Juvenile idiopathic arthritis |
AMI | Acute myocardial infarction | LP | Lichen planus |
AML | Acute myeloid leukemia | LN | Lupus nephritis |
AS H2H | Ankylosing spondylitis head-to-head study versus adalimumab | mCRPC | Metastatic castration-resistant prostate cancer |
BC | Breast cancer | MDR | Multi-drug resistant |
C3G | C3 glomerulopathy | MDS | Myelodysplastic syndrome |
CDx | Companion diagnostics | MS | Multiple sclerosis |
CCF | Congestive cardiac failure | nAMD | Neovascular (wet) age-related macular degeneration |
CLL | Chronic lymphocytic leukemia | NASH | Non-alcoholic steatohepatitis |
CML | Chronic myeloid leukemia | nr-axSpA | Non-radiographic axial spondyloarthritis |
CRC | Colorectal cancer | NRDL | National reimbursement drug list |
COPD | Chronic obstructive pulmonary disease | NSCLC | Non-small cell lung cancer |
COSP | Chronic ocular surface pain | PDR | Proliferative diabetic retinopathy |
CSU | Chronic spontaneous urticaria | PEF | Preserved ejection fraction |
CVRR-Lp(a) | Secondary prevention of cardiovascular events in patients with elevated levels of lipoprotein (a) | PNH | Paroxysmal nocturnal haemoglobinuria |
CVRR-LDLC | Secondary prevention of cardiovascular events in patients with elevated levels of LDLC | PsA H2H | Psoriatic arthritis head-to-head study versus adalimumab |
DME | Diabetic macular edema | RCC | Renal cell carcinoma |
DLBCL | Diffuse large B-cell lymphoma refractory | PROS | PIK3CA related overgrowth spectrum |
FL | Follicular lymphoma | RA | Rheumatoid arthritis |
GCA | Giant cell arteritis | rMS | Relapsing multiple sclerosis |
GVHD | Graft-versus-host disease | ROP | Retinopathy of prematurity |
HCC | Hepatocellular carcinoma | RP | Retinitis pigmentosa |
HFpEF | Chronic heart failure with preserved ejection fraction | RVO | Retinal vein occlusion |
HF-rEF | Chronic heart failure with reduced ejection fraction | SAA | Severe aplastic anemia |
HNSCC | Head and neck squamous cell carcinoma | SjS | Sjögren's syndrome |
HS | Hidradenitis suppurativa | SLE | Systemic lupus erythematosus |
IgAN | IgA nephropathy | SMA | Spinal muscular atrophy type 1 (IV formulation) |
iMN | Membranous nephropathy | SpA | Spondyloarthritis |
IOL | Iron overload | SPMS | Secondary progressive multiple sclerosis |
ITP | Immune thrombocytopenia | TNBC | Triple negative breast cancer |
IPF | Idiopathic pulmonary fibrosis | T1DM | Type 1 Diabetes melitus |
66 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Clinical Trials Update
Includes selected ongoing or recently concluded global trials of Novartis development programs/products which are in confirmatory development or marketed (typically Phase 2 or later).
For further information on all Novartis clinical trials, please visit: www.novartisclinicaltrials.com
Key changes vs. Q3-2019 presentation
New trials added
Study | Program | Indication | Phase | Patients |
NCT04156620 INVIGORATE-1 (CAIN457P12301) | Cosentyx® | Axial spondyloarthritis | Phase 3 | 500 |
NCT04065841 ELIVATE (CLJN452D12201C) | LJN452 | Non-alcoholic steatohepatitis (NASH) | Phase 2 | 210 |
NCT03373461 (CLNP023X2203) | LNP023 | IgA nephropathy | Phase 2 | 146 |
NCT03439839 (CLNP023X2201) | LNP023 | Paroxysmal nocturnal hemoglobinuria | Phase 2 | 15 |
NCT03832114 (CLNP023X2202) | LNP023 | C3 glomerulopathv | Phase 2 | 27 |
NCT03896152 (CLNP023X2204) | LNP023 | Paroxysmal nocturnal hemoglobinuria | Phase 2 | 10 |
NCT03955445 (CLNP023B12001B) | LNP023 | C3 glomerulopathy | Phase 2 | 27 |
NCT04154787 (CLNP023D12201) | LNP023 | Idiopathic membraneous nephropathy | Phase 2 | 72 |
NCT04109313 (CLOU064A2201E1) | LOU064 | Chronic spontaneous urticaria (CSU) | Phase 2 | 250 |
NCT03988608 (CETB115E2202) | Promacta®/ | Previously untreated or relapsed/refractory severe aplastic anemia | Phase 2 | 20 |
Revolade® | or recurrent aplastic anemia | |||
NCT04047472 HOBBY (CRTH258A2307) | RTH258 | Macular degeneration | Phase 3 | 494 |
Trials removed (operational decision-points achieved)
Study | Program | Indication | Phase | Patients | |
NCT02059291 CLUSTER (CACZ885N2301) | Ilaris® | Hereditary periodic fevers | Phase 3 | 203 | |
NCT03578367 ASC4MORE (CABL001E2201) | ABL001 | Chronic myeloid leukaemia (CML) | Phase 2 | 80 |
68 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cardiovascular, Renal and Metabolic
Entresto®- Angiotensin receptor neprilysin inhibitor (ARNI)
Study | NCT02678312 PANORAMA HF (CLCZ696B2319) | NCT03785405 (CLCZ696B2319E1 - extension study) |
Indication | Heart failure in pediatric patients | Heart failure in pediatric patients |
Phase | Phase 2/3 | Phase 3 |
Patients | 360 | 240 |
Primary Outcome | Part 1: Pharmacodynamics and pharmacokinetics of | Number of participants with Adverse Events (AEs) and |
sacubitril/valsartan LCZ696 analytes | ||
Measures | Serious Adverse Events (SAEs) | |
Part 2: Efficacy and safety compared with enalapril | ||
• Part 1: Sacubitril/valsartan 0.8 mg/kg or 3.1 mg/kg or | ||
both; 0.4 mg/kg or 1.6 mg/kg or both (single doses). | ||
• Part 2: enalapril/placebo 0.2 mg/kg bid (ped. formulation | • Single arm, open label sacubitril/valsartan (pediatric | |
Arms/Intervention | 1mg/ml) and adult formulation (2.5, 5, 10 mg bid); | formulation granules (12.5, 31.25 mg in capsules); liquid |
Sacubitril/valsartan (LCZ696)/placebo: Ped. formulation | formulation (1mg/ml and 4mg/ml concentration) and | |
granules (12.5, 31.25 mg in capsules); liquid formulation | adult formulation (50, 100, 200 mg bid)) | |
(1mg/ml and 4mg/ml concentration) and adult | ||
formulation (50, 100, 200 mg bid) | ||
Pediatric patients from 1 month to < 18 years of age with | Pediatric patients with heart failure due to systemic left | |
Target Patients | heart failure due to systemic left ventricle systolic | ventricle systolic dysfunction who have completed study |
dysfunction | CLCZ696B2319 | |
H2-2021; (Analysis of 110 pts from Part 2 formed the basis | ||
for pediatric submission in Apr-2019 and approval by the US | ||
Expected Completion | FDA in Oct-2019 for the treatment of symptomatic HF with | 2022 |
systemic left ventricular systolic dysfunction in children aged | ||
1 year and older) | ||
Publication | TBD | TBD |
70 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Entresto®- Angiotensin receptor neprilysin inhibitor (ARNI)
Study | NCT02554890 PIONEER-HF (CLCZ696BUS01) | NCT02661217 TRANSITION (CLCZ696B2401) | ||
Indication | Heart failure, reduced ejection fraction | Heart failure, reduced ejection fraction | ||
Phase | Phase 3B/4 | Phase 4 | ||
Patients | 881 | 1,002 | ||
Primary Outcome | Percentage change from baseline in N-terminalpro-brain | Assessing the percentage of patients who achieve the target | ||
dose of 200 mg bid LCZ696 at 10 weeks after | ||||
Measures | natriuretic peptide (NT-proBNP) | |||
randomization | ||||
• | Sacubitril/valsartan (LCZ696) 24/26 mg, 49/51 mg or | • | Pre-discharge treatment initiation - LCZ696 (50, 100, | |
Arms/Intervention | 97/103 mg bid or matching placebo | 200 mg bid) | ||
• Enalapril (2.5 mg, 5 mg, and 10 mg) bid or matching | • | Post-discharge treatment initiation - LCZ696 (50, 100, | ||
placebo | 200 mg bid) | |||
Target Patients | Patients with HFrEF (LVEF<40%) hospitalized for ADHF | Heart failure patients with reduced ejection-fraction | ||
and stable for more than 24 hours | hospitalized for an acute decompensation event | |||
Expected Completion | Q3-2018(actual) | Q4-2018(actual) | ||
• Mar-2019 ACC: 4wk OLE data, and core study data on | • | 28-May-2019 TRANSITION primary publication - | ||
published EJHF | ||||
biomarkers, de novo HF, hospitalizations, & prior | ||||
• | Secondary data presentations: de novo HF and | |||
exposure | ||||
ACEi/ARB naive sub-groups presented at ESC-HF | ||||
Publication | • | Apr-2019 Circulation: Research letter on composite | ||
Congress in May 2019 and submitted to EJHF in Jun- | ||||
endpoint (Circulation. 2019;139:00-00) | ||||
2019 (expected publication Q3-2019) | ||||
• Q3-2019 ESC: Secondary abstracts submitted | ||||
• | Planned in Q4-2019:26-weeks data presentation at | |||
• | Planned in Q1-2020: RAAS naive and de novo HF | |||
ESC-2019 in Paris | ||||
71 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Entresto®- Angiotensin receptor neprilysin inhibitor (ARNI)
Study | NCT02884206 PERSPECTIVE (CLCZ696B2320) | NCT02468232 PARALLEL-HF (CLCZ696B1301) | |
Indication | Heart failure | Heart failure, reduced ejection fraction | |
Phase | Phase 3 | Phase 3 | |
Patients | 592 | 225 | |
Primary Outcome | Change from baseline in the CogState Global Cognitive | Time to the first occurrence of the composite endpoint - | |
either cardiovascular (CV) death or heart failure (HF) | |||
Measures | Composite Score (GCCS) | ||
hospitalization | |||
• Sacubitril/valsartan 50, 100, and 200 mg bid with | • | Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid/placebo | |
Arms/Intervention | placebo of valsartan | of enalapril | |
• Valsartan 40, 80, and 160 mg bid tablets with placebo | • | Enalapril 2.5 mg, 5 mg, 10 mg bid / placebo of | |
for sacubitril/valsartan | sacubitril/valsartan | ||
Target Patients | Patients with chronic heart failure with preserved ejection | Japanese heart failure patients (NYHA Class II-IV) with | |
fraction | reduced ejection fraction | ||
Expected Completion | 2022 | Q1-2019(actual);H1-2021(open-label extension) | |
Publication | TBD | Planned in H1-2020: Core study primary manuscript in Circ | |
J | |||
72 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Entresto®- Angiotensin receptor neprilysin inhibitor (ARNI)
Study | NCT01920711 PARAGON-HF (CLCZ696D2301) | NCT03066804 PARALLAX (CLCZ696D2302) | |
Indication | Heart failure, preserved ejection fraction | Heart failure, preserved ejection fraction | |
Phase | Phase 3 | Phase 3 | |
Patients | 4,822 | 2,577 | |
Primary Outcome | Cumulative number of primary composite events of | Change in NT-proBNP from baseline to week 12 | |
cardiovascular (CV) death and total (first and recurrent) HF | and change in 6 minute walk distance (6MWD) from | ||
Measures | |||
hospitalizations | baseline to Week 24 | ||
• Sacubitril/valsartan 50 mg, 100 mg and 200 mg bid and | |||
• | Sacubitril/valsartan or placebo 50 mg, 100 mg, and 200 | matching placebo | |
• Enalapril 2.5 mg, 5 mg and 10 mg bid and matching | |||
Arms/Intervention | mg bid | ||
placebo | |||
• | Valsartan or placebo 40 mg, 80 mg, and 160 mg bid | ||
• Valsartan 40 mg, 80 mg, 160 mg bid and matching | |||
placebo | |||
Target Patients | Heart failure patients (NYHA Class II-IV) with preserved | Heart failure patients (NYHA Class II-IV) with preserved | |
ejection fraction | ejection fraction | ||
Expected Completion | Q3-2019(actual) | Q4-2019(actual) | |
• Sep-2019: Primary manuscript published (Angiotensin- | |||
Neprilysin Inhibition in Heart Failure with Preserved | |||
Publication | Ejection Fraction. Solomon S et al; NEJM. DOI: | TBD | |
10.1056/NEJMoa1908655) | |||
• Sep-2019: ESC: Late breaker presentation of primary | |||
results | |||
73 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Entresto®- Angiotensin receptor neprilysin inhibitor (ARNI)
Study | NCT03909295 (CLCZ696D1301E1 - extension study) | NCT02924727 PARADISE-MI (CLCZ696G2301) |
Indication | Heart failure chronic | Post-acute myocardial infarction |
Phase | Phase 3 | Phase 3 |
Patients | 63 | 5,650 |
Primary Outcome | Number of participants with Adverse Events (AEs) and | Time to the first occurrence of a confirmed composite |
endpoint (cardiovascular (CV) death, heart failure (HF) | ||
Measures | Serious Adverse Events (SAEs) | |
hospitalization, or outpatient heart failure) | ||
• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid / placebo | ||
Arms/Intervention | • Sacubitril/valsartan 50 mg,100 mg,200 mg film coated | of ramipril/valsartan |
tablets | • Ramipril 1.25 mg, 2.5 mg, and 5 mg bid / placebo of | |
sacubitril/valsartan / placebo for valsartan | ||
Japanese heart failure patients (NYHA Class II-IV) with | Post-AMI patients with evidence of LV systolic dysfunction | |
Target Patients | preserved ejection fraction after CLCZ696D2301 | and/or pulmonary congestion, with no known prior history of |
(PARAGON-HF) | chronic HF | |
Expected Completion | Q4-2019(actual) | H1-2021 |
Publication | TBD | TBD |
74 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
LNP023 - Factor B inhibition of the complement alternative pathway
Study | NCT03373461 (CLNP023X2203) | NCT04154787 (CLNP023D12201) |
Indication | IgA nephropathy (IgAN) | Idiopathic membranous nephropathy (iMN) |
Phase | Phase 2 | Phase 2 |
Patients | 146 | 72 |
Primary Outcome | Change from baseline of log transformed UPCR derived |
Measures | from the 24h urine collections at Baseline and Day 90 |
Change from baseline of UPCR derived from 24hr urine collections at Baseline and Week 24
Arms/Intervention
Target Patients
Expected Completion
Publication
- Placebo
• LNP023 Dose 1 - 10mg bid | • LNP023 Dose - 200mg bid | ||
• LNP023 Dose 2 - 50mg bid | • LNP023 Dose - 50mg bid | ||
• | LNP023 Dose 3 | - 200mg bid | • Rituximab |
• | LNP023 Dose 4 | - 100mg bid (Part 2 only) | |
Patients with biopsy proven iMN who are at high risk of | |||
Patients with biopsy-verified IgA nephropathy | disease progression defined on the basis of antibody anti- | ||
PLA2R titre and proteinuria | |||
2021 | 2022 | ||
TBD | TBD |
75 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
LNP023 - Factor B inhibition of the complement alternative pathway
Study | NCT03832114 (CLNP023X2202) | NCT03955445 (CLNP023B12001B) |
Indication | C3 glomerulopathy (C3G) | C3 glomerulopathy (C3G) |
Phase | Phase 2 | Phase 2 (open-label extension) |
Patients | 27 | 27 (from ongoing Phase 2, potential patient from Ph3) |
Primary Outcome Measures
Cohort A: Ratio to Baseline of UPCR to Week 12 derived from 24hr urine collection
Cohort B: Change from Baseline in C3 Deposit Score (based on immunofluorescence microscopy) at Week 12
Characterize the effect of LNP023 treatment on a composite renal response endpoint at 9 months (1. a stable or improved eGFR and, 2. a reduction in proteinuria and 3. an increase in C3 compared to the CLNP023X2202 baseline visit)
Arms/Intervention
Target Patients
Expected Completion
Publication
Increasing doses of LNP023 up to 200mg bid: | |
• Cohort A: Native kidney patients | • Open-label LNP023 200mg bid |
• Cohort B: Kidney transplanted patients | |
Patients with C3 glomerulopathy | Patients with C3 glomerulopathy |
2021 | 2025 |
TBD | TBD |
76 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
LNP023 - Factor B inhibition of the complement alternative pathway
Study | NCT03439839 (CLNP023X2201) | NCT03896152 (CLNP023X2204) |
Indication | Paroxysmal nocturnal hemoglobinuria (PNH) | Paroxysmal nocturnal hemoglobinuria (PNH) |
Phase | Phase 2 | Phase 2 |
Patients | 15 | 10 |
Primary Outcome | Reduction of chronic hemolysis, based on LDH level at | Reduction of PNH associated hemolysis, based on | |
percentage of patients with 60% reduction in LDH or LDH | |||
Measures | Week 13 | ||
below upper limit of normal up to 12 weeks of treatment. | |||
• Cohort 1: 10 patients receiving LNP023 200mg bid, in | |||
addition to SoC, for 13 weeks with 3yr treatment extension | • | Arm 1: 4wks treatment LNP023 25mg bid followed by | |
period | 8wk treatment LNP023 100mg bid and 2yr extension | ||
Arms/Intervention | • Cohort 2: 5 patients receiving LNP023 50mg bid, in | LNP023 100mg bid | |
addition to SoC, for minimum 2 weeks with 3yr treatment | • | Arm 2: 4wks treatment LNP023 50mg bid followed by | |
extension period. Dose may be increased D15 onwards to 200mg bid if LDH not within limit of normal or reduced by at least 60% compared to Baseline.
8wk treatment LNP023 200mg bid and 2yr extension LNP023 200mg bid
Patients with PNH, showing signs of active hemolysis | Patients with PNH, showing signs of active hemolysis, not | |
Target Patients | despite treatment with SoC (defined as an antibody with anti | treated with any other complement inhibitor less than 3 |
C5 activity). | months prior to study start Day 1 | |
Expected Completion | Primary endpoint: 2020 | Primary endpoint: 2020 |
Extension period: 2023 | Extension period: 2022 | |
Publication | In preparation | TBD |
77 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Immunology, Hepatology & Dermatology
CFZ533 - Blocking, non-depleting,Fc-silent,anti-CD40 monoclonal antibody
Study | NCT03663335 CIRRUS I (CCFZ533A2201) | NCT03905525 TWINSS (CCFZ533B2201) |
Indication | Kidney transplantation | Sjögren's syndrome |
Phase | Phase 2B | Phase 2B |
Patients | 676 | 260 |
Primary Outcome Measures
Arms/Intervention
Composite event (BPAR, Graft Loss or Death) over 12 | Change in EULAR Sjögren's syndrome Disease Activity |
months post-transplantation and post conversion (for | Index (ESSDAI) score and EULAR Sjögren's syndrome |
maintenance cohort) | Patient Reported Index (ESSPRI) score |
• Two cohorts: de novo TX and maintenance | • Three dose arms of CFZ533 |
• Test Arms: CFZ533 + MMF + corticosteroids | |
• Placebo | |
• Standard of Care: TAC + MMF + corticosteroids | |
Target Patients | Kidney transplant recipients | Patients with Sjögren's syndrome |
Expected Completion | 2022 | 2022 |
Publication | Manuscript of PoC trial to be submitted in Q1-2020 | Manuscript of PoC trial to be published in Q1-2020 |
79 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
CFZ533 - Blocking, non-depleting,Fc-silent,anti-CD40 monoclonal antibody
Study | NCT03781414 CONTRAIL I (CCFZ533A2202) |
Indication | Liver transplantation |
Phase | Phase 2 |
Patients | 128 |
Primary Outcome | Proportion of patients with composite event (BPAR, Graft |
Measures | Loss or Death) over 12 months |
• Control/Standard of Care: TAC + MMF + Corticosteroids | |
Arms/Intervention | • CFZ533 dose A + MMF + Corticosteroids |
• CFZ533 dose B + MMF + Corticosteroids | |
Target Patients
Expected Completion
Publication
Liver transplant recipients
2022
TBD
80 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT03504852 (CAIN457A2324) | NCT03589885 MATURE (CAIN457A2325) |
Indication | Psoriasis | Psoriasis |
Phase | Phase 3B | Phase 3 |
Patients | 331 | 122 |
Primary Outcome | PASI 90 response and IGA mod 2011 0 or 1 response after | PASI 75 response and IGA mod 2011 0 or 1 response after |
Measures | 16 weeks of treatment | 12 weeks of treatment |
Arms/Intervention
Target Patients
Expected Completion
Publication
• | Secukinumab 300 mg every 2 weeks after weekly doses | • | Secukinumab 2 mL (300 mg) auto-injector |
till Week 4 | • | Secukinumab 2 x 1 mL (150 mg each) prefilled syringe | |
• | Secukinumab 300 mg every 4 weeks after weekly doses | • | Placebo 2 mL auto-injector |
till Week 4 | • | Placebo 2 x 1 mL prefilled syringe | |
Subjects (≥90kg) with moderate to severe plaque psoriasis | Subjects with moderate to severe plaque psoriasis | ||
Q3-2020 | Q4-2020 | ||
TBD | TBD |
81 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT02471144 (CAIN457A2310) | NCT03668613 (CAIN457A2311) | ||
Indication | Psoriasis | Psoriasis | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 162 | 84 | ||
Primary Outcome | Psoriasis Area and Severity Index (PASI) 75 response and | Psoriasis Area and Severity Index (PASI) 75 response and | ||
Investigators' Global Assessment (IGA) 0 or 1 response at | Investigators' Global Assessment (IGA) 0 or 1 response at | |||
Measures | ||||
week 12 | week 12 | |||
• | Secukinumab low dose | |||
Arms/Intervention | • | Secukinumab high dose | • | Secukinumab low dose |
• | Placebo | • | Secukinumab high dose | |
• | Etanercept (comparator) | |||
Target Patients | Patients from 6 to less than 18 years of age with severe | Pediatric patients of age 6 to <18 years, with moderate to | ||
chronic plaque psoriasis | severe plaque psoriasis | |||
Expected Completion | 2023 | 2023 | ||
Publication | TBD | TBD | ||
82 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT03066609 (CAIN457A2318) |
Indication | Psoriasis |
Phase | Phase 3 |
Patients | 543 |
Primary Outcome | Psoriasis Area and Severity Index (PASI) 75 response and |
Measures | Investigators' Global Assessment (IGA) 0 or 1 response at |
week 12 | |
Arms/Intervention
Target Patients
Expected Completion
Publication
- Secukinumab 300 mg
- Secukinumab 150 mg
- Placebo
Patients with moderate to severe chronic plaque-type psoriasis with or without psoriatic arthritis comorbidity
Q1-2019(actual)
- Week 16 results: Poster presented at: 2019 American Academy of Dermatology (AAD) Annual Meeting,
- March1-5, 2019, Washington, D.C.
- 52-weekresults: Poster at EADV 2019, Madrid 9-13 October, 2019
83 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT03031782 (CAIN457F2304) | NCT03769168 (CAIN457F2304E1 - extension study) | ||
Indication | Psoriatic arthritis | Psoriatic arthritis | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 80 | 64 | ||
Primary Outcome | Time to 33 flares | Number of participants with JIA ACR30 response | ||
Measures | ||||
Arms/Intervention | • | Secukinumab (pre-filled syringe) 75 mg | • | Secukinumab 75 mg/0.5 ml |
• | Placebo | • | Secukinumab 150 mg/1.0 ml | |
Target Patients | Juvenile idiopathic arthritis subtypes of psoriatic and | Patients with juvenile idiopathic arthritis subtypes of juvenile | ||
enthesitis-related arthritis | psoriatic arthritis and enthesitis related arthritis | |||
Expected Completion | H1-2021 | 2025 | ||
Publication | TBD | TBD | ||
84 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT01892436 FUTURE 1 extension (CAIN457F2306E1) | NCT01649375 MEASURE 2 (CAIN457F2310) | ||
Indication | Psoriatic arthritis | Ankylosing spondylitis | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 460 | 219 | ||
Primary Outcome | Proportion of subjects that have a positive clinical response | Assessment of SpondyloArthritis International Society / | ||
to treatment (individual improvement) in disease activity | ||||
Measures | ASAS 20 response | |||
according to ACR20 (or ACR50 or ACR 70) | ||||
• | Secukinumab 75 mg | • | Secukinumab 75 mg | |
Arms/Intervention | • | Secukinumab 150 mg | ||
• | Secukinumab 150 mg | |||
• | Placebo | |||
Target Patients | Patients with active psoriatic arthritis | Patients with active ankylosing spondylitis | ||
Expected Completion | Q1-2018(actual) | Q4-2018(actual) | ||
• Primary 52 week results: Baeten D & Sieper J, et al. N | ||||
• 3 year results: ACR 2016; Mease PJ et al. Arthritis | Engl J Med 2015;373:2534-48 | |||
• | 2 year results: Marzo-Ortega, et al. Arthritis Care Res | |||
Rheumatol. 2016; 68 (suppl 10) | ||||
2017 Feb 24. doi: - 10.1002/acr.23233 | ||||
• | 3 years results: Manuscript published in September | |||
• | 3 year results: Marzo-Ortega, et al. RMD 2017 | |||
Publication | 2018 (Mease PJ, et al. RMD Open 2018;4:e000723. | |||
• | 5 year results: EULAR 2019; Marzo-Ortega H, et al. | |||
doi:10.1136/rmdopen-2018-000723) | ||||
FRI0379. Annals of the Rheumatic Diseases | ||||
• | 5 year results: accepted for publication in ACR open | |||
2019;78:873. | ||||
Rheumatology in September 2019 | ||||
• | 5 year results; manuscript target submission Oct 2019 | |||
85 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT01752634 FUTURE 2 (CAIN457F2312) | NCT02008916 MEASURE 3 (CAIN457F2314) | ||
Indication | Psoriatic arthritis | Ankylosing spondylitis | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 399 | 222 | ||
Primary Outcome | Proportion of subjects achieving American College of | Assessment of Spondyloarthritis International Society | ||
Measures | Rheumatology 20 (ACR20) response criteria | criteria / ASAS 20 response | ||
• | Secukinumab (AIN457) 150 mg s.c. | • | Secukinumab 10 mg/kg / 300 mg | |
• | Secukinumab (AIN457) 75 mg s.c. | |||
Arms/Intervention | • | Secukinumab 10 mg/kg / 150 mg | ||
• | Secukinumab (AIN457) 300 mg s.c. | |||
• | Placebo | |||
• | Placebo s.c. | |||
Target Patients | Patients with active psoriatic arthritis | Patients with active ankylosing spondylitis | ||
Expected Completion | Q1-2019(actual) | Q1-2018(actual) | ||
• 16 weeks results: PANLAR congress in Apr-2016 | ||||
• Primary results: McInnes IB, et al. Lancet. | • 52 weeks results: Pavelka et al. Arthritis Research & | |||
2015;386:1137-46 | Therapy 2017 | |||
Publication | • | 2 years results: McInnes et al, Rheumatology | • 2 year results: Presented at ACR in Nov-2017 | |
2017;56:1993-2003 | • | 3 year (EOS) results: To be presented (ORAL) at | ||
• | 5 year (EOS) manuscript ongoing; to be submitted in | PANLAR April 2019 | ||
Oct-2019 | • | 3 year (EOS) manuscript submitted in May-2019; | ||
awaiting journal decision | ||||
86 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation |
Cosentyx®- Anti IL-17
Study | NCT01989468 FUTURE 3 (CAIN457F2318) | NCT02159053 MEASURE 4 (CAIN457F2320) | ||
Indication | Psoriatic arthritis | Ankylosing spondylitis | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 416 | 350 | ||
Primary Outcome | American College of Rheumatology 20 (ACR20) response in | Assessment of Spondyloarthritis International Society | ||
Measures | subjects treated with secukinumab vs. placebo | criteria / ASAS 20 at week 16 | ||
• | Secukinumab (AIN457) 150 mg s.c. | • | Secukinumab 150 mg s.c. with loading | |
Arms/Intervention | • | Secukinumab (AIN457) 300 mg s.c. | • | Secukinumab 150 mg s.c. without loading |
• | Placebo | • | Placebo | |
Target Patients | Patients with active psoriatic arthritis | Patients with active ankylosing spondylitis | ||
Expected Completion | Q2-2018(actual) | Q2-2018(actual) | ||
Publication | 52 week results: Nash et al, Arthritis Research & Therapy | • | Week 104 (EOS) manuscript: Kivitz et al, Rheumatol | |
2018, 20:47 | Ther https://doi.org/10.1007/s40744-018-0123-5 | |||
87 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT02294227 FUTURE 4 (CAIN457F2336) | NCT02404350 FUTURE 5 (CAIN457F2342) | ||
Indication | Psoriatic arthritis | Psoriatic arthritis | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 342 | 990 | ||
Primary Outcome | Assessment of American College of Rheumatology 20 | American College of Rheumatology 20 (ACR20) response at | ||
Measures | (ACR20) | Week 16 | ||
• | Secukinumab 150 mg with loading | • | Secukinumab 150 mg load | |
• | Secukinumab 150 mg no load | |||
Arms/Intervention | • | Secukinumab 150 mg without loading | ||
• | Secukinumab 300 mg load | |||
• | Placebo | |||
• | Placebo | |||
Target Patients | Patients with active psoriatic arthritis | Patients with active psoriatic arthritis | ||
Expected Completion | Q1-2018(actual) | Q1-2019(actual) | ||
• 24 week results published: Mease P, et al. Annals of the | ||||
• | 52 week results: abstract presented at PANLAR | Rheumatic Diseases 2018;77:890-897. | ||
Publication | congress (Apr-2018) | • 52 week results presented at EULAR and ACR 2018 | ||
• | 2 year (EOS) results published: Rheumatology | • | 52 week manuscript accepted (Rheumatology, October | |
Therapy. 2019 Jun 21. doi: 10.1007/s40744-019-0163-5. | 2019, in press) | |||
• | 2 year (EOS) results presented at EULAR 2019 |
88 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT01863732 (CAIN457F2305E1 - extension study) | NCT02745080 EXCEED (CAIN457F2366) | ||
Indication | Ankylosing spondylitis | Psoriatic arthritis | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 300 | 850 | ||
Primary Outcome | Assessment of spondyloarthritis international society criteria | American College of Rheumatology 20 (ACR20) response | ||
Measures | / ASAS 20 response | |||
Arms/Intervention | • | Secukinumab 75 mg in PFS | • | Secukinumab 300 mg s.c. |
• | Secukinumab 150 mg in PFS | • | Adalimumab 40 mg s.c. | |
Target Patients | Patients with active ankylosing spondylitis | Patients with active psoriatic arthritis | ||
Expected Completion | Q2-2018(actual) | Q1-2020 | ||
• 3-year results: Manuscript published in Clinical and | ||||
Experimental Rheumatology in May-2017 | ||||
• 4-year results: Presented at ACR in Nov-2017 | ||||
• 4 year results manuscript published; Rheumatology, | ||||
Publication | Volume 58, Issue 5, May 2019, Pages 859-868, | • | Manuscript target submission Jan-2020 | |
• | 5 year (EOS) results manuscript published; Baraliakos X, |
et al. RMD Open 2019;5:e001005. doi: 10.1136/rmdopen-2019-001005
89 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT02696031 PREVENT (CAIN457H2315) | NCT03259074 SURPASS (CAIN457K2340) | ||
Indication | Non-radiographic axial spondyloarthritis | Ankylosing spondylitis | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 555 | 837 | ||
Primary Outcome | The proportion of participants who achieved an ASAS 40 | No radiographic structural progression as measured by | ||
response (Assessment of SpondyloArthritis International | modified Stoke Ankylosing Spondylitis Spine Score | |||
Measures | ||||
Society criteria); | (mSASSS) | |||
• | Secukinumab 150 mg load | • | Secukinumab 150/300 mg | |
Arms/Intervention | • | Secukinumab 150 mg no load | ||
• | Adalimumab biosimilar 40 mg | |||
• | Placebo | |||
Target Patients | Patients with non-radiographic axial spondyloarthritis | Patients with active ankylosing spondylitis | ||
Expected Completion | Week 52: Q3-2019(actual); Final: H1-2021 | 2022 | ||
• Abstract (16 week results) submitted as a late breaker to | ||||
Publication | ACR 2019 | TBD | ||
• | Manuscript target submission Jan-2020 | |||
90 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT03713619 SUNSHINE (CAIN457M2301) | NCT03713632 SUNRISE (CAIN457M2302) | ||
Indication | Hidradenitis Suppurativa (HS) | Hidradenitis Suppurativa (HS) | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 471 | 471 | ||
Primary Outcome | Proportion of participants with Hidradenitis Suppurativa | Proportion of patients with Hidradenitis Suppurativa Clinical | ||
Measures | clinical response (HiSCR) | Response (HiSCR) | ||
• | Secukinumab 300 mg every 2 weeks | • | Secukinumab 300 mg every 2 weeks | |
Arms/Intervention | • | Secukinumab 300 mg every 4 weeks | • | Secukinumab 300 mg every 4 weeks |
• | Placebo (every 2 weeks) | • | Placebo (every 2 weeks) | |
• | Placebo (every 4 weeks) | • | Placebo (every 4 weeks) | |
Target Patients | Subjects with moderate to severe Hidradenitis Suppurativa | Subjects with moderate to severe Hidradenitis Suppurativa | ||
Expected Completion | H2-2021 | H2-2021 | ||
Publication | Preliminary results in EADV (most likely) in 2021 | Preliminary results in EADV (most likely) in 2021 | ||
91 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Cosentyx®- Anti IL-17
Study | NCT04156620 INVIGORATE-1 (CAIN457P12301) |
Indication | Axial spondyloarthritis |
Phase | Phase 3 |
Patients | 500 |
Primary Outcome | The proportion of subjects achieving an ASAS40 |
(Assessment of SpondyloArthritis International Society | |
Measures | |
criteria) response | |
Arms/Intervention | • Secukinumab intravenous (i.v.) regimen |
• Placebo intravenous (i.v.) regimen | |
Target Patients | Patients with active axial spondyloarthritis |
Expected Completion | 2022 |
Publication | TBD |
92 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Ilaris®- Anti IL-1β
Study | NCT02296424 (CACZ885G2306) | |
Indication | SJIA - Systemic Juvenile Idiopathic Arthritis | |
Phase | Phase 3B/4 | |
Patients | 182 | |
Proportion of patients in clinical remission on | ||
Primary Outcome | canakinumab who are able to remain in remission | |
following canakinumab dose tapering (reduced | ||
Measures | ||
canakinumab dose or prolonged canakinumab dosing | ||
interval) | ||
Arms/Intervention | • | Canakinumab dose reduction |
• | Canakinumab dose interval prolongation | |
Target Patients | Patients with Systemic Juvenile Idiopathic Arthritis (SJIA) | |
(Pediatric) | ||
Expected Completion | 2018(actual) |
• Remission & flexible dosing - presented at ISSAID &
Publication | EULAR in Q2-2019 |
• Planned manuscript in 2019: Remission & flexible | |
dosing to be submitted in Q4-2019 |
93 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
LJN452 - FXR Agonist
Study | NCT02855164 (CLJN452A2202) | NCT04065841 ELIVATE (CLJN452D12201C) | |
Indication | Non-alcoholic steatohepatitis (NASH) | Non-alcoholic steatohepatitis (NASH) | |
Phase | Phase 2 | Phase 2 | |
Patients | 345 | 210 | |
Adverse event profile of different doses; determine the dose | |||
relationship of LJN452 on markers of hepatic inflammation | Proportion of patients with resolution of NASH and no | ||
Primary Outcome | in NASH (ALT and AST); determine dose-response | worsening of fibrosis OR improvement in fibrosis by at least | |
Measures | relationship of LJN452 on liver fat content by changes in | one stage without worsening of NASH at Week 48 | |
quantitative MRI; determine effect of LJN452 on liver fibrosis | compared with baseline | ||
by biopsy | |||
• Arm A: combination therapytropifexor + licogliflozin | |||
• Arm B: tropifexor monotherapytropifexor (+ licogliflozin | |||
Arms/Intervention | • | Multiple LJN452 doses and placebo | placebo) |
• Arm C: licogliflozin monotherapylicogliflozin (+ tropifexor | |||
placebo) | |||
Target Patients | Patients with non-alcoholic steatohepatitis (NASH) | Adult patients with non-alcoholic steatohepatitis (NASH) | |
and liver fibrosis | |||
Expected Completion | Q2-2020 | 2022 | |
• Primary (interim) data abstract submitted to AASLD in | |||
Publication | Q3-2019 | TBD | |
• | Manuscript to be submitted in H2-2020 | ||
94 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
LOU064 - Bruton's tyrosine kinase (BTK) inhibitor
Study | NCT03926611 (CLOU064A2201) | NCT04109313 (CLOU064A2201E1) | ||
Indication | Chronic spontaneous urticaria (CSU) | Chronic spontaneous urticaria (CSU) | ||
Phase | Phase 2 | Phase 2 | ||
Patients | 308 | 250 | ||
Primary Outcome Measures | Change from baseline in weekly Urticaria Activity Score (UAS7) at Week | • | Long-term safety and tolerability | |
4 | ||||
• | Arm 1 Low dose of LOU064 orally in the morning (once daily) and | |||
matching placebo in the evening from Day 1 to 85 | ||||
• | Arm 2 Medium dose of LOU064 orally in the morning (once daily) and | |||
matching placebo in the evening from Day 1 to 85 | ||||
Arms/Intervention | • | Arm 3 High dose of LOU064 orally in the morning (once daily) and | • | Selected dose of LOU064 taken orally twice a day |
matching placebo in the evening from Day 1 to 85 | (morning and evening) from day 1 to week 52 | |||
• Arm 4 Low dose of LOU064 orally, twice daily from Day 1 to 85 | ||||
• Arm 5 Medium dose of LOU064 orally, twice daily from Day 1 to 85 | ||||
• Arm 6 High dose of LOU064 orally, twice daily from Day 1 to 85 | ||||
• | Placebo arm Matching placebo, orally, twice daily from Day 1 to 85 | |||
Target Patients | Adults with CSU inadequately controlled by H1-antihistamines | Patients with CSU who have participated in preceding | ||
studies with LOU064 | ||||
Expected Completion | Q3-2020 | 2022 | ||
Publication | TBD | TBD | ||
95 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
LJC242 - FXR agonist + CCR2/CCR5 inhibitor
Study | NCT03517540 TANDEM (CLJC242A2201J) |
Indication | Non-alcoholic steatohepatitis |
Phase | Phase 2 |
Patients | 200 |
Primary Outcome | • Evaluation of safety and tolerability of combination |
Measures | therapy (tropifexor + cenicriviroc) by monitoring adverse |
event profile, vital signs and laboratory parameters | |
Arms/Intervention
Target Patients
Expected Completion
Publication
- Tropifexor
- Cenicriviroc
- Tropifexor + cenicriviroc
Adult patients with non-alcoholic steatohepatitis (NASH) and liver fibrosis
Q4-2020
Manuscript to be submitted in H1-2021
96 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
QGE031 - Anti-IgE
Study | NCT02477332 (CQGE031C2201) | NCT02649218 (CQGE031C2201E1) | |
Indication | Chronic spontaneous urticaria / Chronic idiopathic urticaria | Chronic spontaneous urticaria / Chronic idiopathic urticaria | |
Phase | Phase 2B | Phase 2B | |
Patients | 382 | 226 | |
Primary Outcome | Establish dose-response relationship of QGE031 with | Long-term safety; number of participants with treatment- | |
respect to achievement of complete hives response at week | |||
Measures | emergent adverse events | ||
12 | |||
• | Ligelizumab 24mg q4wks for 20 weeks | ||
• | Ligelizumab 72mg q4wks for 20 weeks | ||
Arms/Intervention | • | Ligelizumab 240mg q4wks for 20 weeks | Ligelizumab 240 mg q4wks open label for 52 weeks |
• Ligelizumab 120mg single dose | |||
• | Omalizumab 300mg q4wks for 20 weeks | ||
• | Placebo q 4wks for 20 weeks | ||
Adult patients with chronic spontaneous urticaria | Adult patients with chronic spontaneous urticaria | ||
Target Patients | inadequately controlled with H1-antihistamines at approved | inadequately controlled with H1-antihistamines at approved | |
or increased doses, alone or in combination with H2- | or increased doses, alone or in combination with H2- | ||
antihistamines or leukotriene receptor antagonists. | antihistamines or leukotriene receptor antagonists. | ||
Expected Completion | 2017(actual) | Q3-2019(actual) | |
• Primary results: Presented at EAACI 2018, EADV 2018, | • Primary results: AAD 2019; | ||
Publication | and GUF 2018; NEJM publication (Oct. 3rd); | • Secondary results presented in 2019 at: AAD, EAACI, | |
• Secondary results presented in 2019 at: AAD, EAACI, | WCD, EADV, PAAM, ACAAI, UCARE; manuscript | ||
WCD, EADV, PAAM, ACAAI, UCARE. | planned in H1/2020 | ||
97 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
QGE031 - Anti-IgE
Study | NCT03437278 (CQGE031C2202) | |
Indication | Chronic spontaneous urticarial / Chronic idiopathic urticaria | |
Phase | Phase 2 | |
Patients | 48 | |
Primary Outcome | Change in the 7 day Urticaria Activity Score (UAS7) | |
Measures | ||
• Ligelizumab high dose q4wks for 24 weeks | ||
Arms/Intervention | • | Ligelizumab low dose q4wks for 24 weeks |
• | Placebo / ligelizumab high dose q4wks for 8 / 16 weeks |
Target Patients
Expected Completion
Adolescents from 12 to <18 years of age, with chronic spontaneous urticaria
H2-2021
• Study design was presented at PAAM (Peds Allergy & | ||
Asthma Meeting) and at UCARE meeting 2019 | ||
Publication | • | Primary results to be presented in 2022 (e.g. EAACI, |
PAAM, EADV) | ||
• | Manuscript to be submitted in 2022 | |
98 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
QGE031 - Anti-IgE
Study | NCT03580369 Pearl 1 (CQGE031C2302) | NCT03580356 Pearl 2 (CQGE031C2303) |
Indication | Chronic spontaneous urticaria | Chronic spontaneous urticaria |
Phase | Phase 3 | Phase 3 |
Patients | 1,050 | 1,050 |
Primary Outcome | Absolute change from baseline in UAS7 (Urticaria Activity | Absolute change from baseline in UAS7 (Urticaria Activity | ||
Measures | Score) at week 12 | Score) at week 12 | ||
• | Ligelizumab dose A q4w for 52 weeks | • | Ligelizumab dose A q4w for 52 weeks | |
• | Ligelizumab dose B q4w for 52 weeks | • | Ligelizumab dose B q4w for 52 weeks | |
Arms/Intervention | • | Omalizumab 300 mg q4w for 52 weeks | • | Omalizumab 300 mg q4w for 52 weeks |
• | Placebo q4w from randomization to wk20, then | • | Placebo q4w from randomization to wk20, then | |
ligelizumab dose B from wk24 to wk52 | ligelizumab dose B from wk24 to wk52 | |||
Target Patients | Adolescents and adults with chronic spontaneous urticaria | Adolescents and adults with chronic spontaneous urticaria | ||
inadequately controlled with H1-antihistamines | inadequately controlled with H1-antihistamines | |||
Expected Completion | H2-2021 | H2-2021 | ||
• Study design presented at UCARE 2018 | ||||
Publication | • | Primary results to be presented in 2022 (e.g. EAACI, PAAM, EADV) | ||
• | Manuscript to be submitted in 2022 | |||
99 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
VAY736 - Fully human IgG1/κ anti-BAFF-R mAb
Study | NCT02962895 (CVAY736A2201) | NCT03217422 AMBER (CVAY736B2201) |
Indication | Primary Sjögren's syndrome | Autoimmune hepatitis |
Phase | Phase 2B | Phase 2/3 |
Patients | 180 | 80 |
Primary Outcome | Safety and efficacy of VAY736 in primary Sjögren's | Alanine aminotransferase (ALT) normalization | |
Measures | syndrome (pSS) | ||
Arms/Intervention | • | VAY736 | • VAY736 |
• | Placebo | • Placebo control with conversion to active VAY736 | |
Target Patients | Patients with moderate to severe primary Sjögren's | Autoimmune hepatitis patients with incomplete response or | |
syndrome (pSS) | intolerant to standard treatment of care | ||
Expected Completion | Q2-2020 | 2023 | |
• Late Breaking Abstract to be submitted to American | |||
Publication | College of Rheumatology 30-Sep-2019 | TBD | |
• | Manuscript to be submitted in 2020 | ||
100 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
ZPL389 - H4 receptor antagonist
Study | NCT03517566 ZEST (CZPL389A2203) | NCT03948334 ZESTExt (CZPL389A2203E1 - extension | ||
study) | ||||
Indication | Atopic dermatitis | Atopic dermatitis | ||
Phase | Phase 2 | Phase 2 | ||
Patients | 360 | 360 | ||
Primary Outcome | IGA (Investigator's global assessment) response at week 16 | Frequency of Adverse Events (AEs) and Serious Adverse | ||
Measures | Events (SAEs) | |||
• | ZPL389 dose 1 | • | ZPL389 Dose 1 + Topical Corticosteroids (TCS) and /or | |
• | ZPL389 dose 2 | Topical Calcineurin Inhibitors (TCI) | ||
Arms/Intervention | • | ZPL389 dose 3 | • | ZPL389 Dose 2 + Topical Corticosteroids (TCS) and /or |
• | ZPL389 dose 4 | Topical Calcineurin Inhibitors (TCI) | ||
• | Placebo | |||
Target Patients | Patients with moderate to severe atopic dermatitis | Adult patients with atopic dermatitis | ||
Expected Completion | H1-2021 | 2023 | ||
Publication | TBD | TBD | ||
101 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Neuroscience
Zolgensma®- SMN1 gene replacement therapy
Study | NCT03461289 STRIVE-EU(CL-302) | NCT03306277 STRIVE (CL-303) |
Indication | Type 1 spinal muscular atrophy | Type 1 spinal muscular atrophy |
Phase | Phase 3 | Phase 3 |
Patients | 30 | 20 |
Primary Outcome | • Achievement of independent sitting for at least 30 | |
Proportion of participants sitting without support | seconds | |
Measures | ||
• Event-free survival | ||
Arms/Intervention | Open-label,single-arm,single-dose, intravenous | Open-label,single-arm,single-dose, intravenous |
Target Patients | Patients with spinal muscular atrophy Type 1 | Patients with Spinal Muscular Atrophy Type 1 |
Expected Completion | H2-2020 | Q4-2019(actual) |
Publication | TBD | TBD |
103 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Zolgensma®- SMN1 gene replacement therapy
Study | NCT03505099 SPR1NT (CL-304) | NCT03837184 STRIVE Asia Pacific (CL-306) |
Indication | Spinal muscular atrophy | Type 1 spinal muscular atrophy |
Phase | Phase 3 | Phase 3 |
Patients | 27 | 6 |
• Percentage of participants achieving functional | ||
Primary Outcome | independent sitting for at least 30 seconds at any visit | Proportion of participants sitting without support |
Measures | • Percentage of participants achieving the ability to stand | |
without support for at least 3 seconds at any visit | ||
Arms/Intervention | Open-label,single-arm,single-dose, intravenous | Open-label,single-arm,single-dose, intravenous |
Target Patients | Pre-symptomatic patients with spinal muscular atrophy and | Patients with spinal muscular atrophy Type 1 |
multiple copies SMN2 | ||
Expected Completion | H2-2021 | H2-2021 |
Publication | TBD | TBD |
104 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Zolgensma®- SMN1 gene replacement therapy
Study | NCT03381729 STRONG (CL-102) |
Indication | Type 2 spinal muscular atrophy |
Phase | Phase 1 |
Patients | 27 |
Primary Outcome | • | Safety and tolerability, incidence of adverse events |
• | Proportion of patients achieving Standing Milestone | |
Measures | ||
• Change in Hammersmith Functional Motor Scale | ||
Arms/Intervention | Open-label,single-arm,single-dose, intrathecal | |
Target Patients | Patients with spinal muscular atrophy with 3 copies of SMN2 | |
Expected Completion | Q4-2019 [Cohort B] | |
Publication | TBD | |
105 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Aimovig®- CGRP receptor antagonist
Study | NCT03096834 LIBERTY (CAMG334A2301) | NCT03333109 EMPOWER (CAMG334A2302) | ||
Indication | Migraine | Migraine | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 246 | 900 | ||
Primary Outcome | Percentage of patients with a 50% response in the reduction | Change from baseline in monthly migraine days at the last | ||
Measures | of Monthly Migraine Days (MMD) | month (Month 3) of the double-blind treatment period | ||
• | Subcutaneous injection of AMG334 (erenumab) | • | AMG334 (erenumab) Dose 1 | |
Arms/Intervention | • | AMG334 (erenumab) Dose 2 | ||
• | Subcutaneous injection of placebo | |||
• | Placebo | |||
Target Patients | Adult episodic migraine patients who have failed prophylactic | Adult episodic migraine patients | ||
migraine treatments | ||||
Expected Completion | 2017 DBT phase (actual); H1-2021 OLE phase (final DBL) | Q2-2020 | ||
• Planned for Q1-2020 (Neurology): PROs and | ||||
prespecified subgroup analysis (DBT phase) | ||||
Publication | • Planned for Q2-2020: 1Y OLE | Planned for H2-2020 | ||
• Planned for Q4 2020: 2Y OLE - TBC. Potentially | ||||
abstract only | ||||
106 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Aimovig®- CGRP receptor antagonist
Study | NCT03867201 DRAGON (CAMG334A2304) |
Indication | Migraine |
Phase | Phase 3 |
Patients | 550 |
Primary Outcome | Change from baseline in monthly migraine days during the |
Measures | last 4 weeks of the 12-week treatment period |
Arms/Intervention | • Subcutaneous injection of AMG334 (erenumab) 70 mg |
• Subcutaneous injection of placebo | |
Target Patients | Adult chronic migraine patients |
Expected Completion | 2022 DBT phase; 2024 OLE phase |
Publication | Planned in Q4-2023 (DBT) |
107 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Gilenya®- S1P-R modulator
Study | NCT01633112 ASSESS (CFTY720D2312) | NCT01201356 LONGTERMS (CFTY720D2399) | |
Indication | Relapsing remitting multiple sclerosis (RRMS) | Relapsing multiple sclerosis (RMS) | |
Phase | Phase 3B | Phase 3 | |
Patients | 1,064 | 4,125 | |
Primary Outcome | Comparison of 2 doses (0.25 mg and 0.5 mg) of fingolimod | ||
to glatiramer acetate (20 mg) in reducing the annualized | Long-term safety and tolerability | ||
Measures | |||
relapse rate up to 12 months | |||
• Fingolimod 0.5 mg orally | |||
Arms/Intervention | • | Fingolimod 0.25mg orally | Single-arm study of fingolimod 0.5 mg/day |
• Copaxone®20 mg s.c. | |||
Target Patients | Patients with relapsing-remitting multiple sclerosis | Patients with relapsing multiple sclerosis | |
Expected Completion | 2018(actual) | 2018(actual) | |
• Cohen J et al. Extended treatment with fingolimod for | |||
• Primary data presentation at AAN in 2019 | relapsing multiple sclerosis: the 14-year LONGTERMS | ||
Publication | study results (Therapeutic Advances in Neurological | ||
• | Primary manuscript - submission planned in Q4-2019 | ||
Disorders 2019.12:eCollection 2019, doi: | |||
10.1177/1756286419878324) |
108 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
LMI070 - SMN2 RNA splice modulator
Study | NCT02268552 (CLMI070X2201) |
Indication | Type 1 spinal muscular atrophy |
Phase | Phase 1/2 |
Patients | 39 |
Primary Outcome | Number of participants with adverse events (AEs), serious |
Measures | adverse events (SAEs) and deaths |
Branaplam oral, once weekly: | |
• Part 1: 5 ascending doses | |
Arms/Intervention | • Part 2: 2 different dose levels |
• Part 3: patients continue on initial dose assigned in Part | |
1 or Part 2 | |
Target Patients | Patients with type 1 spinal muscular atrophy |
Expected Completion | Q3-2020 |
Publication | TBD |
109 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Mayzent ®- S1P-R modulator
Study | NCT01665144 -EXPAND (CBAF312A2304) |
Indication | Secondary progressive multiple sclerosis |
Phase | Phase 3 |
Patients | 1,652 |
Primary Outcome Measures | The delay in time to confirmed disability progression as |
measured by EDSS (Expanded Disability Status Scale) | |
Arms/Intervention
Target Patients
Expected Completion
Publication
-
BAF312(5-day titration: 0.25mg to 1.25mg; Maintenance
dose: 2mg (day 6)) - Placebo
Patients with secondary progressive multiple sclerosis
Core in 2016/Extension in 2023
The Lancet Neurology, Volume 39, No.10127, p1237-1330, March 2018
110 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
OMB157 - Anti-CD20
Study | NCT02792218 Asclepios I (COMB157G2301) | NCT02792231 Asclepios II (COMB157G2302) |
Indication | Multiple sclerosis | Multiple sclerosis |
Phase | Phase 3 | Phase 3 |
Patients | 900 | 900 |
Primary Outcome | Annualized Relapse Rate (ARR) - number of confirmed | Annualized Relapse Rate (ARR) - number of confirmed | ||
relapses in a year calculated based on cumulative number | relapses in a year calculated based on cumulative number | |||
Measures | ||||
of relapses by patient adjusted for time-in-study by patient | of relapses by patient adjusted for time-in-study by patient | |||
Arms/Intervention | • | Ofatumumab subcutaneous | • | Ofatumumab subcutaneous |
• | Teriflunomide oral | • | Teriflunomide oral | |
Target Patients | Patients with relapsing forms of multiple sclerosis | Patients with relapsing forms of multiple sclerosis | ||
Expected Completion | Q3-2019(actual) | Q3-2019(actual) | ||
Publication | Primary manuscript planned in H1-2020 | Primary manuscript planned in H1-2020 | ||
111 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
OMB157 - Anti-CD20
Study | NCT03249714 APOLITOS (COMB157G1301) | NCT03650114 ALITHIOS (COMB157G2399) |
Indication | Multiple sclerosis | Multiple Sclerosis |
Phase | Phase 2 | Phase 3 |
Patients | 60 | 2010 |
Primary Outcome | Reduced cumulative number of Gd-enhanced T1 lesions | Evaluate the long-term safety and tolerability of ofatumumab | |
across 4 MRI scans at week 12, 16, 20 and 24 (ofatumumab | 20 mg subcutaneous (sc) once every 4 (q4) weeks in | ||
Measures | |||
vs placebo) | subjects with RMS from the first dose of ofatumumab | ||
Arms/Intervention | • | Ofatumumab 20 mg subcutaneous injections | • Ofatumumab 20 mg every 4 weeks |
• | Placebo | ||
Target Patients | Patients with relapsing forms of multiple sclerosis | Patients with relapsing MS | |
Expected Completion | Q1-2020 | 2025 | |
Publication | TBD | TBD | |
112 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Oncology
ABL001 - Specific, allosteric Bcr-Abl kinase inhibitor
Study | NCT03106779 ASCEMBL (CABL001A2301) |
Indication
Phase
Patients
Primary Outcome Measures
Arms/Intervention
Target Patients
Expected Completion
Publication
Chronic myeloid leukaemia (CML)
Phase 3
233
Major Molecular Response (MMR) rate at 24 weeks
- ABL001 40 mg bid
- Bosutinib 500 mg
Patients with chronic myelogenous leukemia in chronic phase, previously treated with 2 or more tyrosine kinase inhibitors
H2-2020
Manuscript submission in H2-2020 (journal TBD)
114 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
ACZ885 - IL-1β inhibitor
Study | NCT03447769 CANOPY-A (CACZ885T2301) | NCT03631199 CANOPY-1 (CACZ885U2301) |
Indication | Adjuvant NSCLC | 1stLine Non-small cell lung cancer (NSCLC) |
Phase | Phase 3 | Phase 3 |
Patients | 1,500 | 627 |
Primary Outcome Measures
Arms/Intervention
Target Patients
Expected Completion
Publication
• Safety run-in part: Incidence of dose limiting toxicities | |||
Disease free survival (primary), overall survival (key | • | Double-blind, randomized, placebo-controlled part: | |
secondary) | Progression free survival (PFS) | ||
• | Overall survival (OS) | ||
• | Canakinumab 200mg q3w sc for 18 cycles | • | Canakinumab or matching placebo in combination with |
pembrolizumab and platinum-based doublet | |||
• | Placebo q3w sc for 18 cycles | ||
chemotherapy | |||
Patients with: | Patients with | ||
• | High-risk NSCLC (AJCC/UICC v.8 stage II-IIIA and IIIB | • | Histologically confirmed Stage IIIB, IV NSCLC with no |
(T>5cm N2)) after complete resection and standard of | prior systemic anticancer therapy | ||
care adjuvant cisplatin-based chemotherapy | • | Squamous and non-squamous NSCLC | |
• | All histologies | • No EGFR mutation and ALK rearrangement | |
2022 | H1-2021 | ||
TBD | Johnson B et al. Abstract accepted for presentation at | ||
AACR-NCI-EORTC Oct 2019 (safety run-in) | |||
115 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
ACZ885 - IL1β inhibitor
Study | NCT03626545 CANOPY-2 (CACZ885V2301) |
Indication
Phase
Patients
Primary Outcome Measures
Arms/Intervention
Target Patients
Expected Completion
Publication
2nd/ 3rdLine Non-small cell lung cancer (NSCLC)
Phase 3
240
- Safetyrun-in part: Incidence of dose limiting toxicities
- Double-blind,randomized, placebo-controlled part: Overall Survival
- canakinumab in combination with docetaxel
- canakinumabmatching-placebo in combination with docetaxel
Patients with:
- Stage IIIB or IV NSCLCwithout EGFR, ALK,ROS-1 or B- RAF mutation
- Previously treated with platinum therapy and PD(L)1- inhibitor
H1-2021
TBD
116 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
BYL719 - Alpha-specific PI3K inhibitor
Study | NCT02437318 SOLAR-1 (CBYL719C2301) |
Indication | HR+/HER2- advanced breast cancer with PIK3CA mutation |
Phase | Phase 3 |
Patients | 572 |
Primary Outcome | Progression-free survival (PFS) for patients with PIK3CA |
Measures | mutant status |
Arms/Intervention | • Fulvestrant 500 mg + alpelisib 300 mg |
• Fulvestrant 500 mg + placebo | |
Men and postmenopausal women with hormone receptor | |
Target Patients | positive, HER2-negative advanced breast cancer which |
progressed on or after aromatase inhibitor treatment | |
Expected Completion | Q3-2018(actual) |
• Andre F, et al. Presentation at ESMO 2018 | |
Publication | • Andre et al. Manuscript N Engl J Med 2019;380:1929- |
1940. | |
117 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Exjade®- Iron chelation of bis-hydroxy-phenyl triazole type
Study | NCT00940602 TELESTO (CICL670A2302) | |
Indication | Iron overload | |
Phase | Phase 2 | |
Patients | 224 | |
Primary Outcome | To compare deferasirox to placebo with regard to event-free | |
survival in low and int-1 risk MDS patient with transfusional | ||
Measures | ||
iron overload | ||
Arms/Intervention | • | Deferasirox, iron chelator |
• | Placebo | |
Target Patients | Patients with myelodysplastic syndromes (low/int-1 risk) and | |
transfusional iron overload | ||
Expected Completion | Q3-2018(actual) | |
Publication | • | Angelucci E, et al. Presentation at ASH 2018 |
• | Angelucci E, et al. Manuscript submitted Q3-2019 | |
118 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
INC280 - MET Inhibitor
Study | NCT02414139 (CINC280A2201) | NCT03647488 (CINC280D2201) | ||
Indication | EGFR Wild-type, ALK negative advanced Non-small Cell | Non-small cell lung cancer | ||
Lung Cancer (NSCLC) | ||||
Phase | Phase 2 | Phase 2 | ||
Patients | 364 | 105 | ||
Primary Outcome | Run in part: Assess safety and tolerability of capmatinib and | |||
Overall Response Rate (ORR) | spartalizumab combination. | |||
Measures | ||||
Randomized part: Overall Survival (OS) | ||||
• | Pre-treated pts. with MET GCN: ≥ 6; ≥ 4 and < 6; < 4 | |||
• Pre-treated pts. with MET mutations regardless of | ||||
cMET GCN as second or third line | • | Capmatinib plus spartalizumab | ||
Arms/Intervention | • | Treatment-naïve pts. with MET dysregulation | ||
• | Docetaxel | |||
• Pre-treated pts with MET dysregulation - second line | ||||
• Treatment-naïve pts with cMET mutations regardless of | ||||
cMET GCN | ||||
Pre-treated adult patients with EGFR wild-type ALK | ||||
Adult patients with EGFR wild-type (wt), ALK-negative | rearrangement negative advanced/metastatic non-small cell | |||
Target Patients | advanced/ metastatic NSCLC with either MET | lung cancer, that has demonstrated progression following one | ||
amplification or MET mutations | prior platinum doublet and one prior PD-(L)1 checkpoint | |||
inhibitor | ||||
Expected Completion | Q2-2019(actual) | 2022 | ||
Publication | • | Wolf J, et al. Presented at ASCO 2019 | TBD | |
• | Wolf J, et al. Manuscript submitted Q3-2019 | |||
119 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Jakavi®- JAK1/2 inhibitor
Study | NCT02913261 REACH2 (CINC424C2301) | NCT03112603 REACH3 (CINC424D2301) | ||
Indication | Steroid-refractory acute graft vs. host disease (SR aGVHD) | Steroid-refractory chronic graft vs. host disease (SR cGVHD) | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 308 | 324 | ||
Primary Outcome | Overall Response Rate (ORR) at 28 Days | Overall Response Rate (ORR) at 183 Days | ||
Measures | ||||
Arms/Intervention | • | Ruxolitinib 10mg bid | • | Ruxolitinib 10mg bid |
• | Best available therapy (BAT) | • | Best available therapy (BAT) | |
Target Patients | Patients with SR aGVHD | Patients with SR cGVHD | ||
Expected Completion | Q3-2019(actual) | Interim Analysis: Q3-2019(actual); Final:Q3-2020 | ||
Publication | • | Manuscript submission in Q4-2019 | • | Manuscript submission in H2-2020 |
• | Zeiser R, et al. Abstract submitted Q4-2019 | • | Abstract submission to congress in H2-2020 | |
120 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Jakavi®- JAK1/2 inhibitor
Study | NCT03491215 REACH4 (CINC424F12201) | NCT04097821 ADORE (CINC424H12201) |
Indication | Acute graft versus host disease | Myelofibrosis |
Phase | Phase 2 | Phase 1/2 |
Patients | 45 | 130 |
Primary Outcome Measures
Arms/Intervention
Target Patients
Expected Completion
Publication
• Measurement of PK parameters | • Incidence of dose limiting toxicities within the first 2 | |
cycles | ||
• Overall Response Rate (ORR) | ||
• Response rate at the end of cycle 6 | ||
• | Ruxolitinib | |
• Ruxolitinib | • | Ruxolitinib+Siremadlin |
• | Ruxolitinib+Crizanlizumab | |
• | Ruxolitinib+MBG453 | |
Pediatric patients with grade II-IV acute graft vs. host disease | Patients with Myelofibrosis (MF) | |
after allogeneic hematopoietic stem cell transplantation | ||
2023 | 2024 | |
TBD | TBD |
121 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Kisqali®- CDK 4/6 inhibitor
Study | NCT03701334 NATALEE (CLEE011O12301C) | |
Indication | Adjuvant treatment of hormone receptor (HR)-positive, | |
HER2-negative, early breast cancer (EBC) | ||
Phase | Phase 3 | |
Patients | ~4,000 | |
Primary Outcome | Invasive Disease-Free Survival for using STEEP criteria | |
(Standardized Definitions for Efficacy End Points in adjuvant | ||
Measures | ||
breast cancer trials) | ||
Arms/Intervention | • | Ribociclib + endocrine therapy |
• | Endocrine therapy | |
Pre and postmenopausal women and men with HR-positive, | ||
Target Patients | HER2-negative EBC, after adequate surgical resection, who | |
are eligible for adjuvant endocrine therapy | ||
Expected Completion | Interim Analysis: H1-2021;Final: 2026 | |
Publication | TBD | |
122 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Kymriah®- CAR-T therapy
Study | NCT02445248 JULIET (CCTL019C2201) | NCT03568461 ELARA (CCTL019E2202) |
Indication | Relapsed / refractory DLBCL | Relapsed / refractory follicular lymphoma (FL) |
Phase | Phase 2 | Phase 2 |
Patients | 128 | 113 |
Primary Outcome Measures
Arms/Intervention
Target Patients
Expected Completion
Publication
Overall response rate; efficacy and safety of CTL019 | Complete Response Rate (CRR) |
Single-arm study of single dose of CTL019 | Single-arm study of tisagenlecleucel |
Adult patients with relapsed or refractory diffuse large B-cell | Adult patients with relapsed or refractory FL |
lymphoma (DLBCL) | |
2017(actual) | Interim Analysis: Q3-2020 |
- Schuster et al. Presentations at ICML 2017; at EHA 2017; at ASH 2017; at ASH 2018; Borchmann et al.
Presentation at EHA 2018; Bachanova et al. | TBD |
Presentation at ICML 2019 | |
- Schuster et al. N Engl J Med.2019;380(1):45-56. doi: 10.1056/NEJMoa1804980. Epub 2018 Dec 1.
123 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Kymriah®- CAR-T therapy
Study | NCT03876769 CASSIOPEIA (CCTL019G2201J) | NCT03570892 BELINDA (CCTL019H2301) |
Indication | 1stline high risk acute lymphoblastic leukemia (ALL) | 2ndline Diffuse large B-cell lymphoma (DLBCL) |
Phase | Phase 2 | Phase 3 |
Patients | 160 | 318 |
Primary Outcome Measures
Arms/Intervention
Target Patients
Expected Completion
5 year Disease Free Survival (DFS) | Event-free Survival (EFS) |
Single-arm study of tisagenlecleucel; retreatment allowed | Tisagenlecleucel versus standard of care |
Adult patients with aggressive B-cellNon-Hodgkin | |
Pediatric and young adult patients with 1stline high risk ALL | Lymphoma after failure of rituximab and anthracycline- |
containing frontline immunochemotherapy | |
2025 | H2-2021 |
Publication | TBD | TBD |
124 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
MBG453 - TIM-3 antagonist
Study | NCT03946670 STIMULUS MDS-1 (CMBG453B12201) |
Indication | Myelodysplastic syndrome |
Phase | Phase 2 |
Patients | 120 |
Primary Outcome | Complete Remission (CR) rate and Progression Free |
Measures | Survival (PFS) |
Arms/Intervention | • Experimental: MBG453 + hypomethylating agents |
• Placebo comparator: Placebo + hypomethylating agents | |
Target Patients | Adult subjects with intermediate, high or very high risk |
Myelodysplastic Syndrome (MDS) as per IPSS-R criteria | |
Expected Completion | H2-2021 |
Publication | TBD |
125 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
PDR001 - PD-1 checkpoint inhibitor
Study | NCT02967692 COMBI-i (CPDR001F2301) |
Indication
Phase
Patients
Primary Outcome Measures
Arms/Intervention
Target Patients
Expected Completion
Publication
BRAFV600 mutant metastatic melanoma
Phase 3
538
Part 1 (safety-run in): 9; Part 2 (biomarker cohort): 27; Part 3
(Phase III, randomized, placebo controlled): 532
Progression-Free Survival (PFS)
- Spartalizumab 400mg i.v. Q4W + Tafinlar 150mg bid + Mekinist 2 mg
- Placebo + Tafinlar 150 mg bid + Mekinist 2 mg
Previously untreated patients with unresectable or metastatic BRAF V600 mutant melanoma
Q3-2020
Abstract submission to congress in H2-2020
126 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Rydapt®- Multi-targeted kinase inhibitor
Study | NCT03280030 (CPKC412A2220) | NCT03591510 (CPKC412A2218) |
Indication | Acute myeloid leukemia | Acute myeloid leukemia |
Phase | Phase 2 | Phase 2 |
Patients | 66 | 50 |
Primary Outcome | Incidence of safety events and event free survival | Occurrence of dose limiting toxicities |
Measures | Event Free Survival ( EFS) | |
Arms/Intervention
Target Patients
Expected Completion
• | Midostaurin 50 mg | • Chemotherapy followed by Midostaurin |
• | Placebo | |
Newly diagnosed patients with FLT3-mutated acute myeloid | Newly diagnosed pediatric patients with FLT3 mutated acute | |
leukemia (AML) from pan-Asia countries | myeloid leukemia (AML) | |
H1-2020 | H2-2022 | |
Publication | Abstract submission to congress in H2-2020 | TBD |
127 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
PDR001 - PD-1 checkpoint inhibitor
Study | NCT03484923 (CPDR001J2201) | |
Indication | Previously treated unresectable or metastatic melanoma | |
Phase | Phase 2 | |
Patients | 230 | |
Primary Outcome | ||
Objective Response Rate (ORR) | ||
Measures | ||
• | PDR001 400mg i.v. Q4W + LAG525 600 mg i.v. Q4W | |
• | PDR001 400mg i.v. Q4W + capmatinib 400 mg bid orally | |
Arms/Intervention | • | PDR001 400mg i.v. Q4W + canakinumab 300 mg (s.c) |
Q4W | ||
• PDR001 400mg i.v. Q4W + ribociclib 600 mg p.o QD on Days 1 to 21 of a 28-day cycle
Target Patients
Expected Completion
Publication
Adult patients with previously treated unresectable or metastatic melanoma
H2-2021
TBD
128 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Promacta®/Revolade®- Thrombopoetin receptor agonist
Study | NCT03025698 (CETB115E2201) | NCT03988608 (CETB115E2202) |
Indication | Previously untreated or relapsed/refractory severe aplastic | Previously untreated or relapsed/refractory severe aplastic |
anemia or recurrent aplastic anemia | anemia or recurrent aplastic anemia | |
Phase | Phase 2 | Phase 2 |
Patients | 60 | 20 |
Primary Outcome | PK of eltrombopag at steady state in pediatric patients with | Hematologic response rate |
Measures | SAA | |
Arms/Intervention
Target Patients
Expected Completion
- Eltrombopag 12.5, 25, 50, 75 mg FCT & 25 mg pFOS
- Arm B: previously untreatedSAA-hATG/cyclosporine +
eltrombopag | • Eltrombopag 25 mg film-coated tablets |
• Arm A: relapsed/refractory SAA or AA: | |
hATG/cyclosporine + eltrombopag or cyclosporine + | |
eltrombopag | |
Pediatric patients from age 1 <18 years with | Chinese patients with refractory or relapsed severe aplastic |
relapsed/refractory SAA or recurrent AA after IST or | |
anemia | |
previously untreated SAA | |
2025 | 2023 |
Publication | TBD | TBD |
129 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
SEG101 - p-Selectin inhibitor
Study | NCT03264989 SOLACE-Adults (CSEG101A2202) | NCT03474965 SOLACE-Kids (CSEG101B2201) |
Indication | Prevention of Vaso-Occlusive Crises (VOC) in patients with | Prevention of VOC in pediatric patients with SCD |
Sickle Cell Disease (SCD) | ||
Phase | Phase 2 | Phase 2 |
Patients | 55 | 100 |
Primary Outcome | PK/PD and safety of SEG101 (crizanlizumab) at 5 mg/kg | PK/PD and safety of SEG101 at 5 mg/kg |
Measures | ||
SEG101 (crizanlizumab) at a dose of 5.0 mg/kg (or 7.5 | SEG101 (crizanlizumab) at a dose of 5 mg/kg by IV infusion | |
Arms/Intervention | mg/kg for exploratory group) by IV infusion, ± | |
± Hydroxyurea/Hydroxycarbamide | ||
Hydroxyurea/Hydroxycarbamide | ||
Target Patients | Adult SCD patients with VOC | Pediatric SCD patients with VOC |
Expected Completion | Q4-2018(actual) | H2-2021 (pediatric patients ≥6 year old) |
2022 (pediatric patients 6 months - 6 year old) | ||
Publication | Abstract submission to congress in H1-2020 | TBD |
130 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
SEG101 - p-Selectin inhibitor
Study | NCT03814746 STAND (CSEG101A2301) |
Indication | Prevention of Vaso-Occlusive Crises (VOC) in patients with |
Sickle Cell Disease (SCD) | |
Phase | Phase 3 |
Patients | 240 |
Primary Outcome | Rate of VOC events leading to healthcare visit |
Measures | |
Arms/Intervention
Target Patients
Expected Completion
Publication
- Crizanlizumab 5.0 mg/kg
- Crizanlizumab 7.5 mg/kg
- Placebo
Adolescent and adult SCD patients (12 years and older)
H1-2022
TBD
131 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Tafinlar®- BRAF inhibitor
Study | NCT01677741 (CDRB436A2102) |
Indication | BRAFV600 mutant cancers |
Phase | Phase 1/2 |
Patients | 85 |
Primary Outcome | Safety, tolerability and pharmacokinetics |
Measures | |
Arms/Intervention | Single-arm study of oral dabrafenib (dose based on age |
and weight) | |
Target Patients | Pediatric subjects aged 1 year to <18 years with advanced |
BRAF V600-mutation positive solid tumors | |
Expected Completion | Q3-2020 |
• Kieran MW et al. Manuscript Clin Cancer Res; (accepted | |
Q3-2019, not yet published) (PK analysis) | |
Publication | • Hargrave D et al. Manuscript Clin Cancer Res; (accepted |
Q3-2019, not yet published) (safety/efficacy in low-grade | |
gliomas) | |
132 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Tafinlar®+Mekinist®- BRAF inhibitor and MEK inhibitor
Study | NCT02684058 (CDRB436G2201) |
Indication | BRAFV600 mutant gliomas |
Phase | Phase 2 |
Patients | 142 |
Primary Outcome | |
Objective response rate | |
Measures | |
Arms/Intervention | Dabrafenib + trametinib (dose based on age and weight) |
Children and adolescent patients with BRAF V600 mutation | |
Target Patients | positive relapsed or refractory high grade glioma (HGG) or |
BRAF V600 mutation positive low grade glioma (LGG) | |
Expected Completion | H2-2021 |
Publication | TBD |
133 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Tafinlar®+Mekinist®- BRAFV600 inhibitor and MEK inhibitor
Study | NCT02124772 (CTMT212X2101) |
Indication | BRAFV600 mutant solid tumors |
Phase | Phase 1/2A |
Patients | 142 |
Primary Outcome | |
Safety, tolerability and pharmacokinetics and clinical activity | |
Measures | |
Arms/Intervention | Trametinib (dose based on age and weight) |
Dabrafenib + trametinib (dose based on age and weight) | |
Target Patients | Pediatric Subjects Aged 1 Month to <18 Years with |
Advanced V600-Mutation Positive Solid Tumors | |
Expected Completion | H1-2021 |
Publication | Abstract submission to congress in H1-2020 |
134 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Zykadia®- ALK inhibitor
Study | NCT02299505 ASCEND-8 (CLDK378A2112) |
Indication | ALK activated NSCLC |
Phase | Phase 2 |
Patients | 306 |
Primary Outcome | Part 1: Pharmacokinetics when taken with food |
Measures | Part 2: Overall Response Rate (ORR) when taken with food |
• Oral LDK378 450 mg once daily taken with food | |
Arms/Intervention | • Oral LDK378 600 mg once daily taken with food |
• Oral LDK378 750 mg once daily fasted | |
Target Patients | Adult patients with ALK-rearranged(ALK-positive) advanced non-small cell |
lung cancer | |
Part 1 (PK): 2016 (actual) | |
Expected Completion | Part 2 (ORR): Q2-2018(actual) |
Final (ORR): Q4-2019 | |
• Part 1 (PK): Cho BC, et al. J Thorac Oncol. 2017 Sep; 12(9) 1357-1367 | |
Publication | • Part 2 (ORR): Cho B et al. J Thorac Oncol. 2019 Jul; 14(7) 1255-1265 |
• Final (ORR): TBD | |
135 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
177Lu-PSMA-617 - Lu-labelled prostate specific membrane antigen (PSMA)
Study | NCT03511664 VISION (PSMA-617-01) |
Indication | PSMA-positive Metastatic Castration-resistant Prostate |
Cancer (mCRPC) | |
Phase | Phase 3 |
Patients | 814 |
Primary Outcome | • | Radiographic Progression Free Survival |
Measures | • | Overall Survival |
Arms/Intervention | • | 177Lu-PSMA-617 plus BS/BSC |
• | BS/BSC alone | |
Adult patients with PSMA-positive Metastatic Castration- | ||
Target Patients | resistant Prostate Cancer (mCRPC) | |
Expected Completion | H2-2020 | |
Publication | TBD | |
136 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Ophthalmology
Lucentis®- Anti-VEGF
Study | NCT02375971 RAINBOW (CRFB002H2301) | NCT02640664 RAINBOW Extension (CRFB002H2301E1) | ||
Indication | Retinopathy of Prematurity (ROP) | Retinopathy of Prematurity (ROP) | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 224 | 180 | ||
Absence of active Retinopathy of Prematurity (ROP) and | ||||
Primary Outcome | unfavorable structural outcome at Week 24, defined as, 1) | To evaluate the visual function of patients by assessing the | ||
survival, 2) no intervention with a second modality for ROP, | visual acuity in the better-seeing eye at the patient's fifth | |||
Measures | ||||
3) absence of active ROP and 4) absence of unfavorable | birthday. | |||
structural outcome | ||||
• | Ranibizumab 0.2 mg (up to 3 injections max) | • | Ranibizumab 0.2 mg (up to Week 40, if warranted) | |
Arms/Intervention | • | Ranibizumab 0.1 mg (up to 3 injections max) | ||
• | Ranibizumab 0.1 mg (up to Week 40, if warranted) | |||
• | Laser therapy | |||
Target Patients | Male and female preterm infants with bilateral retinopathy of | Male and female preterm infants with bilateral retinopathy of | ||
prematurity (ROP) who require treatment. | prematurity (ROP) who completed RAINBOW . | |||
Expected Completion | Q1-2018(actual) | 2023 | ||
• | EURETINA: Sep-2018 | |||
• | AAO: Oct-2018 | |||
Publication | • | Primary manuscript published online by The Lancet in | TBD | |
Sep-2019 | ||||
(https://www.thelancet.com/pdfs/journals/lancet/PIIS0140 | ||||
-6736(19)31344-3.pdf) | ||||
138 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation |
RTH258 - Anti-VEGF
Study | NCT02434328 HARRIER (CRTH258A2302) | NCT02307682 HAWK (CRTH258A2301) | ||
Indication | Neovascular age-related macular degeneration (nAMD) | Neovascular age-related macular degeneration (nAMD) | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 743 | 1,082 | ||
Primary Outcome | Change in Best Corrected Visual Acuity (BCVA) from | Change in Best Corrected Visual Acuity (BCVA) from | ||
Measures | baseline at week 48 | baseline at week 48 | ||
• | Brolucizumab (RTH258) 6 mg/50 µL | • | Brolucizumab (RTH258) 3 mg/50 µL | |
Arms/Intervention | • | Brolucizumab (RTH258) 6 mg/50 µL | ||
• | Aflibercept 2 mg/50 µL | |||
• | Aflibercept 2 mg/50 µL | |||
Target Patients | Subjects with exudative age-related macular degeneration | Subjects with exudative age-related macular degeneration | ||
Expected Completion | Q1-2018(actual) | Q2-2018(actual) | ||
• Oral presentations including both primary endpoint and key 2nd superior anatomic outcomes at AAO meetings in Nov- | ||||
2017 (1styear results) and Nov-2018 (2ndyear results) | ||||
• Year 1 Manuscript: Dugel P, et al. Ophthalmology 2019 Apr 12; HAWK and HARRIER: Phase 3, Multicenter, | ||||
Publication | Randomized, Double-Masked Trials of Brolucizumab for Neovascular Age-Related Macular Degeneration. |
- Abstracts submissions on superior anatomic outcomes/Fluid/PostHoc results are planned for key retinal congresses (Angiogenesis/Mac Soc inFeb-2019; WRC in Mar-2019; ARVO in April-2019; ASRC July-2019; EURETINA Sept-2019; AAO Oct-2019 and APVRS Dec-2019
139 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
RTH258 - Anti-VEGF
Study | NCT03386474 (CRTH258A2301E1) | NCT03481634 KESTREL (CRTH258B2301) | ||
Indication | Neovascular age-related macular degeneration (nAMD) | Diabetic eye disease | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 150 | 534 | ||
Primary Outcome | Number of treatment-emergent adverse events | Change from baseline in best-corrected visual acuity | ||
Measures | (BCVA) | |||
• | Brolucizumab (RTH258) 6 mg/50 µL | • | Brolucizumab (RTH258) 3 mg/50 µL | |
Arms/Intervention | • | Brolucizumab (RTH258) 6 mg/50 µL | ||
• | Aflibercept 2 mg/50 µL | |||
• | Aflibercept 2mg/50 uL | |||
Target Patients | Patients with neovascular age-related macular degeneration | Patients with visual impairment due to diabetic macular | ||
who have completed the CRTH258A2301 study | edema (DME) | |||
Expected Completion | Q3-2018(actual) | H2-2021 | ||
Publication | TBD | TBD | ||
140 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
RTH258 - Anti-VEGF
Study | NCT03481660 KITE (CRTH258B2302) | NCT04058067 KINGLET (CRTH258B2304) | ||
Indication | Diabetic eye disease | Diabetic macular edema | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 356 | 268 | ||
Primary Outcome | Change from baseline in best-corrected visual acuity | Change in best-corrected visual acuity (BCVA) | ||
Measures | (BCVA) | |||
Arms/Intervention | • | Brolucizumab (RTH258) 6 mg/50 µL | • | Brolucizumab (RTH258) 6 mg/50 µL |
• | Aflibercept 2 mg/50 µL | • | Aflibercept 2 mg/50 µL | |
Target Patients | Patients with visual impairment due to diabetic macular | Patients with visual impairment due to diabetic macular | ||
edema (DME) | edema | |||
Expected Completion | H2-2021 | 2022 | ||
Publication | TBD | TBD | ||
141 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
RTH258 - Anti-VEGF
Study | NCT03917472 KINGFISHER (CRTH258B2305) | NCT03802630 RAPTOR (CRTH258C2301) | ||
Indication | Diabetic macular edema | Retinal vein occlusion | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 500 | 500 | ||
Primary Outcome | Change in best-corrected visual acuity (BCVA) from | Change from baseline in best-corrected visual acuity | ||
Measures | baseline up to week 52 | (BCVA) at week 24 | ||
Arms/Intervention | • | Brolucizumab (RTH258) 6 mg/50 µL | • | Brolucizumab (RTH258) 6 mg/50 µL |
• | Aflibercept 2 mg/50 µL | • | Aflibercept 2 mg/50 µL | |
Target Patients | Patients with visual impairment due to diabetic macular | Adult patients with visual impairment due to macular edema | ||
edema | secondary to branch retinal vein occlusion | |||
Expected Completion | H2-2021 | 2022 | ||
Publication | TBD | TBD | ||
142 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
RTH258 - Anti-VEGF
Study | NCT03810313 RAVEN (CRTH258C2302) | NCT04047472 HOBBY (CRTH258A2307) |
Indication | Retinal vein occlusion | Macular degeneration |
Phase | Phase 3 | Phase 3 |
Patients | 750 | 494 |
Primary Outcome Measures
Arms/Intervention
Change from baseline in best-corrected visual acuity | Change from baseline in best-corrected visual acuity | ||
(BCVA) at week 24 | (BCVA) at week 48 | ||
• | Brolucizumab (RTH258) 6 mg/50 µL | • | Brolucizumab (RTH258) 6 mg/50 µL |
• | Aflibercept 2 mg/50 µL | • | Aflibercept 2 mg/50 µL |
Target Patients | Adult patients with visual impairment due to macular edema | Chinese patients with neovascular age-related macular |
secondary to central retinal vein occlusion | degeneration | |
Expected Completion | 2023 | 2023 |
Publication | TBD | TBD |
143 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
UNR844 - Disulfide bonds modulator
Study | NCT03809611 (CUNR844A2203) | |
Indication | Presbyopia | |
Phase | Phase 2 | |
Patients | 124 | |
Primary Outcome | Change in binocular distance-corrected near visual acuity | |
Measures | (DNCVA) from baseline | |
Arms/Intervention | • | 1.5% solution UNR844-Cl |
• | Placebo | |
Target Patients | Patients with presbyopia | |
Expected Completion | Q1-2020 | |
Publication | Planned in ASRCS in 2020 | |
144 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Respiratory
QAW039 - DP2 receptor antagonist
Study | NCT02555683 LUSTER-1 (CQAW039A2307) | NCT02563067 LUSTER-2 (CQAW039A2314) | ||
Indication | Asthma | Asthma | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 846 | 846 | ||
Primary Outcome | Reduction in the rate of moderate-to-severe asthma | Reduction in the rate of moderate-to-severe asthma | ||
Measures | exacerbations | exacerbations | ||
• | QAW039 Dose 1 | • | QAW039 Dose 1 | |
Arms/Intervention | • | QAW039 Dose 2 | • | QAW039 Dose 2 |
• | Placebo | • | Placebo | |
Target Patients | Patients with uncontrolled severe asthma | Patients with uncontrolled severe asthma | ||
Expected Completion | Q4-2019(actual) | Q3-2019(actual) | ||
Publication | Planned in 2020 | Planned in 2020 | ||
146 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
QAW039 - DP2 receptor antagonist
Study | NCT03215758 ZEAL-1 (CQAW039A2316) | NCT03226392 ZEAL-2 (CQAW039A2317) | ||
Indication | Asthma | Asthma | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 650 | 650 | ||
Primary Outcome | ||||
Pre-dose forced expiratory volume in 1 second (FEV1) | Pre-dose forced expiratory volume in 1 second (FEV1) | |||
Measures | ||||
Arms/Intervention | • | QAW039 | • | QAW039 |
• | Placebo | • | Placebo | |
Target Patients | Patients with uncontrolled asthma | Patients with uncontrolled asthma | ||
Expected Completion | Q3-2019(actual) | Q3-2019(actual) | ||
Publication | Planned in 2020 | Planned in 2020 | ||
147 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
QAW039 - DP2 receptor antagonist
Study | NCT03052517 SPIRIT (CQAW039A2315) | NCT03650400 (CQAW039B2201) |
Indication | Asthma | Asthma |
Phase | Phase 3 | Phase 2 |
Patients | 1,900 - 2,300 | 24 |
Primary Outcome Measures
Arms/Intervention
Long term safety: treatment emergent adverse event (AE),
SAE and AE leading to discontinuation from study (52 wks | Pharmacokinetics, safety and tolerability | |
and 160 wks) | ||
• | QAW039 Dose 1 | • Fevipiprant Cohort A; Fevipiprant Cohort B; Chewable |
• | QAW039 Dose 2 | |
tablet | ||
• | Placebo | |
Target Patients | Patients with moderate to severe asthma | Children aged 6 to < 12 years with asthma |
Expected Completion | For Submission: Q4-2019(actual);Final: 2022 | Q3-2020 |
Publication | TBD | TBD |
148 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
QBW251 - CFTR potentiator
Study | NCT04072887 (CQBW251B2201) | |
Indication | Chronic obstructive pulmonary disease (COPD) | |
Phase | Phase 2 | |
Patients | 900 | |
Primary Outcome | Trough FEV1 (Forced Expiratory Volume in 1 second) | |
Measures | change from baseline after 12 weeks of treatment | |
• | QBW251 450 mg | |
• | QBW251 300 mg | |
Arms/Intervention | • | QBW251 150 mg |
• | QBW251 75 mg | |
• | QBW251 25 mg | |
• | Placebo | |
Target Patients
Expected Completion
Publication
COPD patients on background triple inhaled therapy (LABA / LAMA / ICS)
H2-2021
TBD
149 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
QMF149 - Long-acting beta2 agonist and inhaled corticosteroid
Study | NCT02892019 (CQMF149G2202) |
Indication | Asthma |
Phase | Phase 2 |
Patients | 80 |
Primary Outcome | Trough FEV1 |
Measures | |
Arms/Intervention | • Indacaterol acetate 75 μg od (via Concept1 inhaler) |
• Indacaterol acetate 150 μg od (via Concept1 inhaler) | |
Target Patients | Children ≥ 6 to < 12 years of age with asthma |
Expected Completion | Q3-2019(actual) |
Publication | Planned in H2-2020 |
150 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
QVM149 - Long-acting beta2 agonist, Long-acting muscarinic antagonist and inhaled corticosteroid
Study | NCT02554786 PALLADIUM (CQVM149B2301) | NCT02571777 IRIDIUM (CQVM149B2302) | ||
Indication | Asthma | Asthma | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 2,216 | 3,092 | ||
Primary Outcome | ||||
Trough FEV1 | Trough FEV1 | |||
Measures | ||||
• | QMF149 150/160 µg od | • | QVM149 150/50/160 µg od | |
• | QMF149 150/320 µg od | • QVM149 150/50/80 µg od | ||
Arms/Intervention | • MF 400 µg od | • QMF149 150/160 µg od | ||
• MF 400 µg bid | • QMF149 150/320 µg od | |||
• | Salmeterol 50 µg /fluticasone 500 µg bid | • | Salmeterol 50 µg /fluticasone 500 µg bid | |
Adult and adolescent (≥12 years) patients with asthma | Adult (≥18 years) patients with asthma inadequately | |||
Target Patients | inadequately controlled on medium/high-dose ICS or low- | |||
controlled on medium/high-dose of LABA/ICS (GINA step ≥4) | ||||
dose LABA/ICS (GINA step ≥ 3) | ||||
Expected Completion | Q3-2019(actual) | Q3-2019(actual) | ||
Publication | • | Planned in H1-2020 | • | Planned in H1-2020 |
• | Abstract: van Zyl-Smit et al, presented at BTS Dec-2019 | • | Abstract ATS Q2-2020 | |
151 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
QVM149 - Long-acting beta2 agonist, Long-acting muscarinic antagonist and inhaled corticosteroid
Study | NCT03100500 (CQVM149B1305) | NCT03100825 (CQVM149B1304) |
Indication | Asthma | Asthma |
Phase | Phase 3 | Phase 3 |
Patients | 51 | 94 |
Primary Outcome | Long-term safety/tolerability: Incidence and severity of | Long-term safety/tolerability: Incidence and severity of |
treatment emergent adverse events during the 52 weeks | treatment emergent adverse events during the 52 weeks | |
Measures | ||
study | study | |
Arms/Intervention | • Single arm: QMF149 150/320 μg od | • Single Arm: QVM149 150/50/160 μg od |
Target Patients | Japanese patients with asthma inadequately controlled | Japanese patients with asthma inadequately controlled |
Expected Completion | Q1-2019(actual) | Q2-2019(actual) |
Publication | • Planned in H1-2020 | • Planned in H1-2020 |
• Abstract for ATS in Q2-2020 | • Abstract for ATS in Q2-2020 | |
152 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
QVM149 - Long-acting beta2 agonist, Long-acting muscarinic antagonist and inhaled corticosteroid
Study | NCT02892344 QUARTZ (CQVM149B2303) | NCT03158311 ARGON (CQVM149B2306) | ||
Indication | Asthma | Asthma | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 802 | 1,251 | ||
Primary Outcome | Trough FEV1 | Non-inferiority of Asthma Quality of Life Questionnaire | ||
Measures | (AQLQ) | |||
• | QMF149 150/80 µg od | • QVM149 150/50/80 μg od | ||
Arms/Intervention | • | QVM149 150/50/160 μg od | ||
• MF 200 µg od | ||||
• | Salmeterol/fluticasone 50/500 μg bid + tiotropium 5 μg od | |||
Adult and adolescent (≥12 years) patients with mild asthma | ||||
Target Patients | inadequately controlled on low-dose ICS or low-dose | Patients with uncontrolled asthma | ||
LABA/ICS (Gina step 2-3) | ||||
Expected Completion | Q1-2019(actual) | Q3-2019(actual) | ||
Publication | • | O. Kornmann et al. Respiratory Medicine 161 (2020) | • | Planned in H1-2020 |
• Abstract: D'Andrea et al, presented at ERS Sep-2019 | • | Abstract ATS Q2-2020 | ||
153 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Xolair ®- anti-IgE antibody
Study | NCT03369704 (CIGE025F1301) | |
Indication | Seasonal allergic rhinitis: Severe Japanese Cedar Pollinosis | |
Phase | Phase 3 | |
Patients | 337 | |
Primary Outcome Measures | Mean nasal symptom score, consists of severity of sneezing, rhinorrhea and nasal congestion. | |
In addition to standard of care: | ||
Arms/Intervention | • | Omalizumab per approved allergic asthma dosing table for IgE/body weight combinations |
• | Placebo |
Target Patients
Expected Completion
Publication
Patients with severe Japanese cedar pollinosis, whose symptoms were inadequately controlled with current recommended therapies
Q1-2019(actual)
- Late breaking abstract was published at AAAAI (American Association of Allergy, Asthma and Immunology) annual meeting, Feb 2019
- Poster published at EAACI (the European Academy of Allergy and Clinical Immunology), Jun 2019
- Oral presentations were made at JRS (Japanese Rhinologic Society), Oct 2019, and Asian Pacific Society of Respirology congress, Nov 2019
- Planned oral/poster presentation at Japan society of Immunology & Allergology in Otolaryngology, Feb 2020
- Planned manuscript to be submitted to JACI in Practice, Q1 2020
154 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Sandoz Biopharmaceuticals
Hyrimoz®- Biosimilar adalimumab
Study | NCT02744755 ADMYRA (GP17-302) | |
Indication | Immunology | |
Phase | Phase 3 | |
Patients | 353 | |
Primary Outcome | Change in DAS28-CRP score from baseline to week 12 in | |
patients treated with GP2017 and patients treated with | ||
Measures | ||
Humira® | ||
Arms/Intervention | • | GP2017 |
• | US licensed Humira®adalimumab | |
Target Patients
Expected Completion
Publication
Patients with moderate to severe active rheumatoid arthritis
2018(actual)
- Wiland, P. et al., presented at EULAR 2019
- Wiland, P. et al., BioDrugs, Q4 2019
156 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
GP2411 - Biosimilar denosumab
Study | NCT03974100 (CGP24112301) | |
Indication | Osteoporosis | |
Phase | Phase 3 | |
Patients | 522 | |
Primary Outcome | Percent change from baseline (%CfB) in lumbar spine Bone | |
Measures | Mineral Density | |
• | GP2411 60 mg /mL subcutaneous injection every 6 | |
Arms/Intervention | months | |
• | Prolia®60 mg /mL subcutaneous injection every 6 | |
months
Target Patients
Expected Completion
Postmenopausal women with osteoporosis
2022
Publication | Study data publications expected for 2024 and beyond |
157 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
Global Health
KAF156 - Plasmodium Falciparum Inhibitor - PfCARL mediated
Study | NCT03167242 (CKAF156A2202) |
Indication | Malaria |
Phase | Phase 2 |
Patients | 512 |
Primary Outcome | PCR-corrected adequate clinical and parasitological |
Measures | response (ACPR) |
Arms/Intervention | • KAF156 and LUM-SDF (different combinations) |
• Coartem | |
Target Patients | Adults and children with uncomplicated Plasmodium |
Falciparum Malaria | |
Expected Completion | H1-2021 |
Publication | TBD |
159 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
KAE609 - Plasmodium Falciparum Inhibitor - spiroindolone against PfATP4
Study | NCT03334747 (CKAE609A2202) | |
Indication | Malaria | |
Phase | Phase 2 | |
Patients | 210 | |
Primary Outcome | CTCAE grades increase from baseline in alanine | |
aminotransferase (ALT) or aspartate aminotransferase | ||
Measures | ||
(AST) | ||
Arms/Intervention | • | KAE609 |
• | Coartem | |
Target Patients | Adults with uncomplicated Plasmodium Falciparum malaria | |
Expected Completion | Q4-2020 | |
Publication | TBD | |
160 Novartis Q4 and FY 2019 Results | January 29, 2020 | Novartis Investor Presentation
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Novartis AG published this content on 29 January 2020 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 29 January 2020 09:09:05 UTC