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Tolero Pharmaceuticals, Inc. MAKING MEANINGFUL MEDICINES BIO Utah : November 2014.

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Presentation on theme: "Tolero Pharmaceuticals, Inc. MAKING MEANINGFUL MEDICINES BIO Utah : November 2014."— Presentation transcript:

1 Tolero Pharmaceuticals, Inc. MAKING MEANINGFUL MEDICINES BIO Utah : November 2014

2 Company Overview Founded:2011 Series A:2013 $3.3M Series B:2014 $22.4M Headquarters:Lehi, UT / Salt Lake City, UT Employees:~25 (70% R&D) Focus:Oncology: Hematology & Solid Tumors Business Model:Discover, Develop & Commercialize New Chemical Entities for the treatment of cancer and other serious human diseases. 1

3 Tolero Pipeline Lead product candidate, Alvocidib, advancing toward approval in front-line and Relapsed/Refractory AML Experience with 400 patients across five Phase II clinical trials Most recent trial in 165 patients revealed striking CR rates vs. SOC (p<0.003) Tolero has a rich pre-clinical pipeline and is advancing three additional product candidates into the clinic Addressing orphan diseases and large unmet needs Expect to file 2 IND’s in ProgramIndication(s)Next Milestone Clinical Alvocidib Front-line AML Relapse/Refractory AML End of Phase II meeting with FDA Initiate Phase III clinical trial Preclinical BMP Signaling Hepcidin-Driven Anemia, (Anemia of Chronic Inflammation, Cancer) IND enabling studies prior to Phase I clinical trial AXL Inhibitor Head and Neck, Lung Cancer, AML IND enabling studies prior to Phase I clinical trial PIM Inhibitor Psoriasis, AML, Prostate Cancer, Multiple Sclerosis IND enabling studies prior to Phase I clinical trial

4 Experienced Management Team 3 Name/TitleExperience David Bearss, PhD CEO  Founder, Chief Scientific Officer at Montigen Pharmaceuticals  Chief Scientific Officer at Supergen (Nasdaq: SUPG)  Co-director of the Center for Investigational Therapeutics at the Huntsman Cancer Institute Dallin Anderson, MBA Chairman & President  Founder, Chairman, Chief Executive Officer and President at Montigen Pharmaceuticals  Senior Vice President of Development at Supergen (Nasdaq: SUPG)  MBA, Harvard Steve Weitman, MD, PhD Senior Advisor, Clinical & Regulatory Strategy  Physician and Director of the Institute for Drug Development, UTHSC-San Antonio  Chief Medical Officer and SVP at Ilex Oncology  Led team for FDA approval of Clofarabine Michael McCullar, PhD, MBA COO  Senior Vice President of Business Development, Astex Pharmaceuticals (Acquired by Otsuka Pharmaceuticals)  Vice President of Strategy and Development, SuperGen, led approval of Dacogen in US, acquisitions of Montigen Pharmaceuticals and Astex Therapeutics, LLC Steve Warner, PhD VP, Discovery & Development  Translational Genomics Research Institute  Manager, Discovery Biology at Supergen (Nasdaq: SUPG)  Senior Manager, Drug Discovery at the Huntsman Cancer Institute Michael Bernstein, MPH VP, Regulatory Affairs  11 years at the FDA as Project Manager, Administrative Assistant to the Division Director and Executive Secretary to the PCNS and PDAC  Senior Director of Regulatory Affairs at Ilex Oncology  Career to date includes over 20 INDs/CTXs and 9 NDAs/BLAs/MAAs submissions Joe Nilson Director, Business Development  20 years of product development, commercialization and licensing of healthcare and consumer products  Vice President of Business Development at various subsidiaries of a privately held company

5 Alvocidib Overview Exclusive license from Sanofi in 2013 Potent pan-CDK inhibitor Significant activity across 400 AML patients Potential for a meaningful advance in the treatment of AML 4

6 Alvocidib Mechanism of Action 5 Kinase Inhibition of CDK Family Kinase Alvocidib IC50 (nM) CDK96 CDK4/69 CDK723 CDK1157 CDK5110 CDK8120 CDK3410 CDK2550

7 BRD4 mediates the recruitment of P-TEFb (Cdk9/CyclinT) 6 7SK RNA Bound Inactive P-TEFb Brd4 Bound Active P-TEFb 7SK HEXIM1 CyclinT / Cdk9 Brd4 CyclinT / Cdk9 Brd4 CyclinT / Cdk9 RNA PII CTD 2P

8 Alvocidib in Acute Leukemias Alvocidib has demonstrated both clear single-agent activity and also striking efficacy in seqence with ara-c and mitoxantrone (FLAM regimen) Single Agent Activity Phase 1 single agent study at Ohio State University Three-days dosing of alvocidib resulted in an average of 87%, 76% and 82% reduction of circulating blast counts in untreated, relapse and refractory patients, respectively. Overall response rate of 83%. Combination Activity Phase II FLAM in front-line int/high-risk adult AML (N=45) showed a 67% CR Phase II FLAM in front-line int/high-risk adult AML (N=78) showed a 67% CR 7

9 NCI-8972 Final Results: % Complete Remission FLAM 7+3P Value Primary Endpoint: CR rate 70%46%0.003 CR by Risk Factors: No Adverse25/25 (100%)13/18 (72%)0.009 Adverse Genetics Cytogenetics Complex Monosomal FLT3 ITD+ 30/58 (52%) 22/48 (46%) 14/30 (47%) 13/24 (54%) 9/13 (69%) 10/27 (37%) 6/21 (29%) 3/16 (19%) 3/12 (25%) 3/6 (50%) Secondary AML31/52 (60%)9/26 (35%)0.05 >1 High-risk feature 51/84 (61%)13/38 (34%)0.01 Randomized Phase II Study 2:1 165 Patients FLAM (109 patients) Alvocidib: 50 mg/m 2 IV day 1-3 Ara-C 2 g/m 2 IV over 72 hr, day 6-8 Mitoxantrone 40 mg/m 2 IV day (56 patients) Ara-C 100 mg/m 2 /day IV days 1-7 Daunorubicin 90 mg/m 2 IV days 1-3 In intermediate and high risk patient populations, alvocidib in combination shows significant improvement over 7+3, the current AML standard of care. Randomized Phase II trial provides conclusive data to support further development Alvocidib in Combination in AML 8

10 Alvocidib/FLAM in Relapse/Refractory AML Clinical experience to-date from 5 trials: 156 patients treated with FLAM across 4 relapsed/refractory AML trials 19 patients treated with monotherapy alvocidib in 1 relapsed/refractory trial 175 patients treated in Relapse/Refractory AML Alvocidib/FLAM has produced dramatic Complete Remissions in Relapsed AML: Bolus alvocidib dose: CR 75% Hybrid bolus/infusion dosing: CR 92% 9

11 Alvocidib Development Plan Frontline and Relapse/Refractory AML Convene FDA End-of-Phase II meeting to discuss the planned Phase III studies Launch concurrently 2015 Chronic Lymphocytic Leukemia Combination Solid Tumors 10

12 Thank You!


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