Venture Philanthropy Models: The Leukemia & Lymphoma Society's Therapy Acceleration Program. A FasterCures Webinar June 19, 2013

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1 Venture Philanthropy Models: The Leukemia & Lymphoma Society's Therapy Acceleration Program A FasterCures Webinar June 19, 2013

2 What is FasterCures? Founded in 2003, our mission is to save lives by saving time. We create opportunities and provide a platform for nontraditional allies to come together to share ideas and find partners. We work across diseases, sectors, and disciplines. We are a center of the Milken Institute.

3 Brings together dozens of disease research organizations with results oriented research methods virtually connected through TRAIN Central Station, an open source accessible platform featuring Web events, key tools and resources, organization specific profiles, case studies, and more amplifies lessons learned from TRAIN organizations in an effort to promote innovative approaches and their sense of urgency in speeding the path to cures

4 New venture philanthropy resources help us spread the word!

5 Speakers Louis DeGennaro, Ph.D. Chief Mission Officer, Leukemia & Lymphoma Society Lawrence Mayer, Ph.D. Founder, President and Head of Research, Celator Pharmaceuticals, Inc. Moderator: Kristin Schneeman Program Director, FasterCures

6 Confronting a Broad Spectrum of Diseases AML MM NHL CLL CML Average Age of Onset Incidence 58,000 4,300 Adult ALL MDS HL Pediatric ALL MPN Median 5-year Survival Rate SEER database, scientific literature

7 Players in the Innovation Ecosystem Patients Patient Groups Academic Research Biotech FDA Third-Party Payors Pharma

8 A Better Solution for Patients Patient Support Academic Research Research Grants Patient Programs Patients Third-Party Payors Patient Groups Biotech Pharma Venture Philanthropy Advocacy FDA Policy

9 Cures & Access Discovery Development Commercialize New Therapies $60M Research Grants $20M Therapy Acceleration $44M Co-Pay $5M Policy Agenda: approval & access $20M Services to Patients

10 Therapy Acceleration Program (TAP) More Therapies to More Patients, Faster Cures & Access Biotechnology Accelerator Academic Concierge Clinical Trials Program

11 New Opportunity Diligence Process STAGE 1 Initial Inquiry and Non Confidential Presentation PASS STAGE 2 Champion (TAP staff) Assigned and Project Reviewed by LLS staff Execute CDA STAGE 3 Confidential Presentation Need More Data, Follow Up PASS STAGE 4 Expert Review and Reference Check PASS STAGE 5 Request Preliminary Proposal PASS STAGE 6 Request Full Proposal and Invite for Presentation to TAP Committee

12 Post Approval Implementation Process STAGE 1 Present to TAP Committee Approved Conditionally Approved Not Approved STAGE 2 Negotiate contract with mutually agreed to deliverables, milestones, go/no go decisions and an ROI for LLS STAGE 3 Kick off Meeting Quarterly joint research advisory committee meetings Update to TAP Committee

13 Diligence Questions Unmet medical need avoiding crowded areas supported by others Quality of the science and the asset, level of innovation Competency and enterprise readiness (financial) of partner Value of LLS as a partner More recently: Strategic fit with LLS research agenda Potential for a return on investment

14 TAP Due Diligence Activity And Deal Flow Partnerships Evaluated Outcome YTD Declined Under Review Approved Total

15 Contract Structure Field Timeline, milestones, deliverables Payment tranches Commercially reasonable efforts Interruption clause ROI Approval & Commercialization Transfer payments: M & A, licensing Technical failure

16 Measuring Impact Successful: Direct impact for patients Demonstrated clinical response throughout the development process Demonstrated clinical proof of concept Partnered Achieved expected endpoint (preclinical programs) On going Programs: Potential patient impact Timely progress through milestones Discontinued Programs: Technical failure; No further path forward Have not demonstrated clinical utility; Lack of patient impact Have not met milestones

17 Therapy Acceleration Program Pipeline INDICATION(S) PRECLINICAL PHASE I PHASE II PHASE III MDS AML Myeloma B-cell Lymphoma Myeloma AML B-Cell Cancers Licensed by Acquired by $15 M Investment by CTCL MLL Leukemia Licensed by Recent IPO AML/MDS Leukemia Acute Leukemia Acute Leukemia DLBCL Partnership with

18 The Multiplier Effect: New Dollars Driving for a Cure 106x 30x

19 Celator Partnership

20 Celator-LLS Partnership Accelerates Clinical Testing of New Leukemia Drug CPX-351 Celator is developing a novel drug combination nanomedicine named CPX-351 for acute myelogenous leukemia (AML) Lipid spherules 100-times smaller than a red blood cell act like a nano-scale trojan horse, selectively delivering two drugs to leukemia cells in the bone marrow Phase 1 clinical trial results were very promising Celator wanted to expand the use of CPX-351 by testing it in multiple Phase 2 clinical trials The LLS Therapy Acceleration Program (TAP) provided funds that allowed Celator to evaluate CPX-351 in two large, randomized AML Phase 2 trials Results in both Phase 2 trials were very positive More patients had complete remission of their AML with CPX-351 More patients lived longer when treated with CPX-351 The benefits of CPX-351 were greatest in AML patients that typically have a poor prognosis Additional TAP funding is helping Celator to complete the final clinical trial required to make CPX-351 commercially available to AML patients

21 Complete Leukemia Remission Rate in Secondary AML 7+3 group: 16/50 CPX-351 group: 29/50

22 Deaths During 60-Day Treatment Period 7+3 group: 16/50 CPX-351 group: 3/50

23 Patients Alive 1 Year After Treatment 7+3 group: 11/50 CPX-351 group: 26/50

24 Key Elements to the Success of the Celator-TAP Partnership Clear role and high impact of LLS funding for important clinical trials Able to conduct two randomized Phase 2 studies instead of just one Able to size the studies appropriately (125 patients in each) to generate definitive results Very efficient LLS review process Clear delineation of TAP mandate, review process and expectations Rapid and decisive evaluation and final contract negotiation Realistic (i.e. executable) goals, milestones and costs Plan established by knowledgeable individuals with past experience/track record Feasibility supported by supplemental clinical & commercial analyses Appropriate clinical operations structure (i.e. internal resources and external CROs) TAP funding played key role in Celator s business interactions Small patient numbers for many heme-onc indications often limits Pharma enthusiasm; two randomized Phase 2 studies attracted greater interest and confidence in CPX-351 Support of Phase 3 study enabled Celator to initiate the trial independently which facilitated completion of financing sufficient to take Celator past completion of Phase 3

25 Targets, Leads & Candidates Program Targets, Leads & Candidates Discovery Development Commercialize New Therapies Research Grants Therapy Acceleration Co-Pay Policy Agenda: approval & access Services to Patients & Patient Registry

26 Targets, Leads & Candidates Program A contract framework for partnerships between LLS and the pharmaceutical industry Enables third party participation in and support of LLS research programs while safeguarding the LLS mission Industry partner provides funding for academic grant and/or LLS Therapy Acceleration Program LLS provides process and infrastructure for identifying high quality research; project management

27 LLS / Celgene Partnership Joint Steering Committee establishes research focus areas RFP mechanism to solicit projects from academia and the private sector Projects are vetted by LLS peer review and TAP vetting mechanisms Funded projects have timelines, milestones & deliverables; LLS oversees project management Celgene has right to first negotiation

28 Quest For Cures RFP Solicitation Novel approaches for measuring and monitoring clonal heterogeneity in the clinical setting at both the genomic and epigenetic levels and assessing its contribution to response or resistance to therapy. New molecular stratification mechanisms to identify key subsets of patients with B cell malignancies; application of these tools should be tractable to prospectively identify patients predicted to have poor outcomes with current standard therapies, aiming to direct these patients to novel therapeutic interventions that specifically address the identified cause(s) of treatment failure. Enhancing the understanding of the role of the microenvironment in initiation and maintenance of hematologic malignancies with emphasis on the mechanisms by which the elements of the microenvironment contribute to chemoresistance.

29 A Better Solution for Patients Patient Support Academic Research Research Grants Patient Programs Patients Third-Party Payors LLS Biotech Pharma Venture Philanthropy Advocacy FDA Policy

30 Q&A Louis DeGennaro, Ph.D. Chief Mission Officer, Leukemia & Lymphoma Society Lawrence Mayer, Ph.D. Founder, President and Head of Research, Celator Pharmaceuticals, Inc. Moderator: Kristin Schneeman Program Director, FasterCures

31 View an archive of this Webinar

32 Save the date for medical research's most forward thinking event. Mark your calendars to be in New York for the 5th Partnering for Cures on November 3-5, 2013.

33

34 @fastercures follow like learn read link watch share connect

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