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Aficamten

Phase 3

Obstructive Hypertrophic Cardiomyopathy | Small molecule | Cardiovascular |Milestone Pharmaceuticals Inc.|Last Updated: Jan 21, 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
UNCONTROLLED
Total Trials1
Total Enrollment40
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06116968An Open-Label Study of Aficamten for Chinese Patients With Symptomatic oHCMPHASE3 COMPLETED 40 — — Nov 14, 2023Oct 20, 2025Jan 21, 202612 China
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Study Endpoints
Primary Endpoints
Patient incidence of reported adverse events (AEs)
Baseline to End of study, up to 2 years
Patient incidence of reported serious adverse events (SAEs )
Baseline to End of study, up to 2 years
Patient incidence of LVEF<50% & LVEF <40%
Baseline to End of study, up to 2 years
Secondary Endpoints
Peak LVOT-G at rest and with Valsalva provocation
Change from baseline values to week 48 at 12-week intervals;
Proportion of patients with resting LVOT-G <50 mmHg
Change from baseline values to week 48 at 12-week intervals;
Proportion of patients with resting LVOT-G <30 mmHg
Change from baseline values to week 48 at 12-week intervals;
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AficamtenEXPERIMENTALPatients in this arm take daily dose of Aficamten. Each patient will start at the lowest dose (5 mg) in the pre-specified dose range and undergo dose titration up to a maximum of 20 mg.
Interventions
NameTypeDescription
AficamtenDRUG5-20mg
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Eligibility Criteria
Age Range18 Years — 85 Years
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: 1. Completion of a Cytokinetics trial investigating CK-3773274 2. LVEF ≥55% at the Screening Visit Exclusion Criteria: 1. Has participated in another investigational device or drug study or received an investigational device or drug \<1 month (or 5 half-lives for drugs, whiche...

Countries:China
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