MMRF and MMRC. Collaborative Research Model for a Rare Disease. Susan L. Kelley, M.D. MMRF and MMRC 11/23/2009. Collaborative Model

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1 Collaborative Research Model for a Rare Disease Susan L. Kelley, M.D. 1

2 OVERVIEW Multiple myeloma is an orphan blood cancer with a low 5-year survival rate. African American incidence is twice that of Caucasians. Prostate Breast 5 Year Survival All Cancers 68% Non-Hodgkin s Lymphoma Colon Leukemia Ovary Brain Multiple Myeloma Lung Pancreas 99% 89% 68% 67% 54% 44% 36% 36% 15% 5% Incidence rates for Myeloma (per 100,000) 4.1 Caucasian Men Women African American 2

3 CURRENT TREATMENT The treatment paradigm has changed with 4 new drug approvals by FDA since s-1990s: Cytotoxic chemotherapy Steroids 2009: FDA Approvals : -Bortezomib (Proteasome Inhibitor) -Thalidomide, Lenalidomide (IMIDs) -Liposomal Doxorubicin High dose chemotherapy with stem cell rescue (early or late option after induction therapy) 3

4 MULTIPLE MYELOMA TREATMENT OUTCOMES 4

5 MODEL The are sister organizations contributing to all phases of drug development. Funds worldwide research Builds myeloma community Partners with MMRC Supports 14 institutions Conducts tissue banking Facilitates Phase I/II trials 5

6 MODEL The serve as an end-to-end solution for drug development; the model is unique, compelling, and proven. 6

7 MODEL The MMRF builds business solutions to solve scientific problems. 7

8 LEADERSHIP MMRF/C leadership reflects a focus on drug development and commercialization; external partners provide specialized expertise. HBS Bain CRO Labvantage Broad Institute TGen Patient Navigator Medical Education Social Networking Online Strategies 8

9 RESEARCH FUNDING Since inception, the MMRF has invested heavily in supporting senior and junior researchers, as well as in extensive education programming. 120 Senior Research, 80 Research Fellows 53 Targeted RFA 270 Grants Overall $40M Funded Multi-media patient education CME symposia at scientific meetings Clinical trial accrual $15+ million invested 9

10 CLINICAL The MMRF Biotech Investment Award advances drugs into myeloma by filling funding gaps in the early development process. Company MMRF Funding Scope and Results Q Announcements $1.0M 2006 $1.0M 2007 $1.0M 2007 $1.0M 2008 $1.0M 2008 $1.0M 2009 Phase I trial of PI3K drug in MMRC nearing completion. Novel discovery program for first-in-class drug ongoing. ACE-011 bone-growth stimulator Phase II nearing completion. Phase I targeted for Targeting Phase I of novel cell-cycle inhibitor in MMRC 4Q09. Two awards planned. 10

11 MMRC The MMRC integrates 14 leading academic centers to facilitate tissue banking and Phase I/II trials. Current MMRC Members Dana-Farber Cancer Institute Mayo Clinic H. Lee Moffitt Cancer Center UHN/Princess Margaret Hospital City of Hope National Medical Center Emory University Hackensack University Medical Center Indiana University Ohio State University Saint Vincent Catholic Medical Center University of California, San Francisco University of Chicago University of Michigan Washington University in St. Louis Integrate research efforts Create critical mass of tissue and patients Conduct early phase trials with correlative studies 11

12 MMRC The MMRC has opened 20 trials with 12 investigational agents, and accelerated the timeline to trial opening by more than 35%. Compounds/Drugs Evaluated Carfilzomib PD Elotuzumab NPI-0052 Panobinostat Perifosine Pomalidomide SF1126 Tipifarnib TKI258 Torisel Zolinza Proteolix Pfizer Facet/BMS Nereus Novartis Keryx Celgene Semafore Centocor Ortho Biotech Novartis Wyeth Merck Final Protocol to Patient Enrolled % reduction 158 Published Benchmark MMRC Post Business Solutions (5 trials) 12

13 MMRC ACCRUAL Patient accrual to MMRC trials in 2009 increased versus Tissue collection will be comparable with 2008 number of 600 samples Patients Tissue Samples Jan Feb Mar April May June July Aug Sep Oct Nov Dec 13

14 TISSUE BANK The MMRC Tissue Bank generates high quality samples for genomics and correlative studies in support of biomarker discovery

15 GENOMICS INITIATIVE The MMRF Genomics Initiative was the first to sequence the myeloma tumor genome to identify new targets and biomarkers. Partners Achievements 250 samples comprehensively analyzed Open access data portal Data available pre-publication Twelve myeloma patient samples sequenced 15

16 PERSONALIZED MEDICINE The MMRC Personalized Medicine initiative will explore approaches to tailored therapy in myeloma Genomics/Proteomics Tissue Bank Biomarker Discovery Workshop Oct 09 MMRC Pilot Study in High- Risk Patients Patient Risk Segmentation Study 2 16

17 DATABASE The MMRF s database of more than 200,000 enables targeted and rapid communications. Constituent Total % s Patients/caregivers 31,000 70% Clinicians 9,000 45% Nurses 5,000 45% Initiatives Market Research Clinical Trial Recruitment Breaking News Updates from Medical Meetings Educational Programs 17 17

18 CLINICAL TRIAL ACCRUAL The MMRF is enhancing the Patient Navigator program to drive clinical trial accrual with specific benefits for late-stage trials. Program Highlights Up-to-date searchable database Trained clinical specialists Late-stage trials prioritized Site support Marketing Targeted communications to stage of disease and geography MMRF Newsletter articles, ads Ads in MMRC, cancer publications Social networking/communities 18 18

19 PARTNERSHIP The drive collaboration to ensure new treatments and optimal outcomes for patients. Industry MMRF/C Academia Driven By Shareholder Driven By Patient Driven By Research Treatments to Patients Market Success Continued Scientific Innovation 19 19