2015-05-08T13:35:40-05:00 2014-12-31 231518888 MAILLIE LLP 624 WILLOWBROOK LANE WEST CHESTER PA 19382 990 2014-01-01 522122720 FRIEDREICH'S ATAXIA RESEARCH ALLIANCE FRIE 4848796160 PO BOX 1537 SPRINGFIELD VA 22151 JENNIFER FARMER EXECUTIVE DIRECTOR 4848796160 2015-04-20 1 EDWARD FRONCZKOWSKI CPA P01259092 6106964353 2014 2016-03-07 17:11:31Z JENNIFER FARMER 533 W UWCHLAN AVENUE DOWNINGTOWN PA 19335 5996377 0 X WWW.CUREFA.ORG X 1998 VA TO TREAT AND CURE FRIEDREICH'S ATAXIA BY ADVANCING RESEARCH, AWARENESS AND PARTNERSHIPS. 16 15 6 350 0 0 3852500 4958831 0 0 59864 287653 75480 -3737 3987844 5242747 2806246 4113902 0 0 451977 462636 0 0 96708 211229 233456 3469452 4809994 518392 432753 3688078 4011934 381693 290640 3306385 3721294 X TO MARSHAL AND FOCUS THE RESOURCES AND RELATIONSHIPS NEEDED TO CURE FA BY RAISING FUNDS FOR RESEARCH, PROMOTING PUBLIC AWARENESS AND ALIGNING SCIENTISTS, PATIENTS, CLINICIANS, GOV'T AGENCIES AND PHARMACEUTICAL COMPANIES DEDICATED TO CURING FA AND RELATED DISORDERS. 0 0 4337835 4103902 RESEARCH & GRANT PROGRAM: FARA'S COMMITMENT TO FUNDING HIGH QUALITY RESEARCH PROJECTS THAT MOVE US CLOSER TO EFFECTIVE TREATMENTS CONTINUED AT AN ACCELERATED PACE IN 2014. IN TOTAL, FARA PROVIDED >$4M IN BASIC AND TRANSLATIONAL AND CLINICAL RESEARCH GRANTS, A NEW FRIEDREICH'S ATAXIA CENTER OF EXCELLENCE, SCIENTIFIC CONFERENCE SUPPORT AND CLINICAL RESEARCH INFRASTRUCTURE FUNDING. OVER THE PAST YEAR, >35 NEW LETTERS OF INTENT FOR FUNDING REQUESTS WERE SUBMITTED, 21 FULL APPLICATIONS WERE SUBMITTED AND AFTER RIGOROUS PEER REVIEW, 15 WERE DETERMINED MERITORIOUS AND AWARDED FUNDING IN 2014 AND EARLY 2015. IN ADDITION, BASED ON EXCELLENT PROGRESS, CONTINUATION FUNDING WAS PROVIDED FOR 14 PROJECTS, FOR A TOTAL OF 29 RESEARCH GRANTS AWARDED. FARA ALSO AWARDED TWO CONFERENCE GRANTS TO SUPPORT SCIENTIFIC MEETINGS WITH CONTENT FOCUSED ON FRIEDREICH'S ATAXIA. THESE FUNDED PROJECTS ADDRESS NUMEROUS IMPORTANT RESEARCH QUESTIONS THAT DIRECTLY TARGET FARA'S STRATEGIC RESEARCH INITIATIVES, INCLUDING EVALUATING OF MECHANISMS OF DISEASE, DRUG DISCOVERY, PRE-CLINICAL AND CLINICAL STUDIES OF LEAD CANDIDATES IN THE TREATMENT PIPELINE, ADVANCES IN GENE AND CELL THERAPY APPROACHES, ADVANCES IN DIFFERENTIATION OF INDUCED PLURIPOTENT STEM (IPS) CELL LINES FROM FA PATIENTS INTO SENSORY NEURONS AND CARDIOMYOCYTES, DEVELOPMENT OF NEW ANIMAL MODELS, CARDIAC RESEARCH AND EVALUATION OF NOVEL BIOMARKERS. SEVERAL ARE CO-FUNDED WITH OUR FRIEDREICH'S ATAXIA ADVOCACY GROUP PARTNERS; THIS COLLABORATIVE APPROACH BOTH INCREASES THE NUMBER OF AVAILABLE RESEARCH DOLLARS AND HELPS TO MINIMIZE DUPLICATION OF EFFORT. OF NOTE, FA SHARES SIMILAR SYMPTOMS AND DISEASE MECHANISMS WITH OTHER DISEASES, BOTH RARE AND COMMON. RESEARCH INTO FA IS PROVIDING INSIGHTS AND ADVANCES IN OTHER DISEASES SUCH AS MITOCHONDRIAL DISEASES, MUSCULAR DYSTROPHIES, DIABETES, AND CARDIOMYOPATHY. NEW IN 2014 - FRIEDREICH'S ATAXIA CENTER OF EXCELLENCE, PHILADELPHIA PAIN MARCH, FARA ANNOUNCED THE ESTABLISHMENT OF THE PENN MEDICINE/ CHILDREN'S HOSPITAL OF PHILADELPHIA FRIEDREICH'S ATAXIA CENTER OF EXCELLENCE. THE CENTER WAS LAUNCHED WITH A GIFT OF $3.25 MILLION (OVER 3 YEARS) PRESENTED BY FARA IN PARTNERSHIP WITH THE HAMILTON AND FINNERAN FAMILIES. THIS CENTER WILL HELP TO EXPEDITE BASIC SCIENCE AND DRUG DISCOVERY FINDINGS FOR NEW TREATMENTS, PROVIDE RESOURCES FOR CLINICAL RESEARCH AND CARE THAT WILL HELP TO INCREASE UNDERSTANDING OF THE DISEASE, AND IMPROVE OUTCOMES FOR INDIVIDUALS LIVING WITH FRIEDREICH'S ATAXIA. PROJECTS AND INVESTIGATORS BEING FUNDED INCLUDE DR. ROB WILSON- DRUG DISCOVERY, DR. DAVID LYNCH- CLINICAL AND NEUROSCIENCE RESEARCH, DR, IAN BLAIR- METABOLIC BIOMARKERS, AND DR. KIM LIN- CARDIAC RESEARCH.A FEW HIGHLIGHTED NEW RESEARCH GRANTS:2014 KEITH MICHAEL ANDRUS MEMORIAL AWARD (FOR CARDIAC RESEARCH), TITLE: IDENTIFICATION OF THERAPEUTIC COMPOUNDS ON A CARDIAC DROSOPHILA MODEL OF FRIEDREICH'S ATAXIA, PRINCIPAL INVESTIGATOR, DR. VERONIQUE MONNIER (UNVERSITE' PARIS)DR. VERONIQUE MONNIER AND HER TEAM HAVE DEVELOPED A DROSOPHILA CARDIAC MODEL OF FRIEDREICH'S ATAXIA THAT RECAPITULATES THE DEFECTS OF CARDIAC FUNCTION OBSERVED IN PATIENTS AND FRDA MOUSE MODELS, IN PARTICULAR HEART DILATATION AND IMPAIRED SYSTOLIC FUNCTION. IN A RECENT PUBLICATION DESCRIBING THIS FRDA FLY MODEL, THEY SHOWED THAT METHYLENE BLUE, A CLINICALLY APPROVED DRUG, WAS ABLE TO PREVENT THE HEART DYSFUNCTION (TRICOIRE H, PALANDRI A, BOURDAIS A, CAMADRO JM, MONNIER V. HUMAN MOLECULAR GENETICS 23:968- 979, 2014). THE MAJOR GOAL OF THIS PROJECT IS TO USE THE DROSOPHILA CARDIAC MODEL TO IDENTIFY NEW THERAPEUTIC COMPOUNDS THROUGH A MEDIUM-SCALE UNBIASED DRUG SCREENING OF 1280 COMPOUNDS. CARDIAC IMAGING WILL BE PERFORMED IN VIVO TO SELECT COMPOUNDS THAT IMPROVE THE HEART FUNCTION OF THE FLY. THIS DRUG SCREEN HAS GREAT POTENTIAL FOR IDENTIFYING NOVEL COMPOUNDS THAT ARE LIKELY TO LEAD TO THERAPEUTIC INTERVENTIONS FOR FRDA. 2014 KYLE BRYANT TRANSLATIONAL RESEARCH AWARD, TITLE: SMALL MOLECULE INDUCED EXON SKIPPING OF MLH3 TO SLOW REPEAT EXPANSION IN AN FRDA MOUSEL MODEL, PRINCIPAL INVESTIGATOR, DR. EDWARD GRABCZYK, LOUSIANA STATE UNIVERSITY. DR. GRABCZYK FOUND THAT A PROTEIN CALLED MSH3 CAUSES GAA REPEATS IN THE FXN GENE TO EXPAND. HE THEN DISCOVERED THAT TRANSCRIPTION THROUGH THE REPEAT AND THE SEQUENTIAL ACTIONS OF SEVERAL DNA MISMATCH REPAIR PROTEINS CALLED MUTSBETA (MSH2/MSH3 HETERODIMER) AND THEN MUTLGAMMA (MLH1/MLH3 HETERODIMER) ARE LINKED TO CELL LOSS OR REPEAT EXPANSION IN FRDA PATIENT TISSUE SAMPLES. MUTLGAMMA IS THE PROTEIN COMPLEX THAT CUTS THE DNA IN THE REPEAT TO START THE EXPANSION. WITHOUT THE CUT, THERE IS NO EXPANSION. WHEN TREATING CELLS WITH SPLICE-SWITCHING OLIGONUCLEOTIDES (SSOS), THE REPEAT STOPS EXPANDING. DR. GRABCZYK WILL EXPLORE AND TEST THE USE SSOS TO BLOCK THIS EXPANSION IN MOUSE MODELS OF FA AS A FIRST STEP HEADING TO HUMAN TRIALS. TITLE: ACTIVATION OF FRATAXIN EXPRESSION BY DUPLEX RNA, PRINCIPAL INVESTIGATOR, DR. DAVID COREY,(UT SOUTHWESTERN DR. DAVID COREY DESIGNED DUPLEX RNAS TO RECOGNIZE THE REPEAT REGION IN FA AND POTENTIALLY INTERFERE WITH CHROMATIN CONTACTS THAT MIGHT CONTRIBUTE TO DECREASED TRANSCRIPTION. HE HAS DISCOVERED THAT INTRODUCTION OF THESE DUPLEX RNAS INTO FA-PATIENT DERIVED CELLS CAUSED AN INCREASE OF FXN MRNA AND PROTEIN TO LEVELS SIMILAR TO THOSE IN NORMAL CELLS. THESE DATA SUGGEST THAT DUPLEX RNAS CAN BE USED TO UP-REGULATE FXN EXPRESSION AND MAY PROVIDE A NEW STRATEGY FOR THERAPY. TITLE: EARLY AND LONGITUDINAL ASSESSMENT OF NEURODEGENERATION IN THE BRAIN AND SPINAL CORD IN FRIEDREICH'S ATAXIA CO-PRINCIPAL INVESTIGATORS: PIERRE-GILLES HENRY AND CHRISTOPHE LENGLET (UNIVERSITY OF MINNESOTA) THE OVERALL GOAL OF THIS PROJECT IS TO DEMONSTRATE THE FEASIBILITY OF IDENTIFYING AND TRACKING STRUCTURAL AND NEUROCHEMICAL CHANGES IN THE SPINAL CORD AND BRAIN OF PATIENTS WITH FRDA, ESPECIALLY AT AN EARLY STAGE OF THE DISEASE. DRS. HENRY AND LENGLET WILL USE BOTH MAGNETIC RESONANCE SPECTROSCOPY AND DIFFUSION MAGNETIC RESONANCE IMAGING, WHICH PROVIDE COMPLEMENTARY INFORMATION ON NEUROCHEMICAL AND MICROSTRUCTURAL CHANGES. THESE STUDIES MAY RESULT IN NOVEL AND UNIQUE TOOLS FOR THE QUANTITATIVE ASSESSMENT OF DISEASE PROGRESSION AND TREATMENT EFFICACY IN PROSPECTIVE THERAPEUTIC TRIALS OF FRDA.ADDITIONALLY, FARA CONTINUES TO SUPPORT OF THE DEVELOPMENT OF NEW MOUSE MODELS OF FA WITH RESEARCH FUNDING THE JACKSON LABORATORIES, BAR HARBOR, MAINE AND DR. VIJAY CHANDRAN AT THE UNIVERISTY OF CALIFORNIA LOS ANGELES.RESULTS REPORTED FROM FARA AWARDED GRANTS - IN 2014, THERE WERE >20 MEDICAL AND SCIENTIFIC PUBLICATIONS THAT WERE DETAILED REPORTS OF FARA FUNDED RESEARCH. HERE ARE TWO HIGHLIGHTED PUBLICATIONS:DR. HELENE PUCCIO A RESEARCH DIRECTOR AT THE FRENCH INSTITUTE OF HEALTH AND MEDICAL RESEARCH (INSERM) AND FARA FUNDED SCIENTIST, HAS BEEN TESTING A GENE-THERAPY APPROACH IN THE CARDIAC FA MOUSE MODEL THAT SHE DEVELOPED IN HER LAB AT THE INSTITUTE DE GENETIQUE MOLECULAIRE ET CELLULAIRE. THE RESULTS SHOW THAT A SINGLE INTRAVENOUS INJECTION OF AAVRH10 EXPRESSING FRATAXIN IS NOT ONLY CAPABLE OF PREVENTING THE DEVELOPMENT OF HEART DISEASE IN THE MOUSE, BUT ALSO, FULLY AND RAPIDLY TREATS THE MICE WITH ADVANCED STAGES OF HEART DISEASE, RETURNING THE HEART TO NORMAL FUNCTION. THE RESULTS OF THIS RESEARCH WERE FULLY REPORTED IN NATURE MEDICINE, APRIL 2014. FARA HAS CONTINUED TO FUND BOTH CARDIAC AND NEURONAL GENE THERAPY RESEARCH IN DR. PUCCIO'S LABORATORY. DR. DAVID LYNCH, AT THE CHILDREN'S HOSPITAL OF PHILADELPHIA, COMPLETED IN 2103 AND 2014 A SMALL PILOT PHASE 2A OF STUDY INTERFERON GAMMA IN CHILDREN WITH FA, WITH FUNDING FROM FARA AND SUPPORT FROM VIDARA (NOW HORIZON PHARMA) WHICH DONATED THE DRUG FOR THE INITIAL STUDY. THE PRIMARY PURPOSE WAS TO EVALUATE SAFETY AND TOLERABILITY OF INTERFERON GAMMA AND TO SEE IF THERE WAS AN INCREASE IN FRATAXIN LEVELS. THE FULL RESULTS WERE PRESENTED AT THE CHOP FA SYMPOSIUM AND PUBLISHED IN ACTA NEUROLOGICA SCANDIVANICA IN OCTOBER 2014. IN SUMMARY, WHILE THERE WAS NO SIGNIFICANT CHANGE IN FRATAXIN LEVELS ACROSS THE GROUP, INDIVIDUAL DATA IN CERTAIN TISSUES (BUCCAL CELLS) DISPLAYS A LOT OF "NOISE" - LEVELS SPIKING UP AND DOWN, WHICH IS SOMETHING THAT DOES NOT NORMALLY OCCUR. MORE INTRIGUING, THE CLINICAL DATA OBTAINED FROM THE STUDY SHOWED A SIGNIFICANT IMPROVEMENT IN NEUROLOGICAL FUNCTION MEASURED BY THE FRIEDREICH ATAXIA RATING SCALE (FARS). IT IS IMPORTANT TO NOTE THAT THIS STUDY DID NOT HAVE A CONTROL OR PLACEBO GROUP.. THESE RESULTS ARE SUPPORTIVE OF THE NEED FOR FURTHER INVESTIGATION. HORIZON PHARMA, THE COMPANY THAT SELLS ACTIMMUNE (INTERFERON GAMMA) IN THE UNITED STATES, IS COMMITTED TO MOVING FORWARD. THEY ARE PRESENTLY IN PLANNING STAGES OF WHAT WE HOPE WILL BE A DEFINITIVE PHASE 3 STUDY (PLACEBO CONTROLLED IN A MUCH LARGER GROUP OF INDIVIDUALS WITH FA). 109639 10000 RESEARCH CONFERENCES: FARA ORGANIZES AND SUPPORTS A NUMBER OF SCIENTIFIC CONFERENCES TO IMPROVE SHARING OF KNOWLEDGE, INSIGHTS AND ADVANCES AND BUILD COLLABORATIONS AND SYNERGISTIC CONNECTIONS BETWEEN FA RESEARCHERS. MAY 2014 - FARA AND GOFAR (ITALIAN FA ADVOCACY ORGANIZATION) CO-HOSTED A MEETING ON FRIEDREICH'S ATAXIA MOUSE MODELS IN PHILADELPHIA. ACADEMIC AND BIOPHARMACEUTICAL SCIENTISTS MADE UP THE 50 PARTICIPANTS IN THE MEETING. NEW MOUSE MODELS IN DEVELOPMENT WERE PRESENTED BY M.POOK AND V.CHANDRAN (FARA GRANTEES). FURTHER CHARACTERIZATION OF MITOCHONDRIAL DYSFUNCTION AND NEURODEGENERATION EXISTING MODELS WAS HIGHLIGHTED BY RESEARCH FROM J. MAGRENE AND C. LUTZ (FARA GRANTEES). C. LUTZ FROM THE JACKSON LABORATORIES ALSO LED A DISCUSSION ON FURTHER MOUSE MODEL DEVELOPMENT WORK NEEDED TO FULL GAPS SO THAT MODELS WITH MORE ROBUST AND DISEASE RELEVANT PHENOTYPE WOULD BE AVAILABLE FOR DRUG DEVELOPMENT. AFTER THE MEETING FARA AND GOFAR AWARDED FUNDS TO JACKSON LABORATORIES TO BEGIN NEW MODEL DEVELOPMENT.OCTOBER 2014 - FARA ORGANIZED A MEETING HOSTED AT THE NATIONAL INSTITUTES OF HEALTH ON ALL ASPECTS OF BIOMARKER EXPLORATION IN FA AND THEIR IMPORTANCE TO ADVANCING TREATMENTS. SCIENTISTS, GOVERNMENT OFFICIALS AND PHARMACEUTICAL REPRESENTATIVES MADE UP THE 60 PARTICIPANTS IN THE MEETING. A BIOMARKER CONSORTIUM WAS CREATED OUT OF THE MEETING TO HELP TAKE IDENTIFIED PROJECTS/ NEEDS FORWARD.IN 2014, FARA BEGAN PLANNING THE NEXT FA INTERNATIONAL SCIENTIFIC CONFERENCE. FARA DECIDED TO PARTNER WITH OTHER INTERNATIONAL ATAXIA RESEARCH ORGANIZATIONS TO COMBINE EFFORTS AND CONFERENCE NEEDS. THE 2015 INTERNATIONAL ATAXIA RESEARCH CONFERENCE WILL BE IN WINDSOR, ENGLAND, MARCH 25-28, 2015. FARA IS CO-ORGANIZING AND HOSTING THE MEETING WITH ATAXIA UK, ATAXIA IRELAND AND GOFAR. CONFERENCE STEERING COMMITTEE WAS ESTABLISHED IN EARLY 2014, PRELIMINARY PROGRAM AND INVITED SPEAKERS WERE CONFIRMED AND REGISTRATION AND SCIENTIFIC ABSTRACTS OPENED IN JULY 2014. AT THE END OF 2014 THERE WERE >150 ABSTRACTS SUBMITTED AND >250 CONFIRMED DELEGATES INCLUDING REPRESENTATIVES FROM THE EUROPEAN MEDICAL ASSOCIATION. THE CONFERENCE PROGRAM CAN BE FOUND AT HTTP://WWW.CUREFA.ORG/CONFERENCE.HTML 181540 EDUCATION, AWARENESS & OUTREACH PROGRAMS: FRIEDREICH'S ATAXIA (FA) IS A RARE DISEASE; AFFECTING 1 IN 50,000 INDIVIDUALS. FARA IS DEDICATED TO RAISING AWARENESS FOR FA. FARA HAS UTILIZED BOTH TRADITIONAL AND SOCIAL MEDIA STRATEGIES TO BRING GREATER AWARENESS TO FA IN THE GENERAL PUBLIC AND TO ENGAGE AND EDUCATE THE FA COMMUNITY. FOR EXAMPLE, FARA CONDUCTED A SOCIAL MEDIA CAMPAIGN THAT ENCOURAGED COMMUNITY PARTICIPATION LEADING UP TO RARE DISEASE DAY AND FA AWARENESS DAY. FARA ALSO HOSTED WEBINARS TO INFORM THE COMMUNITY ABOUT THE LATEST DEVELOPMENTS IN FA RESEARCH AND SOME OF THE RESULTS OF FUNDED GRANTS.FARA CO-ORGANIZED TWO PATIENT SYMPOSIUMS WITH CLINICAL RESEARCH NETWORK SITES, CHILDREN'S HOSPITAL OF PHILADELPHIA (CHOP) AND UNIVERSITY OF SOUTH FLORIDA (USF). THE CHOP AND FARA TEAM LED AN ALL-DAY SYMPOSIUM AT IN KING OF PRUSSIA, PA ON OCTOBER 13TH, 2014. THERE WERE MORE THAN 175 PATIENT FAMILIES WHO PARTICIPATED. THE SYMPOSIUM PROVIDED:" CLINICAL UPDATES, INCLUDING CARDIOLOGY AND NEUROLOGY, IN PATIENTS WITH FA;" UPDATES ON CURRENT RESEARCH AND CLINICAL TRIALS, INCLUDING GENE THERAPY, INTERFERON GAMMA, RNA BASED APPROACHES TO UNSILENCING THE GENEAND" PANEL DISCUSSION ON HOME MODIFICATIONS TO IMPROVE ACCESSIBILITY.THE SYMPOSIUM AT USF WAS HELD ON SEPT 4, 2014 HAD ABOUT 200 ATTEND AND WAS STREAMED LIVE ON FACEBOOK AND THERE WERE SEVERAL HUNDRED MORE VIEWS. THE USF SYMPOSIUM FEATURED A DISTINGUISHED RESEARCH PANEL PROVIDING UPDATES ON UNDERSTANDING HOW AND WHY THE GENE MUTATIONS LEADS TO DECREASED FRATAXIN AND SPECIFIC THERAPEUTIC APPROACHES INCLUDING, HDAC INHIBITORS, ADVANCES IN GENE THERAPY. THE PATIENT PANEL DISCUSSION SHARED EXPERIENCES RELATED TO PARTICIPATION IN CLINICAL TRIALS AND RESEARCH. THE FARA AMBASSADOR PROGRAM WHICH WAS LAUNCHED IN 2011 WITH >20 PARTICIPANTS HAD A YEAR OF GROWTH AND TRANSFORMATION IN 2014. THE MISSION OF THE FARA AMBASSADORS IS TO BE POSITIVE, SUPPORTIVE, PEER REPRESENTATIVES FOR THE FA COMMUNITY; ACTIVELY RAISING AWARENESS AND FUNDS FOR FARA.IN 2014, FARA FACILITATED FORMAL TRAINING FOR THE AMBASSADOR LEADERSHIP TEAM AND AMBASSADORS. THE AMBASSADOR BLOG PROJECT TEAM CONSISTS OF THREE ADMINS AND 6 MORE TEAM MEMBERS. IN 2014, THEY ACHIEVED >100 POSTS WITH ~40,000 VIEWS. THE AMBASSADORS ALSO STARTED A CARD PROGRAM IN WHICH THEY DESIGNED CARDS TO SEND TO THE VARIOUS STAKEHOLDERS AND COMMUNITY MEMBERS THROUGHOUT THE YEAR TO SAY THANK YOU OR TO OFFER ENCOURAGEMENT. THEY ARE AVERAGING ABOUT 20 CARDS PER MONTH. FINALLY, THE AMBASSADORS HAVE DONE IMPORTANT OUTREACH IN VISITING >8 ACADEMIC LABORATORIES AND BIOPHARMACEUTICAL COMPANIES TO PROVIDE THE PATIENT PERSPECTIVE OF FA, AND SHARING THEIR PERSONAL EXPERIENCES AT FA SYMPOSIA. 4629014 1 1 0 1 0 0 0 0 0 0 1 0 1 0 0 1 1 0 0 0 1 1 0 0 1 0 0 1 0 1 0 0 0 0 0 0 0 0 1 0 0 0 0 0 0 0 0 1 3 0 1 6 1 0 0 0 0 0 1 1 0 0 0 0 X 16 15 0 0 1 0 0 0 0 1 1 0 0 1 1 1 1 1 1 1 1 0 AL AK AZ AR CA CO CT FL GA IL KS KY ME MD MS MI MN MS MO NH NJ NM NY NC ND OH OK OR PA RI SC TN UT VA WA WV WI MA X X X JENNIFER FARMER 4848796160 533 W UWCHLAN AVENUE DOWNINGTOWN PA 19335 RONALD BARTEK PRESIDENT/DIRECTOR 40.00 X X X 160000 0 10492 EDWARD RAMSEY CHAIRMAN 0.00 X X 0 0 0 MARILYN E DOWNING SECRETARY 0.00 X X 0 0 0 RUTH DEWITT TREASURER 0.00 X X 0 0 0 JENNIFER GOOD DIRECTOR 0.00 X 0 0 0 PAUL AVERY DIRECTOR 0.00 X 0 0 0 PETER CRISP DIRECTOR 0.00 X 0 0 0 DR EARL GILLER SCIENTIFIC DIRECTOR 0.00 X 0 0 0 DR HOLLY HEDRICK DIRECTOR 0.00 X 0 0 0 NICHOLAS A JOHNSON DIRECTOR 0.00 X 0 0 0 GEOFFREY LEVITT DIRECTOR 0.00 X 0 0 0 STEVE KLASKO DIRECTOR 0.00 X 0 0 0 BILL ALEFANTIS DIRECTOR 0.00 X 0 0 0 THOMAS HAMILTON DIRECTOR 0.00 X 0 0 0 BERNARD RAVINA SCIENTIFIC DIRECTOR 0.00 X 0 0 0 DR SANJAY BIDICHANDANI SCIENTIFIC DIRECTOR 0.00 X 0 0 0 JENNIFER FARMER EXECUTIVE DIRECTOR 40.00 X 89494 0 7765 249494 0 18257 1 0 1 0 0 2953189 2005642 4958831 14051 14051 410253 136651 273602 273602 273602 613242 2953189 616979 -3737 -3737 5242747 0 0 283916 3258047 3258047 855855 855855 272613 205676 18250 48687 190023 150410 11468 28145 11250 11250 16250 9282 749 6219 34331 34331 21990 12989 8609 392 18292 10453 2613 5226 99979 80981 18953 45 6511 4611 1900 754 754 5906 4223 1683 CREDIT CARD & BANK FEES 5519 404 2120 2995 BOOKS, SUBSCRIPTIONS & 4969 3975 994 BUSINESS REGISTRATION F 3825 570 3255 MISCELLANEOUS 2587 2000 587 1293 1232 61 4809994 4629014 84272 96708 1357092 961837 277365 973135 109720 111200 6719 3260 5833 3510 949 2323 836233 868679 1100000 1091500 3688078 4011934 381693 290640 381693 290640 X 2972678 3393277 333707 328017 3306385 3721294 3688078 4011934 432753 -17844 0 X X 0 1 X 1 0 X 2980429 2831171 3174652 4460547 5572073 19018872 2980429 2831171 3174652 4460547 5572073 19018872 1040178 17978694 57570 12704 21555 9955 14051 115835 19134707 0.93960 0.93510 X RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED RESTRICTED 0 0 5100 4000 9100 5457873 5466973 423349 105837 0 0 302630 350101 423349 1076080 1614120 9100 9100 9100 27300 75658 87525 105837 269020 403530 3564 5100 5100 5100 18864 5833 3510 2323 2323 INVESTMENT IN EDISON PHARMACEUTICALS, INC. 1091500 C 1091500 X 5841882 -17844 616979 599135 5242747 0 5242747 5426973 616979 616979 4809994 0 4809994 PART X, LINE 2: FARA BELIEVES IT HAS APPROPRIATE SUPPORT FOR ANY TAX POSITIONS TAKEN AND, AS SUCH, DOES NOT HAVE ANY UNCERTAIN TAX POSITIONS THAT ARE MATERIAL TO THE FINANCIAL STATEMENTS. FARA'S FEDERAL RETURN OF ORGANIZATION EXEMPT FROM INCOME TAX (FORM 990) FOR TAX YEARS 2011, 2012 AND 2013 IS SUBJECT TO EXAMINATION BY THE IRS, GENERALLY FOR THREE YEARS AFTER THE TAX RETURNS WERE FILED. PART XI, LINE 2D - OTHER ADJUSTMENTS: SPECIAL EVENTS EXPENSE PART XII, LINE 2D - OTHER ADJUSTMENTS: SPECIAL EVENTS EXPENSE 1 EUROPE GRANTS TO RECIPIENTS LOCATED IN REGION RESEARCH AND GRANT PROGRAM 666338 EAST ASIA AND THE PACIFIC GRANTS TO RECIPIENTS LOCATED IN REGION RESEARCH AND GRANT PROGRAM 189517 0 0 0 0 0 0 855855 0 855855 EUROPE MEDICAL RESEARCH 101000 WIRE EUROPE MEDICAL RESEARCH 100000 WIRE EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 75000 WIRE EUROPE MEDICAL RESEARCH 295299 WIRE EUROPE MEDICAL RESEARCH 21195 WIRE EUROPE MEDICAL RESEARCH 23844 WIRE EUROPE MEDICAL RESEARCH 125000 WIRE EAST ASIA AND THE PACIFIC MEDICAL RESEARCH 114517 WIRE 8 0 0 0 0 0 0 FARA ENERGY BALL 1674795 1182594 492201 236892 RIDE ATAXIA PHILADELPHIA 264673 252798 11875 47561 65 1626963 1517797 109166 332526 3566431 2953189 613242 616979 616979 -3737 FELICIA DEROSA 533 W UWCHLAN AVENUE DOWNINGTOWN PA 19335 1 UNIVERSITY OF ROCHESTER 515 HYLAN BUILDING RC BOX 270140 ROCHESTER NY 14627 160743209 501(C)(3) 121783 MEDICAL RESEARCH UNIVERSITY OF SOUTH FLORIDA FOUNDATION 4202 EAST FOWLER AVENUE TAMPA FL 33620 590879015 501(C)(3) 150000 MEDICAL RESEARCH CHILDREN'S HOSPITAL OF PHILADELPHIA 34TH ST CIVIC CENTER BLVD PHILADELPHIA PA 19104 231352166 501(C)(3) 138621 MEDICAL RESEARCH OHIO STATE UNIVERSITY 333 W 10TH AVE COLUMBUS OH 43210 316025986 501(C)(3) 5307 MEDICAL RESEARCH SCRIPPS RESEARCH INSTITUTE 10550 NORTH TORREY PINES ROAD OSP-TPC7 LA JOLLA CA 92037 330435954 501(C)(3) 205510 MEDICAL RESEARCH THE JACKSON LABORATORY 600 MAIN STREET BAR HARBOR ME 04609 010211513 501(C)(3) 21360 MEDICAL RESEARCH THE REGENTS OF THE UNIVERSITY OF CALIFORNIA LOS ANGELES 10920 WILSHIRE BLVD 5TH FLOOR LOS ANGELES CA 90024 956006143 501(C)(3) 245500 MEDICAL RESEARCH UNIVERSITY OF IOWA DEPT OF PEDIATRICS 200 HAWKINS DRIVE IOWA CITY IA 52242 426004813 501(C)(3) 7700 MEDICAL RESEARCH THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA 3451 WALNUT STREET RM P 221 FRANKLIN BLDG PHILADELPHIA PA 19104 231352685 501(C)(3) 1264178 MEDICAL RESEARCH INDIANA UNIVERSITY 1044 WEST WALNUT INDIANAPOLIS IN 46202 356001673 501(C)(3) 100000 MEDICAL RESEARCH UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER 5323 HARRY HINES BLVD BL 2100 DALLAS TX 753909020 756002868 501(C)(3) 78071 MEDICAL RESEARCH UNIVERSITY OF FLORIDA - DEPT OF NEUROLOGY L3-100 MCKNIGHT BRAIN INSTITUTE NEWELL DRIVE GAINESVILLE FL 32611 596002052 501(C)(3) 15800 MEDICAL RESEARCH ALBANY RESEARCH INSTITUTE INC 113 HOLLAND AVE ALBANY NY 12208 141716021 501(C)(3) 73492 MEDICAL RESEARCH DUKE UNIVERSITY 134 CHAPEL DRIVE DURHAM NC 27708 560532129 501(C)(3) 100000 MEDICAL RESEARCH FEDERATION OF AMERICAN SOCIETIES FOR EXPERIMENTAL BIOLOGY 9650 ROCKVILLE PIKE BETHESDA MD 20814 520700497 501(C)(3) 5000 MEDICAL RESEARCH LSU HEALTH SCIENCE CENTER 433 BOLIVAR ST NEW ORLEANS LA 70112 726087770 501(C)(3) 147049 MEDICAL RESEARCH REGENTS OF THE UNIVERSITY OF MINNESOTA 200 SE OAK ST 600 MINNEAPOLIS MN 55455 416007513 501(C)(3) 150000 MEDICAL RESEARCH WEILL MEDICAL COLLEGE OF CORNELL UNIVERSITY 445 E 69TH ST NEW YORK NY 10021 131623978 501(C)(3) 99276 MEDICAL RESEARCH AMERICAN HEART ASSOCIATION 1617 JOHN F KENNEDY BLVD 700 PHILADELPHIA PA 19103 135613797 501(C)(3) 13000 MEDICAL RESEARCH BAYLOR COLLEGE OF MEDICINE 1 BAYLOR PLAZA HOUSTON TX 77030 741613878 501(C)(3) 125000 MEDICAL RESEARCH NATIONAL ATAXIA FOUNDATION 2600 FERNBROOK LANE NORTH PLYMOUTH MN 55447 410832903 501(C)(3) 5000 MEDICAL RESEARCH UNIVERSITY OF VIRGINIA UVA HEALTH SYSTEM CHARLOTTESVILLE VA 22908 540485595 501(C)(3) 125000 MEDICAL RESEARCH THE REGENTS OF UNIVERSITY OF CA DAVIS PO BOX 989062 WEST SACRAMENTO CA 95798 946036494 501(C)(3) 37500 MEDICAL RESEARCH PART I, LINE 2: GRANTS ARE REVIEWED BY INDEPENDENT SCIENTIFIC ADVISORS AND APPROVED BY BOARD. X 0 0 0 0 0 0 0 0 0 RONALD BARTEK PRESIDENT/DIRECTOR 160000 0 0 0 0 0 0 0 10492 0 170492 0 0 0 X 5 164419 MARKET PRICE 0 0 0 FORM 990, PART III, LINE 4A CONTINUED FIRST EVER, CONCENSUS CLINICAL MANAGEMENT GUIDELINES FOR FRIEDREICH ATAXIA PUBLISHED. FARA'S GOAL IS TO IMPROVE TREATMENT AND MEDICAL OUTCOMES FOR INDIVIDUALS LIVING WITH FA. ONE WAY TO DO THIS IS BY HAVING EDUCATIONAL MATERIALS AVAILABLE TO PHYSICIANS AND PEOPLE LIVING WITH FA. DRS. LOUISE CORBEN AND MARTIN DELATYCKI LED A COMMITTED TEAM OF INTERNATIONAL PHYSICIANS AND RESEARCHERS, MOST FROM FARA'S COLLABORATIVE CLINICAL RESEARCH NETWORK, WHO HAVE CRITICALLY EVALUATED MEDICAL LITERATURE, OUTCOMES RESEARCH AND CLINICAL PRACTICE FOR TREATING SYMPTOMS OF FA, AND HAVE COME TOGETHER TO PREPARE THE FIRST COMPREHENSIVE CONSENSUS CLINICAL MANAGEMENT GUIDELINES. THESE GUIDELINES WERE PUBLISHED IN ORPHANET JOURNAL OF RARE DISEASES, NOV 2014, AND A FULL VERSION FOR PHYSICIANS AND INDIVIDUALS WITH FA IS AVAILABLE ON THE FARA WEBSITE, HTTP://WWW.CUREFA.ORG/_PDF/CLINICALMANAGEMENTGUIDELINESFORFA.PDF THESE GUIDELINES ARE A GIANT STEP FORWARD IN DOCUMENTING DIAGNOSIS, TREATMENT AND MANAGEMENT OF FA. IN ADDITION TO RESEARCH GRANTS FARA FUNDS THE ONGOING DEVELOPMENT OF VITAL CLINICAL RESEARCH INFRASTRUCTURE. CLINICAL RESEARCH INFRASTRUCTURE REFERS TO THE RESOURCES NEEDED TO FACILITATE ANY TYPE OF RESEARCH, INCLUDING CLINICAL TRIALS THAT INVOLVE PATIENTS. THESE RESOURCES CAN INCLUDE THINGS LIKE: PATIENT REGISTRY : THE ONLY INTERNATIONAL FRIEDREICH ATAXIA PATIENT REGISTRY WITH MORE THAN 2200 INDIVIDUALS ENROLLED. THIS REGISTRY CAPTURES DEMOGRAPHIC AND CLINICAL INFORMATION ON INDIVIDUALS WITH FA AND IS USED TO RECRUIT INDIVIDUALS FOR CLINICAL TRIALS WORLD WIDE. FARA PATIENT REGISTRY : WWW.CUREFA.ORG/REGISTRY COLLABORATIVE CLINICAL RESEARCH NETWORK IN FA (CCRN IN FA): AN INTERNATIONAL NETWORK OF 10 CLINICAL RESEARCH CENTERS THAT WORK TOGETHER TO ADVANCE TREATMENTS AND CLINICAL CARE FOR INDIVIDUALS WITH FRIEDREICH'S ATAXIA. HAVING SUCH A NETWORK MEANS THAT THERE ARE TRAINED PHYSICIANS AND RESEARCH COORDINATORS READY TO DO CLINICAL RESEARCH STUDIES AND TRIALS. ALSO, THIS NETWORK IS BACKED BY A DATA COORDINATION CENTER THAT FACILITATES ALL ASPECTS OF DATA COLLECTION, DATABASE MANAGEMENT, AND STATISTICAL ANALYSIS OF STUDY DATA. TO LEARN MORE, VISIT WWW.CUREFA.ORG/NETWORK.HTML NATURAL HISTORY STUDY: LONGITUDINAL DATA (USUALLY ABOUT 10 YEARS) ON INDIVIDUALS WITH A DISEASE THAT DESCRIBES AND QUANTIFIES THE PROGRESSION OF THE DISEASE ALONG WITH THE SYMPTOMS AND MANIFESTATIONS OF THE DISEASE. NATURAL HISTORY CAN SOMETIMES SERVE AS THE BASIS FROM WHICH MEASUREMENTS CAN BE MADE TO DETERMINE EFFECTS OF NEW TREATMENTS, DRUGS OR INTERVENTIONS. CLINICAL OUTCOME MEASURES : FUNCTIONAL PERFORMANCE TESTS (E.G., TIMED PEGBOARD OR WALK TESTS, VISION, HEARING OR SPEECH TESTS) THAT QUANTIFY HOW MUCH CHANGE TAKES PLACE IN A SPECIFIC AMOUNT OF TIME AND ARE USED IN CLINICAL TRIALS TO MEASURE WHETHER A DRUG IS ALTERING THE COURSE OF THE DISEASE. BIOMARKERS: ANYTHING THAT CAN BE USED AS AN INDICATOR OF A PARTICULAR DISEASE STATE - USUALLY PROTEINS, ENZYMES, GENETIC VARIANTS, IMAGING (MRI, CT OR PET SCANS). BIOMARKERS CAN BE USED TO ASSESS RISK OF DISEASE, DIAGNOSIS, OR OUTCOMES. USE OF BIOMARKERS IN DRUG DEVELOPMENT IS OF GREAT INTEREST BECAUSE BIOMARKERS CAN PROVIDE EVIDENCE OF BIOLOGICAL ACTIVITY, POTENTIALLY DEMONSTRATING THERAPEUTIC BENEFIT MORE QUICKLY THAN TRADITIONAL OUTCOME MEASURES. BIOREPOSITORY: A REPOSITORY OR BANK OF STORED BIOLOGICAL MATERIALS SUCH AS BLOOD SAMPLES, DNA, ORGANS, AND TISSUES (SUCH AS SKIN, MUSCLE, HEART) THAT CAN BE USED FOR RESEARCH. THROUGH THE CCRN IN FA WE HAVE COLLECTED TEN YEARS OF NATURAL HISTORY DATA (ONGOING) IN MORE THAN 800 INDIVIDUALS WITH FA, VALIDATED CLINICAL OUTCOME MEASURES AND THE FARS SCALE, STUDIED SPEECH, VISION AND HEARING, LAUNCHED BIOMARKER STUDIES, ESTABLISHED DNA AND RNA REPOSITORIES, AND PROVIDED MANY BLOOD SAMPLES TO RESEARCHERS AROUND THE WORLD. THE CCRN IN FA INVESTIGATORS HAVE BEEN INVOLVED IN MULTIPLE CLINICAL TRIALS INCLUDING A FEW THAT WERE DESIGNED AND CONDUCTED SOLELY THROUGH NETWORK SITES. FARA CONTRIBUTED MORE THAN $550,000 IN 2014 TO CLINICAL RESEARCH INFRASTRUCTURE IN DIRECT FUNDING TO THE CCRN IN FA CENTERS AND RELATED CLINICAL RESEARCH ACTIVITIES. TITLES AND SUMMARIES OF MOST OF THE PROJECTS PRESENTLY FUNDED BY FARA ARE AVAILABLE AT: WWW.CUREFA.ORG/RPMP/PUBLIC/PGGRANTLIST.ASPX AND COMPLETE LISTINGS OF GRANTS AWARDED BY YEAR CAN BE ACCESSED AT: WWW.CUREFA.ORG/GRANTS-AWARDED.HTML FORM 990, PART VI, SECTION A, LINE 4 THE BYLAWS WERE AMENDED AT THE END OF 2014 TO MODIFY THE NUMBER OF DIRECTORS FROM 18 TO 20. A VICE-CHAIR WAS ADDED AND INCLUDED AS AN EX OFFICIO MEMBER OF THE EXECUTIVE COMMITTEE. FORM 990, PART VI, SECTION B, LINE 11 COPIES OF 990 ARE DISTRIBUTED TO BOARD MEMBERS FOR REVIEW AND APPROVAL PRIOR TO FILING. FORM 990, PART VI, SECTION B, LINE 12C ALL NEW AND EXISTING BOARD MEMBERS REQUIRED TO ANNUALLY COMPLETE A CONFLICT OF INTEREST STATEMENT FORM 990, PART VI, SECTION B, LINE 15 EVALUATION AND COMPENSATION COMMITTEE PERFORMS EMPLOYEE EVALUATIONS AND DETERMINES SALARY INCREASES ON A YEARLY BASIS FOR ALL EMPLOYEES. COMMITTEE EXAMINES BENCHMARK DATA IN DETERMINING SALARIES FOR PRESIDENT AND EXECUTIVE DIRECTOR. FORM 990, PART VI, SECTION C, LINE 18 FORM 990 IS AVAILABLE ON THE ORGANIZATION'S WEBSITE AS WELL AS GUIDESTAR.ORG AND CHARITYNAVIGATOR.ORG FORM 990, PART VI, SECTION C, LINE 19 FINANCIAL STATEMENTS ARE AVAILABLE ON THE ORGANIZATION'S WEBSITE. THE GOVERNING DOCUMENTS, CONFLICT OF INTEREST POLICY AND FORM 1023 ARE AVAILABLE UPON REQUEST. FORM 990, PART VI LINE 17 THE ORGANIZATION IS ALSO REGISTERED TO SOLICIT CONTRIBUTIONS IN THE STATES LISTED. FORM 990, PART XII, LINE 2C FARA'S AUDIT COMMITTEE CONSISTS OF THE EXECUTIVE COMMITTEE, EXECUTIVE DIRECTOR AND ONE AT-LARGE BOARD MEMBER. EACH YEAR THE AUDIT COMMITTEE SEEKS THE SERVICES OF AN OUTSIDE ACCOUNTING FIRM AND CONTRACTS FOR A FULL AUDIT, PREPARATION OF FINANCIAL STATEMENTS AND FILING OF THE 990. THE AUDIT COMMITTEE IS RESPONSIBLE FOR REVIEWING RECOMMENDATIONS FROM THE AUDIT AND PROPOSING NEW POLICIES AND PROCEDURES AS NECESSARY. THE AUDIT COMMITTEE ALSO PARTICIPATES IN DETAILED REVIEW OF FINANCIAL STATEMENTS AND 990 PRIOR TO SHARING WITH THE FULL BOARD FOR A VOTE. FARA'S BOARD OF DIRECTORS RECEIVES THE FINANCIAL STATEMENTS AND 990 FOR REVIEW AND VOTES TO APPROVE PRIOR TO PUBLIC FILING.