Verastem, Inc. · NASDAQ:VSTM
Verastem — Investor Presentation (September 2017)
26 slides from Verastem's investor presentation, Sep 19, 2017
Slide 1: 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 2: Verastem at a Glance
VERASTEM AT A GLANCE SCIENTIFIC FOUNDATION Novel drugs targeting malignant cells both directly and through modulation of the tumor microenvironment VALUE DRIVERS Presentation of full DUO data Duvelisib NDA filing targeted 1H 2018 Clinical POC of FAK/I-O combinations in 2018 NASDAQ: VSTM DUVELISIB PI
Slide 3: Advancing Portfolio of Cancer Programs
ADVANCING PORTFOLIO OF CANCER PROGRAMS PHASE 1 / 1B PHASE 2 PHASE 3 COLLABORATOR line data available * - Investigator Sponsored Trial (IST) Duvelisib and defactinib are investigational agents available for clinical trial use only. Safety and efficacy have not been established. Page Verastem, Inc. 4
Slide 4: DUO: A Positive Phase 3 Study of Duvelisib in R/R CLL
DUO™: A POSITIVE PHASE 3 STUDY OF DUVELISIB IN RELAPSED/REFRACTORY CHRONIC LYMPHOCYTIC LEUKEMIA POSITIVE PHASE 3 STUDY, TOP LINE DATA ANNOUNCED SEPTEMBER 2017 Treatment arm 1 Study end points Duvelisib 25 mg BID • Primary: ‒ Progression-free survival (PFS) Secondary: Relapsed or Refractory CLL/SLL p
Slide 5: DUO Met Its Primary Endpoint of PFS by IRC in ITT and del(17p)
DUO™ MET ITS PRIMARY ENDPOINT OF PFS BY IRC IN BOTH THE ITT AND DEL(17P) SUBPOPULATION TOP LINE DATA Duvelisib monotherapy had a manageable safety profile, with results from this study consistent with the well-characterized safety profile of duvelisib monotherapy observed to date in patients with ad
Slide 6: DUO May Open an Initial Commercial Opportunity for Duvelisib
DUO™ MAY OPEN AN INITIAL COMMERCIAL OPPORTUNITY FOR DUVELISIB IN A GROWING LYMPHOID MALIGNANCY MARKET $25 • CLL is the fastest growing subtype of NHL (18% CAGR), as multiple new kinase inhibitors transform treatment away from chemotherapy1 Average lines of therapy per patient may be increasing, as e
Slide 7: Unmet Need Remains for Patients with CLL
UNMET NEED REMAINS FOR PATIENTS WITH CLL, THE MAJORITY OF WHICH PROGRESS FOLLOWING 1L THERAPY Average CLL patient 1st Line 72 y.o. male treated outside of academic institutions, often with multiple comorbidities1,2,3 First line therapies:5 Additional Relapsed/Refractory therapies:5 • • • Chemo + ant
Slide 8: Physicians Envision a Chemo-Free Future for CLL Patients
PHYSICIANS ENVISION A CHEMO-FREE FUTURE FOR CLL PATIENTS DRIVEN BY ADDITIONAL TARGETED ORAL MONOTHERAPY OPTIONS US oncologist opinion, academic (n = 16) and non-academic (n = 61-63): Disagree (1-3) Neither Agree nor Disagree (4) Agree (5-7) “In the future, many CLL patients will be treated with targ
Slide 9: Increase in Therapy Options Offers CLL Patients More Personalized Treatment
AN INCREASE IN THERAPY OPTIONS OFFERS CLL PATIENTS OPPORTUNITY FOR MORE PERSONALIZED TREATMENT PLANS Imbruvica® (ibrutinib) Zydelig® (idelalisib) + Rituxan (rituximab) Venclexta™ (venetoclax) WEEK 2 DAILY Rituxan® DOSE “IMBRUVICA may increase the risk of hemorrhage in patients receiving antiplatelet
Slide 10: Duvelisib Well-Positioned as Differentiated Therapy Option for R/R CLL
DUVELISIB TARGETED IS WELL-POSITIONED TO BE A DIFFERENTIATED THERAPY OPTION FOR R/R CLL PATIENTS D U V E L I S I B Novel, dual kinase inhibitor with an alternative safety profile to currently approved oral monotherapies Simple, at home, oral monotherapy dosing enables convenient treatment in the com
Slide 11: Positive DUO Results Establish Foundation for Potential Hematological Franchise
POSITIVE TOP-LINE RESULTS FROM THE PHASE 3 DUO™ STUDY ESTABLISH THE FOUNDATION FOR A POTENTIAL HEMATOLOGICAL FRANCHISE The positive DUO™ study supports: Pursuit of first FDA approval: duvelisib monotherapy in R/R CLL/SLL NDA filing targeted 1H 2018 Establishment of US commercial capability Plann
Slide 12: DYNAMO: Phase 2 Study of Duvelisib 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* iNHL patients (N = 129) Duvelisib 25 mg BID • Key secondar
Slide 13: 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 per IRC at mature follow up 80% 70% 60% 50% 40% 30% 20% 10% 0% •ORR by IRC at per-protocol final analysis: (p=0.0001) 68% 43% Secondary endpoints, at mature follow-up by IRC: •Me
Slide 14: 88% of Patients on DYNAMO Had Reduction in Target Lymph Nodes
AT MATURE FOLLOW UP, 88% OF PATIENTS ON DYNAMO™ HAD REDUCTION IN TARGET LYMPH NODES BY IRC 88% of patients had reduction in target lymph nodes (IRC data) Zinzani et al., ICML 2017 Duvelisib is an investigational agent available for clinical trial use only. Safety and efficacy have not been establish
Slide 15: Duvelisib Generally Well Tolerated, Manageable Safety Profile
DUVELISIB HAS SHOWN A GENERALLY WELL TOLERATED, MANAGEABLE SAFETY PROFILE WITH APPROPRIATE RISK MITIGATION ADVERSE EVENTS OF INTEREST, IRC data on mature follow up • • • Few discontinuations due to severe AEs of interest Serious opportunistic infections < 4%: PCP (unconfirmed) (n=1); CMV (n=2); fung
Slide 16: 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 treatment options have low response rates with limited durability NCCN guidelines still recommend clinical trials for relapsed patient
Slide 17: Duvelisib: Value Proposition
D U V E L I S I B :Va luepr oposition VISION A chemo-free future is envisioned for CLL patients NEED +CLL patients to enable this vision VALUE Duvelisib, if approved, may offer a simple oral monotherapy option to maintain treatment in the community SUPPORT filing opportunity for duvelisib as a poten
Slide 18: Defactinib Combination Strategy to Enhance Checkpoint Inhibitor Efficacy
D E F A C T I N I B COMBINATION STRATEGY TO ENHANCE CHECKPOINT INHIBITOR EFFICACY IP: COM 2028 before extensions; Orphan Designation: Ovarian & mesothelioma in the US & EU Clinical stage FAK inhibitor BOOST IMMUNE ATTACK to increase proportion of responders and duration of response REDUCE STROMAL DE
Slide 19: 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 20: Preclinical Insights Translated into Clinical I-O Combination Trials
PRECLINICAL INSIGHTS HAVE TRANSLATED DIRECTLY INTO MULTIPLE CLINICAL I-O COMBINATION TRIALS In preclinical studies, FAK inhibition has been observed to… …reduce stromal density, enabling therapies & immune cells to penetrate tumors …boost immune attack, supporting combination with immunotherapies Se
Slide 21: Executive Management
EXECUTIVE MANAGEMENT Robert Forrester President/CEO, BOD CEO/CFO - CombinatoRx, COLY MeesPierson, Barclays, UBS Daniel Paterson Chief Operating Officer CEO - The DNA Repair Co. (now On-Q-ity) PharMetrics (now IMS), Axion Julie Feder Chief Financial Officer CFO, Clinton Health Access Initiative VP, F
Slide 22: Board of Directors
BOARD OF DIRECTORS Michael Kauffman, M.D., Ph.D. Robert Forrester President/CEO Verastem (VSTM) Lead Director CEO Karyopharm (KPTI), former CMO Onyx Timothy Barberich Former CEO/Chair Sepracor (SEPR) Alison Lawton COO, Aura Biosciences, former Genzyme (now Sanofi) Louise Phanstiel BOD: Cedars Sinai,
Slide 23: Key Financial Stats
KEY FINANCIAL STATS DUVELISIB TERMS •No up front payment •Verastem will pay to Infinity up to $28 million in milestones -First milestone of $6 million payable upon positive data from the DUO study -$22 million milestone payable upon the first regulatory approval in any territory •Milestones are paya
Slide 24: Duvelisib Key Milestones
DUVELISIB KEY MILESTONES Page Verastem, Inc. 26 Potential Publications & Presentations: Present DUO results/DUO extension study Publish Duvelisib Phase 1 data Present PTCL combo study Publish Phase 1 CLL, TCL, DYNAMO and CONTEMPO 2017 FDA Pre-NDA Meeting Initiate PTCL study 2018 File NDA (H1) Accept
Slide 25: Verastem at a Glance
VERASTEM AT A GLANCE SCIENTIFIC FOUNDATION Novel drugs targeting malignant cells both directly and through modulation of the tumor microenvironment VALUE DRIVERS Presentation of full DUO data Duvelisib NDA filing targeted 1H 2018 Clinical POC of FAK/I-O combinations in 2018 NASDAQ: VSTM DUVELISIB PI
Slide 26: Verastem at a Glance
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