Presentation is loading. Please wait.

Presentation is loading. Please wait.

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

Similar presentations

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!

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

Similar presentations

Ads by Google