MULTIPLE MYELOMA RESEARCH FOUNDATION INC
383 MAIN AVE NO 5TH FL, NORWALK, CT 06851 www.themmrf.org

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Organizations Filed Purposes: THE MULTIPLE MYELOMA RESEARCH FOUNDATION (MMRF) SUPPORTS INVESTIGATORS AND SCIENTIFIC PROJECTS THROUGH A NUMBER OF RESEARCH GRANTS. THE GOAL OF THIS INITIATIVE IS TO ACCELERATE THE DEVELOPMENT OF THERAPEUTIC APPROACHES FOR MYELOMA THROUGH OUR UNIQUE END-TO-END MODEL THAT SUPPORTS EFFORTS IN BASIC SCIENCE OR TRANSLATION RESEARCH.IN 2017 THE MMRF AWARDED MULTI-YEAR GRANTS FOR THREE NEW PROGRAMS. IN DATA ANALYTICS & ANALYSIS, MMRF AWARDED $2 MILLION TO INVESTIGATORS FOR OUR ANSWER FUND, WHICH WILL LEVERAGE THE LANDMARK MMRF COMMPASS STUDY TO IDENTIFY THE NEXT GENERATION OF THERAPEUTIC TARGETS AND/OR PATHWAYS OR BIOMARKERS FOR HIGH-RISK MULTIPLE MYELOMA (MM) PATIENTS. IN IMMUNOTHERAPY, $7 MILLION WAS AWARDED TO CREATE THE IMMUNE NETWORKS OF EXCELLENCE. THESE MULTICENTER, AND MULTINATIONAL, RESEARCH PARTNERSHIPS OF WORLD-CLASS SCIENTISTS AND CLINICIANS WILL ACCELERATE RESEARCH INTO THE ROLE OF THE PATIENT IMMUNE SYSTEM IN MYELOMA BIOLOGY AND DISEASE PROGRESSION, CHARACTERIZE THE CHANGES IN THE IMMUNE SYSTEM ASSOCIATED WITH THE PATIENT'S RESPONSE OR RESISTANCE TO THERAPY AND IDENTIFY AND DEVELOP NOVEL IMMUNOTHERAPIES AND IMMUNOTHERAPY STRATEGIES. THESE INVESTIGATIVE NETWORKS WILL ALSO COLLECT AND ANALYZE A WIDE RANGE OF KEY IMMUNOLOGICAL DATA FROM PATIENTS AT DIFFERENT DISEASE STAGES AND IMMUNOTHERAPIES THAT WILL BE THE BASIS OF IMMUNE DATABANK. IN 2019 THE MMRF LAUNCHED THE IMMUNE ATLAS, A RESEARCH INITIATIVE TO CREATE A COMPREHENSIVE IMMUNOLOGICAL MAP OF MULTIPLE MYELOMA. THIS INITIATIVE WILL GENERATE HIGH-DIMENSIONAL DATA (DOWN TO THE SINGLE CELL LEVEL) DESCRIBING THE HOW PATIENTS' IMMUNE SYSTEMS CHANGE DURING THE DEVELOPMENT AND PROGRESSION OF MULTIPLE MYELOMA, AND DURING CLINICAL RESPONSE OR NON-RESPONSE TO THERAPY. THE IMMUNE ATLAS INITIATIVE BUILDS UPON THE SCIENTIFIC FOUNDATIONS OF THE COMMPASS GENOMIC STUDY AND THE IMMUNE NETWORKS OF EXCELLENCE PROGRAM, TO PROVIDE A HOLISTIC VIEW OF HOST-DISEASE BIOLOGY AND INTERACTIONS. THE IMMUNE ATLAS INITIATIVE WILL BE CRITICAL TO THE APPLICATION OF IMMUNE THERAPY FOR THE TREATMENT FOR MULTIPLE MYELOMA, FOR DEVELOPING NOVEL BIOMARKERS FOR PATIENT SELECTION, STRATIFICATION AND SEQUENCING OF THERAPIES, FOR SPECIFIC IMMUNE THERAPIES, FOR MONITORING RESPONSE TO TREATMENT AND FOR DRIVING THE IDENTIFICATION OF NOVEL DRUG. ALMOST HALF A MILLION DOLLARS WAS SPENT IN 2019 FOR THIS PROGRAM EXPECTED TO COST $4M OVER THE NEXT TWO YEARS.THE THIRD NEW INITIATIVE IS THE MMRF PREVENTION PROJECT, WHICH FOCUSES ON DETERMINING WHY THE PRECURSOR CONDITIONS MGUS AND SMOLDERING (SMM) QUICKLY PROGRESS TO MM IN SOME PATIENTS AND HOW TO IDENTIFY THIS HIGH-RISK POPULATION. $4 MILLION WAS AWARDED TO FIVE INSTITUTIONS FOR WORK ON THE MMRF PREVENTION PROJECT. AT THE END OF 2019, THE MMRF LAUNCHED THE PRECISION PREVENTION INITIATIVE UNDER THE MMRF PREVENTION PROGRAM. THIS IS A COLLABORATIVE EFFORT BETWEEN THE MMRF CURE CLOUD AND DFCI PRECURSOR STUDIES (PROMISE AND PCROWD). THE MUTUAL GOAL OF THIS 5-YEAR COLLABORATIVE INITIATIVE IS TO FOLLOW SMOLDERING PATIENTS LONGITUDINALLY AND GENERATE A ROBUST GENOMIC/IMMUNE/CLINICAL DATASET WHICH WOULD HELP US REDEFINE THE RISK STRATIFICATION MODELS AND IDENTIFY COMPREHENSIVE PROGNOSTIC AND PREDICTIVE BIOMARKERS OF DISEASE PROGRESSION. THE PREVENTION PROGRAM WILL PROVIDE FUNDING OF UP TO $3.966 MILLION TO SUPPORT THE CORRELATIVE STUDIES AND ASSOCIATED COSTS.

THE MULTIPLE MYELOMA RESEARCH CONSORTIUM, LLC. (MMRC) IS A SUBSIDIARY OF THE MULTIPLE MYELOMA RESEARCH FOUNDATION. THE MMRC IS THE FIRST RESEARCH MODEL THAT BRINGS TOGETHER 25 LEADING ACADEMIC CENTERS WITH INDUSTRY PARTNERS TO CONDUCT HIGHLY COLLABORATIVE PHASE 1 AND PHASE 2 CLINICAL TRIALS OF NOVEL COMPOUNDS AND COMBINATION TREATMENTS FOR MULTIPLE MYELOMA. ALL CENTERS ARE DRIVEN BY HIGHLY DEFINED METRICS THAT MANDATE ACCOUNTABILITY AND STRONGLY PROMOTE TEAM SCIENCE. IN 2019, THE MMRF THROUGH THE MMRC OPENED FIRST-EVER PLATFORM CLINICAL TRIAL FOR MULTIPLE MYELOMA AND IS THE IND (INVESTIGATIONAL NEW DRUG) HOLDER, THE MYELOMA-DEVELOPING REGIMENS USING GENOMICS (MYDRUG, NCT03732703). MYDRUGSCREENS PATIENTS' TUMOR SAMPLES FOR GENETIC SIGNATURES AND RANDOMIZE THEM TO APPROPRIATE TARGETED THERAPIES, ALLOWING FOR GREATER EFFICIENCY IN ASSESSING TREATMENT OPTIONS. TOWARD THE END OF 2019, A SECOND PLATFORM STUDY CALLED MYCHECKPOINT (NCT04150965) FOR WHICH THE MMRC IS ALSO THE IND HOLDER WAS APPROVED BY OUR PHARMA PARTNER AND MOVED TOWARD ACTIVATION. WHILE MYDRUG TARGETS UPFRONT HIGH RISK PATIENTS, MYCHECKPOINT IS BRINGING NEW IMMUNOTHERAPIES TO VERY ADVANCED RELAPSED PATIENTS WHO ARE RUNNING OUT OF OPTIONS. AN ADDITIONAL THREE IST CLINICAL TRIALS OPENED IN 2019 AND THE MMRC ENROLLED OVER 160 PATIENTS ACROSS 20 TRIALS. FROM ITS INCEPTION, THE MMRC HAS ENROLLED OVER 2,150 PATIENTS ACROSS MORE THAN 75 CLINICAL TRIALS

THE MMRF LONGITUDINAL STUDY (COMMPASS) COMMENCED IN 2011 AND HAS BEEN EXTENDED THROUGH 2023. MMRF IS THE SPONSOR OF A PERSONALIZED MEDICINE INITIATIVE IN WHICH 1,153 NEWLY DIAGNOSED PATIENTS HAVE BEEN ENROLLED TO DATE THROUGH 108 SITES (I.E., HOSPITALS, ACADEMIC MEDICAL CENTERS AND OTHER COMMUNITY HEALTH CENTERS) ACROSS NORTH AMERICA AND EUROPE. THIS IS AN OBSERVATIONAL STUDY, RATHER THAN AN INTERVENTIONAL ONE WITH EXPERIMENTAL DRUGS BEING TESTED. BIOSPECIMENS (LIKE BLOOD AND BONE MARROW SAMPLES) ARE COLLECTED FROM THE PATIENTS PERIODICALLY OVER THE COURSE OF THEIR TREATMENT ALONG WITH CORRELATING CLINICAL DATA. THE STANDARD OF CARE (I.E., DRUGS AND TREATMENT) FOR EACH PATIENT IS DETERMINED BY SUCH PATIENT'S PERSONAL PHYSICIAN. THE TISSUE SAMPLES COLLECTED ARE PLACED IN A BIOBANK. AN UNRELATED, THIRD PARTY, NOT-FOR-PROFIT BIOMEDICAL RESEARCH INSTITUTE THEN PERFORMS LABORATORY TESTS ON A PORTION OF EACH OF THE SAMPLES RESULTING IN GENOMIC DATA ABOUT EACH PATIENT. OUR GOAL IS TO BUILD AND ANALYZE THE LARGEST FULLY ACCESSIBLE CLINICO-GENOMIC DATASET OF ANY CANCER. THE FINDINGS CANNOT BE PATENTED AND ALL THE DATA ARE PLACED ON A PUBLIC PORTAL (THE MMRF RESEARCHER GATEWAY). THE RESULTING DATA REPOSITORY IS THE MOST COMPREHENSIVE CATALOG OF MULTIPLE MYELOMA AND CONTAINS THE GREATEST AMOUNT OF WHOLE GENOME SEQUENCING DATA OF ANY CANCER, ACCESSIBLE TO ANY RESEARCHER IN THE WORLD. IT WILL PROVIDE FAR MORE INFORMATION THAN IS AVAILABLE FROM CURRENT CANCER TISSUE BANKS THAT TYPICALLY INCLUDE ONLY ONE SAMPLE PER PATIENT.IN 2015, COMMPASS WAS CLOSED TO NEW ENROLLMENT WITH THE AFOREMENTIONED 1,153 PATIENTS WHO ARE BEING FOLLOWED FOR A PERIOD OF 8 YEARS UNTIL 2023. IN DECEMBER 2016, THE MMRF LAUNCHED A SIMILAR SAMPLE/DATA SHARING COLLABORATION WITH ECOG (THE "EASTERN COOPERATIVE ONCOLOGY GROUP"). THE INITIATIVE IS NOW CLOSED TO ENROLLMENT AND ACCRUED APPROXIMATELY 100 NEW SAMPLES.

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