Traws Pharma, Inc. · NASDAQ:TRAW
Traws Pharma — Onconova Therapeutics — 34th Annual Cowen Healthcare Conference 2014
26 slides from Traws Pharma's conference presentation, Mar 7, 2014
Slide 1: 34th Annual Cowen Healthcare Conference March 5, 2014
34th Annual Cowen Healthcare Conference March 5, 2014
Slide 2: Safe Harbor Summary
Safe Harbor Summary 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 includin
Slide 3: Investment Highlights
Investment Highlights Rigosertib – PI3K/PLK targeted drug for a range of cancers Phase 3 top-line data in higher risk MDS patients released Next step is discussions with regulators in 2Q 2014 Phase 3 trial of Oral rigosertib in lower risk MDS in 2H 2014 Partnered in Europe (Baxter) and Japan (SymBio
Slide 4: Rigosertib Mechanism of Action and Safety Profile
Rigosertib Mechanism of Action and Safety Profile
Slide 5: Rigosertib: Dual Pathway Inhibitor
Rigosertib: Dual Pathway Inhibitor Targets PI3K + PLK pathways Kinomescan: PIK3CA and RIOK1 Right panel shows “polo” effect Interacts with Ras Binding Domain Multiple signaling pathways affected Composition of matter valid until 2026 Orphan designation for MDS Mitotic Pathway Mediated Effects
Slide 6: Overview of Safety Findings
Overview of Safety Findings Favorable risk-benefit profile overall to date (>1,000 patients) Lack of significant myelosuppression, cardiotoxicity, or neurotoxicity Generally, no need for premedication during the studies Potential safety signals are being monitored on an ongoing basis Risk-benefit an
Slide 7: Rigosertib in Higher Risk MDS
Rigosertib in Higher Risk MDS
Slide 8: Treating Higher-risk MDS
Higher-risk myelodysplastic syndromes (MDS) are defined by patients who fall into higher-risk group categories in the original or revised International Prognostic Scoring System. Survival for these patients is dismal, and treatment should be initiated rapidly. Standard therapies include the hypometh
Slide 9: Marrow Blast Response in 2nd-Line Higher Risk MDS Patients
Marrow Blast Response in 2nd-Line Higher Risk MDS Patients Bone marrow biopsy from 30/39 rigosertib treated higher risk MDS patients who failed Vidaza/Dacogen Change in bone marrow blast after receiving rigosertib IV These patients similar to patients enrolled in pivotal Phase 3 MDS trial Earlier St
Slide 10: Overall Survival Data in Post-HMA MDS Patients
Overall Survival Data in Post-HMA MDS Patients Overall survival for patients after hypomethylating agents failure is 17-22 weeks (4 publications) Median OS of 35 weeks for patients on rigosertib in an ITT analysis of 39 patients 0 0 WEEKS SURVIVAL PROBABILITY (%) Intent-to-Treat Analysis: Median OS
Slide 11: Design of US/Europe Pivotal Phase HR-MDS Trial Under SPA
Design of US/Europe Pivotal Phase 3 HR-MDS Trial Under SPA Rigosertib IV as a single agent in patients with MDS after failed prior azacitidine or decitabine therapy Continuous infusion using a portable pump; 1800 mg daily dose MDS relapsed, refractory or intolerant to azacitidine or decitabine Rando
Slide 12: Post-HMA MDS Subgroups Per 04-21 Protocol
Progression (according to 2006 IWG criteria) at any time after initiation of azacitidine or decitabine treatment during the past 2 years; Failure to achieve complete or partial response or hematological improvement (according to 2006 IWG) after at least six 4 - week cycles of azacitidine or either f
Slide 13: Overall Survival Estimation From Top-line Analysis
Overall Survival Estimation From Top-line Analysis Median Overall Survival (months) N Hazard Ratio p Value Total Rigosertib 8.2 199 0.86 0.27 (NS) BSC 5.8 100 Subgroup Rigosertib 8.5 127 0.67 0.02 BSC 4.7 57 Subgroup of patients who do not respond to HMAs
Slide 14: Preliminary Safety Analysis of ONTIME Trial at Top-line Analysis
Preliminary Safety Analysis of ONTIME Trial at Top-line Analysis Treatment Emergent All Events (%)
Slide 15: Completed and Ongoing Trials
Completed and Ongoing Trials Phase 3 ONTIME trial 299 patients 199 + 100 89 sites in 6 countries Low drop-out rate Drug well tolerated Median treatment 10 weeks Most treated at full dose SPA in the US Enrolled post-HMA Patients who did not respond to HMAs Patients who responded and then relapsed Pha
Slide 16: Phase 1/2 First-line Combination Study in MDS
Phase 1/2 First-line Combination Study in MDS Up to 40 patient multi-site study oral rigosertib + azacitidine combination Phase 1 stage: ascending dose 3/6 cohorts Phase 2 stage: Simon Minimax two-stage design 15 patients followed by 13 patients Objectives: Determine safety and PK Assess efficacy (I
Slide 17: Rigosertib in Lower Risk MDS
ONCONOVA THERAPEUTICS | Rigosertib in Lower Risk MDS
Slide 18: Lower-risk Myelodysplastic Syndromes Treatment Overview
Lower-risk myelodysplastic syndromes (MDSs) are defined as having low or intermediate 1 risk by the International Prognostic Scoring System and are characterized mainly by anemia in most cases. Supportive care--primarily red blood cell transfusions--remains an important component of their treatment,
Slide 19: Durable Transfusion Independence in LR-MDS Patients Treated With Oral Rigosertib
0 10 20 30 40 50 60 70 80 01-10 01-41 01-45 01-34 01-02 01-37 01-39 01-27 01-23 01-14 01-25 01-35 01-20 01-01 Duration of Transfusion Independence (weeks) Patient ID Transfusion Independence in 14 /36 (39%) evaluable patients (8 weeks+ treatment) with intermittent 560 mg bid Durable Transfusion Inde
Slide 20: Transfusion Independence in LR-MDS Patient Previously Treated With ESA and Lenalidomide
Transfusion Independence in LR-MDS Patient Previously Treated With ESA and Lenalidomide Week 6.0 7.0 8.0 9.0 10.0 11.0 12.0 -6 -2 1 3 5 7 9 11 13 15 17 19 20 22 24 26 28 30 32 34 36 38 40 42 44 46 48 50 52 54 56 58 60 Hb (g/dL) ESA (1 dose) RBC 2U 2U 2U
Slide 21: DNA Methylation Analysis Identifies Hypomethylated Loci in Responder LR-MDS Patients
AML MDS r MDS nr BM PBL DNA Methylation Analysis Identifies Hypomethylated Loci in Rigosertib Responder LR-MDS Patients Functional annotation of hypo- and hypermethylated genes which best distinguished responders from non-responders The genes most affected by methylation were related to regulation o
Slide 22: Phase 3 Trial for LR-MDS
Phase 3 Trial for LR-MDS Discussions with FDA May seek an SPA Design Placebo controlled ESA refractory patients Oral 560/280 dosing; 2/3 weeks schedule Response based Transfusion Independence lasting at least 8 weeks Plan to start in H2-2014 EU consultation following protocol completion
Slide 23: Unmet Needs in Higher and Lower Risk MDS
Unmet Needs in Higher and Lower Risk MDS Market Est. 2011 US incidence ~15,600* Est. 2011 US prevalence ~52,000* Vidaza and Dacogen 2012 sales US sales of Vidaza + North American sales of Dacogen reported > $550** million Last FDA approval in 2006 (Dacogen) No approved second-line treatment availabl
Slide 24: Rigosertib MDS Trials and Milestones
Rigosertib MDS Trials and Milestones Regulatory Discussion of Results 2Q14 Advance to Phase 3 Phase 2 Dosing in H2-2014 Rigosertib Single-agent 1st-line in Lower Risk MDS (Oral) 2nd-line in Higher Risk MDS (IV) Rigosertib in Combination Phase 1 Phase 2 Phase 3 Milestones Top-line data available Tria
Slide 25: Appendix
ONCONOVA THERAPEUTICS Appendix
Slide 26: Three Clinical Programs and Deep Non-clinical Pipeline
Three Clinical Programs and Deep Non-clinical Pipeline Phase 3 in higher risk MDS; Phase 2 in lower risk MDS Phase 3 Phase 2 Phase 1 Pre-clinical Four Phase 1 studies funded with non-dilutive support; Seeking additional government funding Acute Radiation Syndrome (ARS) Recilisib Cyclin D targeted ON
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