Approval Probability
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Aficamten · 5 trials · 6 indications
Effect of aficamten compared with placebo on participant health status
Effect of aficamten compared with placebo on maximal exercise capacity
The effect of CK-3773274 on exercise capacity in participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) was determined through changes in peak oxygen uptake (pVO2) after 24 weeks of treatment. pVO2 was measured by cardiopulmonary exercise testing (CPET) on a treadmill or bicycle. A higher pVO2 indicates better cardiorespiratory fitness.
Patient incidence of reported Adverse Events (AEs)
To determine the dose for Part B based on Part A PK and safety data.
To evaluate the effect of a single oral dose of aficamten on the QTc interval in healthy participants.
| Arm | Type | Description |
|---|---|---|
| Aficamten | EXPERIMENTAL | Participants 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. |
| Placebo | PLACEBO_COMPARATOR | Participants in this arm will receive placebo, for up to 72 weeks. |
| Aficamten up to 20 mg | EXPERIMENTAL | Participants received 5 mg, 10 mg, 15 mg, or 20 mg of aficamten; dose levels were guided by echocardiography assessments. Treatment was administered for up to 24 weeks. |
| Placebo to match aficamten | PLACEBO_COMPARATOR | Participants received placebo for up to 24 weeks. |
| Part A (Dose Finding Cohort 1): Aficamten 50 mg | EXPERIMENTAL | Participants in this arm will receive a single oral dose of 50 mg aficamten. |
| Part A (Dose Finding Cohort 2): Aficamten 75 mg | EXPERIMENTAL | Participants in this arm will receive a single oral dose up to 75 mg aficamten. |
| Part A (Dose Finding Cohort 3): Aficamten 100 mg | EXPERIMENTAL | Participants in this arm will receive a single oral dose up to 100 mg aficamten. |
| Part B (TQT Study): Aficamten | EXPERIMENTAL | Participants will receive a single oral dose of aficamten. The dose will be determined based on review of Part A PK parameters, echocardiogram parameters, safety, and tolerability for aficamten. |
| Part B (TQT Study): Aficamten-matching Placebo | PLACEBO_COMPARATOR | Participants in this arm will receive a single oral dose of aficamten-matching placebo. |
| Part B (TQT Study): Moxifloxacin 400 mg | ACTIVE_COMPARATOR | Participants will receive a single oral dose of 400 mg moxifloxacin |
| Name | Type | Description |
|---|---|---|
| Aficamten | DRUG | Oral Tablet |
| Placebo | DRUG | Oral Tablet |
| Aficamten (5 mg, 10 mg, 15 mg, and 20 mg) | DRUG | Aficamten tablets were administered orally once daily. |
| Placebo to match aficamten | DRUG | Placebo tablets were administered orally once daily. |
| Aficamten (5 - 20 mg) | DRUG | Aficamten tablets administered orally. During titration phase, clinic visits will occur approximately every 2-6 weeks. In the maintenance phase clinic visits will occur every 24 weeks, with safety check-in occurring every 12 weeks between visits by phone or, if desirable, in the clinic. |
| Aficamten-matching Placebo | DRUG | Oral Tablet |
| Moxifloxacin | DRUG | Oral Tablet |
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...
Aficamten is an investigational small molecule being developed for symptomatic hypertrophic cardiomyopathy (HCM), including obstructive hypertrophic cardiomyopathy (oHCM) and non-obstructive hypertrophic cardiomyopathy (nHCM). It is also being studied in pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy and in healthy participants for clinical pharmacology studies.
Aficamten is being developed by Cytokinetics, Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker CYTK. The company is conducting clinical trials of Aficamten in adults with hypertrophic cardiomyopathy and in pediatric patients with obstructive hypertrophic cardiomyopathy.
Aficamten is in clinical development across multiple phases. It has completed a Phase 1 study in healthy participants, and is being evaluated in Phase 2 and Phase 3 trials for hypertrophic cardiomyopathy. Aficamten is investigational and has not been approved by the FDA.
Aficamten is being studied in several trials, including NCT04848506, an open-label extension study in adults with HCM; NCT05924815, a completed Phase 1 QT study in healthy participants; NCT06081894, a Phase 3 placebo-controlled trial in symptomatic non-obstructive HCM; and NCT06412666, a Phase 2 study in pediatric patients with symptomatic obstructive HCM.
Yes, Aficamten is also known as Aficamten (5-20 mg), which refers to the dose range being evaluated in clinical studies. The drug is being tested at doses of 5 to 20 mg in trials for hypertrophic cardiomyopathy.
Aficamten clinical trials are randomized, double-blind, and placebo-controlled, with a total planned enrollment of approximately 900 participants across studies. The largest trial, NCT04848506, is an open-label extension enrolling 900 adults with symptomatic HCM, while the Phase 3 trial NCT06081894 enrolls 500 adults with non-obstructive HCM.