Novartis Investor Relations
Q4 2020 Results
Investor presentation January 26, 2021
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," 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 impact of the COVID-19 pandemic on certain therapeutic areas including dermatology, ophthalmology and the Sandoz retail business, and on drug development operations; or regarding potential future, pending or announced transactions; regarding potential future sales or earnings of the Group or any of its divisions; or by discussions of strategy, plans, expectations or intentions; or regarding the Group's liquidity or cash flow positions and its ability to meet its ongoing financial obligations and operational needs; or regarding our not-for-profit portfolio of 15 medicines from the Sandoz division for symptomatic treatment of COVID-19 and our collaboration with Molecular Partners to develop, manufacture and commercialize potential medicines for the prevention and treatment of COVID-19. 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: liquidity or cash flow disruptions affecting our ability to meet our ongoing financial obligations and to support our ongoing business activities; the impact of the COVID-19 pandemic on enrollment in, initiation and completion of our clinical trials in the future, and research and development timelines; the impact of a partial or complete failure of the return to normal global healthcare systems including prescription dynamics by mid 2021; global trends toward healthcare cost containment, including ongoing government, payer 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 development of the products described in this presentation; the potential that the strategic benefits, synergies or opportunities expected from the transactions described, including BeiGene, may not be realized or may be more difficult or take longer to realize than expected; 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; 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, investigations or disputes; our performance on environmental, social and governance measures; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; 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.
Enbrel® is a registered trademark of Amgen, Inc. Humira® and Skyrizi™ are registered trademarks of Abbvie Inc. Siliq® is a registered trademark Valeant Pharmaceuticals International, Inc. Taltz® is a registered trademark of Eli Lilly and Company. Stelara®, Tremfya® and Simponi® are registered trademarks of Janssen Biotech, Inc. Cimzia® is a registered trademark of UCB Group of Companies.
2 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
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 | Chief Legal Officer |
Susanne Schaffert | |
President, Novartis Oncology |
3 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Vas Narasimhan
Chief Executive Officer
Company overview
4 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
We are transforming Novartis...
Strategy set out in 2018...
Our focus | Our five priorities | |
Focus our | Unleash the | Deliver |
company | power of our | transformative |
and capital | people | innovation |
Accelerate | Embrace | Go big |
certain | operational | on data |
geographies | excellence | and digital |
Strengthen | Build trust | |
our core | with society | |
...is transforming Novartis
100% focused as a medicines company
Leading pipeline, with 4 advanced therapy platforms
Achieved USD 2bn cost savings over 2017-2020
Establishing a leading digital and data science platform
Improving ESG scores, sector-leading across 4 key indices
Record-high engagement score
5 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
...while delivering strong operational performance
Net sales +5% CAGR1 | Core3 | OpInc +10% CAGR1 | Innovative Medicines | ||||||||||
core3 margin up to 35% | |||||||||||||
Continuing operations2, USD bn | |||||||||||||
2017 | 2018 | 2019 | 2020 | 2017 | 2018 | 2019 | 2020 | 2017 | 2018 | 2019 | 2020 | ||
47.4 | 48.7 | 15.4 | 35.0% | ||
44.8 | 14.1 | 33.5% | |||
42.3 | 12.6 | 32.0% |
11.7 | 31.0% | |
1. CAGR % 2017-20 in USD 2. Refers to continuing operations as defined on page 43 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz, as well as the continuing corporate functions 3. Constant currencies (cc) and core results are non-IFRS measures. An explanation of non-IFRS measures can be found on page 55 of the Condensed Financial Report
6 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Q4 sales growth and margin expansion driven by strong performance from key growth drivers
Operational performance
Continuing operations1, % cc vs. PY
Key growth driver sales Q4 2020
Growth | ||
Sales | Growth vs. PY | vs. PY |
USD mn | USD mn | cc |
Key growth drivers and launches, as % of Innovative Medicines sales
Q4 2020 FY 2020
Net | 3% |
sales | |
1% | |
Core2 | 13% |
OpInc | |
2% |
716 | 198 | 35% | |
1,109 | 144 | 13% | |
471 | 91 | 23% | |
376 | 83 | 24% | |
254 | 68 | 33% | |
240 | 62 | 32% | |
408 | 52 | 13% | |
141 | 45 | 42% | |
57 | 40 | nm | |
34 | 33 | nm | |
335 | 32 | 8% | |
184 | 29 | 18% |
52%
43%
34%
28%
Q4 | Q4 | Q4 | Q4 |
2017 | 2018 | 2019 | 2020 |
Adakveo®
Beovu®
Mayzent®
Piqray®
Xiidra®
Lutathera®
Kymriah®
Kisqali®
Ilaris®
Zolgensma®
Jakavi®
Tafinlar+Mekinist®
Promacta®
Entresto®
Cosentyx®
Other3
nm - not meaningful
3. Includes Tasigna®, Xolair®, Aimovig®, Tabrecta ®, Kesimpta®, Luxturna® , Enerzair ® and Atectura®
1. Refers to continuing operations as defined on page 43 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz, as well as the continuing corporate functions 2. Core results are non-IFRS measures. An explanation of non-IFRS measures can be found on page 55 of the Condensed Financial Report
7 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Zolgensma® sales of USD 920m in first full year since launch, now registered in 37 countries
Sales evolution
USD m
291 | ||||||
254 | ||||||
205 | 169 | |||||
170 | ||||||
148 | ||||||
44 | 100 | |||||
126 | 105 | 122 | 106 | |||
Q1'20 | Q2'20 | Q3'20 | Q4'20 | |||
Ex-US | US | |||||
Performance highlights
- Delivered significant growth in 2020 despite COVID-19 delaying new starts and switches; impact expected to continue through H1 2021
- Treatment of choice for newly diagnosed patients
- >800 patients have received Zolgensma®; now registered in 37 countries
- Shift toward broader labeling (e.g. EU, Canada) and use globally - In EU, 15% of patients >2 years old and 2% over 13.5kg
- Access pathways in 9 EU countries (~25% of the population)
Growth opportunities in existing indications
- Approvals expected H1 2021: Switzerland, Australia, Argentina, South Korea
- Formal reimbursement expected in 15 countries by 2022 (.e.g UK, Spain, Canada, Brazil, Argentina)
- Expected increase in newborn screening: >80% in US and 20% in EU by end 20211
Clinical and pipeline
- Confirmatory data in 2021: cumulative IV safety, final STR1VE-EU & STR1VE-US results
- IT partial clinical hold: preclinical studies on track; FDA recommends a pivotal confirmatory study to be initiated after partial clinical hold is lifted
- 10+ early-stage programs with two INDs planned in 2021
1. Newborn screening implementation may be impacted by COVID-related delays
8 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
China: Rich pipeline, NRDL successes, expected to double sales by 2024
Second fastest growing pharmaceutical multinational company in China in 2020
Sales
Continuing operations1, USD bn, % cc
+16% | 2.6 | |
2.2 | ||
2019 FY | 2020 FY |
- China net sales grew +16% despite COVID-19impact
- Key growth drivers continued momentum, FY sales growth YoY: Entresto®, Cosentyx®, Oncology strategic brands2
- NRDL achievements in 2020:
- Successfully listed: Cosentyx® (PsO & AS), Gilenya®, Mayzent®, Tafinlar® & Mekinist®3
- Renewed listing: Tasigna®, Votrient®, Sandostatin® LAR, Zykadia®
- Rich pipeline:
- 2020 NDA approvals: Cosentyx® AS, Mayzent®, Pataday®, Tafinlar®+Mekinist® adj, Zykadia® 1L
- 7 approvals expected in 2021 (NDA submitted in 2020)
- Kesimpta® granted China priority review, TQJ230 obtained 1st BTD
- New launches planned by 2023: Kisqali®, ACZ885
- New indications by 2023: Afinitor® (ER+ 2/3L BC), Tafinlar®+Mekinist® (BRAF+ NSCLC), Revolade® (SAA 2L/1L)
BTD - break through therapy designation 1. Refers to continuing operations as defined on page 43 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz, as well as the continuing corporate functions 2. Oncology strategic brands include: Strategic brands: Revolade®/ Jakavi®/ Tasigna®/ Exjade®/ Votrient®/ Sandostatin® LAR/ Zykadia®/ Tafinlar®+Mekinist® 3. Azarga® and Simbrinza® were also successfully listed in NRDL
9 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Sandoz sales met guidance with significant margin growth, despite pandemic impact
Sales stable, core margin increased by +3.3%pts in 2020
(vs. PY, in cc)
Q4 2020 | FY 2020 |
Net sales
0% 0%
Core OpInc
15%
3% |
Core 20.8% 24.2%
margin:+3.3%pts
Key 2020 performance drivers
Biopharmaceuticals1 growth
+19%, increasing share in maturing European markets
Retail decline
driven by COVID-19 and US oral solids
Margin increase from
product mix, productivity, favorable price effects; reduced spend
Driving growth
Biosimilars
- 15+ pipeline assets, many entering clinical phase soon
- Sales of up to USD 3.5bn expected by 2025
Small molecules
- High LoE coverage (EU: >80%, US: >50%) with approximately 40 US first-to-files until 2024
Driving margin
(mid-to-high 20s in mid-to long term)
- Manufacturing optimization
- Product mix from higher biosimilar share
- Operational excellence including use of digital
1. Biopharmaceuticals include biosimilars, biopharmaceutical contract manufacturing and Glatopa®
10 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Novartis focused on overcoming COVID-19 challenges
Select examples
Dermatology market declines with COVID-19 | |||||||||||||
PsO market weekly patient visits Jan-Aug 2020 | PsO market weekly NBRx Jan-Aug 2020 | ||||||||||||
25 | 4.0 | ||||||||||||
20 | 3.0 | ||||||||||||
Volume (thousands) | 15 | Thousands | |||||||||||
2.0 | |||||||||||||
10 | 1.0 | ||||||||||||
5 | |||||||||||||
Visit | |||||||||||||
0 | 0.0 | ||||||||||||
Jan | Mar | May | Aug | Oct | Dec | Jan | Mar | May | Aug | Oct | Dec | ||
Sources: IQVIA Visits Data. IQVIA National Prescription Audit for Dermatology |
Oncology market declines with COVID-19
Biopsy and surgery rates | CDK4/6 market NBRx |
Key areas impacted in 2020 expected to continue through H1 2021
Dynamic segment of | | Several therapeutic areas, |
market (new/switch | particularly dermatology and | |
patients): less patient | ophthalmology | |
traffic, less F2F | | New launches |
physician access | ||
(e.g. Kesimpta®, Mayzent®) | ||
| Advanced breast cancer | |
(CDK4/6 market and Piqray®) | ||
Hospital initiation (lab | | Kymriah® |
120 | ||||||||
100 | ||||||||
80 | ||||||||
60 | ||||||||
40 | ||||||||
20 | ||||||||
Breast | Lung | Melanoma | ||||||
0 | ||||||||
Pre- | Mar | Apr | May | Jun | Jul | Aug | Sep | Oct |
COVID |
-20%
Pre-COVID baseline | Sep'20-Nov'20 Avg. |
Q3'19-Q1'20 Avg. |
work, diagnostics); | | Lutathera® |
immuno-suppression | ||
concerns | | Zolgensma® |
Lower demand | | Sandoz Retail |
and anti-infectives |
Source: IQVIA APLD, Novartis Analysis | Source: IQVIA projection |
11 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Key innovation milestones in Q4
Approvals | Positive FDA | In-licensing deals | ||||||
AdComm Opinion | ||||||||
Tislelizumab | BeiGene | |||||||
EU for | HFpEF | (anti-PD-1) | ||||||
Hyperlipidemia | ||||||||
Anti-COVID-19 | Molecular | |||||||
EU for Sickle cell | DARPins | |||||||
Positive readout | Partners | |||||||
disease | ||||||||
DME1
US for CRSwNP
LNP023 (iptacopan): FDA breakthrough designation (PNH), Rare Pediatric Disease Designation (C3G)
QGE031 (ligelizumab): FDA breakthrough designation (CSU) granted in December 2020
Leqvio® CRL update
-
CRL due to unresolved facility inspection-related conditions at
3rd party manufacturer in Europe2 - No concerns raised in CRL regarding efficacy or safety of Leqvio®
- Response to CRL based on 3rd party readiness to be submitted Q2-Q3 2021
- Need for FDA inspection under discretion of FDA
- Concurrently working on technology transfer to add Novartis own facility
All abbreviations on slide 144. CRSwNP - Severe chronic rhinosinusitis with nasal polyps. 1. Ph3 KESTREL study 2. Third party site originally scheduled for inspection May 2020; underwent paper based review due to pandemic.
12 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
2021 catalysts maintaining long-term momentum
Potential catalysts | Selected examples | ||
Major approvals | Kesimpta® (EU/JP) | Entresto® (US) | Cosentyx® (US/JP/CN) |
RMS | HFpEF | Pediatric psoriasis | |
Major submissions1 | Alpelisib (BYL719) | Asciminib (ABL001) | Jakavi® |
PROS | CML | Acute and chronic GvHD | |
Beovu® | Leqvio® (US)2 | Kymriah® |
Major readouts
Enabling submission 2021
Enabling submission 2022
Others
Pivotal study starts
DME | Hyperlipidemia | FL |
177Lu-PSMA-617 | Sabatolimab (MBG453) | Canakinumab (ACZ885)3 |
mCRPC | MDS | NSCLC 1L + 2L |
Kymriah® | Entresto® | |
r/r DLBCL 1st relapse | Post-AMI | |
Ligelizumab (QGE031)4 | Cosentyx® | |
CSU | HS | |
Iptacopan (LNP023) | Kisqali® | |
IgAN, C3G (Ph2) | Breast cancer (MONALEESA-2 OS) | |
Iptacopan (LNP023) | Ligelizumab (QGE031) | 177Lu-PSMA-617 |
IgAN, C3G, aHUS | Food allergy, CINDU | pre-taxane |
1. First submission in any market. 2. Novartis received a CRL from the FDA due to unresolved facility inspection-related conditions at a third-party manufacturing facility in Europe. FDA has not raised any concerns related to the efficacy or safety of inclisiran. Response to CRL planned to be submitted Q2 - Q3 2021. 3. Depending on timing of final read-out submission may move to early 2022 4. Q4/2021-Q1/2022 potential COVID impact
13 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Moving forward a breadth of assets to drive long-term growth
Selected opportunities: selection of expected 2021 milestones and additional indications
Lifecycle management
Post-AMI: PARADISE | |
Entresto® | Ph3 readout H1 2021 |
HFpEF: FDA action date Q1 2021
HS: SUNRISE, SUNSHINE
Ph3 readout H2 2021
Cosentyx®
L. Planus, Peds PsO, jPsA/ER, | |
GCA, lupus nephritis | |
Kisqali® | aBC: MONALEESA-2 OS |
readout H2 2021 | |
PROS: submission H2 2021 | |
BYL719 | TNBC, ovarian cancer, HER2+ |
aBC, HNSCC 2/3L | |
Beovu® | DME: submission H1 2021 |
RVO, diabetic retinopathy | |
Pharmaceuticals
Iptacopan | IgAN, C3G: Ph2 readout H1 2021 |
(LNP023) | |
aHUS: Ph3 start. PNH, iMN | |
Iscalimab | Sjögren's, kidney Tx, liver Tx |
(CFZ533) | |
Ligelizumab | CSU: PEARL 1, 2 |
(QGE031) | Ph3 readout H2 2021 |
CINDU, food allergy | |
Ph3 start H2 2021 | |
Pelacarsen | CVRR-Lp(a) |
(TQJ230) | |
Branaplam | HD: Ph2b start H2 2021 |
(LMI070) | |
SMA | |
Oncology
Canakinumab | NSCLC 1L/2L: CANOPY-1/2 |
(ACZ885) | Ph3 readout H1/2 2021 |
NSCLC adjuvant | |
177Lu-PSMA-617 | mCRPC 3L: |
VISION Ph3 readout H1 2021 | |
mCRPC pre-taxane: | |
Ph3 start H1 2021 | |
Sabatolimab | HR-MDS: STIMULUS |
(MBG453) | Ph2 readout H2 2021 |
AML | |
TNO155 | Solid tumors, multiple |
combinations being explored | |
in on-going trials | |
LXH254 | BRAF/NRASm melanoma, |
mRAS/RAF NSCLC | |
'Wild Cards' | ECF843 (Dry eye: Ph2 readout H2 2021), LNA043 (Osteoarthritis: Ph2b start H1 2021), | |
CSJ117 (Asthma), QBW251 (COPD), NIS793 (Solid tumors) | ||
14 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Marie-France Tschudin
President, Novartis Pharmaceuticals
15 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Pharmaceuticals portfolio continues to rejuvenate with 43% sales driven by recent launches and growth drivers
Pharmaceuticals net sales | FY 2020: Net sales up +5% | ||
USD bn, growth in % cc | Growth drivers and launches growing +33%, despite pandemic conditions: | ||
Recent launches1 | now represent 43% of the pharmaceutical sales | ||
Growth drivers2 | |||
Mature products3 | Portfolio shift continues to lay foundation for future growth | ||
+5% | |||
Cosentyx® and Entresto® contributed USD 6.5bn, growing +23% cc YoY | |||
23.3 | 24.3 | ||
Key approvals and launches in 2020 include: | |||
0.7 | 1.9 | ||
- Kesimpta® in US | |||
7.1 | +33% | ||
8.6 | - Leqvio® in EU | ||
15.5 | 13.8 | - Cosentyx® nr-axSpA in US and EU | |
-10% | Launched new products or new major indications in all 5 franchises | ||
FY 2019 | FY 2020 | ||
1. Zolgensma®, Xiidra®, Beovu® , Mayzent®, Aimovig®, Luxturna®, Kesimpta® , Enerzair® and Atectura®. 2. Cosentyx®, Entresto®, Xolair® , Ilaris® 3. All other brands. |
16 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Cosentyx® Q4 sales up 13%, maintaining strong position in dermatology and outgrowing rheumatology market
Sales evolution
USD m, % cc
Ex-US | |
US | 4.0bn (+13%) |
3.6bn | 1,109 | ||||||
937 | 965 | 930 | 944 | 1,012 | |||
858 | 423 | ||||||
791 | 372 | ||||||
336 | 354 | 354 | 330 | ||||
324 | |||||||
317 | |||||||
474 | 534 | 601 | 611 | 576 | 614 | 640 | 686 |
Q1 | Q2 | Q3 | Q4 | Q1 | Q2 | Q3 | Q4 |
2019 | 2020 |
Robust Q4 performance despite continued COVID-19 impact
- COVID-19market impact: patient visits at 70%-80% vs. pre-COVID-191
- Demand driven by clinical profile, strong access, execution excellence
- Q4 YoY TRx grew faster than the market in US for PsO and SpA:
- PsO: +10% vs. market +7%, maintaining strong NBRx share ~16%2
- SpA: +18% vs. market +9%, leading NBRx share ~30%3
Catalysts for continued strong growth
- Increased biologics penetration in existing indications (all regions)
- China NRDL listing for PsO and AS
- 300mg auto-injector & pre-filled syringe approved by EMA
- Clinical data readout (ULTIMATE): significant reduction in synovitis
- Expansion to up to 10 indications/label expansions planned (e.g. HS, JIA)
JIA - Juvenile idiopathic arthritis HS - Hidradenitis suppurativa TRx - Total Prescriptions NBRx - New-to-Brand Prescriptions EMA - European Medicines Agency NRDL - National Reimbursement Drug List PsO - Psoriasis AS - ankylosing spondylitis SpA - spondyloarthritis TRx growth is calculated by comparing product volume across two time periods (YoY refers to Q4 2020 compared with Q4 2019). NBRx share calculated as product NBRx volume divided by market NBRx volume. 1. IQVIA Visits Data; 2. IQVIA National Prescription Audit for Dermatology through December 2020; PsO market includes Enbrel®, Humira®, Siliq®, Skyrizi™, Stelara®, Taltz®, Tremfya® 3. IQVIA National Prescription Audit for Rheumatology through December 2020; SpA market includes Cimzia®, Enbrel®, Humira®, Simponi®, Stelara®, Taltz®, Tremfya®
17 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Entresto® grows 44% in 2020 based on demand as an essential, first-choice treatment in heart failure
Sales evolution | |||||||
USD m, % cc | |||||||
Ex-US | |||||||
US | 2.5bn (+44%) | ||||||
1.7bn | 632 | 716 | |||||
518 | 569 | 580 | |||||
354 | |||||||
357 | 421 | 430 | 272 | 318 | |||
233 | 276 | ||||||
200 | 210 | ||||||
158 | |||||||
221 | 220 | 285 | 293 | 308 | 314 | 362 | |
199 | |||||||
Q1 | Q2 | Q3 | Q4 | Q1 | Q2 | Q3 | Q4 |
2019 | 2020 |
Strong momentum across geographies continues in Q4
- US demand driven growth (+27% YoY) 1
- China (>200% YoY) fueled ex-US growth
- Europe delivered strong double-digit growth (+32% YoY)
Confidence in future growth
- Increasing penetration in HFrEF: only 25% currently treated2
- A direct-to-ARNI approach is now recommended in de novo patients by ACC in the 2021 ECDP3
LCM
- Extended use in the treatment of HFpEF (US, Q1 2021)
- PARADISE-MItrial on track for read-out in Q2 2021
ACC - American College of Cardiology HFrEF - Heart failure with reduced ejection fraction. HFpEF - Heart failure with preserved ejection fraction. CHF - Congestive Heart Failure. Post-AMI - post Acute Myocardial Infarction 1. US NBRx - IMS New to Brand Q4 vs Q3. Weekly NBRx averaged ~4,000 for the Q4 period up to 25 Dec 2020 2. Eligible patients defined as prevalent HFrEF patients within each market's label. G7 = US, CA, JP, DE, FR, IT, UK 3. 2021 Update to the 2017 ACC Expert Consensus Decision Pathway (ECDP) for Optimization of Heart Failure Treatment: Answers to 10 Pivotal Issues About Heart Failure With Reduced Ejection Fraction (in publication). https://www.jacc.org/doi/10.1016/j.jacc.2020.11.022
18 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Positive FDA AdComm supports extending the use of Entresto® to more HF patients, building on leading position in rEF
Unmet need and value of Entresto® confirmed
- Recognition of highest effect in ejection fraction below normal1
- 12:1 vote in favor of extending use
- Understanding of HF is evolving putting dichotomous classification in question2
- Confirms strong unmet need
- Regulatory decision expected Q1 2021
Preparing for launch
- Disease burden significant with frequent HF hospitalizations, emergency rooms and urgent office visits
- Depending on label, US addressable population could be 1.5m to 3m
- Strong experience and footprint in cardiology
- Expect to maintain leading access position
- Uptake likely gradual due to lack of consensus guidelines
1. LVEF pooled analysis PARADIGM/PARAGON. Solomon S et al. 2020 Circulation. 2. Dichotomous classification of HFrEF and HFpEF is not adequately capturing treatment effect as discussed by FDA AdComm on Entresto (15 Dec 2020) and spironolactone (16 Dec 2020), incl. CHARM study (Lund et al. European Journal of Heart Failure (2018) 20, 1230-1239); TOPCAT study (Solomon et al. European Heart Journal (2016) 37, 455-462)
19 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Kesimpta® on track to become a 1st choice high efficacy DMT for patients, physicians and payers; preparing for RoW launches
Solid launch progress during pandemic | 2021 priorities for acceleration | |
Broad payer | >150m (~73%) US commercial lives | Pull-through access, driving conversion |
coverage | have unrestricted coverage or single step edit | to paid product with sales ramp up in H2 |
coverage including big 3 plans1 | ||
Comprehensive | 100% target prescribers reached2,3 | Drive breadth, depth of HCP adoption, increase |
engagement | F2F engagement with pandemic recovery | |
Broad adoption | ~1000 patients treated in 1st 4 months | Intensify patient activation through DTC and |
of launch4, 17% naive5 | omni-channel engagement | |
Positive customer | 80% of free Rx filled within 1 week average6 | Maintain and build |
experience | ||
Pandemic impact | Restricted F2F interactions, patient visits down | Impact expected to continue into H1 |
on MS Market | 15-20%7, NBRx down 12%8 |
1. United Healthcare & OptumRx, CVS Caremark & Aetna, ESI Commercial, based on payer wins & associated lives covered 2. Internal target list having at least one contact through the hybrid F2F/ digital model 3. IQVIA BrandImpact. MS Promotional Activities Analysis. Neurology Panel. End Nov 2020. 4. IQVIA National Prescription Audit NBRx data through W/E 01/01 5. Based on start forms. 6. Novartis HUB and Direct to Specialty Pharmacy data 7. Symphony APLD Jan-Oct 2020 vs PY 8. Internal estimate; IQVIA Weekly NBRx w/e 20-Mar 2020 to 1-Jan 2021 vs PY
20 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Leqvio®: Expect gradual ramp in Europe, building foundation to be stronger out of the gate upon approval in US
Progressing approvals
EC approval December 2020
Completed 10 additional filings in RoW
CRL response plan submission Q2/Q3 2021
Preparing launches
First prescriptions expected February with initial focus on population at high CV risk
On track for Q3 2021 launch in collaboration with NHS England
Advancing infrastructure building with healthcare systems
21 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Focus in 2021 will be launches and preparing for next big bets, building on the strong foundation
Maximize growth drivers
Cosentyx®: deliver nr-axSpA launch, LCM addressing 7m additional patients
Entresto®: drive penetration in CN, launch in JP, expansion into pEF and post-AMI
Deliver on new launches
Kesimpta®: enable broad HCP adoption, fast and simple access
Leqvio®: establish broad and affordable access, addressing adherence, system barriers
Beovu®: restore evidence-based confidence to treat, expansion into
DME/ RVO
Next wave of launches
Iptacopan: educate on complement- driven renal and hematologic diseases
Iscalimab: create awareness on unmet need
Ligelizumab: leverage dermatology / allergology footprint to expand use of biologics in CSU
Pelacarsen: drive Lp(a) awareness and standard protocol testing
Branaplam: drive biomarkers awareness and engage HD community on early treatment
Nr-axSpA - non-radiographic axial spondyloarthritis, pEF - preserved ejection fraction, post-AMI - post acute myocardial infarction, DME - diabetic macular edema, RVO - retinal vein occlusion, Lp(a) - lipoprotein (a)
22 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Susanne Schaffert
President, Novartis Oncology
23 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Continued momentum on growth drivers and launches more than offset generic erosion in Q4 and FY 2020
Oncology net sales | |||
USD bn, % cc | |||
Recent launches1 | |||
Growth drivers2 | |||
Base business4 | |||
Gx3 | |||
+3% | |||
14.4 | .2 | 14.7 | |
1.3 | 2.1 | ||
0.2 | |||
3.9 | +29% | ||
4.6 | |||
3.7 | 0.9 | ||
3.6 | |||
-1% | |||
5.5 | 4.4 | -19% | |
FY 2019 | FY 2020 |
FY 2020: Net sales up +3%
- Growth drivers and launches up +29%, despite pandemic conditions
- Embarked on virtual launches with Adakveo® and Tabrecta®
Q4: Continued momentum of growth drivers and recent launches
- Promacta® / Revolade® (USD 471m, +23%)
- Jakavi® (USD 376m, +24%)
- Tafinlar® + Mekinist® (USD 408m, +13%)
- Kymriah® (USD 141m, +42%)
- Kisqali® (USD 184m, +18%)
Q4: Offset Gx erosion, COVID-19 impact
- Generic impact mainly from Afinitor®, Exjade®
- Lutathera® (USD 109m, +1% cc) continued impact from COVID-19
All growth rates in cc. 1. Recent launches include Kisqali®, Lutathera®,Kymriah®, Piqray®, Adakveo® , Tabrecta® 2.Growth drivers include Promacta®/Revolade®, Tafinlar®+ Mekinist®, Jakavi® (marketed by Novartis ex-US) 3. Gx include Afinitor®, Exjade®, Glivec® and Sandostatin® 4. Base business - other brands
24 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Kisqali®: Strong growth as the only CDK4/6i proven to extend lives in two Ph3 trials
Sales evolution | |
USD m, % cc | |
Ex-US | |
US | |
+45% | |
687 | |
480 | 369 |
230 | |
250 | 318 |
FY 2019 | FY 2020 |
- Delivered strong growth (+45% cc) and market share gains (+4.8%1) in 2020, driven by unprecedented overall survival (OS) benefit from 2 pivotal Ph3 trials
- CDK4/6i market still depressed due to COVID-19 (NBRx down -15% vs. PY)
- Longest median OS among all Ph3 trials in aBC: nearly 5 years in pre- menopausal patients
- Pooled data from MONALEESA studies presented at SABCS confirmed efficacy across luminal and ET resistant HER2-E patient subtypes
- Ability to selectively and preferentially inhibit CDK4 may restore endocrine sensitivity in these more aggressive tumors
- NATALEE adjuvant study in intermediate and high risk populations (70% of adjuvant patients) final readout expected 2022
1. Patient share, October YTD vs PY
25 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Kymriah®: Double-digit growth in all regions despite pandemic
Sales evolution
USD m, % cc
Ex-US
US
+68%
474
278269
119
159205
FY 2019 | FY 2020 |
- Sales grew +68% cc in 2020, despite COVID-19 impact on healthcare systems
- Continued to expand our global footprint:
- 27 countries covering at least 1 indication
- 290+ qualified treatment centers
- FBRI approved for commercial supply (previous approvals: Stein, Les Ulis)
- Increased manufacturing capacity ~70% vs. PY, improved process robustness
- New data at ASH:
- ELARA interim analysis showed Kymriah® is effective in extensively pre-treated patients with r/r FL: CR 65%, ORR 83%; submission expected 2021
- JULIET updated efficacy results showed continued durable responses for patients with r/r DLBCL: 2-yearprogression-free survival rate 33%
- Advanced our CAR-T pipeline (new indications) and CAR-T manufacturing platform
FBRI - Foundation for Biomedical Research and Innovation
26 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Asciminib: Superior efficacy and safety profile confirms potential to transform standard of care in CML
ASCEMBL is the 1st head-to-head pivotal trial vs. a 2nd generation TKI
Major Molecular Response rate at 24 weeks | ||||
25.5% | | |||
of patients | 13.2% | 1.9x1 | ||
n=1 | n=7 | |||
57 | 6 | | ||
% | ||||
Asciminib | Bosutinib |
Ph3 ASCEMBL study in 3L+ CML-CP: demonstrated statistically significant superiority in efficacy vs. bosutinib and a favorable safety profile
MMR rate at 24 weeks: 25.5% vs. 13.2% with bosutinib, meeting primary endpoint; consistent across most major demographic and prognostic subgroups
Complete Cytogenetic response at 24 weeks 40.8%
% of patients | 24.2% | 1.7x |
Asciminib | Bosutinib |
- AEs leading to dose adjustment or interruption with asciminib vs. bosutinib were 37.8% (n=156) and 60.5% (n=76), respectively; AEs leading to discontinuation were 5.8% and 21.1%, respectively
- Global regulatory submissions planned in H1 2021; further plans to expand into earlier lines underway
1. After adjusting for major cytogenetic response (MCyR) status at baseline. CML-CP, chronic myeloid leukemia-chronic phase; MMR, major molecular response; AE, adverse event;
27 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
2021: Maximize momentum for our growth drivers and new launches, while preparing for next big bets
Maximize growth drivers
Kisqali®: Continue strong growth driven by M3 and M7 OS data
Kymriah®: Drive growth in pALL and DLBCL, leveraging a differentiated profile and increased manufacturing capacity and reliability
Lutathera®: Unlock potential in community setting and grow use in earlier lines of treatment
Promacta®/Revolade®: Growth in ITP and uptake in 1L SAA in the US and Japan
Jakavi®: Continue strong growth in PV and MF, while launching GVHD
Tafinlar® + Mekinist®: leverage potential in adjuvant melanoma and NSCLC
Deliver on new launches
Piqray®: Maximize US launch momentum and expand further to EU markets
Adakveo®: Enable access in larger US accounts and continue global expansion
Tabrecta®: Maximize the first mover advantage as the first and only MET inhibitor available for patients in the US
Asciminib: Drive awareness of CML unmet need and the importance of STAMP and launch successfully in US
Next wave of launches
177Lu-PSMA: Further evolve commercial infrastructure for best-in-class launch
Canakinumab: Focus on medical education on pro-tumor inflammation
Sabatolimab: Build awareness of the dual mechanism of action of TIM-3 and its potential in MDS/AML
TNO155: Advance a first-in-class inhibitor of SHP2 across 5 combination trials in NSCLC and
CRC
LXH254: Advance potentially best-in-class B/C- RAF inhibitor in RAS/RAF mutant melanomas and lung cancers
28 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Expanding our pipeline with in-licensing of tislelizumab, a late-stagePD-1 inhibitor, from BeiGene1
Tislelizumab
Anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages
Strategic rationale
Accelerate IO market entry with backbone PD-1
- PD-1market expected to reach USD 63bn by 2026
- Tislelizumab already approved in China; 15 potentially registration-enabling clinical trials currently ongoing, with first ex-China filing expected in 2021
- Key indications include NSCLC, HCC, esophageal and gastric cancer
Unlock combination potential with Novartis assets
- Multiple combination opportunities identified with Novartis portfolio, across all 4 therapeutic platforms
- Open the door to potential broader strategic collaboration with BeiGene
Deal terms
- Upfront payment of USD 650m plus royalties and milestone payments
- Novartis obtains development and commercialization rights in ex-China regions2
- Closing expected H1 2021, deal subject to normal closing conditions1
1. Novartis signed a strategic collaboration agreement to in-license tislelizumab from BeiGene, Ltd. in major markets outside of China on January 11, 2021. Closing of the transaction is subject to receiving antitrust clearance. Until closing, BeiGene will continue to have control of tislelizumab 2. Ex-China regions include the US, Canada, Mexico, the EU, UK, Norway, Switzerland, Iceland, Liechtenstein, Russia, and Japan; BeiGene retains the rights to tislelizumab in China and other countries
29 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Harry Kirsch
Chief Financial Officer
Financial review and 2020 guidance
30 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
2020 financial results in line with guidance
As revised in Q3 investor call
Group full year guidance | FY 2020 vs. PY |
In cc
"Sales expected to grow mid single digit assuming return to normal prescription dynamics.
3 to 4% range in case of resurgence of COVID-19"
"Core operating income to grow low double digit to mid teens"
in cc
3%
13%
31 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Solid FY performance on top and bottom line
Continuing operations | 1 | Q4 | Change vs. PY | |||
USD million | 2020 | % USD | % cc | 2 | ||
Net Sales | 12,770 | 3 | 1 | |||
Core Operating income 2 | 3,501 | 1 | 2 | |||
Operating income | 2,644 | 45 | 51 | |||
Net Income | 2,099 | 86 | 93 | |||
Core EPS (USD) | 2 | 1.34 | 2 | 3 | ||
EPS (USD) | 0.92 | 84 | 93 | |||
Free Cash Flow | 2 | 3,342 | -4 | |||
FY | Change vs. PY | ||
2020 | % USD | % cc | 2 |
48,659 | 3 | 3 | |
15,416 | 9 | 13 | |
10,152 | 12 | 19 | |
8,071 | 13 | 20 | |
5.78 | 9 | 13 | |
3.55 | 14 | 21 |
11,691 -10
- Refers to continuing operations as defined on page 43 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz, as well as the continuing corporate functions
- Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 55 of the Condensed Financial Report
32 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Core margin expansion of +2.8% points FY
Innovative Medicines core margin at 35.0%
Continuing operations1,2
Q4 2020 | FY 2020 | ||||||||
Core operating | Core operating | ||||||||
Net sales | income | Core margin | Net sales | income | Core margin | ||||
change vs. PY | change vs. PY | Core margin | change vs. PY | change vs. PY | change vs. PY | Core margin | change vs. PY | ||
(in % cc) | (in % cc) | (%) | (%pts cc) | (in % cc) | (in % cc) | (%) | (%pts cc) | ||
Innovative Medicines | 1 | 3 | 31.4 | 0.7 | 4 | 11 | 35.0 | 2.2 | |
Sandoz | 0 | 3 | 20.8 | 0.8 | 0 | 15 | 24.2 | 3.3 | |
Continuing Operations | 1 | 2 | 27.4 | 0.4 | 3 | 13 | 31.7 | 2.8 | |
- Refers to continuing operations as defined on page 43 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz, as well as the continuing corporate functions
- Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 55 of the Condensed Financial Report
33 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
FY 2020 free cash flow decreased to USD 11.7bn
Continuing operations1 free cash flow2
USD billion
-10% | Key drivers vs. PY: | |||||||
+ | Higher operating income | |||||||
12.9 | ||||||||
11.7 | (adjusted for non-cash items) | |||||||
− Payments for legal matters | ||||||||
− | Lower divestment proceeds | |||||||
FY 2019 | FY 2020 |
- Refers to continuing operations as defined on page 43 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz, as well as the continuing corporate functions
- Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 55 of the Condensed Financial Report
34 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
2021 Novartis full year guidance
Barring unforeseen events; growth vs. PY in cc
Continuing operations | full year guidance1
vs. PY (cc)
Sales expected to grow low to mid single digit
- IM Division expected to grow mid single digit
- Sandoz expected to be broadly in line with prior year
Core operating income expected to grow mid single digit, ahead of sales
1. Key assumptions:
- Our guidance assumes that we see a return to normal global healthcare systems including prescription dynamics by mid 2021
- In addition, we assume that no Gilenya and no Sandostatin LAR generics enter in 2021 in the US
35 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Q1 2021 Sales expected to decline due to PY COVID-19 forward purchasing of approximately +0.4bn
Sales growth vs. PY
%pts, cc
13
3
2020
Actuals
Q1FY
Low to mid single digit
2021
Illustrative
Low to mid single digit
Q1 2020
0.4bn Forward purchasing (approximately 3%pts of Q1 growth)
Q1 2021
Broadly in line vs. PY excluding PY forward purchasing impact
Key assumption:
Return to normal prescription dynamics of healthcare systems by mid 2021
36 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Expecting margin expansion with Core OpInc growth magnitude lower in 2021 driven by increased launch investments
2021 pushes and pulls
+
Growth drivers and Kesimpta® launch uptake benefiting from return to normal global healthcare systems by early H2
Productivity programs
Continue new ways of working
-
Increased investments
in launches and pre-launches, including Kesimpta® and Leqvio®
Increased development costs
tislelizumab1
Increased investments into growth drivers assuming physician access normalizes as of mid year
1. Novartis signed a strategic collaboration agreement to in-license tislelizumab from BeiGene, Ltd. in major markets outside of China on January 11, 2021. Closing of the transaction is subject to receiving antitrust clearance. Until closing, BeiGene will continue to have control of tislelizumab
37 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
FY 2021 Guidance on other financial KPIs
Barring unforeseen events; growth vs. PY in cc
Continuing operations1 | full year guidance
vs. PY (cc)
Core Net | Expenses expected to be broadly in line vs. 2020 |
Financial Result | |
Core Tax Rate | 2021 Core tax rate expected to be around 16% |
1. Refers to continuing operations as defined on page 43 of the Condensed Financial Report, excludes Alcon, includes the businesses of Innovative Medicines and Sandoz, as well as the continuing corporate functions
38 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Novartis proposes 24th consecutive dividend increase to the AGM: 3.00 CHF / share1
3.5
3.0 | CHF dividend | USD dividend |
2.5
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
2020 dividend yield 3.6%2
2020 dividend growth 1.7%3
CHF 2.95 USD 3.12 | CHF 3.00 USD 3.404 |
2016 2017 2018 2019 2020
1. | Proposal to shareholders at the 2021 Annual General Meeting, taking place on March 2, 2021 2. Based on closing share p rice of CHF 83.65 at end of business year 2020 (December 30, 2020) 3. In CHF |
4. | Converted at historic exchange rates at the dividend payment dates as per Bloomberg for 2020, assumes an exchange rate of USD/CHF of 0.88106 as of December 31, 2020 |
39 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Expected currency impact for full year 2021
Currency impact vs. PY
%pts, assuming late-January exchange rates prevail in 2021
FX impact on Net sales | |||
2 | 4 | 3 to 4 | |
0 | |||
Q4 | FY | Q1 | FY |
20202021
Actual Simulation
FX impact on Core operating income
2 3
-1-4
Q4 FY Q1 FY
20202021
40 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Vas Narasimhan
Chief Executive Officer
41 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Made significant strides in building trust with society and in integrating ESG across every aspect of our company
Key ESG developments over the past 12 months and selected aspirations
Ethical | Pricing | Global Health | Corporate | Governance & | ||||
Standards | & Access | Challenges | Citizenship | Transparency | ||||
Resolved material legacy | Introduced 100+ | Expanded our Africa sickle | Strong COVID-19 response | ESG indexlaunched | |||
compliance issues | Emerging Market Brands | cell disease program | Integrating D&I efforts | Reporting in line with TCFD | |||
Launched Code of Ethics | Issued sustainability- | Advancing pipeline of | across our operations | Working towards integrated | |||
linked bond | novel malaria treatments | reporting | |||||
Select 200% increase in SITs | 50% increase in our | Full carbon, plastic and | ESG targets embedded into | ||||
commitments patient reach by 20251 | Flagship Programs' reach | water neutrality by 2030 | executive remuneration | ||||
by 20251 |
Significant improvements recognized by third party ESG rating agencies
SIT - Strategic innovative therapies 1. As defined in ESG bond prospectus More details in Novartis in Society Report: www.novartis.com/nisreport2020
42 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Confident that we will grow top and bottom line every year to 2025 and meet external expectations
Analyst consensus sales | ||||||
USD billion | ||||||
+4% CAGR | ||||||
60 | ||||||
49 | ||||||
2020 | Growth Drivers1 | Launches2 | Other/Pipeline | Sandoz | Gx Brands3 | 2025 |
Actual | Consensus | |||||
IM margin | ||||||
IM margin | ||||||
External expectations based | ||||||
35.0% | ||||||
on analyst consensus | ||||||
37.6% | ||||||
1. Cosentyx®, Entresto®, Zolgensma®, Kisqali®, Mayzent®, Tafinlar+Mekinist®, Jakavi®, Beovu®, Xiidra®, Aimovig®, Xolair®. 2. Lutathera®, Kymriah®, Piqray®, Adakveo®, Kesimpta®, Leqvio®, Tabrecta®, Asciminib. 3. Brands with 2024 | ||||||
consensus sales lower than 2019 actual sales (Glivec®, Tasigna®, Afinitor®, Votrient®, Promacta®, Exjade®, Sandostatin®, Galvus®, Gilenya®, Lucentis®). | Source: Novartis Investor Relations in-house consensus as of November 12, 2020. |
43 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Conclusion
Delivered on our strategic and operational commitment in 2020
Third successive year of increases in sales, core operating income and margin
Progressing the pipeline and key approvals in 2020: Kesimpta® in US, Leqvio®, Zolgensma® in EU, Tabrecta® in US, new indications for Cosentyx®
Expecting top and bottom line growth every year through 2025
44 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Appendix
Strong FY operational performance from growth drivers
Key growth driver sales FY 2020
Sales | Growth vs. PY | Growth vs. PY | |
USD Million | USD Million | cc | |
2,497 | 771 | 44% | |
920 | 559 | 151% | |
3,995 | 444 | 13% | |
1,738 | 322 | 23% | |
1,339 | 225 | 20% | |
687 | 207 | 45% | |
1,542 | 204 | 16% | |
320 | 204 | 176% | |
873 | 202 | 31% | |
474 | 196 | 68% | |
376 | 184 | 95% | |
190 | 155 | nm | |
nm - not meaningful
Key growth drivers and launches, as % of Innovative Medicines sales
49%
Adakveo® | ||||||||
Mayzent® | ||||||||
40% | Beovu® | |||||||
Piqray® | ||||||||
Xiidra® | ||||||||
32% | Lutathera® | |||||||
26% | Kymriah® | |||||||
Kisqali® | ||||||||
Ilaris® | ||||||||
Zolgensma® | ||||||||
Jakavi® | ||||||||
Tafinlar+Mekinist® | ||||||||
Promacta® | ||||||||
Entresto® | ||||||||
Cosentyx® | ||||||||
FY 2017 FY 2018 FY 2019 | FY 2020 | |||||||
Other1 | ||||||||
1. Includes Tasigna®, Xolair®, Aimovig®, Tabrecta®, Luxturna® , Kesimpta®, Enerzair ® and Atectura®
46 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Net debt increased by USD 8.6bn mainly due to
The Medicines Company acquisition
-8.6 | ||||||
-15.9 | ||||||
-7.0 | -2.1 | -1.2 | -24.5 | |||
-10.0 | 11.7 | |||||
Dec. 31, 2019 | Dividends | M&A | Free Cash Flow2 | Treasury share | Others | Dec. 31, 2020 |
transactions1 | transactions, net |
1. Mainly the acquisition of The Medicines Company for USD 9.6bn. 2. Free cash flow is a non-IFRS measure. An explanation of non-IFRS measures can be found on page 55 of the Condensed Financial Report.
47 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Advancing our innovation agenda over the last 3 years
Major
approvals1
Major
submissions2
Major
readouts (pivotal)
2018 | 2019 | 2020 | ||||||||||
DLBCL | SPMS | HR+ BC | Sickle cell | Relapsing MS | NSCLC | Hyperlipidemia | ||||||
Migraine | ||||||||||||
Lutathera® | ||||||||||||
Adj Melanoma | NET | SMA | nAMD | Asthma | Nr-axSpA | |||||||
1st line SAA | ||||||||||||
SPMS | HR+ BC | nAMD | NSCLC | asthma | HFpEF | Hyperlipidemia | ||||||
SMA | 1st line SAA | Sickle cell | Relapsng MS | Nr-axSpa | ||||||||
Asciminib | ||||||||||||
Advanced BC | HR+ BC | nr-axSpA | Relapsing MS | HFpEF | Chronic GvHD | CML 3L | DME | |||||
Fevipiprant | ||||||||||||
NSCLC | SMA presymptomatic | Asthma | Follicular lymphoma | |||||||||
1. First approval in any market. 2. First submission in any market.
48 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
2020 pipeline milestones
H1 2020 | H2 2020 | ✓ Achieved | ✕ Missed | |||||
Regulatory | Beovu® | nAMD (EU/JP) | ✓ | Adakveo® | Sickle cell disease (EU) | ✓ | ||
Cosentyx® | nr-axSpA (EU/US) | ✓ | Tabrecta® (capmatinib) | NSCLC (US/JP) | ✓ | |||
decisions and | ||||||||
Cosentyx® | AS (CN) | ✓ | Cosentyx® | Pediatric psoriasis (EU) | ✓ | |||
opinions | ||||||||
Kesimpta® | Relapsing MS (US) | ✓ | Cosentyx® | nr-axSpA (JP) | ✓ | |||
(H2 2020) | ||||||||
Piqray® | HR+/HER2- aBC with PIK3CA | ✓ | Entresto® | HFpEF (US) | Q1 20212 | |||
mutation (EU) | ||||||||
Enerzair® | Asthma (EU/JP) | ✓ | Leqvio (inclisiran) | Hyperlipidemia (US) | ✕4 | |||
Tafinlar® & Mekinist® | Adjuvant melanoma (CN) | ✓ | Hyperlipidemia (EU) | ✓ | ||||
Xiidra® | DED (EU) | ✕ | Xolair® | Nasal Polyposis (US/EU) | ✓ | |||
Zolgensma® IV | SMA (EU/JP) | ✓ | ||||||
Major | Entresto® | HFpEF (US) | ✓ | Alpelisib (BYL719) | PROS (US) | H2 2021 | ||
Inclisiran (KJX839) | Hyperlipidemia (EU) | ✓ | AVXS-101 IT | SMA (US) | ✕3 | |||
expected | ||||||||
Cosentyx® | Juvenile PsA / enthesitis-related | |||||||
submissions | Q2 2021 | |||||||
arthritis (US/EU) | ||||||||
Spartalizumab (PDR001) | Metastatic melanoma (US/EU) | Negative | ||||||
and Tafinlar® & Mekinist® | read-out | |||||||
177Lu-PSMA-617 | mCRPC (US) | Q4 2021 | ||||||
Major | Entresto® | Post-acute MI1 | ✓ | Asciminib (ABL001) | CML 3L | ✓ | ||
Tropifexor (LJN452) | NASH | ✓ | Beovu® | DME | ✓ | |||
expected trial | ||||||||
UNR844 | Presbyopia | ✓ | Jakavi® | chronic GVHD | ✓ | |||
readouts* | ||||||||
Kisqali® | aBC (MONALEESA-2 OS) | Q4 2021 | ||||||
177Lu-PSMA-617 | mCRPC | H1 2021 | ||||||
*Achieved = on-time readout of data, irrespective of trial outcome | 1. Planned study readout 2021; preplanned DMC interim analysis readout completed in March 2.Received positive FDA Advisory Committee recommendation for use in | |||||||
patients with HFpEF Dec 2020 | 3. FDA recommended pivotal confirmatory study to supplement existing STRONG data | 4. Novartis received a CRL from the FDA due to unresolved facility inspection-related conditions at a third-party | ||||||
manufacturing facility in Europe. FDA has not raised any concerns related to the efficacy or safety of inclisiran. Response to CRL planned to be submitted Q2 - Q3 2021 |
49 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
2021 key pipeline milestones1
H1 2021 | H2 2021 | ✓ Achieved ✕ Missed |
Regulatory decisions and opinions
Major expected submissions
Major expected trial readouts*
Entresto® | HFpEF (US) | Cosentyx® | Pediatric psoriasis (US / CN / JP) | ||
Kesimpta® | Relapsing MS (EU / JP) | ||||
Leqvio® | Hyperlipidemia (US)2 | Asciminib (ABL001) | CML 3L (JP) | ||
Jakavi® | Acute and chronic GvHD (EU, JP) | Beovu® | DME (JP) | ||
Tabrecta® | NSCLC (EU) | Alpelisib (BYL719) | PROS (US) | ||
Beovu® | DME (US / EU) | Kymriah® | r/r Follicular lymphoma (US / EU / JP) | ||
Asciminib (ABL001) | CML 3L (US /EU) | ||||
Iptacopan (LNP023) | Ph2 - IgAN | Canakinumab (ACZ885) | Ph3 - NSCLC 1L | ||
Iptacopan (LNP023) | Ph2 - C3G | ECF843 | Ph2 - Dry eye | ||
Entresto® | Ph3 - Post-AMI | Ligelizumab (QGE031) | Ph3 - CSU3 | ||
Canakinumab (ACZ885) | Ph3 - NSCLC 2L | Kisqali® | aBC (MONALEESA-2 OS) | ||
177Lu-PSMA-617 | Ph3 - mCRPC | Remibrutinib (LOU064) | Ph2 - CSU | ||
Cosentyx® | Ph3 - Juvenile PsA | Cosentyx® | Ph3 - HS | ||
Sabatolimab (MBG453) | Ph2, MDS | ||||
Kymriah® | Ph3, r/r DLBCL 1st relapse |
*Achieved = on-time readout of data, irrespective of trial outcome
1. 2021 Key milestone table may evolve based on read-out outcomes as well as BD&L activities 2. Novartis received a CRL from the FDA due to unresolved facility inspection-related conditions at a third-party manufacturing facility in Europe. FDA has not raised any concerns related to the efficacy or safety of inclisiran. Response to CRL planned to be submitted Q2 - Q3 2021 3. Q4/2021-Q1/2022 potential COVID impact
50 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
COVID-19 outlook for 2021
Therapeutic areas with highest potential for ongoing COVID-19 impact in 2021
Ophthamology | Respiratory Diseases |
Kidney Disease /
TransplantDermatology
To date, minimal impact (<3 months) expected for majority of regulatory submissions through 20251
Uncertainty remains about FDA's ability to conduct foreign
manufacturing inspections
Robust mitigations in place for clinical trial execution:
- COSSET (COVID-19Site Surveillance Tool): predictive tool for proactively adapting to dynamic COVID-19 impacts at site level
- > 35k remote monitoring visits since March
- > 2k direct-to-patient medical delivery shipments
- < 24 hours to detect / respond to site level actions
- Active home nursing and virtual safety assessments
- Novartis Digital Recruitment (NDR) implemented for new trial starts
1. As of end of December 2020
51 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Our pipeline projects at a glance
Phase 1/2 | Phase 3 | Registration | Total | |
ONCOLOGY | 51 | 22 | 0 | 73 |
PHARMACEUT ICALS | 67 | 21 | 5 | 93 |
Cardiovascular, Renal, Metabolism | 12 | 4 | 2 | 18 |
Immunology, Hepatology, Dermatology | 27 | 9 | 1 | 37 |
Neuroscience | 7 | 2 | 1 | 10 |
Ophthalmology | 6 | 3 | 0 | 9 |
Respiratory | 9 | 2 | 0 | 11 |
Global Health | 6 | 1 | 1 | 8 |
BIOSIMILARS | 0 | 1 | 0 | 1 |
Total | 118 | 44 | 5 | 167 |
52 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Novartis submission schedule
New Medical Entities: Lead and supplementary indications
2021 | 2022 | 2023 | 2024 | ≥2025 | ||||||||||||||||
177Lu-PSMA-617 | Lead | ligelizumab | Lead | ECF843 | Lead | Icenticaftor | Lead | 177Lu-NeoB | Lead | iscalimab | Lead | NIS793 | Lead | |||||||
AAA617 | QGE031 | Dry eye | QBW251 | AAA603 | CFZ533 | Solid tumors | ||||||||||||||
mCRPC 3L | CSU | COPD | Multiple Solid Tumors | Renal Tx | ||||||||||||||||
LEAD INDICATIONS
asciminib | Lead | iptacopan |
ABL001 | LNP023 | |
CML 3L | PNH | |
sabatolimab | Lead | |
MBG453 | ||
HR-MDS | ||
Lead | SAF312 | Lead | 177Lu-PSMA-R2 | Lead | ianalumab | Lead | OAV201 | Lead |
COSP | AAA602 | VAY736 | AVXS-201 |
Prostate cancer | Sjögren's syndrome | Rett syndrome |
UNR844 | Lead | CEE321 | Lead | LMI070 | Lead | pelacarsen | Lead | ||
Presbyopia | Atopic Dermatitis | Huntington's disease | TQJ230 | ||||||
CVRR-Lp(a) | |||||||||
cipargamin | Lead | LNA043 | Lead | remibrutinib | |||||
KAE609 | Osteoarthritis | LOU064 | |||||||
Malaria severe | CSU | ||||||||
CPK850 | Lead | LXE408 | Lead | spartalizumab | Lead | ||||
RP | Visceral leishmaniasis | PDR001 | |||||||
Malignant melanoma (combo) | |||||||||
CSJ117 | Lead | LXH254 | Lead | TNO155 | Lead | ||||
Asthma | Solid tumors | Solid tumors | |||||||
tropifexor&cenicriviroc | |||||||||
ganaplacide | Lead | mavoglurant | Lead | Lead | |||||
KAF156 | AFQ056 | LJC242 | |||||||
NASH | |||||||||
Malaria uncomplicated | Cocaine use disorder | ||||||||
gevokizumab | Lead | MIJ821 | Lead | ||||||
VPM087 | Depression | ||||||||
1st line CRC / 1st line RCC | |||||||||
INDICATIONS | canakinumab | LCM | 177Lu-PSMA-617 | |
ACZ885 | AAA617 | |||
NSCLC 2L | Pre-taxane | |||
canakinumab1) | LCM | canakinumab | ||
ACZ885 | ACZ885 | |||
NSCLC 1L | Adjuvant NSCLC | |||
iptacopan | ||||
LNP023 | ||||
C3G | ||||
NEW | iptacopan | |||
LNP023 | ||||
IgAN | ||||
1. Depending on timing of final read-out submission may move to early 2022.
LCM | crizanlizumab | LCM | cipargamin | LCM | iptacopan | LCM | LMI070 | LCM | ||||
SEG101 | KAE609 | LNP023 | SMA | |||||||||
Sickle cell anaemia with crisis ped | Malaria uncomplicated | aHUS | ||||||||||
LCM | ligelizumab | LCM | iscalimab | LCM | ianalumab | LCM | remibrutinib | LCM | ||||
QGE031 | CFZ533 | VAY736 | LOU064 | |||||||||
CINDU | Liver Tx | AIH | Sjögren's syndrome | |||||||||
LCM | sabatolimab | LCM | iscalimab | LCM | ligelizumab | LCM | tropifexor&licogliflozin LCM | |||||
MBG453 | CFZ533 | QGE031 | LJN452 | |||||||||
Unfit AML | Sjögren's syndrome | Food allergy | NASH (combos) | |||||||||
LCM | iptacopan | LCM | ||||||||||
LNP023 | ||||||||||||
iMN | ||||||||||||
53 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Novartis submission schedule
Supplementary indications for existing brands
20211) | 2022 | 2023 | 2024 | ≥2025 |
alpelisib, BYL719 | LCM | Cosentyx | LCM | Cosentyx | LCM | Coartem | LCM | Aimovig | LCM | Jakavi | LCM | Mayzent | LCM |
PROS | secukinumab, AIN457 | secukinumab, AIN457 | artemether + lumefantrine, CCA566 | erenumab, AMG334 | ruxolitinib, INC424 | siponimod, BAF312 |
PsA IVIV | AS IVIV | Malaria uncompl., formula for <5kg | Pediatric Migraine | Myelofibrosis (combination) | Pediatric MS |
Beovu | LCM | Cosentyx | LCM | Beovu | LCM | Cosentyx | LCM | Cosentyx | LCM | Jakavi | LCM | Piqray | LCM |
brolucizumab, RTH258 | secukinumab, AIN457 | brolucizumab, RTH258 | secukinumab, AIN457 | secukinumab, AIN457 | ruxolitinib, INC424 | alpelisib, BYL719 |
DME | AS H2H | Diabetic retinopathy | GCA | Lichen Planus | Pediatrics Chronic GVHD | HNSCC 2/3L |
Cosentyx | LCM | Cosentyx | LCM | Beovu | LCM | Jakavi | LCM | Cosentyx | LCM | Kymriah | LCM | Piqray | LCM |
secukinumab, AIN457 | secukinumab, AIN457 | brolucizumab, RTH258 | ruxolitinib, INC424 | secukinumab, AIN457 | tisagenlecleucel, CTL019 | alpelisib, BYL719 |
Juvenile idiopathic arthritis | Hidradenitis suppurativa | RVO | Pediatrics Acute GVHD | Lupus Nephritis | 1L high risk ALL, pediatrics & young adults | HER2+ adv BC |
Entresto
sacubitril/valsartan, LCZ696 Post-AMI
LCM | Entresto EU3) | LCM | denosumab | BioS | Tafinlar + Mekinist | LCM | leqvio | LCM |
sacubitril/valsartan, LCZ696 | GP2411 | dabrafenib + trametinib, DRB436 | KJX839 |
Pediatric HF | anti RANKL mAb | Thyroid cancer | CVRR-LDLC |
Jakavi | LCM | Promacta | LCM | Kisqali | LCM | Tabrecta | LCM |
ruxolitinib, INC424 | eltrombopag, ETB115 | ribociclib, LEE011 | capmatinib, INC280 |
Chronic GVHD | Radiation sickness syndrome | HR+/HER2- BC (adj) | Solid tumors |
Jakavi | LCM | Promacta | LCM | Lutathera | LCM | ||
ruxolitinib, INC424 | eltrombopag, ETB115 | 177Lu-oxodotreotide2) | |||||
Acute GVHD | Food effect free formulation | GEP-NET 1L G3 | |||||
Kymriah | LCM | Tafinlar + Mekinist | LCM | Piqray | LCM | ||
tisagenlecleucel, CTL019 | dabrafenib + trametinib, DRB436 | alpelisib, BYL719 | |||||
r/r DLBCL 1st relapse | HGG/LGG - Pediatrics | TNBC | |||||
Kymriah | LCM | Xolair | LCM | Piqray | LCM | ||
tisagenlecleucel, CTL019 | omalizumab, IGE025 | alpelisib, BYL719 | |||||
r/r Follicular lymphoma | Food allergy | Ovarian cancer | |||||
Xolair | LCM | ||||||
omalizumab, IGE025 | |||||||
Auto-injector | |||||||
1. OAV101 (AVXS-101) IT filing timelines TBC based on HA feedback, preclinical studies to address partial clinical hold are on track 2. 177Lu-dotatate in US 3. Approved in US
54 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Novartis pipeline in registration
1 lead indications
Lead indication
Immunology, Hepatology, Dermatology | Cardiovascular, Renal, Metabolism | ||||||||||||
Code | Name | Mechanism | Indication(s) | Code | Name | Mechanism | Indication(s) | ||||||
AIN457 | Cosentyx® | IL17A inhibitor | 300 mg AI | KJX839 | Leqvio® | siRNA (regulation of LDL-C) | Hyperlipidemia2) | ||||||
LCZ696 | Entresto® | Angiotensin receptor/neprilysin | HFpEF | ||||||||||
inhibitor |
Neuroscience | Global Health | ||||||||||||
Code | Name | Mechanism | Indication(s) | Code | Name | Mechanism | Indication(s) | ||||||
OMB157 | ofatumumab | CD20 antagonist | r MS1) | LAM320 | Lamprene® | SMPD1 inhibitor | Tuberculosis3) |
1. Approved in US as Kesimpta® 2. Novartis received a CRL from the FDA due to unresolved facility inspection-related conditions at a third-party manufacturing facility in Europe, FDA has not raised any concerns related to the efficacy or safety of inclisiran. Response to CRL planned to be submitted Q2 - Q3 2021. 3. WHO Pre-Qualification
55 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Novartis pipeline in Phase 3
Oncology
Code | Name | Mechanism | Indication(s) | ||||
AAA617 | 177Lu-PSMA-617 | Targeted radioligand therapy | mCRPC | mCRPC pre-taxane | |||
AAA6011) | Lutathera® | Targeted radioligand therapy | GEP-NET 1L G3 | ||||
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 | ||
lymphoma | ALL, pediatrics | relapse | |||||
and young | |||||||
adults | |||||||
DRB436 | Tafinlar® + | BRAF inhibitor + MEK inhibitor | Thyroid cancer | ||||
Mekinist® | |||||||
ETB115 | Promacta® | Thrombopoietin receptor (TPO-R) | Radiation sickness syndrome | Food effect free formulation | |||
agonist | |||||||
INC280 | capmatinib | Met inhibitor | NSCLC EU2) | ||||
INC424 | Jakavi® | JAK1/2 inhibitor | Acute GVHD | Chronic GVHD | |||
LEE011 | Kisqali® | CDK4 Inhibitor | HR+/HER2- BC (adj) | ||||
MBG453 | sabatolimab | TIM3 antagonist | HR-MDS |
Immunology, Hepatology, Dermatology
Code | Name | Mechanism | Indication(s) | |||
AIN457 | Cosentyx® | IL17A Inhibitor | Lupus Nephritis | Juvenile idiopathic arthritis | AS H2H | |
IV regimen in PsA | IV regimen in AS | HS | ||||
QGE031 | ligelizumab | IgE Inhibitor | CSU | CINDU | Food allergy |
Ophthalmology
Code | Name | Mechanism | Indication(s) | ||
RTH258 | Beovu® | VEGF Inhibitor | Diabetic retinopathy | RVO | DME |
1. 177Lu-dotatate in US 2. Approved in US & JP 3. Approved in US
6 lead indications
Lead indication
Neuroscience
Code | Name | Mechanism | Indication(s) | |||
AMG334 | Aimovig® | CGRPR antagonist | Ped Migraine | |||
BAF312 | Mayzent® | S1P1,5 receptor modulator | Ped MS |
Respiratory Disease
Code | Name | Mechanism | Indication(s) | |||
IGE025 | Xolair® | IgE inhibitor | Food allergy | Auto-injector |
Cardiovascular, Renal, Metabolism
Code | Name | Mechanism | Indication(s) | |||
KJX839 | Leqvio® | siRNA (regulation of LDL-C) | CVRR-LDLC | |||
LCZ696 | Entresto® | Angiotensin receptor/neprilysin | Post-AMI | Pediatric HF3) | ||
inhibitor | ||||||
TQJ230 | pelarcasen | ASO targeting Lp(a) | CVRR-Lp(a) |
Global Health
Code | Name | Mechanism | Indication(s) |
COA566 | Coartem® | - | Malaria uncomplicated, new formulation <5kg patients |
Biosimilars
Code | Name | Mechanism | Indication(s) | ||
GP2411 | denosumab | anti RANKL mAb | Denosumab BioS |
56 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Novartis pipeline in Phase 2
Oncology
Code | Name | Mechanism | Indication(s) | ||||||
BYL719 | alpelisib | PI3Kα inhibitor | PROS | ||||||
BLZ945 | BLZ945 | CSF-1 Inhibitor | Solid tumors | ||||||
DRB436 | Tafinlar® + Mekinist® | BRAF inhibitor + MEK inhibitor | HGG/LGG - Pediatrics | ||||||
INC280 | capmatinib | Met inhibitor | Solid tumors | NSCLC | NSCLC | ||||
(Combo) | (Combo) | ||||||||
INC424 | Jakavi® | JAK1/2 inhibitor | Myelofibrosis (combination) | Pediatrics acute | Pediatrics | ||||
GVHD | chronic GVHD | ||||||||
LXH254 | LXH254 | cRAF inhibitor | Melanoma (combo) | ||||||
MBG453 | sabatolimab | TIM3 antagonist | Unfit AML | ||||||
NIR178 | NIR178, spartalizumab | Ad2AR inhibitor, PD1 inhibitor | Cancers | ||||||
NIS793 | NIS793 | TGFB1 inhibitor | Pancreatic cancer | ||||||
PDR001 | spartalizumab | PD1 inhibitor | Metastatic melanoma (combo) | ||||||
SEG101 | crizanlizumab | P-selectin Inhibitor | Ped sickle cell anaemia with crisis |
Immunology, Hepatology, Dermatology
Code | Name | Mechanism | Indication(s) | ||||
ADPT02 | ADPT02 | - | NASH (Combos) | ||||
AIN457 | Cosentyx® | IL17A inhibitor | GCA | Lichen planus | |||
CFZ533 | iscalimab | CD40 inhibitor | Renal Tx | Sjögren's | HS | Liver Tx | |
LJC242 | tropifexor & cenicriviroc | FXR agonist, CCR2 inhibitor | NASH (combos) | ||||
LJN452 | tropifexor & licogliflozin | FXR agonist | NASH (combos) | ||||
LNA043 | LNA043 | ANGPTL3 agonist | Osteoarthritis | ||||
LOU064 | remibrutinib | BTK inhibitor | CSU | Sjögren's | |||
LRX712 | LRX712 | - | Osteoarthritis | ||||
LYS006 | LYS006 | Anti-inflammatory | Acne | Colitis ulcerative | HS | ||
MAS825 | MAS825 | - | NLRC4-GOF indications | ||||
VAY736 | ianalumab | BAFF-R inhibitor | Sjögrens | AIH | SLE |
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 |
1. Preclinical studies to address partial clinical hold are on track
31 lead indications | |||||||||||
Lead indication | |||||||||||
Neuroscience | |||||||||||
Code | Name | Mechanism | Indication(s) | ||||||||
BAF312 | Mayzent® | S1P1,5 receptor modulator | Stroke | ||||||||
BLZ945 | BLZ945 | CSF-1 Inhibitor | ALS | ||||||||
LMI070 | branaplam | mRNA splicing modulator | SMA | ||||||||
MIJ821 | MIJ821 | NR2B Inhibitor | Depression | ||||||||
OAV101 | AVXS-101 | Survival motor neuron (SMN) | SMA IT1) | ||||||||
gene therapy | |||||||||||
Respiratory Disease | |||||||||||
Code | Name | Mechanism | Indication(s) | ||||||||
CMK389 | CMK389 | IL-18 inhibitor | Pulmonary sarcoidosis | ||||||||
CSJ117 | CSJ117 | TSLP inhibitor | Asthma | ||||||||
DFV890 | DFV890 | - | COVID-19 related pneumonia | ||||||||
LOU064 | remibrutinib | BTK inhibitor | Asthma | ||||||||
MAS825 | MAS825 | - | COVID-19 related pneumonia | ||||||||
QBW251 | icenticaftor | CFTR potentiator | COPD | ||||||||
VAY736 | ianalumab | BAFF-R inhibitor | IPF | ||||||||
Cardiovascular, Renal, Metabolism | |||||||||||
Code | Name | Mechanism | Indication(s) | ||||||||
CFZ533 | iscalimab | CD40 inhibitor | Lupus nephritis | T1DM | |||||||
HSY244 | HSY244 | - | Atrial fibrillation | ||||||||
LCZ696 | Entresto® | Angiotensin receptor/neprilysin | nHCM | ||||||||
inhibitor | |||||||||||
LMB763 | nidufexor | FXR agonist | Diabetic nephropathy | ||||||||
LNP023 | iptacopan | CFB inhibitor | PNH | IgAN | C3G | iMN | aHUS | ||||
LTW980 | LTW980 | - | Hypertriglyceridemia | ||||||||
Global Health | |||||||||||
Code | Name | Mechanism | Indication(s) | ||||||||
AFQ056 | AFQ056 | mGluR5 Antagonist | Cocaine use disorder | ||||||||
KAE609 | cipargamin | PfATP4 inhibitor | Malaria severe | Malaria uncomplicated | |||||||
KAF156 | ganaplacide | - | Malaria uncomplicated | ||||||||
LXE408 | LXE408 | Protozoan inhibitor | Visceral leishmaniasis |
57 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Novartis pipeline in Phase 1 (1 of 2)
36 lead indications
Lead indication
Oncology
Code | Name | Mechanism | Indication(s) | |||
AAA603 | 177Lu-NeoB | Radioligand therapy target GRPR | Multiple solid tumors | |||
AAA602 | 177Lu-PSMA-R2 | Radioligand therapy target PSMA | Prostate cancer | |||
ADPT01 | ADPT01 | - | TNBC (combos) | Colorectal cancer (combos) | ||
ADPT03 | ADPT03 | BCL11A | Sickle cell anemia | |||
CSJ137 | CSJ137 | Growth factor inhibitor | Anaemia | |||
CTL019 | Kymriah® | CD19 CART | Lymphoma | |||
DKY709 | DKY709 + spartalizumab | Novel immunomodulatory agent | Cancers | |||
EGF816 | nazartinib + LXH254, ribociclib, capmatinib, opdivo, mekinist | EGFR inhibitor | NSCLC (combo) | |||
HDM201 | HDM201 + MBG453, venetoclax | MDM2 inhibitor | Haematological malignancy | |||
JBH492 | JBH492 | - | Haematological malignancy | |||
JEZ567 | JEZ567 | CD123 CART | AML | |||
KAZ954 | KAZ954 | - | Solid tumors | |||
LHC165 | LHC165 + spartalizumab | TLR7 agonist | Solid tumors | |||
LXF821 | LXF821 | EGFR CART | Glioblastoma multiforme | |||
LXH254 | LXH254 (combos) | cRAF inhibitor | Solid tumors | Solid tumors | ||
MAK683 | MAK683 | EED inhibitor | Cancers | |||
MCM998 | MCM998, LXG250 | BCMA CART, CD19 CART | Multiple myeloma | |||
MIK665 | MIK665 | MCL1 inhibitor | AML (combo) | |||
NIS793 | NIS793, spartalizumab | TGFB1 inhibitor | Solid tumors | |||
NIZ985 | NIZ985, spartalizumab | IL-15 agonist | Solid tumors | |||
NZV930 | NZV930, spartalizumab, NIR178 | CD73 antagonist | Solid tumors | |||
PDR001 | spartalizumab (combos) | PD1 inhibitor | AML | Solid tumors (combo) | ||
PHE885 | PHE885 | BCMA cell therapy | Multiple myeloma | |||
SQZ622 | SQZ622 | CD123xCD3 modulator | AML | |||
TNO155 | TNO155 | SHP2 inhibitor | Solid tumors (single agent) | Solid tumors (combo) | Solid tumors (combo) | |
VAY736 | ianalumab + ibrutinib | BAFF-R inhibitor | Haematological malignancy | |||
VOB560 | VOB560 | - | Cancers | |||
VPM087 | gevokizumab | IL1B Antagonist | CRC 1st line | |||
WNT974 | WNT974 + spartalizumab | Porcupine Inhibitor | Solid tumors | |||
WVT078 | WVT078 | - | Multiple myeloma | |||
YTB323 | YTB323 ± ibrutinib | CD19 CART | Haematological malignancy |
58 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Novartis pipeline in Phase 1 (2 of 2)
36 lead indications
Lead indication
Immunology, Hepatology, Dermatology
Code | Name | Mechanism | Indication(s) | |||
CEE321 | CEE321 | Pan JAK Inhibitor | AD | |||
DFV890 | DFV890 | - | Anti-inflammatory therapy | |||
FIA586 | FIA586 | - | NASH | |||
MHS552 | MHS552 | - | Autoimmune indications | |||
MHV370 | MHV370 | - | Sjögren's | SLE | ||
NGI226 | NGI226 | - | Tendinopathy |
Neuroscience
Code | Name | Mechanism | Indication(s) | |||
OAV201 | OAV201 (AVXS-201) | MECP2 gene therapy | Rett syndrome | |||
LMI070 | branaplam | mRNA splicing modulator | Huntington |
Ophthalmology
Code | Name | Mechanism | Indication(s) | ||
MHU650 | MHU650 | - | Diabetic eye diseases |
Respiratory Disease
Code | Name | Mechanism | Indication(s) | ||
LTP001 | LTP001 | - | Respiratory diseases | ||
NCJ424 | NCJ424 | - | Respiratory diseases |
Cardiovascular, Renal, Metabolism
Code | Name | Mechanism | Indication(s) | ||
MBL949 | MBL949 | - | Obesity related diseases |
Global Health
Code | Name | Mechanism | Indication(s) | ||
KAF156 | ganaplacide | - | Malaria prophylaxis |
59 Novartis Q4 Results | January 26, 2021 | 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 2b or later).
For further information on all Novartis clinical trials, please visit: www.novartisclinicaltrials.com
Cardiovascular, Renal and Metabolic
Entresto® - Angiotensin II 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 | Phase 3 |
Patients | 360 | 240 |
Primary Outcome | Part 1: Pharmacodynamics and pharmacokinetics of | Number of participants with Adverse Events (AEs) and |
Measures | sacubitril/valsartan LCZ696 analytes | Serious Adverse Events (SAEs) |
Part 2: Efficacy and safety compared with enalapril | ||
Arms/Intervention | • Part 1: Sacubitril/valsartan 0.8 mg/kg or 3.1 mg/kg or both; | • Single arm, open label sacubitril/valsartan (pediatric |
0.4 mg/kg or 1.6 mg/kg or both (single doses). | formulation granules (12.5, 31.25 mg in capsules); liquid | |
• Part 2: enalapril/placebo 0.2 mg/kg bid (ped. formulation | formulation (1mg/ml and 4mg/ml concentration) and | |
1mg/ml) and adult formulation (2.5, 5, 10 mg bid); | adult formulation (50, 100, 200 mg bid)) | |
Sacubitril/valsartan (LCZ696)/placebo: Ped. formulation | ||
granules (12.5, 31.25 mg in capsules); liquid formulation | ||
(1mg/ml and 4mg/ml concentration) and adult formulation (50, | ||
100, 200 mg bid) | ||
Target Patients | Pediatric patients from 1 month to < 18 years of age with heart | Pediatric patients with heart failure due to systemic left |
failure due to systemic left ventricle systolic dysfunction | ventricle systolic dysfunction who have completed study | |
CLCZ696B2319 | ||
Read-out Milestone(s) | 2022; (Analysis of 110 pts from Part 2 formed the basis for | 2022 |
pediatric submission in Apr-2019 and approval by the US FDA in | ||
Oct-2019 for the treatment of symptomatic HF with systemic left | ||
ventricular systolic dysfunction in children aged 1 year and older) | ||
Publication | TBD | TBD |
62 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Entresto® - Angiotensin II 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 |
Measures | Composite Score (GCCS) | cardiovascular (CV) death or heart failure (HF) hospitalization |
• Sacubitril/valsartan 50, 100, and 200 mg bid with placebo | • Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid/placebo of | |
Arms/Intervention | of valsartan | enalapril |
• Valsartan 40, 80, and 160 mg bid tablets with placebo for | • Enalapril 2.5 mg, 5 mg, 10 mg bid / placebo of | |
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 | |
Read-out Milestone(s) | 2022 | Primary: Q1-2019(actual); Extension (open-label):H1-2021 |
Publication | TBD | Submitted for Q4-2020: Primary manuscript in Circ J |
63 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Entresto® - Angiotensin II 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 | 2,572 | ||
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 baseline | |||
Measures | ||||
hospitalizations | to Week 24 | |||
• | Sacubitril/valsartan or placebo 50 mg, 100 mg, and 200 mg | • | Sacubitril/valsartan 50 mg, 100 mg and 200 mg bid and | |
matching placebo | ||||
Arms/Intervention | bid | |||
• | Enalapril 2.5 mg, 5 mg and 10 mg bid and matching 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 | |||
Read-out Milestone(s) | 2019 (actual) | 2019 (actual) | ||
• | Sep-2019: Primary manuscript (ARNI in HFpEF. Solomon S | |||
et al; NEJM. DOI: 10.1056/NEJMoa1908655) | • Study design (Wachter et al; ESC-HF),May-2020 | |||
• | Mar-2020: Published (NTproBNP, putative placebo analysis) | • | Primary data presented at ESC latebreaker, Aug-2020 | |
Publication | • Jun-2020: Submitted (renal outcomes, cognitive function) | • Baseline data publication in EJHF (expected publication Q4- | ||
• Q4-2020 Planned: Urgent HF visits, regional differences, | 2020), accepted Sep-2020 | |||
win ratio, adjudicated vs reported endpts; Subgroups (mode | • Planned Primary Publication High Tier Journal in H1-2021 | |||
of death, MRA, age, gender) | ||||
64 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Entresto® - Angiotensin II Receptor Neprilysin Inhibitor (ARNI)
Study | NCT02924727 PARADISE-MI (CLCZ696G2301) |
Indication | Post-acute myocardial infarction |
Phase | Phase 3 |
Patients | 5,670 |
Primary Outcome | Time to the first occurrence of a confirmed composite |
Measures | endpoint (cardiovascular (CV) death, heart failure (HF) |
hospitalization, or outpatient heart failure) | |
• Sacubitril/valsartan 50 mg, 100 mg, 200 mg bid; placebo | |
Arms/Intervention | for ramipril ; placebo for valsartan |
• Ramipril 1.25 mg, 2.5 mg, and 5 mg bid; placebo for | |
sacubitril/valsartan; placebo for valsartan | |
Target Patients
Read-out Milestone(s)
Publication
Post-AMI patients with evidence of LV systolic dysfunction and/or pulmonary congestion, with no known prior history of chronic HF
H1-2021
- Q4-2020- Planned: PARADISE-MI study design / baseline characteristics
- Planned primary data presentation and publication in H2- 2021
65 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
KJX839 - siRNA (regulation of LDL-C)
Study | NCT03060577 ORION-3 (CKJX839A12201E1) | NCT03705234 ORION-4 (CKJX839B12301) | ||
Hypercholesterolemia inc. Atherosclerotic Cardiovascular | Hypercholesterolemia inc. Heterozygous Familial | |||
Indication | Disease (ASCVD) and ASCVD risk equivalents Heterozygous | |||
Hypercholesterolaemia (HeFH) | ||||
Familial Hypercholesterolaemia (HeFH) | ||||
Phase | Phase 2 | Phase 3 | ||
Patients | 490 | ~15,000 | ||
LDL-C reduction at Day 210 for Group 1 subjects | A composite of major adverse cardiovascular events, defined as: | |||
• | Coronary heart disease (CHD) death; | |||
Primary Outcome | Changes in other lipids and lipoproteins and reduction of LDL- | |||
• | Myocardial infarction; | |||
Measures | C of more than 50% for patients that are above LDL-C goal ; | |||
• Fatal or non-fatal ischaemic stroke; or | ||||
longer term exposure and safety. | ||||
• | Urgent coronary revascularization procedure | |||
• | Group 1 - inclisiran 300mg sc on Day 1 and every 180 days | Arm 1: every 6 month treatment KJX839 300mg (given by | ||
subcutaneous injection on the day of randomization, at 3 months | ||||
thereafter for up to 4 years. | ||||
and then every 6-months) for a planned median duration of | ||||
• | Group 2- Evolocumab 140mg s.c. injection on Day 1 and | |||
Arms/Intervention | about 5 years | |||
every 2 weeks until Day 336, followed by inclisiran 300mg | ||||
Arm 2: matching placebo (given bysubcutaneous injection on the | ||||
on Day 360, Day 450 and then every 6 months for a | ||||
day of randomization, at 3 months and then every 6- | ||||
planned duration of 4 years. | ||||
months) for a planned median duration of about 5 years. | ||||
Patients with HeFH or pre-existing atherosclerotic | Patient population with mean baseline LDL-C ≥ 100mg/dL | |||
Target Patients | cardiovascular disease (ASCVD) on background statin +/- | |||
ezetimibe therapy | ||||
Read-out Milestone(s) | 2022 | 2025 | ||
Publication | TBD | TBD | ||
66 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
KJX839 - siRNA (regulation of LDL-C)
Study | NCT03851705 ORION-5 (CKJX839A12302) | NCT03814187 ORION-8 (CKJX839A12305B) | |
Hypercholesterolemia inc. Homozygous Familial | Hypercholesterolemia inc. Heterozygous Familial | ||
Indication | Hypercholesterolaemia (HeFH) and Homozygous Familial | ||
Hypercholesterolemia (HoFH) | |||
Hypercholesterolemia (HoFH) | |||
Phase | Phase 3 | Phase 3 | |
Patients | 56 randomized 2:1 (inclisiran: placebo) | 2,991 entered the study | |
Primary Outcome | • | LDL-C reduction at Day 150 | • Proportion of subjects achieving prespecified low density |
lipoprotein cholesterol (LDL-C) targets at end of study | |||
Measures | • | Changes in PCSK9, other lipids and lipoproteins | |
• Safety and tolerability profile of long term use of inclisiran | |||
• Part 1: inclisiran 300mg on Day 1 and Day 90 or placebo | Inclisiran 300mg on day 1 (placebo patients entered into study | ||
on Day 1 and Day 90 | from ORION 9, 10 & 11) or placebo on Day 1 (inclisiran | ||
Arms/Intervention | • Part 2: inclisiran on Day 180 for patients who were | patients entered into study from ORION 9, 10 & 11) then | |
randomized to the placebo group only, inclisiran on Day | inclisiran 300mg on Day 90 and every 6 months for a planned | ||
270 and then every 6 months for a planned duration of 2 | duation of 3 years | ||
years for all patients | |||
Patients with HoFH with background statin +/- ezetimibe | Patients with HeFH or pre-existing atherosclerotic | ||
cardiovascular disease (ASCVD) on background statin +/- | |||
Target Patients | therapy | ||
ezetimibe therapy and risk equivalents (patients from ORION | |||
9, 10 & 11 studies) | |||
Read-out Milestone(s) | Primary: Q3-2020(actual); Final: H2-2021 | 2023 | |
Publication | TBD | TBD | |
67 Novartis Q4 Results | January 26, 2021 | 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 | 112 | 72 |
Primary Outcome | Change from baseline of log transformed UPCR derived from | Change from baseline of UPCR derived from 24hr urine |
Measures | the 24h urine collections at Baseline and Day 90 | collections at Baseline and Week 24 |
• Placebo | ||
• LNP023 Dose 1 - 10mg bid | • LNP023 Dose - 200mg bid | |
Arms/Intervention | • 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 | ||
Target Patients | Patients with biopsy-verified IgA nephropathy | disease progression defined on the basis of antibody anti- |
PLA2R titre and proteinuria | ||
Read-out Milestone(s) | H1-2021 (IA) | 2022 |
Publication | TBD | TBD |
68 Novartis Q4 Results | January 26, 2021 | 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) |
27 patients from ongoing Ph2 (sample size from Ph3 pending | ||
Patients | 27 | HA discussions Q1 2021), total patients for this study will |
increase | ||
Cohort A: Ratio to Baseline of UPCR to Week 12 derived from | Characterize the effect of LNP023 treatment on a composite | |
24hr urine collection | ||
Primary Outcome | renal response endpoint at 9 months (1. a stable or improved | |
Cohort B: Change from Baseline in C3 Deposit Score (based | ||
Measures | eGFR and, 2. a reduction in proteinuria and 3. an increase in | |
on immunofluorescence microscopy) at Week 12 | ||
C3 compared to the CLNP023X2202 baseline visit) | ||
Increasing doses of LNP023 up to 200mg bid: | ||
Arms/Intervention | • Cohort A: Native kidney patients | • Open-label LNP023 200mg bid |
• Cohort B: Kidney transplanted patients | ||
Target Patients | Patients with C3 glomerulopathy | Patients with C3 glomerulopathy |
Read-out Milestone(s) | H1-2021 | 2024 |
Publication | Interim analysis data from Cohort-A presented at American | TBD |
Society of Nephrology (ASN 2020) | ||
69 Novartis Q4 Results | January 26, 2021 | 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 | 16 | 13 | ||
Primary Outcome | Reduction of chronic hemolysis, based on LDH level at Week | Reduction of PNH associated hemolysis, based on | ||
percentage of patients with 60% reduction in LDH or LDH | ||||
Measures | 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 8wk | ||
period | treatment LNP023 100mg bid and 2yr extension LNP023 | |||
Arms/Intervention | • | Cohort 2: 5 patients receiving LNP023 50mg bid, in addition | 100mg bid | |
to SoC, for minimum 2 weeks with 3yr treatment extension | • | Arm 2: 4wks treatment LNP023 50mg bid followed by 8wk | ||
period. Dose may be increased D15 onwards to 200mg bid | treatment LNP023 200mg bid and 2yr extension LNP023 | |||
if LDH not within limit of normal or reduced by at least 60% | 200mg bid | |||
compared to Baseline. | ||||
Patients with PNH, showing signs of active hemolysis despite | Patients with PNH, showing signs of active hemolysis, not | |||
Target Patients | treatment with SoC (defined as an antibody with anti C5 | treated with any other complement inhibitor less than 3 | ||
activity). | months prior to study start Day 1 | |||
Read-out Milestone(s) | Primary: Q2-2020(actual) | Primary: Q2-2020(actual) | ||
Extension: 2023 | Extension: 2022 | |||
Publication | Antonio M. Risitano, MD, PhD1 et al. Presented at EBMT | TBD | ||
2020 congress | ||||
70 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
TQJ230 - Antisense oligonucleotide targeting apolipoprotein(a) mRNA
Study | NCT04023552 Lp(a)HORIZON (CTQJ230A12301) |
Indication | Cardiovascular risk reduction |
Phase | Phase 3 |
Patients | 7,680 |
Primary Outcome | Time to the first occurrence of MACE (cardiovascular death, |
non-fatal MI, non-fatal stroke and urgent coronary re- | |
Measures | |
vascularization) | |
Arms/Intervention | TQJ230 80 mg injected monthly subcutaneously or matched |
placebo | |
Patients with a history of Myocardial infarction or Ischemic | |
Target Patients | Stroke, or a clinically significant symptomatic Peripheral Artery |
Disease, and Lp(a) ≥ 70 mg/dL | |
Read-out Milestone(s) | 2024 |
Publication | TBD |
71 Novartis Q4 Results | January 26, 2021 | 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 2 | Phase 2 | ||
Patients | 681 | 260 | ||
Primary Outcome | Cohorts 1 and 2-mean iBox risk prediction score at 12 months. | Change in EULAR Sjögren's syndrome Disease Activity Index | ||
Integrative score that will provide a prediction of graft survival | (ESSDAI) score and EULAR Sjögren's syndrome Patient | |||
Measures | ||||
at year 5 | Reported Index (ESSPRI) score | |||
• | Two cohorts: de novo TX and maintenance | • | Three dose arms of CFZ533 | |
Arms/Intervention | • | Test Arms: CFZ533 + MMF + corticosteroids | ||
• | Placebo | |||
• | Standard of Care: TAC + MMF + corticosteroids | |||
Target Patients | Kidney transplant recipients | Patients with Sjögren's syndrome | ||
Read-out Milestone(s) | 2022 | 2022 | ||
Publication | TBD | TBD | ||
73 Novartis Q4 Results | January 26, 2021 | 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 Loss | |
Measures | 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
Read-out Milestone(s)
Publication
Liver transplant recipients
2023
TBD
74 Novartis Q4 Results | January 26, 2021 | 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 16 | PASI 75 response and IGA mod 2011 0 or 1 response after 12 |
Measures | weeks of treatment | weeks of treatment |
• Secukinumab 300 mg every 2 weeks after weekly doses till | • Secukinumab 2 mL (300 mg) auto-injector | |
Arms/Intervention | Week 4 | • Secukinumab 2 x 1 mL (150 mg each) prefilled syringe |
• Secukinumab 300 mg every 4 weeks after weekly doses till | • Placebo 2 mL auto-injector | |
Week 4 | • Placebo 2 x 1 mL prefilled syringe | |
Target Patients | Subjects (≥90kg) with moderate to severe plaque psoriasis | Subjects with moderate to severe plaque psoriasis |
Read-out Milestone(s) | Q3-2020(actual) | Final: Q4-2020(actual) |
Publication | Publication (primary efficacy) planned in H1-2021 | Publication (16 week primary results) planned in H1-2021 |
75 Novartis Q4 Results | January 26, 2021 | 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 | |||
Read-out Milestone(s) | 2023 | 2023 | ||
Publication | Published Q4 2020 JEADV | Publication planned in H1-2021 | ||
76 Novartis Q4 Results | January 26, 2021 | 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 | |
Investigators' Global Assessment (IGA) 0 or 1 response at | ||
Measures | ||
week 12 | ||
• | Secukinumab 300 mg | |
Arms/Intervention | • | Secukinumab 150 mg |
• | Placebo | |
Target Patients | Patients with moderate to severe chronic plaque-type | |
psoriasis with or without psoriatic arthritis comorbidity | ||
Read-out Milestone(s) | Q1-2019(actual) | |
• | Week 16 results: Poster presented at: 2019 American | |
Academy of Dermatology (AAD) Annual Meeting, | ||
Publication | • | March 1-5, 2019, Washington, D.C. |
• | 52-week results: Poster at EADV 2019, Madrid 9-13 | |
October, 2019 | ||
• | Manuscript publication H1-2021 | |
77 Novartis Q4 Results | January 26, 2021 | 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 enthesitis- | Patients with juvenile idiopathic arthritis subtypes of juvenile |
related arthritis | psoriatic arthritis and enthesitis related arthritis | |
Read-out Milestone(s) | H1-2021 | 2025 |
Publication | Planned publication in 2021 | TBD |
78 Novartis Q4 Results | January 26, 2021 | 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 | ||||
Measures | modified Stoke Ankylosing Spondylitis Spine Score (mSASSS) | |||
Society criteria); | ||||
• | 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 | ||
Read-out Milestone(s) | Week 52: Q3-2019(actual); Final: H1-2021 | 2022 | ||
• | Abstract (16 week results) presented at ACR 2019 | |||
• | Abstract (52 week results) presented at EULAR 2020 | • Study design manuscript published. Baraliakos et al. Clinical | ||
Publication | • | Manuscript published in Aug 2020 in Arthritis and | ||
Drug Investigation (2020) 40:269-278. | ||||
Rheumatology | ||||
• | Further publications planned |
79 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
Study | NCT03713619 SUNSHINE (CAIN457M2301) | NCT04179175 (CAIN457M2301E1) | ||
Indication | Hidradenitis Suppurativa (HS) | Hidradenitis Suppurativa (HS) | ||
Phase | Phase 3 | Phase 3 | ||
Patients | 471 | 745 | ||
Primary Outcome | Proportion of participants with Hidradenitis Suppurativa clinical | Proportion of patients with Hidradenitis Suppurativa Clinical | ||
Measures | response (HiSCR) | Response (HiSCR) | ||
• | Secukinumab 300 mg every 2 weeks | |||
Arms/Intervention | • | Secukinumab 300 mg every 4 weeks | • | Secukinumab 300 mg every 2 weeks |
• Placebo (every 2 weeks) | • | Secukinumab 300 mg every 4 weeks | ||
• | Placebo (every 4 weeks) | |||
Patients with moderate to severe hidradenitis suppurativa | ||||
Target Patients | Patients with moderate to severe Hidradenitis Suppurativa | completing either of the core trials AIN457M2301 (NCT | ||
0313632) or AIN567M2302 (NCT03713619) | ||||
Read-out Milestone(s) | Primary (week 16): H2-2021; Final: 2022 | 2025 | ||
Publication | Study design SHSA 2020; Primary 2022 | Study design SHSA 2020 | ||
80 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
Study | NCT03713632 SUNRISE (CAIN457M2302) |
Indication | Hidradenitis Suppurativa (HS) |
Phase | Phase 3 |
Patients | 471 |
Primary Outcome | Proportion of patients with Hidradenitis Suppurativa Clinical |
Measures | Response (HiSCR) |
• Secukinumab 300 mg every 2 weeks | |
Arms/Intervention | • Secukinumab 300 mg every 4 weeks |
• Placebo (every 2 weeks) | |
• Placebo (every 4 weeks) | |
Target Patients
Read-out Milestone(s)
Publication
Subjects with moderate to severe Hidradenitis Suppurativa
Primary (week 16): H2-2021; Final: 2022
StudStudy design SHSA 2020; Primary 2022
81 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
Study | NCT04156620 INVIGORATE-1 (CAIN457P12301) | NCT04209205 INVIGORATE-2 (CAIN457P12302) |
Indication | Axial spondyloarthritis | Psoriatic Arthritis (PsA) |
Phase | Phase 3 | Phase 3 |
Patients | 500 | 380 |
Primary Outcome | The proportion of subjects achieving an ASAS40 (Assessment | The proportion of subjects achieving American College of |
Measures | of SpondyloArthritis International Society criteria) response | Rheumatology 50 (ACR50) response criteria |
Arms/Intervention | • | Secukinumab intravenous (i.v.) regimen | • | Secukinumab intravenous (i.v.) regimen |
• | Placebo intravenous (i.v.) regimen | • | Placebo intravenous (i.v.) regimen | |
Target Patients | Patients with active axial spondyloarthritis | Patients with active psoriatic arthritis (PsA) despite current or | ||
previous NSAID, DMARD and/or anti-TNF therapy | ||||
Read-out Milestone(s) | Primary (week 16): 2022; Final: 2023 | 2022 | ||
Publication | TBD | TBD | ||
82 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Cosentyx® - Anti IL-17
Study | NCT04181762 SELUNE (CAIN457Q12301) | NCT04300296 PRELUDE (CAIN457S12201) | |
Indication | Lupus Nephritis | Lichen Planus | |
Phase | Phase 3 | Phase 2 | |
Patients | 460 | 108 | |
Primary Outcome | Proportion of patients achieving Investigator's Global | ||
Proportion of subjects achieving protocol-defined CRR | Assessment (IGA 0/1) score at 16 weeks +30% delta vs | ||
Measures | |||
placebo | |||
Arms/Intervention | • Secukinumab 300 mg s.c. | • | Secukinumab 300 mg s.c. |
• Placebo s.c. | • | Placebo s.c. | |
Target Patients | Patients with active lupus nephritis (ISN/RPS Class III or IV, | Adult patients with biopsy-proven lichen planus not adequately | |
with or without co-existing class V features) | controlled by topical therapies | ||
Read-out Milestone(s) | 2026 | 2022 | |
Publication | TBD | TBD | |
83 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
LJC242 - FXR agonist + CCR2/CCR5 inhibitor
Study | NCT03517540 TANDEM (CLJC242A2201J) | |
Indication | Non-alcoholic steatohepatitis | |
Phase | Phase 2 | |
Patients | 193 | |
Primary Outcome | Evaluation of safety and tolerability of combination therapy | |
(tropifexor + cenicriviroc) by monitoring adverse event profile, | ||
Measures | ||
vital signs and laboratory parameters | ||
• | Arm A: tropifexor (LJN452) dose 1 | |
Arms/Intervention | • | Arm B: cenicriviroc (CVC) |
• | Arm C: LJN452 dose 1 + CVC | |
• | Arm D: LJN452 dose 2 + CVC |
Target Patients
Read-out Milestone(s)
Publication
Adult patients with non-alcoholic steatohepatitis (NASH) and liver fibrosis
Q4-2020
Abstract planned in H1-2021
84 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
LJN452 - FXR Agonist
Study | NCT04065841 ELIVATE (CLJN452D12201C) |
Indication | Non-alcoholic steatohepatitis (NASH) |
Phase | Phase 2 |
Patients | 380 |
Proportion of patients with resolution of NASH and no | |
Primary Outcome | worsening of fibrosis OR improvement in fibrosis by at least |
Measures | one stage without worsening of NASH at Week 48 compared |
with baseline |
• Arm A: combination therapy tropifexor + licogliflozin
• Arm B: tropifexor monotherapytropifexor + licogliflozin
Arms/Intervention | placebo | |
• | Arm C: licogliflozin monotherapylicogliflozin + tropifexor | |
placebo | ||
• | Arm D: licogliflozin placebo + tropifexor placebo |
Target Patients
Read-out Milestone(s)
Adult patients with biopsy based non-alcoholic steatohepatitis (NASH) and liver fibrosis
2022
Publication | Planned in H1-2023 |
85 Novartis Q4 Results | January 26, 2021 | 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 | Change from baseline in weekly Urticaria Activity Score (UAS7) at Week 4 | • | Long-term safety and tolerability | |
Measures | ||||
• | 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 | • | Selected dose of LOU064 taken orally twice | ||
• | Arm 3 High dose of LOU064 orally in the morning (once daily) and matching | |||
Arms/Intervention | a day (morning and evening) from day 1 to | |||
placebo in the evening from Day 1 to 85 | ||||
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 | ||||
Read-out Milestone(s) | H2-2021 | 2022 | ||
Publication | Primary resuls: EADV2021 synchronized with manuscript (NEJM), ACAAI2021 | TBD | ||
Secondary results: AAAAI, AAD 2022 | ||||
86 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
QGE031 - Anti-IgE
Study | NCT02477332 (CQGE031C2201) | NCT02649218 (CQGE031C2201E1) | ||
Indication | Chronic spontaneous urticaria | Chronic spontaneous urticaria | ||
Phase | Phase 2 | Phase 2 | ||
Patients | 382 | 226 | ||
Primary Outcome | Establish dose-response relationship of QGE031 with respect | Long-term safety; number of participants with treatment- | ||
Measures | to achievement of complete hives response at week 12 | emergent adverse events | ||
• | 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 inadequately | Adult patients with chronic spontaneous urticaria inadequately | |||
Target Patients | controlled with H1-antihistamines at approved or increased | controlled with H1-antihistamines at approved or increased | ||
doses, alone or in combination with H2-antihistamines or | doses, alone or in combination with H2-antihistamines or | |||
leukotriene receptor antagonists. | leukotriene receptor antagonists. | |||
Read-out Milestone(s) | 2017 (actual) | 2019 (actual) | ||
• | H1-2021 3 Manuscripts: Angioedema, Sleep/DLQI | • | H1-2021 manuscript: primary results extension trial (NEJM) | |
(including also ext. data), Data visualization | ||||
Publication | • | 2021 Congresses: exploratory data AAAAI, AAD, EAACI, | ||
• | 2021 Congresses: exploratory data EAACI, EADV, ACAAI, | |||
EADV, ACAAI, encores at GUF | ||||
encores at GUF | ||||
87 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
QGE031 - Anti-IgE
Study | NCT03437278 (CQGE031C2202) | NCT04210843 (CQGE031C2302E1) | ||
Indication | Chronic spontaneous urticaria | Chronic spontaneous urticaria | ||
Phase | Phase 2 | Phase 3 | ||
Patients | 48 | 800 | ||
Primary Outcome | Change in the 7 day Urticaria Activity Score (UAS7) | The proportion of subjects with well-controlled disease | ||
Measures | (UAS7 ≤ 6) at week 12 | |||
• | Ligelizumab high dose q4wks for 24 weeks | • | Ligelizumab Dose 1 and 3 | |
Arms/Intervention | • | Ligelizumab low dose q4wks for 24 weeks | ||
• | Ligelizumab Dose 2 and 3 | |||
• | Placebo / ligelizumab high dose q4wks for 8 / 16 weeks | |||
Target Patients | Adolescents from 12 to <18 years of age, with chronic | Patients who completed studies CQGE031C2302, | ||
spontaneous urticaria | CQGE031C2303, CQGE031C2202 or CQGE031C1301 | |||
Read-out Milestone(s) | H2-2021 | 2026 | ||
• | Study design was presented at PAAM (Peds Allergy & | |||
Asthma Meeting) and at UCARE meeting 2019 | ||||
Publication | • | Baseline characteristics 2020/21 | Study design presented at 2020 EAACI | |
• Primary results to be presented in late 2021/2022 (e.g. | ||||
EAACI, PAAM, EADV) | ||||
• | Manuscript to be submitted in 2022 | |||
88 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
QGE031 - Anti-IgE
Study | NCT03580369 Pearl 1 (CQGE031C2302) | NCT03580356 Pearl 2 (CQGE031C2303) | |
Indication | Chronic spontaneous urticarial / Chronic idiopathic urticaria | Chronic spontaneous urticarial / Chronic idiopathic 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 ligelizumab | • Placebo q4w from randomization to wk20, then ligelizumab | ||
dose B from wk24 to wk52 | 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 | ||
Read-out Milestone(s) | 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 | |||
89 Novartis Q4 Results | January 26, 2021 | 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 2 | Phase 2 | ||
Patients | 180 | 80 | ||
Primary Outcome | Safety and efficacy of VAY736 in primary Sjögren's syndrome | Alanine aminotransferase (ALT) normalization | ||
Measures | (pSS) | |||
Arms/Intervention | • | VAY736 | • | VAY736 |
• | Placebo | • | Placebo control with conversion to active VAY736 | |
Target Patients | Patients with moderate to severe primary Sjögren's syndrome | Autoimmune hepatitis patients with incomplete response or | ||
(pSS) | intolerant to standard treatment of care | |||
Read-out Milestone(s) | Q2-2020(actual) | 2026 | ||
Publication | • | Manuscript Q4-2020 | TBD | |
90 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Neuroscience
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 | ||||
Read-out Milestone(s) | Double-blind: 2017 (actual); | Q1-2020(actual) | ||
Extension (open-label):H1-2021 | ||||
• | PROs and prespecified subgroup analysis (Double-blind | |||
phase) submitted to JNNP accepted Aug-2020 | • Primary analysis manuscript submitted end 2020 | |||
Publication | • | Submitted May 28, 2020 1 year Open-label extension to | • | Abstracts accepted for MTIS in 2020 |
Neurology | • Secondary analysis to be submitted to multiple congresses | |||
• | Planned for Q4-2020: 2Y Open-label extension Abstracts | in 2021 | ||
completed for EAN, AHS, EHF and MTIS in 2020 |
92 Novartis Q4 Results | January 26, 2021 | 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 last |
Measures | 4 weeks of the 12-week treatment period |
Arms/Intervention
Target Patients
Read-out Milestone(s)
Publication
- Subcutaneous injection of AMG334 (erenumab) 70 mg
- Subcutaneous injection of placebo
Adult chronic migraine patients
Double-blind:2021;
Extension (open-label): 2024
Planned in H2-2022 for double-blind phase and H1-2025 for open-label extension phase
93 Novartis Q4 Results | January 26, 2021 | 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
Read-out Milestone(s)
Publication
Patients with type 1 spinal muscular atrophy
Study Part 2: Q3-2020(actual)
Study Part 3: 2023
TBD
94 Novartis Q4 Results | January 26, 2021 | 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 subjects | ||
Measures | |||
vs placebo) | 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 | |
Read-out Milestone(s) | Q1-2020(actual) | 2028 | |
Publication | Publication planned for H1-2021 | TBD | |
95 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
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 | 33 | 22 | |
Primary Outcome | Proportion of participants sitting without support | • | Achievement of independent sitting for at least 30 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 | |
Read-out Milestone(s) | Q4-2020(actual) | Q4-2019(actual) | |
Publication | Final results at the 2021 annual meeting of the | Publication of full results in top-tier neurology journal in | |
European Academy of Neurology (EAN, June 19‒22) | February 2021 | ||
96 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Zolgensma® - SMN1 gene replacement therapy
Study | NCT03505099 SPR1NT (CL-304) | NCT03837184 STR1VE Asia Pacific (CL-306) |
Indication | Spinal muscular atrophy | Type 1 spinal muscular atrophy |
Phase | Phase 3 | Phase 3 |
Patients | 30 | 2 |
• [2 copies of SMN2] Percentage of participants achieving | ||
functional independent sitting for at least 30 seconds at any | ||
Primary Outcome | visit | Proportion of participants sitting without support |
Measures | • [3 copies of SMN2] 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 | ||
Read-out Milestone(s) | H2-2021 | H2-2021 |
(Muscular Dystrophy Association) MDA 2021 (March 15‒18) | ||
Publication | and (American Academy of Neurology) AAN 2021 | TBD |
(April 17‒22) | ||
97 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Zolgensma® - SMN1 gene replacement therapy
Study | NCT03381729 STRONG (CL-102) | |
Indication | Type 2 spinal muscular atrophy | |
Phase | Phase 1 | |
Patients | 51 | |
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 | |
Read-out Milestone(s) | Cohort B: Q4-2019(actual); Cohort C1: TBC | |
Publication | TBD | |
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
98 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
Oncology
ABL001 - Specific, allosteric Bcr-Abl kinase inhibitor
Study | NCT03106779 ASCEMBL (CABL001A2301) |
Indication | Chronic myeloid leukaemia (CML) |
Phase | Phase 3 |
Patients | 233 |
Primary Outcome | Major Molecular Response (MMR) rate at 24 weeks |
Measures | |
Arms/Intervention | • | ABL001 40 mg bid |
• | Bosutinib 500 mg | |
Target Patients | Patients with chronic myelogenous leukemia in chronic phase, | |
previously treated with 2 or more tyrosine kinase inhibitors | ||
Read-out Milestone(s) | Q3-2020(actual) | |
• | Hochhaus A., et al. [Efficacy and Safety Results from | |
ASCEMBL, a Multicenter, Open-Label, Phase 3 Study of | ||
Asciminib, a First-in-Class STAMP Inhibitor, vs Bosutinib | ||
Publication | (BOS) in Patients (Pts) with Chronic Myeloid Leukemia in | |
Chronic Phase (CML-CP) Previously Treated with ≥2 | ||
Tyrosine Kinase Inhibitors (TKIs), LBA-4] ASH 2020 | ||
• | Manuscript submission Q4-2020 |
100 Novartis Q4 Results | January 26, 2021 | Novartis Investor Presentation
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Novartis AG published this content on 26 January 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 26 January 2021 08:55:01 UTC