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Aficamten

Phase 3

Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy | Small molecule | Cardiovascular |Cytokinetics, Incorporated|Last Updated: Feb 2, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment500
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06081894Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCMPHASE3 ACTIVE NOT_RECRUITING 500Aug 30, 2023Sep 1, 2026Feb 2, 2026180 United States, Argentina +18
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Study Endpoints
Primary Endpoints
Change in Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS)
Baseline to Week 36

Effect of aficamten compared with placebo on participant health status

Change in pVO2
Baseline to Week 36

Effect of aficamten compared with placebo on maximal exercise capacity

Secondary Endpoints
Change in composite of two Z-scores of CPET parameters (pVO2 and VE/VCO2 slope)
Baseline to Week 36
Proportion of participants with ≥ 1 class improvement in NYHA Functional Class
Baseline to Week 36
Change in NT-proBNP
Baseline to Week 36
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AficamtenEXPERIMENTALParticipants in this arm will receive a single daily oral dose of 5 mg, 10 mg, 15 mg, or 20 mg of aficamten with dose levels guided by echocardiography assessments, for up to 72 weeks.
PlaceboPLACEBO_COMPARATORParticipants in this arm will receive placebo, for up to 72 weeks.
Interventions
NameTypeDescription
AficamtenDRUGOral Tablet
PlaceboDRUGOral Tablet
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Eligibility Criteria
Age Range18 Years — 85 Years
SexALL
Healthy VolunteersNo
Study Sites180

Inclusion Criteria: * Between 18-85 years of age * Body mass index \< 40 kg/m2 * Diagnosed with nHCM and has a screening echocardiogram with the following: * End-diastolic left ventricular (LV) wall thickness: * ≥ 15 mm in one or more myocardial segments OR * ≥ 13 mm in one or more wall...

Countries:United StatesArgentinaAustraliaBrazilCanadaChinaDenmarkFranceGermanyGreeceHungaryIcelandIsraelItalyJapanNetherlandsPolandPortugalSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06081894primaryCompletionDate: changed
LOWMay 24, 2026NCT06081894studyFirstPostDate: changed