Approval Probability
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Label indication MYQORZO is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. MYQORZO is a cardiac myosin inhibitor indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. ( 1 ) FDA label
Aficamten · 5 trials · 5 indications
Effect of aficamten compared with placebo on participant health status
Effect of aficamten compared with placebo on maximal exercise capacity
Effect of aficamten compared with metoprolol succinate on exercise capacity in patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Exercise capacity 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.
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)
Patient incidence of reported Serious Adverse Events (SAEs)
Patient incidence of reported LVEF \<50%
| 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 plus placebo for metoprolol | EXPERIMENTAL | Patients will receive doses of 5 mg, 10 mg, 15 mg or 20 mg of aficamten (CK-3773274) plus placebo for metoprolol succinate with dose levels guided by echocardiography assessments for up to 24 weeks. |
| Metoprolol succinate up to 200 mg plus placebo for aficamten | ACTIVE_COMPARATOR | Patients will receive 50 mg, 100 mg, 150 mg or 200 mg of metoprolol succinate plus placebo for aficamten (CK-3773274) with dose levels guided by echocardiography and heart rate assessments for up to 24 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. |
| Name | Type | Description |
|---|---|---|
| Aficamten | DRUG | Oral Tablet |
| Placebo | DRUG | Oral Tablet |
| Aficamten (CK-3773274) (5 mg, 10 mg, 15 mg and 20 mg) | DRUG | Aficamten (CK-3773274) tablets administered orally |
| Placebo to match aficamten | DRUG | Placebo for aficamten (CK-3773274) administered orally |
| Metoprolol succinate (50 mg, 100 mg, 150 mg and 200 mg) | DRUG | Metoprolol succinate tablets administered orally |
| Placebo to match metoprolol succinate | DRUG | Placebo for metoprolol succinate administered orally |
| Aficamten (5 mg, 10 mg, 15 mg, and 20 mg) | DRUG | Aficamten tablets were administered orally once daily. |
| Aficamten (5 - 20 mg) | DRUG | Aficamten tablets, 5-20 mg daily, administered orally. Participants may continue on Aficamten until end of treatment criteria defined in the protocol is met. |
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 hypertrophic cardiomyopathy, including symptomatic obstructive hypertrophic cardiomyopathy (oHCM) and symptomatic non-obstructive hypertrophic cardiomyopathy. It is also being studied in pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy. It is not yet approved for any indication.
Aficamten targets cardiac myosin. By modulating cardiac myosin, it aims to reduce excessive contractility in the heart muscle, which is a characteristic feature of hypertrophic cardiomyopathy. This mechanism is intended to address the underlying pathophysiology of both obstructive and non-obstructive forms of the disease.
Aficamten is being developed by Cytokinetics, Incorporated, which trades on the Nasdaq under the ticker CYTK. Cytokinetics is a biopharmaceutical company focused on discovering and developing muscle-directed therapeutics for cardiovascular and neuromuscular diseases.
Aficamten is in Phase 3 clinical development. Two Phase 3 trials in adults with symptomatic obstructive hypertrophic cardiomyopathy have been completed, and a Phase 3 trial in adults with symptomatic non-obstructive hypertrophic cardiomyopathy is active but not recruiting. A Phase 2 trial in pediatric patients is also active but not recruiting.
Aficamten has been studied in four registered trials. Completed Phase 3 trials include NCT05186818 (versus placebo in oHCM) and NCT05767346 (versus metoprolol succinate in oHCM). Ongoing trials include NCT06081894 (Phase 3 in non-obstructive HCM) and NCT06412666 (Phase 2 in pediatric oHCM).
Yes, aficamten is also known by its development code CK-3773274. The drug has been investigated in multiple dose strengths, including 5 mg, 10 mg, 15 mg, and 20 mg. These alternative names and dose descriptions refer to the same investigational compound.