DRUGS LIFESPAN FOR PATIENTS DECADE TRIPLING THE IN THE LAST SURVIVAL IMPROVEMENT

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2 10 40 % 3 IMPROVEMENT DRUGS IN THE LAST DECADE IN 5 YEAR SURVIVAL X TRIPLING THE LIFESPAN FOR PATIENTS

3 4 Drugs Approved in 2015! FDA APPROVALS IN 2015 Drug FDA Approval Panobinostat (Farydak ) February 23 Daratumumab (Darzalex ) November 16 Ixazomib (Ninlaro ) November 20 Elotuzumab (Empliciti ) November 30

4 THE MORE QUESTIONS WE ANSWER, THE MORE EMERGE With 10 drugs approved, what s the right combination or sequence? Should I be on maintenance therapy if I am in a CR? If I relapse, what treatment should I take?

5 MMRF COMMPASS STUDY AS CORNERSTONE Longitudinal Clinical Data and Molecular Profiling TARGET ENROLLMENT: 1,000 newly diagnosed patients DURATION: ~8 years Flow cytometry & BRAF mutations RNA sequencing expression analysis Whole exome DNA sequencing SITES: ~90 Community and Academic Cancer Centers INDUSTRY PARTNERS: Takeda, Amgen, Janssen, BMS Centralized Molecular Tests Whole genome chromosome analysis Cytospin slides for FISH Biorespository of PBMNC, plasma Tumor

6 WHAT ARE THE GOALS OF COMMPASS? Collect as much information as possible on the disease course of 1000 multiple myeloma patients over 8 years Disease characteristics Clinical information over the course of disease (every 3-6 months) Genomic profile of cancer cells at baseline and relapse Quality of life, adverse events, treatments and outcomes data Share this information with the scientific community Encourage use of this information to build predictive treatment models and develop new therapies/biomarkers

7 MMRF RESEARCHER GATEWAY (RG) The Researcher Gateway affords access to and socialization of unprecedented, rich research CoMMpass and other data. Integrates clinical, laboratory, genomic data; advanced visualization, analytics Enables population stratification, biomarker and target discovery Enables researchers to connect, share analyses, results, insights Incorporates existing data sets; definitive MM research resource 7

8 GNS HEALTHCARE NEW BIG DATA ANALYSIS TOOLS GNS Multiple Myeloma Solution in partnership with MMRF The MMRF and GNS Healthcare are engaged in a collaboration to elucidate novel intervention targets from comprehensive multiple myeloma biological networks to address the unmet needs of patients who do not benefit from life-extending therapeutics introduced in recent years. 8

9 Leveraging Big Data to Deliver Key Insights Treatment Landscape for Multiple Myeloma Effectiveness This axis subsumes genetics, labs, genomics, demographics, drugs, procedures, and their ordering, etc. measures that characterize a patient Along with lifestyle choices

10 THE QUESTIONS WE VE BEEN ABLE TO ANSWER Should I be on 3 drugs? Bortezomib-based TRIPLET Bortezomib-based DOUBLET IMID/Carfilzomib-based TRIPLET IMIDS-based DOUBLET

11 THE QUESTIONS WE VE BEEN ABLE TO ANSWER Should I have a transplant?

12 THE QUESTIONS WE VE BEEN ABLE TO ANSWER Which clinical and genomic variables predict for treatment response? First-ever MM Disease Model Gruber et al., ASH 2015

13 BRAF: DISCOVERY TO CLINIC Today Identify BRAF mutation: 38 genomes Nature Clinicians begin trials with BRAF inhibitor vemurafenib Clinical responses observed and reported at ASH 203 genomes sequenced Several other actionable targets identified Initiating combination drug trials

14 ACTIONABLE TARGETS 50 percent of patients with multiple myeloma have known molecular alterations for which there are drugs on the market or in the clinic right now. FGFR3 5% PI3K-AKT 5% IDH1/2 5% IGF1R and ALK 5% CDKN2C and CCND1 18% MyD88 3% Others 11% KRAS and NRAS 40% BRAF 8%

15 MM PATIENT TUMOR SEQUENCING Two efforts ongoing within the MMRF to provide genomic profiling for multiple myeloma patients: CoMMpass trial relapsed patients will have their tumors sequenced in a clinical laboratory Reports provided to physicians to potentially inform subsequent treatment Molecular Profiling Initiative Patients diagnosed with multiple myeloma or related plasma cell cancers will have their tumors sequenced in a clinical laboratory Reports provided to physicians to potentially inform subsequent treatment

16 MM MASTER PROTOCOL Genomic profiling for alterations Arm 1 Arm 2 Arm 3 Arm 4 Arm 5 Arm 6 Arm 7 RAF or RAS mutations 1p/1q alterations or CCND1 activation Del17p FGFR3 activating mutations and t(4;14) PI3K/AKT activating alterations IGF1R/ALK activating alterations IDH1/2 activating mutations BRAFi and/or MEKi CDKi MDM2i FGFRi PI3Ki or AKTi IGF1Ri and ALKi IDHi

17 THE PATIENT JOURNEY Know your diagnosis & subtype Get a 2 nd opinion Know your numbers Bank your tissue Get genomic testing Get imaging Integrate your team Know standard of care Know your coverage Enroll in trial Join community (online) Share your data

18 MMRF COMMUNITY GATEWAY Patient and Caregiver focused online community Ability for patients/caregivers to connect/share in groups with other patient/caregivers, nurses and myeloma experts Health metrics tracker: genomic data, response, QOL, disease, and therapy management Clinical trail finder based on member profile Educational materials 18

19 MMRF COMMUNITY GATEWAY: CLINICAL TRIAL FINDER This trial tool is user friendly, convenient, and smart it uses each MMRF CoMMunity Gateway member s profile to match patients to appropriate trials. It uses criteria such as myeloma status, location, prior treatment history, and more. It also allows members to save their search and compare trials. By providing patients with customized trial results, we will be able to match patients to potential life-extending tools and therapies 19