2020-11-13T16:48:57-06:00 2019-12-31 133628255 CONDON O'MEARA MCGINTY & DONNELLY LLP ONE BATTERY PARK PLAZA NEW YORK NY 10004 990 2019-01-01 202014968 CHARLEY'S FUND INC CHAR 4135280400 635 MAIN STREET SUITE 3 GREAT BARRINGTON MA 01230 TRACY KRAMER SECKLER CO-FOUNDER/CEO 4135280400 2020-11-13 1 JAMES J REILLY P00183769 2126617777 2019 2022-09-23 18:48:47Z TRACY KRAMER SECKLER 635 MAIN STREET SUITE 3 GREAT BARRINGTON MA 01230 2300337 0 X WWW.CHARLEYSFUND.ORG X 2004 MA TO ACCELERATE THE DEVELOPMENT OF LIFE-SAVING TREATMENTS FOR DUCHENNE MUSCULAR DYSTROPHY "DMD" 8 7 4 8 0 0 2757831 2082116 0 100039 12225 14707 -1312 253 2768744 2197115 1522481 1511340 0 0 478333 521578 0 0 314446 260701 243521 2261515 2276439 507229 -79324 5773778 5842303 224443 372292 5549335 5470011 X CHARLEY'S FUND'S SINGULAR MISSION IS TO SUPPORT THE DEVELOPMENT OF TREATMENTS AND A CURE FOR DUCHENNE MUSCULAR DYSTROPHY (DMD). WE IDENTIFY AND DIRECT FUNDING TO THE MOST COMPELLING RESEARCH AND TREATMENT PROGRAMS RELATED TO DMD AND, (CONT. ON SCH. O.) 0 0 1451037 1200000 SEE SCHEDULE O 386620 311340 SEE SCHEDULE O 100039 AS PART OF CHARLEY'S FUND, INC. GENERAL SUPPORT OF THERAPEUTIC DEVELOPMENT, THIS 2019 REVENUE WAS TRIGGERED BY A MILESTONE DETAILED IN A 2016 FUNDING AGREEMENT WITH AKASHI THERAPEUTICS, INC. 1837657 1 1 0 0 0 0 0 0 0 1 1 0 1 0 0 0 0 1 0 0 1 0 0 0 0 0 0 1 0 1 0 0 0 0 0 0 0 0 1 0 0 0 1 1 1 0 0 0 1 11 0 1 4 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 X 8 7 1 0 0 0 0 0 0 1 1 0 0 0 1 1 1 1 1 1 1 0 CA CT FL IL MA NJ NY OH WA X SARAH DONALDSONTHE FUND 4135280400 635 MAIN ST STE 3 GREAT BARRINGTON MA 01230 BENJAMIN SECKLER MD BOARD CHAIRMAN 15.00 X X 0 0 0 EARL KRAMER TREASURER 2.00 X X 0 0 0 KAREN CLIMO CLERK 2.00 X X 0 0 0 KATHE KRAMER CHASE BOARD MEMBER 2.00 X 0 0 0 RANDY REIFF BOARD MEMBER 2.00 X 0 0 0 TRACY KRAMER SECKLER CO-FOUNDER & CEO 40.00 X 74160 0 0 ELLEN WILNER BOARD MEMBER 2.00 X 0 0 0 MARK AVAGLIANO BOARD MEMBER 2.00 X 0 0 0 LAURA DALLE PAZZE PRESIDENT & COO 55.00 X 235206 0 5979 309366 0 5979 1 0 1 0 0 2082116 95847 2082116 RESEARCH DEVELOPMENT AWARD 900099 100039 100039 100039 14707 14707 95847 95847 0 8069 7375 694 694 LOSS ON INVESTEMNT IN LLC 900099 -441 -441 -441 2197115 100292 0 14707 1208694 1208694 302646 302646 315345 204974 27827 82544 163708 50394 113314 22420 687 6315 15418 20105 955 6196 12954 5175 4228 710 237 22700 650 22050 781 781 20025 5628 14397 49664 2095 1380 46189 23586 8295 3544 11747 18154 17370 105 679 2930 2930 463 463 6706 2236 2235 2235 SCIENTIFIC CONSULTING 65130 65130 OTHER 28207 11139 2336 14732 2276439 1837657 124336 314446 10103 7772 1106648 923480 1432833 1688342 12938 11137 4234 4455 11483 10150 797 1333 3202775 3202334 3450 3450 5773778 5842303 30358 27684 194085 344608 224443 372292 X 4299335 4052119 1250000 1417892 5549335 5470011 5773778 5842303 -79324 0 X 0 1 X 1 0 X 1550470 1054965 1415266 2757831 2082116 8860648 1550470 1054965 1415266 2757831 2082116 8860648 1637257 7223391 1178 3119820 4746 11152 13926 3150822 38 38 12011508 3220734 0.60140 0.56750 X SCHEDULE A, PART II, LINE 10, EXPLANATION OF OTHER INCOME: OTHER - 2017 AMOUNT: $ 38. RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 1250000 979558 811666 1417892 94621 1825000 669621 1250000 35000 205000 145379 94621 64707 40000 69707 35000 130000 65293 64707 1.00000 0 0 11483 10150 1333 1333 INVESTMENT - DMD SOLUTIONS,LLC -6754 C INVESTMENT - TCW CATALYST,LLC -414 C INVESTMENT - WIDE VISION LLC 125000 C INVESTMENT - DART THERAPEUTICS 84502 C INVESTMENT - AKASHI THERAPEU. 3000000 C 3202334 PART V, LINE 4: TEMPORARILY RESTRICTED NET ASSETS CONSIST OF CONTRIBUTIONS THAT ARE RESTRICTED BY THE DONOR FOR A SPECIFIC PURPOSE OR RELATE TO FUTURE PERIODS. 1 EUROPE (INCLUDING ICELAND & GREENLAND) 1 0 CONTRACT TO SUPPORT THE LEIDEN UNIVERSITY MEDICAL CENTER IN DEVELOPING BEST PRACTICE GUIDELI GRANTS 162349 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM 1 0 CONTRACT TO SUPPORT THE UNIVERSITA DEGLI STUDI DI BARI IN DEVELOPING BEST PRACTICE GUIDELI GRANTS 140297 EUROPE (INCLUDING ICELAND & GREENLAND) 1 0 CONTRACT TO RECEIVE SUPPORT FROM DUCHENNE UK TO HELP FUND THE ABOVE TWO PROJECTS FUNDRAISING 191892 3 0 0 0 3 0 494538 0 494538 0 0 0 0 0 0 PART I, LINE 2: THE GRANTEES SEND CHARLEY'S FUND PROGESS REPORTS, AND WHEN AGREED-UPON MILESTONES ARE MET, CHARLEY'S FUND MAKE PAYMENTS PER THE FUNDING AGREEMENT. PART III ACCOUNTING METHOD: 1 UMASS MEMORIAL FOUNDATION 333 SOUTH ST SHREWSBURY MA 01545 043108190 501(C)(3) 1200000 RESEARCH GRANT BETH ISRAEL DEACONESS MEDICAL CENTER 302 E 38TH ST NEW YORK NY 10016 042103881 501(C)(3) 8694 RESEARCH GRANT 2 0 PART I, LINE 2: ALL GRANTS ARE PROVIDED UNDER CONTRACTS WITH THE GRANT RECIPIENTS. THE CONTRACTS REQUIRE GRANTEES TO MEET CERTAIN EVENT AND PERFORMANCE MILESTONES AND PERIODIC PROGRESS REPORTS TO CHARLEY'S FUND, INC. ARE REQUIRED. THE BOARD OF DIRECTORS REVIEWS AND APPROVES GRANTS ACCORDING TO THE ORGANIZATION'S POLICY ON FUNDING AND EXPENSE APPROVALS. 0 0 0 0 0 0 0 0 0 LAURA DALLE PAZZE PRESIDENT & COO 205653 0 29553 0 0 0 0 0 5979 0 241185 0 0 0 X 5 95847 FMV 0 1 0 PART III - LINE 1 IMPORTANTLY, AMPLIFY THE IMPACT OF THAT FUNDING WITH STRATEGIC PROBLEM-SOLVING SUPPORT TO BOTH AWARDEES AND THE BROADER DMD RESEARCH COMMUNITY. TO BEAT DMD, IT'S NOT ENOUGH TO FIND THE BEST RESEARCH; WE ALSO MUST PROACTIVELY IDENTIFY AND KNOCK DOWN HURDLES THAT STAND IN THE WAY OF EFFICIENT DRUG DEVELOPMENT. SINCE INCEPTION, WE HAVE DIRECTED MORE THAN $45M IN FUNDING TO THESE EFFORTS, LEVERAGING BOTH OUR OWN FUNDING AS WELL AS FUNDING FROM OTHER DMD PATIENT ORGANIZATIONS TO IDENTIFY AND SUPPORT THE BEST SCIENCE IN PURSUIT OF TREATMENTS AND A CURE FOR DMD. IN 2019, WE DEDICATED A TOTAL OF $1,837,658 TO OUR PROGRAMMATIC EFFORTS. OF THIS, $1,511,340 WAS DEDICATED TO GRANTS AND ALLOCATIONS, AND $326,318 WAS DEDICATED TO OTHER PROGRAMMATIC EXPENSES. PART III - LINE 4A WE CONTINUED OUR SUPPORT OF THE DUCHENNE PROGRAM AT UMASS MEDICAL SCHOOL, WHICH WE CREATED IN 2018. THE PROGRAM IS POWERED BY A UNIQUE COLLABORATION AMONG THE MEDICAL, RESEARCH, AND PATIENT COMMUNITIES. FOUNDING CLINIC DIRECTOR DR. BRENDA WONG, HOST INSTITUTION UMASS MEDICAL CENTER AND MEDICAL SCHOOL, AND CHARLEY'S FUND WORK JOINTLY TO ENSURE THAT THE DUCHENNE PROGRAM - THE LARGEST DEDICATED DUCHENNE CLINIC IN THE NORTHEAST US - DELIVERS WORLD-CLASS RESULTS ON THREE KEY GOALS: 1) DELIVER COMPREHENSIVE, INTEGRATED CARE TO PATIENTS; 2) HOST CUTTING-EDGE CLINICAL TRIALS; AND 3) DRIVE ORIGINAL RESEARCH AND DISCOVERIES TO POWER THE NEXT WAVE OF BREAKTHROUGHS.THE DUCHENNE PROGRAM, NOW IN ITS SECOND YEAR, HAS MORE THAN 300 PATIENTS IN ITS DIRECT CARE AND CONTINUES TO BENEFIT PATIENTS WORLDWIDE EVEN IF THEY DO NOT ENTER ITS DOORS IN PERSON THROUGH SPONSORED CLINICAL TRIALS, ORIGINAL RESEARCH, AND SHARING BEST PRACTICES IN RESEARCH AND CARE WITH THE BROADER COMMUNITY. THIS YEAR, IN ADDITION TO A ROSTER OF INDUSTRY-SPONSORED CLINICAL TRIALS, THE CLINIC SECURED IRB APPROVAL FOR ONGOING REGISTRY WORK AND LAUNCHED A BIOREPOSITORY. PART III - LINE 4B THROUGH OUR IMPACT INITIATIVE, WE WORK WITH PARTNERS IN THE FIELD TO DEVELOP THEIR SCIENTIFIC PROGRAMS AND TO IDENTIFY RESEARCH ROADBLOCKS AND POTENTIAL SOLUTIONS. THE IMPACT INITIATIVE SPEEDS AND IMPROVES ALL RESEARCH - NOT JUST ONE PARTICULAR THERAPY - BY IDENTIFYING AND THEN SOLVING SYSTEMIC PROBLEMS THAT MAKE DRUG DEVELOPMENT SLOWER, MORE DIFFICULT AND MORE COSTLY. CURRENTLY, THE IMPACT INITIATIVE FOCUSES ON THREE AREAS: 1) IMPROVING OUTCOME MEASURES, 2) IMPROVING PRECLINICAL METHODS AND DECISION-MAKING, AND 3) IMPROVING THE DESIGN, EXECUTION AND ANALYSIS OF CLINICAL TRIALS FOR DMD. WE DEVELOPED THE IMPACT INITIATIVE BECAUSE RIGOROUS SCIENCE - UNDERTAKEN WITH URGENCY AND FOCUS - IS THE ONLY WAY WE GET TO THE OUTCOMES WE AND ALL DUCHENNE FAMILIES WANT: DRUGS THAT WORK, AND WORK WELL. SOLVING THESE FIELD-WIDE PROBLEMS AND IMPROVING OUR TOOLS AND RESOURCES IS AN ABSOLUTE MUST TO GET RESULTS. OUTCOME MEASURES ONE ASPECT OF THE DRUG DEVELOPMENT PROCESS THAT CONCERNS US IS THE LACK OF RELIABLE OUTCOME MEASURES THAT CAN DETERMINE THE EFFICACY OF AN INTERVENTION OVER A REASONABLY SHORT PERIOD OF TIME. IN 2019 CASIMIR, LLC CONTINUED ITS WORK TO ADDRESS THIS IMPORTANT NEED BY DEVELOPING A SERIES OF OUTCOME MEASURES DESIGNED TO QUANTIFY PATIENT QUALITY OF MOVEMENT USING VIDEOS OF BOYS WITH DMD PERFORMING DAILY TASKS. WITH OUR SUPPORT, CASIMIR HAS PURSUED ACTIVITIES TO DEVELOP AND REFINE THEIR SCORING SYSTEM, GATHER KEY BASELINE DATA NEEDED TO DETERMINE ITS RELIABILITY AND CONCEPT VALIDITY. THE FDA CONTINUES TO BE SUPPORTIVE AND RECENTLY 'RENEWED' ITS ENDORSEMENT FOR CASIMIR TO CONTINUE WORK WITH AN FDA-ADJACENT ADVISORY BODY THAT PROVIDES HANDS-ON INPUT DURING DEVELOPMENT. ALSO IN 2019, WE CONTINUED OUR SUPPORT FOR ELECTRICAL IMPEDANCE MYOGRAPHY (EIM), COLLABORATING WITH EIM FOUNDER DR. SEWARD RUTKOVE TO ASSESS ITS SUITABILITY AS AN OUTCOME MEASURE FOR DMD. EIM UTILIZES A SMALL, HAND-HELD DEVICE TO RUN A HIGH-FREQUENCY CURRENT THROUGH A MUSCLE. IT IS PAINLESS, RELATIVELY INEXPENSIVE, TAKES SECONDS TO PERFORM AND CAN BE USED ON PATIENTS OF ANY AGE. WITH OUR SUPPORT, DR. RUTKOVE HAS COMPLETED CRITICAL STUDIES TO CONFIRM EIM'S RELIABILITY, CONSISTENCY AND ABILITY TO DIFFERENTIATE HEALTHY MUSCLE FROM DISEASED MUSCLE. OUR PUBLICATION WITH THE RESULTS OF THE MOST RECENT MULTI-SITE STUDY HAS BEEN PUBLISHED IN ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY. CHARLEY'S FUND ALSO CONTINUES TO EXPLORE A POTENTIAL CLINICAL STUDY TO OBTAIN KEY OUTSTANDING DATA IN A MORE EFFICIENT, EFFECTIVE MANNER THAN TWO SEPARATE APPROACHES, MENTIONED ABOVE. BOTH PROGRAMS ARE AT A POINT WHERE THE CRITICAL NEED IS DATA TO HELP ANSWER A KEY QUESTION: "HOW ACCURATELY DO THESE MEASURES DETECT TREATMENT EFFECT?" SINCE STEROID TREATMENT IS THE ONLY APPROVED INTERVENTION KNOWN TO ALTER DISEASE PROGRESSION, IN 2019 WE STARTED CONVERSATIONS WITH BOTH THE CASIMIR AND EIM TEAMS TO CONSIDER AN OBSERVATIONAL STUDY TO EVALUATE THE TWO EXPLORATORY MEASURES' SENSITIVITY TO CHANGE IN DISEASE UPON INITIATION OF STEROID TREATMENT. PRECLINICAL METHODS ONE MAJOR REASON WE HAVE TROUBLE TRANSLATING PROMISING RESEARCH RESULTS INTO ACTUAL THERAPIES IS THE SHORTCOMINGS OF HOW PRECLINICAL METHODS AND MODULES ARE USED BY DMD RESEARCHERS. OUR "OF MICE AND MEASURES" PROGRAM (OMAM) IS A COLLABORATIVE, ONGOING EFFORT THAT BRINGS TOGETHER EXPERTS FROM ACADEMIA AND INDUSTRY TO IMPROVE THE DECISION-MAKING PROCESS FOR WHICH DRUGS SHOULD ADVANCE TO CLINICAL TESTING. TO THIS PURPOSE, IN 2019 WE COMMITTED $302,646 TO LEIDEN UNIVERSITY MEDICAL CENTER IN THE NETHERLANDS AND UNIVERSITA DEGLI STUDI DI BARI IN ITALY FOR A LARGE NATURAL HISTORY STUDY TO BETTER CHARACTERIZE KEY DMD MODELS. A KEY COMPONENT OF THE STUDY IS TO UNEARTH DISCREPANCIES IN THE PROCESSES DIFFERENT LABS UNDERTAKE AND THEN PUBLICIZE THE BEST METHODOLOGY TO ENABLE RELIABLE, CONSISTENT RESULTS. CLINICAL TRIALS THE COLLABORATIVE TRAJECTORY ANALYSIS PROJECT (CTAP) IS A COLLABORATIVE EFFORT AMONG KEY STAKEHOLDERS IN THE INDUSTRY, ACADEMIC, AND PATIENT COMMUNITIES TO IMPROVE THE WAY WE DESIGN, EXECUTE, AND ANALYZE CLINICAL TRIALS IN DUCHENNE. IT MAKES USE OF A GROWING BODY OF DMD NATURAL HISTORY AND CLINICAL TRIAL DATA AND CUTTING-EDGE BIOSTATISTICAL ANALYSIS APPROACHES TO IMPROVE OUR UNDERSTANDING OF THE NATURAL HISTORY OF DUCHENNE AND THUS, HOW SPONSORS CAN BETTER DESIGN TRIALS TO IDENTIFY WHEN THEIR TREATMENT SUCCEEDS IN CHANGING THAT NATURAL HISTORY. CTAP WORKS TO PROVIDE 1) ONGOING INVESTIGATION AND PUBLICATION ON KEY QUESTIONS FACING THE FIELD AND IMPACTING CLINICAL TRIAL DESIGN, AND 2) TARGETED SUPPORT FOR SPONSORS DESIGNING AND RUNNING TRIALS TODAY. TO SUPPORT CTAP'S WORK, CHARLEY'S FUND PROVIDES BOTH FUNDING AND ONGOING ADVICE AND GUIDANCE IN ROLES ON THE JOINT STEERING AND COMMUNICATIONS COMMITTEES. FORM 990, PART VI, SECTION A, LINE 2 BENJAMIN SECKLER, MD (BOARD CHAIRMAN), IS HUSBAND TO TRACY KRAMER SECKLER (BOARD MEMBER). EARL KRAMER (TREASURER), IS FATHER TO TRACY KRAMER SECKLER (BOARD MEMBER) AND KATHE KRAMER CHASE (BOARD MEMBER). KATHE KRAMER CHASE (BOARD MEMBER), AND TRACY KRAMER SECKLER (BOARD MEMBERS), ARE SISTERS. FORM 990, PART VI, SECTION B, LINE 11B NO REVIEW WAS OR WILL BE CONDUCTED. FORM 990, PART VI, SECTION B, LINE 12C OFFICERS, DIRECTORS AND KEY EMPLOYEES MUST REVIEW THE POLICY ANNUALLY AND SIGN A DECLARATION THAT THEY HAVE COMPLIED WITH THE POLICY. FORM 990, PART VI, SECTION B, LINE 15 COMPENSATION OF TOP MANAGEMENT AND OFFICERS OF THE ORGANIZATION IS DEVELOPED IN CONSIDERATION OF PERFORMANCE AND REVIEW OF RELEVANT COMPETITIVE BENCHMARKS AND SUBMITTED TO FINANCE BOARD MEMBERS FOR APPROVAL. FORM 990, PART VI, SECTION C, LINE 19 THE ORGANIZATION MAKES ITS GOVERNING DOCUMENTS, CONFLICT OF INTEREST POLICY AND FINANCIAL STATEMENTS AVAILABLE TO THE PUBLIC UPON REQUEST. CHARLEY'S FUND VENTURES LLC 635 MAINT STREET GREAT BARRINGTON MA 01230 275009330 FUND RAISING MA 3085615 N/A DARIUS GOES WEST INC C/O LOGAN SMALLEY 135 TOPS COURT ATHENS GA 30606 262174272 FUNDRAISING GA 501(C)(3) 509 (A) (3) N/A 0 WIDE VISION DMD CAPITAL LLC 2266 WEST END NEW YORK NY 10025 INVESTMENT MA N/A RELATED 125000 0 0 DMD SOLUTIONS LLC 635 MAIN STREET GREAT BARRINGTON MA 01230 271107747 INVESTMENT MA N/A RELATED 87 -6754 0 0 TCW CATALYST LLC 4904 MERILANE AVE EDINA MN 55436 208875302 INVESTMENT MN N/A RELATED -179 -414 0 0 DART THERAPEUTICS LLC 635 MAIN STREET GREAT BARRINGTON MA 01230 INVESTMENT MA N/A RELATED -175 84502 0 0 AKASHI THERAPEUTICS INC 245 FIRST ST 18TH FLOOR CAMBRIDGE MA 02142 DRUG DEVELOPMENT MA CHARLEY'S FUND C 3000000 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0