THE TRANSFORMATION OF ONCOPEPTIDES
Citi's 15th Annual BioPharma Virtual Conference
MARTY J DUVALL
Chief Executive Officer, CEO
JAKOB LINDBERG
Chief Scientific Officer, CSO
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TRANSFORMATION INTO A FULLY INTEGRATED BIOPHARMA COMPANY
GROWTH STRATEGY SUPPORTED BY STRATEGIC INITIATIVES
Discovery and IND
generation
- Unique proprietary PDC platform
Portfolio Development and Life Cycle | Launch investment and | ||
Management | geographic expansion | ||
• Initial focus on $ 23 B MM market with a broad | • | Management team strengthened | |
supportive clinical program | • Ramping up for US launch around | ||
• Priority review of first-in class RRMM drug | • | year-end | |
melflufen ongoing | Cash position ~180 MUSD after |
landmark directed share issue
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MARTY J DUVALL
PROFESSIONAL EXPERIENCE
- Executive Leadership experience from public and private companies; CEO, CCO, SVP, Global Commercial and Marketing roles
- Pharma and biotech experience across geographies; Aventis (Sanofi), MGI (Eisai), Abraxis (Celgene), Merck (MSD), ARIAD (Takeda) and Tocagen (Forte)
- Broad and deep oncology experience including; hematology (e.g. MDS, CTCL, CML, AML, MM, etc.), and solid tumors (e.g. breast, lung, prostate, H/N, gastric, GBM, etc.), biologics, small molecules, gene therapy, supportive care
- Launch experience; Taxotere (US, Europe and Asia), Abraxane (China), Dacogen (US and Europe), Sylatron (Global), Iclusig (US, Europe, and Asia) and Alunbrig
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MELFLUFEN IS A FIRST IN CLASS PRODUCT
GENERATED FROM OUR PDC PLATFORM
Melflufen is a first in class peptide-drug
conjugate (PDC) that targets
aminopeptidases and rapidly releases
alkylating agents into tumor cells
5
RECENT EVENTS
VALUE GENERATION AND RISK REDUCTION
6
REGULATORY TIMELINES
NDA REVIEW PROCESS FOR MELFLUFEN
•
•
•
•
•
US FDA Submission* - June 30
Priority Review ** - August 29
No ODAC committee planned
90-day Safety Update - September
PDUFA Date - February 28th, 2021
- Evaluation underway for regulatory timelines in other key geographies **Mid-Cycle Review Meeting in October is driving our "launch readiness" work in order to be ready in November
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MELFLUFEN CLINICAL DEVELOPMENT PROGRAM
FULL CLINICAL DEVELOPMENT PROGRAM IN RRMM
O-12-M1
HORIZON | |||||||||||||||||||
OCEAN | |||||||||||||||||||
ANCHOR | Phase 1 | Phase 2 | |||||||||||||||||
BRIDGE | |||||||||||||||||||
AL-AMYLOIDOSIS | |||||||||||||||||||
LIGHTHOUSE | |||||||||||||||||||
PORT | |||||||||||||||||||
2016 | 2017 | 2018 | 2019 | 2020 | 2021 | 2022 | |||||||||||||
Phase 1 and 2: | Phase 2: | Phase 3: | Phase 1 and 2: | Phase 2: | Phase 1 and 2: | Phase 3: | Phase 2: | ||||||||||||
single-arm study (O- | single-arm study in | randomized head- | triple-combination | study in renally | single-arm study in | randomized | open-label, | ||||||||||||
12-M1) | late stage RRMM | to-head study vs | study (ANCHOR) | impaired patients | AL-amyloidosis | daratumumab | randomized, cross- | ||||||||||||
(HORIZON) | pomalidomide | (BRIDGE) | combination study | over study (PORT) | |||||||||||||||
(OCEAN) | (LIGHTHOUSE) | ||||||||||||||||||
The arrows show estimated Last Patient In, in the studies
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COMPETITIVE MELFLUFEN DATA
TRIPLE CLASS REFRACTORY MULTIPLE MYELOMA PATIENTS
Melflufen | Xpovio | Blenrep | ||||
Oncopeptides | Karyopharm | GSK | ||||
US NDA, June 30, 2020 | US approval, July 2019 | US Approval, Aug 6, 2020 | ||||
Number of patients studied | 119 | 122 | 95 | |||
Overall Response/Clinical Benefit Rate | 26%/39% | 25%/39% | 31%/36%* | |||
mDOR / mPFS responders | 5.5m / 8.5m | 3.8m / 4.0m | NR/NR | |||
Progression-free survival | 3.9 months | 3.7 months | 2.8 months* | |||
Overall survival | 11.2 months | 8.0 months | 14.9m* | |||
Share of patients with EMD | 42% | 22% | 20%* | |||
Serious Adverse Event Rate | 51% | 58% | 40% | |||
Non-hematologic toxicity (grade 3/4) | Pneumonia | 9% | Fatigue | 25% | Keratopathy | 44% |
reported in >5% of patients | Hyponatremia | 20% | Decreased Visual | |||
Nausea | 10% | Acuity | 28% | |||
Pneumonia | 9% | Pneumonia | 7% | |||
Diarrhea | 7% | Pyrexia | 6% | |||
Sepsis | 6% | |||||
Hypokalemia | 6% | |||||
Mental status | 6% | |||||
General det. | 6% | |||||
9 Source: Submission for melflufen, FDA Label documents for Xpovio and Blenrep (items marked with '*' is data from DREAMM-2 as published in Lancet).
ENROLLMENT COMPLETED IN PHASE 3 OCEAN STUDY
495 PATIENTS RECRUITED - TOP-LINE RESULTS IN H1 2021
Head to head study versus standard of care
N = 495
Lenalidomid-
refractory multiple myeloma patients
Randomization
Melflufen + | Primary | |
dexamethason | endpoint: | |
PFS | ||
Secondary | ||
Pomalidomid + | ||
endpoint: | ||
dexamethason | ORR, OS | |
RRMM data from pomalidomide FDA label and O-12-M1 study
Treatment | ORR | CBR | Median PFS | Median DOR | Median OS |
Melflufen + Dexamethason | 31% | 49% | 5.7 months | 8.8 months | 20.7 months |
Pomalidomid + Dexamethason | 24% | NR | 3.6 months | 7.0 months | 12.4 months |
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NEWER PRODUCTS USED IN ON TOP OF OLD AS SURVIVAL IMPROVES
NEED OF NEW MoA's
US MM # of Total Patients by Product
70000
60000
50000
40000
30000
20000
10000
0 Jul-17Oct-17Jan-18Apr-18Jul-18Oct-18Jan-19Apr-19Jul-19Oct-19Jan-20Apr-20
Revlimid
Velcade
Darzalex
Pomalyst
Kyprolis
Ninlaro
Cytoxan
Empliciti
melphalan
Farydak
Xpovio
Source: Intrinsiq MAT, Jun 2020
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SIGNIFICANT OPPORTUNITIES BASED ON DEVELOPMENT PROGRAM
US MARKET
New data to drive label expansion
3x RRMM
20,000+
1-2x RRMM | 1-2x RRMM |
Single drug use | Comb. use |
25,000 | 20,000+ |
EMD/High-risk
18,000
Anticipated label in triple-class refractory patients
Head-to-head superiority study with the most used regimen in RRMM
Combination with PI or anti-CD38 opens up 2L+ combination treatment
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PAVING THE WAY FOR A SUCCESSFUL LAUNCH
GLOBAL ORGANIZATION WITH SIGNIFICANT LAUNCH EXPERIENCE
Mohamed Ladha, General Manager US Business Unit
17 years of industry experience with extensive oncology and launch expertise
Led/built commercial functions at 7 pharma or biotech companies for in-line/ launch products
Sarah Donovan, Head of Marketing
20 years of industry experience in sales, analytics; patient advocacy, US and Global Marketing , 10 years of experience in oncology
Led and built marketing functions for launches and inline brands
Paula O'Connor, MD, Head of Medical Affairs US
17 years industry experience with 30 years oncology experience
Led Clin Dev programs at 3 companies and established Med Affairs at 3 companies
An accomplished US Medical Affairs and Commercial Team with nearly 100 oncology product launches
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PASSIONATE TO MAKE A DIFFERENCE FOR PATIENTS
BUILDING A PATIENT FOCUSED ORGANIZATION
National Medical
Accounts & Science
Reimburs. Liaisons
(~5) (~10)
Oncology
Account (~40) Patient (~10) Nurse
ManagersEducators
(~5) | (~2) |
Area | Key Customer |
Business | |
Marketers | |
Directors | |
"Ensuring that every patient who
potentially could benefit from melflufen gains access"
Marty J Duvall
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DISEASE AWARENESS AND EDUCATION
PAVE THE WAY FOR A NEW CLASS OF DRUG
15
MELFLUFEN AWARENESS CONTINUES TO INCREASE IN THE US
MOMENTUM IS GROWING FOLLOWING SCIENTIFIC MEETINGS
Awareness of Melflufen in the US
50%
20%
10%
% MDs Likely or Very Likely to use Product
X by line of therapy
(7-point scale from "Very Unlikely" to "Very Likely")
53%
49%
38% | 38% |
20%
11%
EHA 2019 (Jun 2019) ASH 2019 (Dec 2019) EHA 2020 (Jun 2020) | 2L | 3L | 4L | 5L | 3x refractory EMD |
Source: US physician market research, n=100
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CONTINUOUS NEWSFLOW
MAJOR EVENTS OVER THE NEXT 12 MONTHS
Q2 2020 | Q3 2020 | |
Q4 2020 | H1 2021 |
EHA data update
First patient in
Potential accelerated | Top-line results |
Amyloidosis study
First patient in PORT
approval in US | OCEAN |
Potential launch in | Last patient in |
NDA submission
study
the US | ANCHOR |
Priority review -
PDUFA date
First patient in sEAPort
program (US)
ASH data update | Last patient in |
BRIDGE | |
EHA data update |
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MELFLUFEN AND THE
PDC PLATFORM
JAKOB LINDBERG
Chief Scientific Officer
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MELFLUFEN - A FIRST IN CLASS DRUG CANDIDATE
A PEPTIDE-DRUG CONJUGATE TARGETING AMINOPEPTIDASES
Amino-peptidase | Alkylating payload |
binding domain |
AcidAcidpayload
- Increased potency of linked toxin due to aminopeptidase targeting with subsequent hydrolysis
- Potency increase over the course of disease, i.e. with degree of malignancy
- Circumvent significant amount of transport associated resistance development
- Circumvent significant amount of programmed cell-death related resistance developed, e.g. p53 deletion or mutation
- Aminopeptidase targeting enables additional
beneficial activity to direct cytotoxic effect, e.g. anti-angiogenesis and metastatic process
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AMINOPEPTIDASES ARE EXCELLENT CANCER TARGETS
KEY ROLE IN CANCER CELL SURVIVAL, PROLIFERATION AND MIGRATION
Amino-peptidases play a key role in protein homeostasis, and in other critical functions such as cell-cycle progression, programmed cell death and cell migration
- Amino-peptidasesare over-expressed in cancer cells
II
Amino-peptidase expression is increased between diagnosis and relapse in patient cancer samples
III
Amino-peptidase expression correlates with mutational burden and poor clinical outcome
21 Note: Structure from Kochan et al, PNAS 108 (19): 7745-50.
PDC PLATFORM
THERAPEUTIC ACTIVITY IN MOST CANCERS
PDC Potentiation
• Melflufen is focused on multiple myeloma and AL-amyloidosis
• New molecules are based on PDC platform
• Potential broadening of indications in AML, Non-Hodgkin Lymphoma and breast cancer
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PEPTIDE DRUG CONJUGATE TECHNOLOGY
VERSATILE PLATFORM WITH MULTIPLE VENUES FOR FUTURE DEVELOPMENT
Amino | Amino | Toxic | • | Alternate toxic payload |
• Alternate reactivity of payload | ||||
Acid | Acid | payload | ||
• | Change membrane | |||
permeability of payload |
- Modify amino-peptidase binding domain to alter
specificity for different amino-peptidases
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PDC PIPELINE
FROM PRE-CLINICAL TO CLINICAL DEVELOPMENT 2020/21
EXPLORATORY | LATE PRECLINICAL | PHASE 1 | PHASE 2 | PHASE 3 | REGISTRATION | MARKET |
DEVELOPMENT | DEVELOPMENT | |||||
Melflufen
OPD 5
OPS 2
- OPD5 - High-dose treatment in i.e. bone-marrow transplantation ready for clinical development late 2020
- OPS2 - Second generation PDC candidate with alkylating payload potentially ready for clinical development in 2021
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FINAL DATA IN TRIPLE CLASS REFRACTORY MULTIPLE MYELOMA
INDEPENDENT REVIEW COMMITTEE DATA
Primary End-Point | Investigator | IRC Data | Incl. unconfirmed |
Assisted Data | Jan14th | responses Jan 14th | |
Jan 14th | |||
Overall Response Rate (ORR) - ITT n=157 | 29% | 30% | 31% (inv. and IRC) |
ORR - 3x RRMM n=119 | 26% | 26% | 27% (inv. and IRC) |
ORR - EMD n=55 | 24% | 27% | NA |
Note: Two unconfirmed responses on January 14th have later been confirmed.
Safety profile demonstrates that hematological toxicities were common but manageable, and non- hematological toxicities were infrequent
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STRONG ACTIVITY IN HIGHLY REFRACTORY MM PATIENTS
RESPONDING PATIENTS PROGRESSION FREE FOR 8.5 MONTHS
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FINANCIAL RESULTS H1 2020
WELL FINANCED WITH MANY DEVELOPMENT COMMITMENTS
Operating Costs Jan-Jun
800
700
87,2
600
500 | 148,9 | |
M | ||
400 | ||
SEK | ||
300 27,4
44,3
200 | 441,4 |
100 239,4
G&A
M&S
R&D
- Operating loss increased to SEK 696.2 M (loss:305.6)
- R&D increase primarily due to increase in clinical & drug supply: SEK 332.5 M (193.9)
- OCEAN SEK 177.2 M (110.7)
- Build-upof commercial and medical affairs explains increase in M&S
- Cash flow from operating activities neg. SEK 598.5 M (neg. 265.8)
- Cash position was SEK 937.8 M (626.8) as of Jun 30, 2020
- Directed share issue raising SEK 1,413.9 M before issue costs of SEK 85.2 M in May 2020 closed in two steps in May and July
- Second step of SEK 673.5 M after issue costs not included in cash
0
as of Jun. 30
20192020
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Oncopeptides AB published this content on 10 September 2020 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 23 September 2020 15:24:02 UTC