Traws Pharma, Inc. · NASDAQ:TRAW
Traws Pharma — Onconova Therapeutics — IPO Roadshow (April 2017)
28 slides from Traws Pharma's IPO roadshow presentation, Apr 21, 2017
Slide 1: Forward Looking Statements
This presentation contains forward-looking statements about Onconova Therapeutics, Inc. based on management’s current expectations which are subject to known and unknown uncertainties and risks. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estim
Slide 2: Onconova at a Glance
Founded-1998; IPO-2013 (Nasdaq: ONTX) Focused on Myelodysplastic Syndromes (MDS) Lead clinical candidate: rigosertib Targets RAS effector pathways (Cell, 2016) Two formulations (IV & Oral) 1,200+ patients treated to date in clinical trials for MDS and other conditions Broad pipeline with several pre
Slide 3: Financial Details
Onconova founded in 1998; public since 2013 Ticker Nasdaq ONTX Stock information 6.76 million shares* Public float 79% 52 week range $2.11-8.17 Average daily volume 85,000 Ownership Tyndall, Tavistock, Sabby, Shire; insiders including management Analyst coverage LifeSci Capital; Maxim; SeeThru Equit
Slide 4: Onconova Highlights
Targeting underserved and growing market in Myelodysplastic Syndromes (MDS) >10,000 patients diagnosed annually in the U.S. No new approved treatments in over 10 years Phase 3 Trial (INSPIRE) is underway on four continents for second line higher risk MDS Patents & orphan designation for MDS in the U
Slide 5: Onconova Cancer Product Pipeline
End of Phase 2 Meeting with FDA conducted, Phase 2 expansion underway Single-agent IV rigosertib Partnered with SymBio in Japan/Korea* 2nd-line Higher-risk (HR-MDS) INSPIRE Phase 3 Trial; interim analysis expected in 2017 Oral rigosertib + azacitidine 1st-line Higher-risk (HR-MDS) Key Focus Indicati
Slide 6: Novel Mechanism of Action
Rigosertib is believed to block downstream signaling by RAS effectors including PI3K and RAF by binding to Ras Binding Domain (RBD) found in many effector proteins Published in Cell, 2016 Rigosertib Rigosertib Rigosertib bound to Ras Binding Domain NOVEL MECHANISM OF ACTION Pg. 7 April 2017
Slide 7: MDS Overlaps with Other Diseases
AML PRCA MDS AA LGL MPN PNH 1Young NS. Ann Intern Med. 2002;136:534-546. Slide credit: clinicaloptions.com MDS has overlap with other hematological disorders A spectrum of risk, from low to very high, measured by IPSS-R scores. US prevalence estimate is 59,000; 18,000 have higher risk MDS Treatment
Slide 8: Rigosertib in Higher-Risk MDS
Higher-risk (~22%, 13.1K patients in 2014) Treated with BMT (15%, ~2.0K patients) HMA non-responders (65%, ~7K patients) HMA relapsers (35%, ~3.7K patients) Ineligible# (25%, ~1.0K patients) Eligible for clinical trial (75%, ~3.2K patients) Eligible for 2nd-line drug (75%, >5K patients) Ineligible#
Slide 9: INSPIRE: Global Phase 3 Trial
Statistical analysis: two analyses planned Power 0.80; Target HR < 0.625; (reduce mortality by > 37.5%) for ITT = 0.04; for IPSS-R VHR = 0.01 Dual primary endpoints: Overall survival in ITT population or IPSS-R Very High Risk Exploratory genomic sequencing of patient samples Commentary on new trial
Slide 10: Patient Population for Phase 3 INSPIRE Trial
Data from ONTIME paper* published in Lancet Oncology ITT for ONTIME Trial Subpopulation for INSPIRE Trial (ONTIME subset) 299 Patients 131 Patients *Guillermo Garcia-Manero, Pierre Fenaux, Aref Al-Kali, Maria R Baer, Mikkael A Sekeres, Gail J Roboz, et al. Rigosertib versus best supportive care for
Slide 11: Global INSPIRE Trial Progress
GLOBAL INSPIRE TRIAL PROGRESS As of February 1, 2017 The INternational Study of Phase III IV RigosErtib, or INSPIRE, designed following feedback from the U.S. Food and Drug Administration and European Medicines Agency and derives from findings of the ONTIME Phase 3 trial. Our partner SymBio is enrol
Slide 12: INSPIRE: Key Opinion Leaders Participating in the Phase 3 Trial
INSPIRE: Key Opinion Leaders Participating in the Phase 3 Trial Pg. 13 Sites in USA Maria R. Baer, MD - University of Maryland Greenebaum Cancer Center, Baltimore Robert H. Collins, Jr., MD, FACP - University of Texas Southwestern Medical Center, Dallas Guillermo Garcia-Manero, MD - University of Te
Slide 13: Timelines for Data Analysis for INSPIRE Trial
Key features of INSPIRE trial Targeted population of post-HMA patients Running in North America, Europe, Israel, Australia and Japan Primary endpoint is overall survival Entire trial (ITT analysis) after 176 death events have occurred If the ITT analysis is negative, a 2nd analysis of IPSS-R VHR sub
Slide 14: Mechanistic Rationale for Combination Therapy with Rigosertib + Azacitidine
Preclinical evidence supports synergism of rigosertib + azacitidine combination Complexity of MDS Defined by IPSS-R categories Certain karyotypes Different types of mutations DNA methylation changes Addressed by HMA inhibitors Early stage events Signal transduction changes Later stage mutations May
Slide 15: Combination Therapy Phase 1/2 Trials
Included a diverse patient population including HMA-naïve front-line patients HMA pre-treated second-line patients AML patients Phase 2 dose: 560 mg qAM, 280 mg qPM Oral rigosertib twice daily on Day 1-21 (28-day cycle) Azacitidine 75 mg/m2/day SC/IV for 7 days starting on Day 8 Analysis: CBC was pe
Slide 16: Efficacy Results for Combination Trial
Response Criteria Response per IWG 2006 Overall evaluable (N=33) No prior HMA (N=20) HMA resistant (N=13) Complete Remission* 8 (24%) 7 (35%)** 1 (8%) Marrow CR + Hematologic Improvement (HI) 10 (30%) 6 (30%) 4 (31%) Marrow CR alone 6 (18%) 3 (15%) 3 (23%) Hematologic Improvement alone 1 (3%) 1 (5%)
Slide 17: Next Steps for Rigosertib + Azacitidine Development
Key Parameters and Milestones for Oral Rigosertib + Azacitidine Phase 3 Program Phase 3 Design Randomized Controlled 1:1 randomization between Aza + placebo and Aza + oral rigosertib Patient Population First-line MDS Higher risk patients indicated for azacitidine (Vidaza) Primary Endpoint Composite
Slide 18: Rigosertib Is Partnered in Japan/Korea Since 2011
RIGOSERTIB IS PARTNERED IN JAPAN/KOREA SINCE 2011 Pg. 19 Partnerships sought in other territories April 2017
Slide 19: Onconova Product Candidate Pipeline
Pg. 20 Patent protected, differentiated small molecule compounds Onconova Product Candidate Pipeline Not including Rigosertib Compound Target Stage Next Step Competition Patents Briciclib eIF4E (Cyclin D) Phase I Phase II Dose 4EGI-1 Issued US Recilisib GSK-3, Akt Phase I Primate efficacy CBLB502 Is
Slide 20: Management Team
Manoj Maniar, Ph.D. Senior VP, Product Development Alcon, SRI Wolfgang Meyer, Ph.D. Sr. VP Regulatory Affairs GM, Onconova GmBh Amgen, Micromet, GPC, Fujisawa Michael Petrone, M.D. VP Clin. Dev. Medical Affairs and Pharmacovigilance GSK, Roberts, GPC Steven M. Fruchtman, M.D. Chief Medical Officer R
Slide 21: Summary
Advanced clinical trials Phase 3 underway (IV rigosertib) Phase 2 complete (Oral combination rigosertib) Funded to deliver key 2017 milestones Oral Phase 2 ready to enter Phase 3 trial in 2017 with additional funding IV Phase 3 interim analysis 2017; top-line data 2018 Underserved and growing market
Slide 22: ir@onconova.us
ir@onconova.us Pg. 23 April 2017
Slide 23: Back-Up Slides
BACK-UP SLIDES Pg. 24 April 2017
Slide 24: Board of Directors
Michael B. Hoffman Chairman Partner, Riverstone Holdings LLC Henry S. Bienen Ph.D. Served as the 15th President of Northwestern University Jerome E. Groopman M.D. Chief of Experimental Medicine at the Beth Israel Deaconess Medical Center, Harvard Ramesh Kumar Ph.D. President and CEO, Onconova Therap
Slide 25: Advisory Board
Ross C. Donehower, M.D. Johns Hopkins Sidney Kimmel Comprehensive Cancer Center James F. Holland, M.D. Mount Sinai School of Medicine Stephen Nimer, M.D. Sylvester Cancer Center at the University of Miami Hospitals and Clinics David R. Parkinson, M.D. Venture Partner at NEA Alan R. Williamson, Ph.D.
Slide 26: Revised IPSS: Prognostic Score Values and Risk Categories/Scores
Risk Score Very low < 1.5 Low > 1.5 to 3.0 Intermediate > 3.0 to 4.5 High > 4.5 to 6.0 Very high > 6 Greenberg PL, et al. Blood. 2012;120:2454-2465. Prognostic Variable Prognostic Score Value 0 0.5 1.0 1.5 2.0 3.0 4.0 Cytogenetics Very good -- Good -- Intermediate Poor Very poor BM blast, % < 2 -- >
Slide 27: Revised IPSS-R in Relation to Survival
REVISED IPSS-R IN RELATION TO SURVIVAL Pg. 28 Greenberg et al. Blood 2012;120:2454-65 April 2017
Slide 28: Revised IPSS-R in Relation to Survival
a REVISED IPSS-R IN RELATION TO SURVIVAL =m VERY LOW 1.0 = Low eit i © HIGH a 9 VERY HIGH 08 06 04 02 00 Greenberg et al. Blood 2012;120:2454-65 6) |