Biopharmaceutical firm Cytokinetics has reported that its experimental heart drug, aficamten, produced strong improvements over standard-of-care therapy in obstructive hypertrophic cardiomyopathy (oHCM) patients. The drug showed enhanced exercise capacity versus metoprolol in a late-stage trial, potentially making aficamten a new first-line treatment.

Key highlights

Strong efficacy results in late-stage study

• Aficamten greatly enhanced exercise-induced oxygen uptake after 24 weeks versus metoprolol, a typical beta-blocker.

• 175 patients with obstructive hypertrophic cardiomyopathy were included in the study.

Safety and tolerability

• The drug had a good safety profile versus metoprolol.

• Complete trial results will be reported at a future medical meeting.

Regulatory timeline and market impact

• The U.S. FDA is due to make a regulatory decision by December 26, 2025.

• RBC Capital analysts estimate aficamten would be able to generate $900 million in peak first-line sales by 2034, with overall revenue potential estimated at $3.9 billion.

Analyst reactions and market potential

• Analysts indicate the strong data would induce earlier use or switching to aficamten, despite access issues related to payers.

• Comparisons with Bristol Myers Squibb's Camzyos indicate aficamten provides a strong efficacy and safety profile.

Therapeutic area and unmet need
Aficamten is being brought forward against obstructive hypertrophic cardiomyopathy (oHCM), a disease that results in abnormal thickening of the heart muscle and elevates the risk for sudden cardiac arrest.With aficamten demonstrating superiority against benchmark therapy in an important heart disease indication, Cytokinetics is on the cusp of a possible oHCM treatment breakthrough. Upon approval, aficamten may transform the therapeutic paradigm by providing a safe, efficacious, and earlier-line therapy alternative in an important cardiac underserved segment.