Verastem, Inc. · NASDAQ:VSTM
Verastem — Investor Presentation (August 2017)
27 slides from Verastem's investor presentation, Aug 9, 2017
Slide 1: Corporate Overview
CORPORATE OVERVIEW NASDAQ: VSTM August 9, 2017
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 Pivotal duvelisib study read out mid 2017 directly and through modulation of the Follow up data from DYNAMO iNHL mid 2017 tumor microenvironment Clinical POC of FAK/I-O combinations in 2018 NASDAQ: VS
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 DUO™ Ph 3 top line data expected mid 2017 Randomized open label vs. ofatumumab Refractory iNHL Single arm, monotherapy DYNAMO™ In long term follow up R
Slide 5: Duvelisib: An Investigational New Treatment Option
DUVELISIB AN INVESTIGATIONAL NEW TREATMENT OPTION WITH BROAD POTENTIAL ACROSS B CELL & T CELL MALIGNANCIES FL CLL PTCL UNIQUE First-in-class dual PI3K-δ,γ inhibitor SIMPLE Oral monotherapy with low pill burden and no food effect CONVENIENT Administration without hospitalization or infusion center TO
Slide 6: DYNAMO Is a Robust Phase 2 Study of Duvelisib Monotherapy
DYNAMOTM IS A ROBUST PHASE 2 STUDY OF DUVELISIB MONOTHERAPY IN DOUBLE REFRACTORY iNHL POPULATIONS PHASE 2 STUDY, FULLY ENROLLED WITH FINAL ANALYSIS COMPLETED Study end points • Primary: Overall response rate (ORR) by Independent Review Committee (IRC) Double refractory* Duvelisib iNHL patients 25 mg
Slide 7: DYNAMO Met Its Primary Endpoint of ORR by IRC
DYNAMOTM MET ITS PRIMARY ENDPOINT OF ORR BY IRC IN DOUBLE REFRACTORY iNHL PATIENTS AT PRIMARY ANALYSIS Response rates remain durable on long term follow up ORR per IRC at 18 month follow up 80% • Primary endpoint at 68% per-protocol primary 70% Overall Response Rate (%) analysis: (p=0.0001) 60% 50%
Slide 8: 88% of Patients on DYNAMO Had Reduction in Target Lymph Nodes
88% OF PATIENTS ON DYNAMOTM HAD REDUCTION IN TARGET LYMPH NODES IRC data on 18 month follow up 88% of patients had reduction in target lymph nodes (IRC data) Zinzani et al., ICML 2017 Page 8 Verastem, Inc.
Slide 9: Generally Well Tolerated, Manageable Safety Profile
GENERALLY WELL TOLERATED, WITH A MANAGEABLE SAFETY PROFILE WITH APPROPRIATE RISK MITIGATION IRC data on 18 month follow up ADVERSE EVENTS OF INTEREST • Few discontinuations due to severe AEs of interest • Serious opportunistic infections < 4%: PCP (unconfirmed) (n=1); CMV (n=2); fungal pneumonia (n=
Slide 10: DYNAMO Supports Further Investigation of Duvelisib Across B Cell Malignancies
DYNAMOTM SUPPORTS THE FURTHER INVESTIGATION OF DUVELISIB BROADLY ACROSS B CELL MALIGNANCIES • Duvelisib monotherapy is clinically active in double refractory iNHL – ORR of 47% per IRC; ORR of 60% per investigator – 88% of patients had tumor reduction – Median PFS of 9.0 months per IRC – Responses we
Slide 11: Phase 1 Duvelisib Activity Supports Lead Indication in R/R CLL
PHASE 1 DUVELISIB MONOTHERAPY ACTIVITY SUPPORTS LEAD INDICATION IN RELAPSED/REFRACTORY CLL For relapsed/refractory CLL patients at 25 mg BID with baseline CT scan (n = 30): • 83% (25/30) had a nodal response (reduction ≥50%) • ORR by iwCLL was 57% (17/30), including 1 CR – ORR for the TP53mt/del(17p
Slide 12: DUO: A Phase 3 Study of Duvelisib in R/R CLL
DUO™: A PHASE 3 STUDY OF DUVELISIB IN RELAPSED/ REFRACTORY CHRONIC LYMPHOCYTIC LEUKEMIA (CLL) ONGOING PHASE 3 STUDY, ENROLLMENT COMPLETE IN NOV 2015 Treatment arm 1 Study end points Duvelisib 25 mg BID • Primary: ‒ Progression-free survival (PFS) Relapsed or • Key secondary: Refractory CLL ‒ Overall
Slide 13: DUO May Open Initial Commercial Opportunity in 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 MARKET OPPORTUNITY $25 • CLL is the fastest growing $20 $19.5 subtype of NHL (18% CAGR), as multiple new kinase inhibitors $15 transform treatment away from 12% CAG
Slide 14: Unmet Need Remains for Patients with CLL
UNMET NEED REMAINS FOR PATIENTS WITH CLL, THE MAJORITY OF WHICH PROGRESS FOLLOWING 1° THERAPY Average CLL patient 72 y.o. male treated outside of 1st Line academic institutions, often with multiple comorbidities1,2 16,200 treated patients in 20163 2nd Line ~70% 9,150 3rd Line treated patients in 201
Slide 15: Positive DUO Results Establish Foundation for a Hematological Franchise
POSITIVE RESULTS FROM THE PHASE 3 DUO™ STUDY ESTABLISH FOUNDATION FOR A HEMATOLOGICAL FRANCHISE With positive DUO™ results: Pursue first FDA approval: duvelisib monotherapy in R/R CLL Establish US commercial capability Partner Ex-US Initiate multiple company-sponsored & investigator-sponsore
Slide 16: Additional Chemo-Free Treatment Options Needed for Follicular Lymphoma
ADDITIONAL CHEMO-FREE TREATMENT OPTIONS ARE NEEDED FOR PATIENTS WITH FOLLICULAR LYMPHOMA (FL) FL 14,000 1st line treatable US patients/year1 ~20 - 40% ~10% of front line treated patients3 of treated patients2 Insensitive to front line Elderly, frail or otherwise chemotherapy: inappropriate for Relap
Slide 17: Standard of Care Remains to Be Established for R/R PTCL
STANDARD OF CARE REMAINS TO BE ESTABLISHED FOR PATIENTS WITH RELAPSED/REFRACTORY PTCL RELAPSED/REFRACTORY PTCL (mOS < 6 months1) • Recently approved 2nd+ line FDA Drug / Trial2,3 ORR CR treatment options have low decision response rates with limited Folotyn (pralatrexate IV) durability 27% 8% AA 200
Slide 18: Duvelisib: Value Proposition
D U V E L I S I B : Value proposition TARGETED FIRST APPROVAL • Despite recent advances, additional treatment options are needed for CLL patients past first line therapy • For the relapsed/refractory CLL patient, duvelisib may offer a simple oral option to R/R CLL maintain their treatment in the com
Slide 19: Defactinib: Combination Agent for Enhanced Checkpoint Inhibitor Efficacy
D E FAC T I N I B COMBINATION AGENT FOR ENHANCED Clinical stage FAK inhibitor CHECKPOINT INHIBITOR EFFICACY IP: COM 2028 before extensions; Orphan Designation: Ovarian & mesothelioma in the US & EU BOOST IMMUNE ATTACK REDUCE STROMAL to increase proportion of responders and duration of DENSITY respon
Slide 20: Clinical Experience Has Established Single Agent Activity and Combinability of Defactinib
CLINICAL EXPERIENCE TO DATE HAS ESTABLISHED SINGLE AGENT ACTIVITY AND COMBINABILITY OF DEFACTINIB Total exposure to date: 300+ patients Safety: Well tolerated and combinable; primary toxicities GI Target coverage: pFAK inhibition and decrease in cancer stem cell (CSC) markers replicated in 2 indepen
Slide 21: FAK Inhibition Reduces Stromal Density & Boosts T Cell Entry
FAK INHIBITION REDUCES STROMAL DENSITY & BOOSTS T CELL ENTRY INTO TUMORS, LEADING TO LONGER SURVIVAL IN PRECLINICAL MODELS Fibroblasts Extracellular Matrix Survival Improved long term survival corresponds with increased entry of CD8+ cytotoxic T cells into tumor: FAP+ Fibroblasts, Fibroblast prolife
Slide 22: Preclinical Insights Translated into Multiple Clinical I-O Combination Trials
PRECLINICAL INSIGHTS HAVE TRANSLATED DIRECTLY INTO MULTIPLE CLINICAL I-O COMBINATION TRIALS FAK inhibition boosts immune attack, supporting FAK inhibition reduces stromal density, enabling combination with immunotherapies therapies & immune cells to penetrate tumors Serrels et al. (2015) Cell 163: 1
Slide 23: Need Remains for Improvement with Monotherapy I-O Treatment
NEED REMAINS FOR IMPROVEMENT OF PATIENT OUTCOMES WITH MONOTHERAPY I-O TREATMENT Current ORR Defactinib combination studies 0% 25% 50% 75% 100% PANCREATIC Phase 1b, Defactinib + Gemcitabine + Gemcitabine1 7% Pembrolizumab (anti PD-1) in patients Ipilimumab2 0% with relapsed pancreatic cancer BMS-9365
Slide 24: Clinical Milestones for Key Ongoing Studies
CLINICAL MILESTONES FOR KEY ONGOING STUDIES ‘16 2017 2018 CLINICAL Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 COLLABORATOR DUVELISIB PROGRAM (PI3K DELTA/PI3K GAMMA INHIBITOR) Go/No Go DUO™: Relapsed/Refractory CLL Ph 3 1° Filing Randomized open label vs. ofatumumab Decision DYNAMO™: Refractory iNHL ASH 2016: iCML &
Slide 25: Executive Management and Board of Directors
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 Julie Feder Jonathan Pachter, Ph.D. Chief Financial Officer Chief Scientific Of
Slide 26: Clinical and Scientific Advisory Board
CLINICAL AND SCIENTIFIC ADVISORY BOARD Robert Weinberg, Ph.D. Co-founder & Chairman of CSAB Whitehead Institute/MIT Greg I. Berk, M.D. Edmund J. Pezalla, M.D., Ph.D. Former Executive/CMO – Verastem, Sideris, BIND Former VP – Pharmaceutical Policy and Strategy at Aetna Intellikine, Abraxis Bioscience
Slide 27: Verastem at a Glance
VERASTEM AT A GLANCE SCIENTIFIC FOUNDATION VALUE DRIVERS Novel drugs targeting malignant cells both Pivotal duvelisib study read out mid 2017 directly and through modulation of the Follow up data from DYNAMO iNHL mid 2017 tumor microenvironment Clinical POC of FAK/I-O combinations in 2018 NASDAQ: VS