Verastem, Inc. · NASDAQ:VSTM
Verastem — Investor Presentation (February 2018)
28 slides from Verastem's investor presentation, Feb 14, 2018
Slide 1: Corporate Overview
CORPORATE OVERVIEW NASDAQ: VSTM 2/14/2018
Slide 2: Forward-Looking Statements
FORWARD-LOOKING STATEMENTS This presentation and other matters discussed today, or answers that may be given today, include forward-looking statements about Verastem’s strategy, future plans and prospects, including statements regarding the development and activity of Verastem’s investigational prod
Slide 3: Verastem at a Glance
VERASTEM AT A GLANCE SCIENTIFIC FOUNDATION VALUE DRIVERS Novel drugs targeting malignant cells both Duvelisib NDA filed February 6, 2018 for directly and through modulation of the relapsed or refractory CLL/SLL and FL tumor microenvironment BD deal for duvelisib ex-US in H1 2018 Clinical POC of FAK/
Slide 4: Advancing Portfolio of Cancer Programs
ADVANCING PORTFOLIO OF CANCER PROGRAMS PHASE 1 / 1B PHASE 2 PHASE 3 COLLABORATOR DUVELISIB (PI3K DELTA/PI3K GAMMA INHIBITOR) Relapsed/Refractory CLL/SLL DUO™ Complete In long term follow up Randomized open label vs. ofatumumab Refractory iNHL DYNAMO™ Complete In long term follow up Single arm, monot
Slide 5: Duvelisib
DUVELISIB AN INVESTIGATIONAL NEW TREATMENT OPTION WITH BROAD POTENTIAL ACROSS B CELL & T CELL MALIGNANCIES CLL/SLL PTCL FL First-in-class dual PI3K-δ,γ inhibitor Met primary PFS endpoint in Phase 3 trial of relapsed/refractory CLL/SLL patients Oral monotherapy with low pill burden and no obser
Slide 6: DUO: A Phase 3 Randomized Study in Relapsed/Refractory CLL/SLL
DUO: A Phase 3 Randomized Study in Relapsed/Refractory CLL/SLL Optional Crossover Study Duvelisib Ofatumumab IV 25 mg BID continuously * Administration same as Relapsed or N=160 DUO Refractory N=8 CLL/SLL patients 319 patients Ofatumumab IV Duvelisib Randomized 1:1 ‐ 300 mg IV infusion on Day 1 25 m
Slide 7: DUO Met Primary Endpoint of PFS
DUO Met Primary Endpoint of PFS Significantly Longer Median PFS with Duvelisib per IRC DUV OFA Median PFS (Months) 13.3 9.9 95% CI 12.1, 9.2, 11.3 16.8 Hazard ratio 0.52 p‐value < 0.0001 Number at Risk 7 Source: Flinn et al., ASH 2017
Slide 8: Significantly Longer PFS with Duvelisib per Investigator Assessment
Significantly Longer PFS with Duvelisib per Investigator Assessment DUV OFA Median PFS (Months) 17.6 9.7 95% CI 15, 22 9, 11 Hazard ratio 0.40 p‐value < 0.0001 Number at Risk • 89 patients on OFA arm received duvelisib in crossover study, achieving an ORR of 73% and a median PFS of 15 months per Inv
Slide 9: Duvelisib Maintained PFS Advantage in All Subgroups Analyzed
Duvelisib Maintained PFS Advantage in All Subgroups Analyzed 9 Source: Flinn et al., ASH 2017
Slide 10: Significantly Higher ORR with Duvelisib per IRC
Significantly Higher ORR with Duvelisib per IRC Overall Response Rate Lymph Node Response Rate DUV OFA 90 85.0% Lymph node response: CR 0.6% 0.6% ≥ 50% decrease in the SPD of 80 73.8% PRwL 0.6% 0 target lymph nodes PR 72.5% 44.7% from baseline 70 p‐value < 0.0001 60 Percent 50 45.3% 40 30 15.7% 20 1
Slide 11: AEs of Special Interest Infrequently Led to Duvelisib Discontinuation
AEs of Special Interest Infrequently Led to Duvelisib Discontinuation Rash All ≥ Grade 3 AEs Pneumonitis AEs leading to discontinuation (any Grade) Transaminitis Colitis Pneumonia Diarrhea Neutropenia 0 10 20 30 40 50 60 70 80 90 100 Percent of Patients Percent of Patients • Severe opportunistic inf
Slide 12: DUO Study Conclusions
DUO Study Conclusions • DUO met the primary endpoint for PFS: duvelisib monotherapy achieved significant improvement in PFS vs OFA (13.3 m vs 9.9 m; HR = 0.52; p < 0.0001) per IRC – PFS per investigator response assessment significantly favored duvelisib vs OFA (17.6 m vs 9.7 m; p < 0.0001) – Simila
Slide 13: DUO May Open an Initial Commercial Opportunity in a Growing Lymphoid Malignancy Market
DUO™ MAY OPEN AN INITIAL COMMERCIAL OPPORTUNITY FOR DUVELISIB IN A GROWING LYMPHOID MALIGNANCY MARKET MAJOR MARKET TOTAL SALES ($B) CLL/SLL MARKET OPPORTUNITY $25 • CLL/SLL is the fastest growing $20 $19.5 subtype of NHL (18% CAGR), as multiple new kinase inhibitors $15 transform treatment away from
Slide 14: Unmet Need Remains for Patients with CLL/SLL
UNMET NEED REMAINS FOR PATIENTS WITH CLL/SLL, THE MAJORITY OF WHICH PROGRESS FOLLOWING 1L THERAPY Average CLL/SLL patient 72 y.o. male treated outside of 1st Line academic institutions, often with multiple comorbidities1,2,3 16,200 treated patients in 20164 2nd Line ~70% 9,120 3rd Line treated patie
Slide 15: Increase in Therapy Options Offers CLL/SLL Patients More Personalized Treatment
AN INCREASE IN THERAPY OPTIONS OFFERS CLL/SLL PATIENTS OPPORTUNITY FOR MORE PERSONALIZED TREATMENT PLANS Imbruvica® (ibrutinib) Zydelig® (idelalisib) Venclexta™ (venetoclax) + Rituxan (rituximab) WEEK 1 DAILY Zydelig® Rituxan® DAILY 2 1.5-2L WATER DAILY INITIAL DAILY Rituxan® DOSE 375 mg/m2 “IMBRUVI
Slide 16: Duvelisib: CLL/SLL Value Proposition
D U V E L I S I B : CLL/SLL value proposition 1st Line Targeted therapy use VISION 2nd Line A chemo-free future is envisioned for CLL/SLL patients CLL/SLL 3rd Line 1st Line + Additional oral targeted treatment options are desired for NEED 2nd Line + CLL/SLL patients to enable this vision CLL/SLL 3rd
Slide 17: DYNAMO: Phase 2 Study of Duvelisib Monotherapy in Double Refractory iNHL
DYNAMOTM: A PHASE 2 STUDY OF DUVELISIB MONOTHERAPY IN DOUBLE REFRACTORY iNHL POPULATIONS PHASE 2 STUDY, FINAL ANALYSIS COMPLETED Study end points • Primary: Overall response rate (ORR) by Independent Review Committee (IRC) Double refractory* Duvelisib iNHL patients 25 mg BID • Key secondary: (N = 12
Slide 18: DYNAMO Met Its Primary Endpoint of ORR by IRC
DYNAMO™ MET ITS PRIMARY ENDPOINT OF ORR BY IRC IN DOUBLE REFRACTORY iNHL PATIENTS AT FINAL ANALYSIS Primary endpoint: • ORR by IRC at per- protocol final analysis: (p=0.0001) Secondary endpoints: • Median PFS on duvelisib: 8.4 months • Median DOR: 10 months Flinn et al., ASH 2016 Duvelisib is an inv
Slide 19: 83% of Patients in DYNAMO Had Reduction in Target Lymph Nodes
83% OF PATIENTS IN THE DYNAMO™ STUDY HAD REDUCTION IN TARGET LYMPH NODES BY IRC Flinn et al., ASH 2016 Duvelisib is an investigational agent available for clinical trial use only. Safety and efficacy have not been established. Page 19 Verastem, Inc.
Slide 20: Duvelisib's Side Effects Are Well Characterized and Manageable
DUVELISIB’S ASSOCIATED SIDE EFFECTS ARE WELL CHARACTERIZED, AND MANAGEABLE ADVERSE EVENTS OF INTEREST, IRC data on mature follow up (18 months post last patient enrolled) • Few discontinuations due to severe AEs of interest • Serious opportunistic infections < 4%: PCP (unconfirmed) (n=1); CMV (n=2);
Slide 21: Unmet Need Remains for Patients with FL
UNMET NEED REMAINS FOR PATIENTS WITH FL, THE MAJORITY OF WHICH PROGRESS FOLLOWING 1L THERAPY Average iNHL patient 67 y.o. male treated outside of 1st Line academic institutions, often with multiple comorbidities1,2,3 11,480 treated patients in 20164 2nd Line ~70% 7,336 3rd Line treated patients in 2
Slide 22: Duvelisib May Offer Convenient Option for Community Doctors
FOR COMMUNITY DOCTORS, DUVELISIB MAY OFFER A CONVENIENT OPTION FOR BOTH THEIR CLL/SLL AND FL PATIENTS UNMET NEED IN CLL/SLL UNMET NEED IN FL Additional options post 1st line therapy are Additional chemo-free treatment options needed for the majority (>50%) of patients, are needed for patients insens
Slide 23: Duvelisib Expansion Planned in R/R PTCL
DUVELISIB EXPANSION IS PLANNED IN R/R PTCL, WHERE STANDARD OF CARE REMAINS TO BE ESTABLISHED RELAPSED/REFRACTORY PTCL (mOS < 6 months1) • Recently approved 2nd+ line Drug / Trial2,3 ORR CR FDA decision treatment options have low response Folotyn (pralatrexate IV) rates with limited durability Single
Slide 24: DUO and DYNAMO Establish Foundation for Potential Hematological Franchise
THE DUO™ AND DYNAMO™ STUDIES ESTABLISH THE FOUNDATION FOR A POTENTIAL HEMATOLOGICAL FRANCHISE • Pursuit of first FDA approval: duvelisib monotherapy in relapsed/refractory CLL/SLL and FL NDA filed February 6, 2018 • Establishment of US commercial capability • Planned partnership Ex-US • Initiation
Slide 25: Executive Management
EXECUTIVE MANAGEMENT Robert Forrester Daniel Paterson President/CEO, BOD Chief Operating Officer CEO/CFO - CombinatoRx, COLY CEO - The DNA Repair Co. (now On-Q-ity) MeesPierson, Barclays, UBS PharMetrics (now IMS), Axion Steven Bloom Julie Feder Chief Strategy Officer Chief Financial Officer CFO, Cl
Slide 26: Key Financial Stats / Top Investors
KEY FINANCIAL STATS Top Investors • BVF Partners, LP • Point72 Asset Management • Ridgeback Capital Management, L.P. • Bessemer Venture Partners • Renaissance Technologies LLC • Vanguard Group, Inc. • Cardinal Partners • BlackRock Institutional Trust Company • Eastern Capital. Ltd. • Bogle Investmen
Slide 27: Duvelisib Key Milestones
DUVELISIB KEY MILESTONES 2018 NDA filed February 6, 2018 Acceptance of NDA filing File MAA BD deal for ex-US 2019 Potential duvelisib approval (Q1) Upcoming Publications & Presentations: Publish DUO Phase 3 data Publish Duvelisib Phase 1 data Publish Phase 1 CLL/SLL, TCL, DYNAMO and CONTEMPO Page
Slide 28: Verastem at a Glance
VERASTEM AT A GLANCE SCIENTIFIC FOUNDATION VALUE DRIVERS Novel drugs targeting malignant cells both Duvelisib NDA filed February 6, 2018 for directly and through modulation of the relapsed or refractory CLL/SLL and FL tumor microenvironment BD deal for duvelisib ex-US in H1 2018 Clinical POC of FAK/