2019-02-27T20:09:24-06:00 2018-06-30 860683059 BEACHFLEISCHMAN PC 1985 E RIVER ROAD SUITE 201 TUCSON AZ 85718 990 2017-07-01 201991334 CRITICAL PATH INSTITUTE CRIT 5205473440 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 STEVEN BROADBENT MBA COO 5205473440 2019-02-14 1 KAREN K MCCLOSKEY CPA P00099644 5203214600 2019-02-14 64.128.200.66 2019-02-14 15:48:27 US P 100786DC4CA6AC734CEB2D953EA696EF9CD62243 8D176FDB9A9846D98182556421F389029B96F31F 2017 2019-02-21 02:37:17Z MARTHA BRUMFIELD PHD 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 15267826 0 X WWW.C-PATH.ORG X 2004 AZ CREATE AND MANAGE INTERNATIONAL COLLABORATIONS AND DEVELOP NOVEL TOOLS, ENABLING THE FASTER DEVELOPMENT OF SAFER MEDICAL PRODUCTS. 15 14 80 13 0 0 10085049 10019550 5531847 5164609 50971 83667 2500 0 15670367 15267826 1853946 1359100 0 0 7188245 8046940 0 0 0 6113896 5105672 15156087 14511712 514280 756114 16508211 14421996 10541940 7699611 5966271 6722385 X THE CRITICAL PATH INSTITUTE IS A CATALYST IN THE DEVELOPMENT OF NEW APPROACHES THAT ADVANCE MEDICAL INNOVATION AND REGULATORY SCIENCE. THIS IS ACHIEVED BY LEADING TEAMS THAT SHARE DATA, KNOWLEDGE AND EXPERTISE RESULTING IN SOUND, CONSENSUS BASED SCIENCE. 0 0 10965542 1359100 5164609 SEE SCHEDULE O 10965542 1 1 0 1 0 0 0 0 0 0 1 0 0 0 0 1 1 0 0 0 1 1 0 0 0 0 0 1 0 1 0 0 0 0 0 0 0 0 0 0 0 0 0 1 1 0 0 0 1 14 0 1 80 1 0 0 0 0 0 0 0 0 0 0 X 15 14 1 0 0 0 0 0 0 1 1 0 0 1 1 1 1 1 1 1 1 0 AZ X ANNA M MIDKIFF CPA 5206478382 1730 EAST RIVER ROAD NO 200 TUCSON AZ 857185898 D CRAIG BRATER MD BOARD MEMBER 1.00 X 0 0 0 THE HONORABLE JAMES C GREENWOOD BOARD MEMBER 1.00 X 0 0 0 KAY HOLCOMBE BOARD MEMBER 1.00 X 0 0 0 MICHAEL KASSER MBA PHD BOARD MEMBER 1.00 X 0 0 0 SHAUN A KIRKPATRICK MA BOARD MEMBER 1.00 X 0 0 0 RICHARD T MYERS JR BOARD MEMBER 1.00 X 0 0 0 CINDY PARSEGHIAN MBA BOARD MEMBER 1.00 X 0 0 0 ALASTAIR JJ WOOD MB CHB BOARD MEMBER 1.00 X 0 0 0 PETER BARTON HUTT LLB LLM BOARD MEMBER 1.00 X 0 0 0 ALAN G LEVIN MS CPA BOARD MEMBER 1.00 X 0 0 0 PAULA OLSIEWSKI PHD BOARD MEMBER 1.00 X 0 0 0 JEFFREY E JACOB SM DEPUTY CHAIRPERSON 1.00 X 0 0 0 M WAINWRIGHT FISHBURN JR VICE CHAIRPERSON 1.00 X X 0 0 0 TIMOTHY R FRANSON MD CHAIRPERSON OF THE BOARD 1.00 X X 0 0 0 MARTHA BRUMFIELD PHD CEO/PRESIDENT 40.00 X X 296591 0 29794 STEVEN BROADBENT MBA COO/TREASURER 40.00 X 194127 0 26519 LEWIS BARBIERI SECRETARY 40.00 X 114661 0 18817 LYNN D HUDSON PHD EXECUTIVE DIRECTOR 40.00 X 200763 0 18056 JOHN-MICHAEL SAUER PHD EXECUTIVE DIRECTOR 40.00 X 191505 0 29988 STEPHEN J COONS PHD EXECUTIVE DIRECTOR 40.00 X 185620 0 21631 STEVE ARNERIC PHD EXECUTIVE DIRECTOR 40.00 X 171353 0 24267 DEBRA HANNA PHD EXECUTIVE DIRECTOR 40.00 X 176101 0 24700 1530721 0 193772 24 0 1 0 RESEARCH TRIANGLE INSTITUTE 200 PARK OFFICES DRIVE RESEARCH TRIANGLE PARK NC 27709 RESEARCH 365350 EPHIBIAN 3180 N SWAN ROAD TUCSON AZ 85712 IT SERVICES 159419 LOTOS NILE PO BOX 90245 NASHVILLE TN 37209 PUBLIC RELATIONS 144000 CLINICAL OUTCOME SOLUTIONS UNIT 68 BASEPOINT SHEARWAY ROAD FOLKESTONE UK CT19 4RH CLINICAL RESEARCH 142370 COVANCE LABORATORIES INC PO BOX 2464 BURLINGTON NC 27216 RESEARCH 123900 14 3480022 6539528 10019550 RESEARCH SUPPORT FEES 541700 5147122 5147122 OTHER REVENUE 900099 17487 17487 5164609 83667 83667 15267826 5164609 0 83667 X 1231975 1231975 127125 127125 697022 150841 546181 5981035 4213271 1767764 422435 281320 141115 493117 330165 162952 453331 304185 149146 17962 13533 4429 123073 90511 32562 60284 57964 2320 2436169 2359796 76373 205483 138274 67209 355357 314960 40397 366686 303966 62720 523915 96591 427324 460119 438449 21670 388900 379065 9835 32303 21675 10628 55137 36997 18140 DUES AND SUBSCRIPTIONS 38894 35154 3740 BAD DEBT 35000 35000 MISCELLANEOUS 6390 4725 1665 14511712 10965542 3546170 0 1969152 3026764 5994076 4831456 1212619 166823 400363 596248 159773 92347 662965 579794 73630 83171 6674273 5615214 24325 9973 16508211 14421996 753078 600978 9788862 7098633 10541940 7699611 X 5966271 6722385 5966271 6722385 16508211 14421996 756114 0 X 0 1 X 1 1 1 X 5370782 10269811 10197464 10085049 10019550 45942656 5370782 10269811 10197464 10085049 10019550 45942656 21404373 24538283 70230 82059 80580 48471 83667 365007 46307663 22488531 0.52990 0.59930 X 60816 60816 14450896 14511712 875586 218897 0 0 809246 883297 909814 875586 3477943 5216915 67262 11731 61348 60816 201157 202312 220824 227454 218897 869487 1304231 PART II-A, LINE 1B C-PATH SPENT A MINOR AMOUNT OF FUNDS ON LOBBYING IN FYE 06/30/2018. GENERALLY, C-PATH DOES NOT LOBBY FOR OR AGAINST ANY SPECIFIC LEGISLATION. C-PATH EXPENDS SOME TIME AND RESOURCES TO EDUCATE MEMBERS OF CONGRESS IN THE AREA OF DRUG DEVELOPMENT. 42014 39338 2676 179139 179139 0 441812 361317 80495 83171 X 15267826 0 15267826 0 15267826 14511712 0 14511712 0 14511712 PART X, LINE 2: FINANCIAL STATEMENT FIN 48 (ASC 740) FOOTNOTE: C-PATH IS EXEMPT FROM INCOME TAXES UNDER BOTH FEDERAL (INTERNAL REVENUE CODE SECTION 501(C)(3)) AND ARIZONA INCOME TAX LAWS, AND IS CLASSIFIED AS OTHER THAN A PRIVATE FOUNDATION UNDER INTERNAL REVENUE CODE SECTION 509(A)(2). INCOME FROM CERTAIN ACTIVITIES NOT DIRECTLY RELATED TO C-PATH'S TAX-EXEMPT PURPOSE, HOWEVER, MAY BE SUBJECT TO TAXATION AS UNRELATED BUSINESS TAXABLE INCOME (UBTI). GAAP REQUIRES MANAGEMENT TO PERFORM AN EVALUATION OF ALL TAX POSITIONS TAKEN OR EXPECTED TO BE TAKEN IN THE COURSE OF PREPARING C-PATH'S TAX RETURNS TO DETERMINE WHETHER THE TAX POSITIONS MEET A "MORE LIKELY THAN NOT" STANDARD OF BEING SUSTAINED UNDER EXAMINATION BY THE APPLICABLE TAXING AUTHORITIES. THIS EVALUATION IS REQUIRED TO BE PERFORMED FOR ALL OPEN TAX YEARS, AS DEFINED BY THE VARIOUS STATUTES OF LIMITATIONS, FOR FEDERAL AND STATE PURPOSES. C-PATH IS REQUIRED TO FILE INFORMATIONAL RETURNS FOR FEDERAL AND STATE PURPOSES AND, IF IT HAS UBTI, FEDERAL AND STATE INCOME TAX RETURNS. MANAGEMENT HAS PERFORMED ITS EVALUATION OF TAX POSITIONS TAKEN ON ALL OPEN TAX YEARS AND HAS DETERMINED THAT THERE WERE NO POSITIONS TAKEN THAT DO NOT MEET THE "MORE LIKELY THAN NOT" STANDARD. SHOULD C-PATH EVER BE SUBJECT TO PENALTIES AND INTEREST ASSESSED BY VARIOUS TAXING AUTHORITIES, THEY WOULD BE CLASSIFIED AS MANAGEMENT AND GENERAL EXPENSES. 1 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM 0 0 GRANTS TO RECIPIENTS LOCATED IN REGION CLINICAL RESEARCH 127125 0 0 0 0 0 0 127125 0 127125 EUROPE (INCLUDING ICELAND & GREENLAND) - ALBANIA, ANDORRA, AUSTRIA, BELGIUM CLINICAL RESEARCH 127125 WIRE TRANSFER 1 0 0 1 0 0 0 PART I, LINE 2: CRITICAL PATH INSTITUTE ENTERS INTO SUBAWARD AGREEMENTS WITH SUBRECIPIENTS AND REVIEWS PROGRESS DELIVERABLES AND COSTS. 1 BAYLOR RESEARCH INSTITUTE 1950 N STEMMONS FREEWAY SUITE 5010 LOCKBOX 846275 DALLAS TX 75207 751921898 501(C)(3) 400459 DEVELOP A HOLLOW FIBER MODEL FOR TUBERCULOSIS RESEARCH REGENTS OF THE UNIVERSITY OF CALIFORNIA 1855 FOLSOM STREET BOX 0897 SAN FRANCISCO CA 94143 946036493 STATE OF CALIFORNIA 173425 DEVELOP A DISEASE PROGRESSION MODEL FOR TUBERCULOSIS UNIVERSITY OF FLORIDA PO BOX 113001 GAINSVILLE FL 32611 596002052 STATE OF FLORIDA 55100 DEVELOP A POPULATION PHARMACOKINETIC/PHARMACODYNAMIC (PK/PD) MODEL FOR TUBERCULOSIS CLINICAL DATA INTERCHANGE STANDARDS CONSORTIUM PO BOX 2068 ROUND ROCK TX 78680 043503931 501(C)(3) 263981 DEVELOP CLINICAL DATA STANDARDS THE TRANSLATIONAL GENOMICS RESEARCH INSTITUTE 445 N FIFTH STREET SUITE 600 PHOENIX AZ 85004 753065445 501(C)(3) 293585 DEVELOP THE RESEQTB INTEGRATED PIPELINE FOUNDATION FOR THE NATIONAL INSTITUTES OF HEALTH INC 9650 ROCKVILLE PIKE SUITE 3411 BETHESDA MD 20814 521986675 501(C)(3) 45425 CLINICAL EVALUATION AND QUALIFICATION OF KIDNEY SAFETY BIOMARKERS STUDY 6 PART I, LINE 2: CRITICAL PATH INSTITUTE ENTERS INTO SUBAWARD AGREEMENTS WITH SUBRECIPIENTS AND REVIEWS PROGRESS DELIVERABLES AND COSTS. X X X X X 0 0 0 0 0 0 0 0 0 MARTHA BRUMFIELD PHD CEO/PRESIDENT 296591 0 0 0 0 0 22600 0 7194 0 326385 0 0 0 STEVEN BROADBENT MBA COO/TREASURER 194127 0 0 0 0 0 15509 0 11010 0 220646 0 0 0 LYNN D HUDSON PHD EXECUTIVE DIRECTOR 200763 0 0 0 0 0 15716 0 2340 0 218819 0 0 0 JOHN-MICHAEL SAUER PHD EXECUTIVE DIRECTOR 191505 0 0 0 0 0 15681 0 14307 0 221493 0 0 0 STEPHEN J COONS PHD EXECUTIVE DIRECTOR 185620 0 0 0 0 0 14729 0 6902 0 207251 0 0 0 STEVE ARNERIC PHD EXECUTIVE DIRECTOR 171353 0 0 0 0 0 13371 0 10896 0 195620 0 0 0 DEBRA HANNA PHD EXECUTIVE DIRECTOR 176101 0 0 0 0 0 14520 0 10180 0 200801 0 0 0 FORM 990, PART III, LINE 4A, PROGRAM SERVICE ACCOMPLISHMENTS THE CRITICAL PATH INSTITUTE (C-PATH) IS AN INDEPENDENT, NON-PROFIT ORGANIZATION UNIQUELY DEDICATED TO ADVANCING MULTIPLE ASPECTS OF THE FDA'S CRITICAL PATH INITIATIVE BY CREATING COLLABORATIONS AMONG REGULATORS (SCIENTISTS FROM THE FDA AND EMA), THE REGULATED MEDICAL PRODUCT INDUSTRY, ACADEMIA, PATIENT GROUPS, AND OTHER ORGANIZATIONS THAT ADVANCE MEDICAL INNOVATION THROUGH THE DEVELOPMENT OF TOOLS AND METHODS BASED UPON SOUND, CONSENSUS-BASED SCIENCE. THE ACCOMPLISHMENTS FOR THIS FISCAL YEAR REPORT INCLUDE: THE COALITION AGAINST MAJOR DISEASES WAS REBRANDED AS THE CRITICAL PATH FOR ALZHEIMER'S DISEASE. IT DEVELOPS TOOLS AND NOVEL METHODOLOGIES TO ACCELERATE PROGRESS IN THE DEVELOPMENT OF PREVENTION AND TREATMENT STRATEGIES FOR ALZHEIMER'S DISEASE. THIS YEAR'S ACCOMPLISHMENTS INCLUDE: (1) RECEIVING A LETTER OF SUPPORT FROM THE EMA ENCOURAGING INDUSTRY SPONSORS TO SHARE WITH CPAD THE PATIENT-LEVEL DATA FROM COMPLETED PHASE II AND III CLINICAL TRIALS, INCLUDING ACTIVE AND CONTROL ARMS, ALLOWING CPAD TO COMPLETE DEVELOPMENT AND VALIDATION OF A PROPOSED QUANTITATIVE MODEL FOR IMPLEMENTATION BY SPONSORS ACTIVELY DESIGNING CLINICAL TRIALS IN AMNESTIC MILD COGNITIVE IMPAIRMENT (AMCI). (2) GENERATED GRAPHICAL USER INTERFACE-BASED UPDATES TO THE AD MILD-TO-MODERATE CLINICAL TRIAL SIMULATION TOOL AND MILD COGNITIVE IMPAIRMENT (MCI) CLINICAL TRIAL SIMULATION TOOL - THE HIPPOCAMPAL NEUROIMAGING-INFORMED AMNESTIC MCI CLINICAL TRIAL SIMULATOR. (3) INCREASED USE OF THE DATABASE AND MODELS. THE CRITICAL PATH FOR PARKINSON'S CONSORTIUM'S GOALS ARE TO DEVELOP QUANTITATIVE MODEL-BASED TOOLS TO ACCELERATE THE TREATMENT OF PARKINSON'S DISEASE, ESPECIALLY FOR THOSE INDIVIDUALS EXPERIENCING THE FIRST CLINICAL SIGNS OF MOTOR SYMPTOM ONSET. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE:(1) SUBMISSION TO EMA AND FDA OF THE FIRST MODELING TOOL FOR PARKINSON'S- "MODEL-BASED CLINICAL TRIAL ENRICHMENT TOOL FOR PARKINSON DISEASE CLINICAL TRIALS". (2) RECEIVED FDA'S FORMAL RESPONSE TO CPP'S LETTER OF INTENT OUTLINING RECOMMENDATIONS FOR WHAT SHOULD BE SUBMITTED IN THE MODELING BRIEFING DOCUMENT. (3) EMA QUALIFICATION ADVICE LETTER RECEIVED FOR THE MODEL-BASED CLINICAL TRIAL ENRICHMENT TOOL FOR PARKINSON'S DISEASE CLINICAL TRIALS. THE MISSION OF THE CRITICAL PATH TO TB DRUG REGIMENS INITIATIVE IS TO ACCELERATE THE DEVELOPMENT OF NOVEL TB DRUG REGIMENS THAT ARE SAFER, SHORTER, AND MORE EFFICACIOUS THAN THE CURRENT STANDARD OF CARE. THEIR MISSION EXPANDED TO ACCELERATE THE DEVELOPMENT OF A CLINICALLY USEFUL IN VITRO RAPID DRUG SUSCEPTIBILITY ASSAY TO SUPPORT TB REGIMEN DEVELOPMENT AND DEPLOYMENT. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) ELEVEN NEW TB CLINICAL DATASETS WERE ACQUIRED, CURATED, AND INTEGRATED INTO THE TB PLATFORM FOR AGGREGATION OF CLINICAL TB STUDIES (TB-PACTS) PLATFORM AND MADE ACCESSIBLE TO QUALIFIED RESEARCHERS. TO DATE, OVER 55 APPLICATIONS HAVE BEEN SUBMITTED AND 42 HAVE BEEN APPROVED TO SUPPORT TB RESEARCH. (2) COMPLETED THE STAGE 2 ANALYSIS OF TB RE-ANALYSIS OF FLUOROQUINOLONE EXECUTED CLINICAL TRIALS (TB-REFLECT), WHICH FOCUSED ON ISONIAZID RESISTANT SUB-COHORT AND EXAMINATION OF SAFETY FINDINGS FOR STANDARD OF CARE REGIMENS WHICH CONTAIN A FLUOROQUINOLONE. (3) COMPLETED OR SIGNIFICANTLY ADVANCED THE FOLLOWING MODELING AND SIMULATION PROJECTS: POPULATION PK/PD, LIQUID CULTURE, QUANTITATIVE SYSTEMS PHARMACOLOGY, AND CARDIAC RISK ASSESSMENT ALGORITHM. ADDITIONALLY, HFS-TB EXPERIMENTS CHARACTERIZING THE PK/PD ATTRIBUTES OF THREE DRUGS AND REGIMENS FOR TB HAVE BEEN COMPLETED, WITH FOUR EXPERIMENTS ONGOING. OTHER MODEL-BASED ANALYSES ARE ONGOING TO IDENTIFY PREFERRED METHODS FOR THE TRANSLATION OF DATA FROM IN VITRO AND IN VIVO (ANIMAL) MODELS TO PREDICT CLINICAL OUTCOMES FOR ANTI-TB DRUGS AND REGIMENS. (4) EXPANDED ANALYSES TO SUPPORT PBPK QUALIFICATION EFFORTS BASED ON FEEDBACK FROM EMA'S SCIENTIFIC ADVICE WORKING PARTY (SAWP). FURTHER ANALYSES DESIGNED TO VERIFY PBPK GRANULOMA MODEL PERFORMANCE ARE IN PROGRESS. THE DATA COLLABORATION CENTER IS A CENTER WITHIN C-PATH TO ENABLE MULTIPLE ORGANIZATIONS TO WORK TOGETHER IN A NEUTRAL SETTING AND SHARE CLINICAL DATA IN ORDER TO OPTIMIZE ITS VALUE IN CREATING NEW INSIGHTS AND TOOLS THAT ACCELERATE DRUG DEVELOPMENT IN AREAS WITH UNMET MEDICAL NEEDS. THE DCC SUPPORTS DATA SHARING PROJECTS ALIGNED WITH SPECIFIC C-PATH CONSORTIA AS WELL AS DATA SHARING INITIATIVES THAT ARE INDEPENDENT OF C-PATH CONSORTIA. ITS ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) LAUNCHED THE DUCHENNE'S REGULATORY SCIENCE CONSORTIUM (D-RSC) DATABASE WHICH WILL BE USED IN THE DEVELOPMENT OF A DMD DISEASE PROGRESSION MODEL AND CLINICAL TRIAL SIMULATION PLATFORM. (2) LAUNCHED THE CATALYSIS FOUNDATION ONLINE DATA REPOSITORY TO PROVIDE A PLATFORM TO MAKE DATASETS FROM A TREATMENT RESPONSE BIOMARKER STUDY OF TB POSITIVE, HIV NEGATIVE PATIENTS AVAILABLE TO QUALIFIED TB RESEARCHERS. (3) LAUNCHED THE FRIEDREICH'S ATAXIA INTEGRATED CLINICAL DATABASE FOR USE BY QUALIFIED RESEARCHERS. (4) RELEASED TB-PACTS VERSION 2.0 CONTAINING DATA FROM 14 NEW TB STUDIES FOR A TOTAL OF 17. THE DUCHENNE REGULATORY SCIENCE CONSORTIUM'S GOAL IS TO SUPPORT COLLABORATIVE RESEARCH AND REGULATORY QUALIFICATION OF NEW DRUG DEVELOPMENT TOOLS FOR DUCHENNE MUSCULAR DYSTROPHY, TO ENABLE THE EARLIEST POSSIBLE PATIENT ACCESS TO NEW TREATMENTS. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) ACQUIRED TWELVE DATASETS, AND DATA SHARING AGREEMENTS FOR ADDITIONAL DATASETS ARE UNDERWAY. PRESENTLY, ~1,100 INDIVIDUAL PATIENT RECORDS ARE INCLUDED IN THE D-RSC DATABASE, EXCLUDING DATA FROM A PATIENT REPORTED REGISTRY THAT WILL NOT BE USED FOR ANALYSIS. APPROXIMATELY 4000-5000 DATA-POINTS ARE IN THE DATABASE FOR EACH MEASURE OF INTEREST. PARTS OF THE DATABASE ARE OPEN FOR USE BY CONSORTIUM MEMBERS AND HAVE BEEN USED BY AT LEAST FOUR COMPANY MEMBERS AND BY PPMD IN SUPPORT OF INITIAL ANALYSIS AROUND THE DEVELOPMENT OF THE DUCHENNE PLATFORM TRIAL. (2) PUBLISHED THE CDISC THERAPEUTIC AREA USER GUIDE. (3) DEVELOPMENT OF THE LETTER OF INTENT AND MODELING PLAN ARE UNDERWAY. THE GOAL OF THE ELECTRONIC PATIENT-REPORTED OUTCOME CONSORTIUM IS TO ADVANCE THE SCIENCE OF CLINICAL TRIAL ENDPOINT ASSESSMENT BY COLLABORATIVELY SUPPORTING AND CONDUCTING RESEARCH, DESIGNING AND DELIVERING EDUCATIONAL OPPORTUNITIES, AND DEVELOPING AND DISSEMINATING BEST PRACTICE RECOMMENDATIONS FOR ELECTRONIC COLLECTION OF CLINICAL OUTCOME DATA. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) CONTRIBUTED TO EMA'S GOOD CLINICAL PRACTICE INSPECTORS WORKING GROUP (GCP IWG) BY OFFERING THE EPRO CONSORTIUM'S STAKEHOLDER PERSPECTIVE DURING PAST MEETING OF THE GCP IWG ON TOPICS SUCH AS: RESPONSIBILITIES OF EPRO PROVIDERS REGARDING SOURCE DATA, USE OF ELECTRONIC INFORMED CONSENT, AND RESPONSIBILITIES OF EPRO PROVIDERS WHEN COLLECTING ELECTRONIC SIGNATURES. C-PATH LAUNCHED A NEW CONSORTIUM IN PARTNERSHIP WITH CHDI FOUNDATION TO WORK ON DRUG DEVELOPMENT TOOLS FOR HUNTINGTON'S DISEASE. THE ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) RECRUITED 14 INDUSTRY PARTNERS AND 16 SCIENTIFIC ADVISORS. (2) DEVELOPED AND PUBLISHED THE CDISC HD-SPECIFIC THERAPEUTIC AREA USER GUIDE. (3) CREATED A COMPREHENSIVE INVENTORY OF HD CLINICAL DATA SETS AND MODELING AND SIMULATION TOOLS. (4) ACQUIRED THE FIRST TWO DATA SETS, ENROLL-HD, AN ONGOING NATURAL HISTORY STUDY THAT INCLUDES 8000 SUBJECTS, AND TRACK-HD, A COMPLETED OBSERVATIONAL STUDY THAT INVESTIGATED POTENTIAL OUTCOME MEASURES IN OVER 300 SUBJECTS. ACTIVE CONVERSATIONS ARE ONGOING WITH FOUR ADDITIONAL DATA CONTRIBUTORS. (5) LAUNCHED WORKING GROUP ACTIVITIES WITH FOCUS ON CONTEXT-OF-USE, CRITERIA FOR REGULATORY READINESS OF BIOMARKERS, AND AN IMMEDIATE ACTION PLAN TO SEEK REGULATORY FEEDBACK ON CLINICAL OUTCOME ASSESSMENTS CURRENTLY IN DEVELOPMENT. FORM 990, PART III, LINE 4A AT THE REQUEST OF THE FDA, C-PATH FORMED AN INTERNATIONAL NEONATAL CONSORTIUM (INC). THE PURPOSE OF THE CONSORTIUM IS TO ACCELERATE THE DEVELOPMENT OF SAFE AND EFFECTIVE THERAPIES FOR NEONATES. INC ENGAGES THE GLOBAL NEONATAL COMMUNITY - FAMILIES, NEONATAL NURSES, ACADEMIC SCIENTISTS, REGULATORS, PHARMACEUTICAL INVESTIGATORS, ADVOCACY ORGANIZATIONS, AND FUNDERS - TO FOCUS ON THE NEEDS OF THE NEONATE. THIS PAST YEAR INC'S CLINICAL PHARMACOLOGY WORKGROUP DEVELOPED A WHITE PAPER TO ASSIST REGULATORS IN PREPARING GUIDANCE ON THE CLINICAL PHARMACOLOGY CONSIDERATIONS FOR NEONATES, INCLUDING RECOMMENDATIONS FOR SAFER FORMULATIONS ENCOMPASSING EASE OF ADMINISTRATION. INC'S BRONCHOPULMONARY DYSPLASIA (BPD) WORKGROUP DESCRIBED POTENTIAL ENDPOINTS FOR TRIALS TO EVALUATE TREATMENTS FOR CHRONIC PULMONARY INSUFFICIENCY OF PREMATURITY (CPIP). INC'S SEIZURE WORKGROUP DEVELOPED RECOMMENDATIONS FOR USE IN A MASTER PROTOCOL TO EVALUATE MEDICATIONS TO TREAT NEONATAL SEIZURES. INC'S DATA WORKGROUP REVIEWED THE VALUE AND NEEDS FOR SHARING NEONATAL DATA AND PROPOSED A GLOBAL APPROACH TO COLLECTING, STANDARDIZING, AND SHARING DATA. INC'S CLINICAL PHARMACOLOGY ADVERSE EVENT SUBGROUP COMPLETED DEVELOPMENT OF THE NEONATAL ADVERSE EVENT SEVERITY GRADING SCALE (NAESS) AND HAVE INCORPORATED THE NEW AE TERMINOLOGY INTO THE NATIONAL CANCER INSTITUTE - ENTERPRISE VOCABULARY SERVICES (NCI-EVS) ONLINE THESAURUS. INC SUBMITTED COMMENTS ON THE NOTICE OF PROPOSED RULEMAKING (NPRM) "FEDERAL POLICY FOR THE PROTECTION OF HUMAN SUBJECTS" TO U.S. HEALTH AND HUMAN SERVICES' OFFICE FOR HUMAN RESEARCH PROTECTIONS AND ALSO SUPPLIED INPUT ON DRUGS AND THERAPEUTIC AREAS THAT REQUIRE FURTHER STUDY IN NEONATES, IN RESPONSE TO NATIONAL INSTITUTE OF CHILD HEALTH AND DEVELOPMENT'S REQUEST FOR INFORMATION ON THE BEST PHARMACEUTICALS FOR CHILDREN ACT PRIORITIES IN PEDIATRIC THERAPEUTICS 2017. THE MULTIPLE SCLEROSIS OUTCOME ASSESSMENTS CONSORTIUM HAS A GOAL TO OBTAIN REGULATORY QUALIFICATION OF AN IMPROVED CLINICAL OUTCOME ASSESSMENT INSTRUMENT AS A PRIMARY OR SECONDARY ENDPOINT IN CLINICAL TRIALS OF MULTIPLE SCLEROSIS (MS) THERAPIES. THEIR ACCOMPLISHMENTS THIS YEAR INCLUDE: (1) EXECUTED THE STATISTICAL ANALYSIS PLAN (FOR A SINGLE MEASURE) DEVELOPED FOR FDA AND SUBMITTED THE FINAL QUALIFICATION PACKAGE. (2) EXECUTED THE STATISTICAL ANALYSIS PLAN (INCLUDING FOUR MEASURES) DEVELOPED FOR EMA AND THE "VOICE OF THE PATIENT" METHODOLOGY AND SUBMITTED THE FINAL QUALIFICATION PACKAGE TO THE EMA. THE POLYCYSTIC KIDNEY DISEASE OUTCOMES CONSORTIUM HAS DEVELOPED AND OBTAINED REGULATORY QUALIFICATION FROM THE U.S. FOOD AND DRUG ADMINISTRATION (FDA) AND THE EUROPEAN MEDICINES AGENCY (EMA) OF TOTAL KIDNEY VOLUME (TKV) AS A PROGNOSTIC BIOMARKER FOR USE IN CLINICAL TRIALS FOR NEW THERAPIES FOR AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE (ADPKD). THE CONSORTIUM CONTINUES TO EXPLORE ALTERNATE CLINICAL ENDPOINTS, INCLUDING A COMPOSITE ENDPOINT, INNOVATIVE CLINICAL TRIAL DESIGNS, AND REGULATORY PATHWAYS TO HELP EXPEDITE NEW THERAPIES IN PKD. THE FDA HAS COMMUNICATED THAT TKV IS A "REASONABLY LIKELY SURROGATE," WHICH MAKES IT ELIGIBLE FOR AN ACCELERATED APPROVAL PROCESS AT THE FDA. SIX NEW COMPANIES ARE NOW WORKING TO DEVELOP THERAPIES FOR THIS DISEASE. THE FDA APPROVED OTSUKA'S DRUG (JYNARQUETM). THIS IS THE FIRST-EVER FDA-APPROVED TREATMENT FOR PKD. ALTHOUGH IT WAS NOT A DIRECT OUTPUT OF PKDOC, THE CONSORTIUM'S WORK POSITIVELY CONTRIBUTED TO ADVANCES IN THE FIELD CONSISTENT WITH THIS SUCCESS STORY. THE PATIENT-REPORTED OUTCOME CONSORTIUM HAS A GOAL TO ESTABLISH AND MAINTAIN A COLLABORATIVE FRAMEWORK WITH APPROPRIATE STAKEHOLDERS FOR THE QUALIFICATION OF PATIENT-REPORTED OUTCOME (PRO) INSTRUMENTS AND OTHER CLINICAL OUTCOME ASSESSMENT (COA) TOOLS THAT WILL BE MADE AVAILABLE FOR USE IN CLINICAL TRIALS WHERE COA-BASED ENDPOINTS ARE USED TO SUPPORT PRODUCT LABELING CLAIMS. TWO PRO MEASURES WERE QUALIFIED BY FDA FOR EXPLORATORY USE, THE SYMPTOMS OF MAJOR DEPRESSIVE DISORDER SCALE (SMDDS) AND THE NON-SMALL CELL LUNG CANCER SYMPTOM ASSESSMENT QUESTIONNAIRE (NSCLC-SAQ). OTHER PRO MEASURES WHICH HAVE BEEN SUBMITTED TO FDA FOR QUALIFICATION FOR EXPLORATORY USE INCLUDE: (1) THE ASTHMA DAILY SYMPTOM DIARY (ADSD) AND (2) THE FUNCTIONAL DYSPEPSIA SYMPTOM DIARY (FDSD). THE PRO CONSORTIUM HAS ISSUED A TOTAL OF 10 LICENSES FOR USE OF PRO CONSORTIUM MEASURES INCLUDING THE SMDDS, THE NSCLCSAQ, AND THE MYELOFIBROSIS SYMPTOM ASSESSMENT FORM V4.0 (MFSAF V4.0). THE FOLLOWING COAS ARE IN VARIOUS STAGES OF THE DEVELOPMENT AND/OR QUALIFICATION PROCESS: UCSD PERFORMANCE-BASED SKILLS ASSESSMENT FOR MILD COGNITIVE IMPAIRMENT (UPSA-MCI), DIARY FOR IRRITABLE BOWEL SYNDROME SYMPTOMS-C (DIBSS-C) FOR CONSTIPATION PREDOMINANT IBS, DIARY FOR IRRITABLE BOWEL SYNDROME SYMPTOMS-D (DIBSS-D) FOR DIARRHEA PREDOMINANT IBS, DIARY FOR IRRITABLE BOWEL SYNDROME SYMPTOMS-M (DIBSS-M) FOR MIXED IBS, AN OPTIMIZED PROMIS PHYSICAL FUNCTION SHORT FORM FOR MS PATIENTS, PEDIATRIC ASTHMA DIARY - OBSERVER (PAD-O), PEDIATRIC ASTHMA DIARY - CHILD (PAD-C), RHEUMATOID ARTHRITIS WG - PROMIS SHORT FORM V1.0 - FATIGUE 10A. THE PREDICTIVE SAFETY TESTING CONSORTIUM HAS A GOAL TO BRING TOGETHER PHARMACEUTICAL COMPANIES TO SHARE AND VALIDATE INNOVATIVE SAFETY TESTING METHODS UNDER ADVISEMENT OF THE U.S. FOOD AND DRUG ADMINISTRATION (FDA), EUROPEAN MEDICINES AGENCY (EMA), AND JAPAN'S PHARMACEUTICALS AND MEDICAL DEVICES AGENCY (PMDA). CURRENTLY PSTC IS FOCUSED ON DEVELOPING AND OBTAINING REGULATORY QUALIFICATION OF IMPROVED CLINICAL SAFETY BIOMARKERS FOR USE IN DRUG DEVELOPMENT. THIS YEAR THE CONSORTIUM'S ACCOMPLISHMENTS INCLUDE: (1) IN COOPERATION WITH THE FDA, LAUNCHED THE PILOT BIOMARKER DATA REPOSITORY (BMDR) PROJECT DESIGNED TO COLLECT AND ANALYZE KIDNEY SAFETY BIOMARKER DATA. (2) RECEIVED A LETTER OF SUPPORT FROM EMA FOR THE USE OF GLUTAMATE DEHYDROGENASE (GLDH), A SPECIFIC BIOMARKER OF DRUG-INDUCED HEPATIC INJURY IN HUMANS. (3) ACHIEVED FDA QUALIFICATION OF A PANEL OF CLINICAL DRUG-INDUCED KIDNEY INJURY BIOMARKERS FOR USE IN PHASE 1 CLINICAL TRIALS. THE TYPE I DIABETES CONSORTIUM (T1D) IS FOCUSING ON QUALIFYING ISLET AUTOIMMUNITY ANTIBODIES AS SUSCEPTIBILITY/RISK BIOMARKERS TO BE USED IN THE DEVELOPMENT OF THERAPIES FOR THE TREATMENT, AND ULTIMATELY, THE PREVENTION, OF TYPE 1 DIABETES. THIS YEAR T1D SECURED THE FOLLOWING DATA SETS AND IS CURRENTLY WORKING TO BUILD AN INTEGRATED DATABASE TO SUPPORT QUALIFICATION: THE COLORADO DIABETES AUTOIMMUNITY STUDY IN THE YOUNG (DAISY), THE ENVIRONMENTAL DETERMINANTS OF DIABETES IN THE YOUNG (TEDDY), TRIALNET NATURAL HISTORY STUDY (TN01-NH), THE GERMAN BABYDIAB AND BABYDIET STUDIES, AND THE DIABETES PREVENTION TRIAL (DPT-1). THE CONSORTIUM SUBMITTED A LETTER OF INTENT TO FDA AND RECEIVED A POSITIVE RESPONSE TO THE LETTER OF INTENT FROM THE FDA AND WAS ASKED TO PROCEED TO THE NEXT STEP IN THE QUALIFICATION PROCESS: THE DEVELOPMENT OF THE QUALIFICATION PLAN. THE CONSORTIUM IS NOW WORKING ON THE DRAFTING OF THE QUALIFICATION PLAN FOCUSING, IN PART, ON THE BUILDING OF AN ASSAY PUBLICATION LIBRARY AND REVIEWING THE ASSAY METHODOLOGY USED FOR ALL DATA TO COMPLETE THE ANALYTICAL CONSIDERATIONS SECTION OF THE DOCUMENT. THE CONSORTIUM HAS INITIATED CONSTRUCTION OF A MODELING ANALYSIS PLAN TO ENABLE THE GENERATION OF THE T1D DISEASE PROGRESSION MODEL REQUIRED TO SUPPORT THE REGULATORY ENDORSEMENT OF THE ISLET AUTOANTIBODIES. THE TRANSPLANTATION THERAPEUTICS CONSORTIUM IS WORKING TO IDENTIFY MECHANISMS AND DRUG DEVELOPMENT TOOLS TO ACCELERATE DRUG DISCOVERY FOR TRANSPLANT PATIENTS, THROUGH THE COLLABORATIVE INVOLVEMENT OF KEY STAKEHOLDERS IN THE FIELD. THE CONSORTIUM IS INITIALLY FOCUSING ON KIDNEY TRANSPLANT BUT MAY EXPAND TO OTHER SOLID ORGAN TRANSPLANTS IN THE FUTURE. THIS YEAR THE TTC INITIATED TWO WORKGROUPS, THE ENDPOINTS AND BIOMARKERS WORKGROUP, AND THE DRUG SAFETY PROFILE CHARACTERIZATION AND LABELING WORKGROUP. THE INITIAL FOCUS OF THE CONSORTIUM IS TO SEEK FDA ENDORSEMENT FOR A QUANTITATIVE DRUG DEVELOPMENT TOOL CAPABLE OF OPTIMIZING CLINICAL TRIAL DESIGNS IN KIDNEY TRANSPLANTATION, BY INFORMING SPONSOR DECISION MAKING REGARDING CLINICAL TRIAL DESIGN, SIZE, AND DURATION, OPTIMIZE DOSING STRATEGIES, PREDICT SAFETY SIGNALS, SUPPORT EVIDENCE OF EFFECTIVENESS, AND ENDPOINT SELECTION. THROUGH FDA'S BROAD AGENCY ANNOUNCEMENT PROCEDURE, TTC RECEIVED FUNDING TO SUPPORT THE INITIAL FOCUS AREA OF DEVELOPING A MODEL. FORM 990, PART VI, SECTION A, LINE 2 MIKE KASSER AND JEFF JACOB HAVE A BUSINESS RELATIONSHIP. PETER HUTT AND JEFF JACOB HAVE A BUSINESS RELATIONSHIP. WAIN FISHBURN AND JEFF JACOB HAVE A BUSINESS RELATIONSHIP. JEFF JACOB AND SHAUN KIRKPATRICK HAVE A BUSINESS RELATIONSHIP. FORM 990, PART VI, SECTION B, LINE 11B FORM 990 IS PREPARED BY AN EXTERNAL CPA FIRM USING INFORMATION PROVIDED BY THE ORGANIZATION. THE FORM IS REVIEWED BY THE ORGANIZATION'S COMPTROLLER, COO, PRESIDENT/CEO, THE BOARD AUDIT, FINANCE, AND RISK COMMITTEE, AND THE BOARD OF DIRECTORS PRIOR TO BEING FINALIZED. FORM 990, PART VI, SECTION B, LINE 12C ALL MEMBERS OF THE BOARD OF DIRECTORS AND ALL OFFICERS OF THE ORGANIZATION ARE REQUIRED TO FILL OUT A CONFLICT OF INTEREST FORM ANNUALLY. THE RESULTS ARE COMPILED AND REVIEWED BY THE BOARD'S AUDIT, FINANCE, AND RISK COMMITTEE. ANY ACTUAL OR PERCEIVED CONFLICTS THAT HAVE THE POTENTIAL TO BIAS ANY DISCUSSIONS OR DECISIONS BY THE BOARD ARE DISCUSSED BY THE BOARD. ANY DIRECTOR WITH A REAL OR POTENTIAL CONFLICT WILL RECUSE THEMSELVES FROM ANY DISCUSSION OR DECISION WHERE THE CONFLICT HAS A BEARING. FORM 990, PART VI, SECTION B, LINE 15 A BOARD OF DIRECTORS COMPENSATION COMMITTEE MEETS AND REVIEWS EXECUTIVE COMPENSATION ANNUALLY. EVERY FEW YEARS THE COMMITTEE SEEKS AN INDEPENDENT COMPENSATION CONSULTING ORGANIZATION. AFTER THE CONSULTING ORGANIZATION HAS BEEN RETAINED, THE COMPENSATION COMMITTEE RECEIVES THE CONSULTANT'S EXECUTIVE COMPENSATION REVIEWS AND RECOMMENDATIONS. THIS INFORMATION IS THEN USED AS A BASIS FOR THE COMPENSATION COMMITTEE'S RECOMMENDATION TO THE BOARD OF DIRECTORS. THE LAST INDEPENDENT COMPENSATION SURVEY WAS CONDUCTED FOR THE CEO IN AUGUST 2011. THE CEO'S SALARY HAS REMAINED CONSTANT SINCE THE TIME OF HER HIRE. AN OVERALL SALARY PROGRAM FOR EMPLOYEES, WHICH IS DEVELOPED BASED ON SALARY SURVEY INFORMATION, MUST BE APPROVED BY THE BOARD OF DIRECTORS COMPENSATION COMMITTEE BEFORE IMPLEMENTATION. ALL EMPLOYEE SALARY INCREASES MUST BE APPROVED BY THE PRESIDENT/CEO. FORM 990, PART VI, SECTION C, LINE 19 ARTICLES OF INCORPORATION CAN BE FOUND ON THE ARIZONA CORPORATION COMMISSION WEBSITE. THE CONFLICT OF INTEREST POLICY AND THE FINANCIAL STATEMENTS (VIA THE ANNUAL REPORT) ARE POSTED ON THE INSTITUTE'S WEBSITE. FORM 990, PART IX, LINE 11G RESEARCH SUPPORT SERVICES: PROGRAM SERVICE EXPENSES 1,404,700. MANAGEMENT AND GENERAL EXPENSES 0. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 1,404,700. CONSULTING: PROGRAM SERVICE EXPENSES 836,851. MANAGEMENT AND GENERAL EXPENSES 68,397. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 905,248. OTHER PROFESSIONAL FEES: PROGRAM SERVICE EXPENSES 118,245. MANAGEMENT AND GENERAL EXPENSES 7,976. FUNDRAISING EXPENSES 0. TOTAL EXPENSES 126,221. CRITICAL PATH FOUNDATION 1730 EAST RIVER ROAD SUITE 200 TUCSON AZ 85718 204780896 RAISING AND CONTRIBUTING FUNDS TO CRITICAL PATH INSTITUTE AZ 501(C)(3) LINE 11 CRITICAL PATH INSTITUTE 1 CRITICAL PATH GLOBAL LTD 20-22 BEDFORD ROW LONDON UK WC1R 4JS FACILITATE WORK WITH EUROPEAN MEDICINES AGENCY UK CRITICAL PATH INSTITUTE 1 CRITICAL PATH INSTITUTE LTD 33 SIR JOHN ROGERSONS QUAY DUBLIN EI 2 FACILITATE WORK WITH EUROPEAN MEDICINES AGENCY EI CRITICAL PATH INSTITUTE 1 0 0 0 0 0 0 0 0 0 0 0 0 0 1 1 0 0 0 0