Loading Curie Briefs...
Getting the latest healthcare news for you
Getting the latest healthcare news for you
A heart drug with a growing footprint — Cytokinetics' aficamten significantly improved symptoms and exercise capacity in patients with non-obstructive hypertrophic cardiomyopathy (nHCM), a genetic heart condition with no currently approved treatments. Late-stage trial data, presented at the ESC Congress, showed 53% of patients on aficamten improved meaningfully across key disease areas vs. just 13% on placebo. The company plans to file for expanded U.S. approval in Q4 2026.
A heart drug with a growing footprint
Cytokinetics' aficamten (Myqorzo) — already approved for obstructive hypertrophic cardiomyopathy (oHCM) — is now eyeing a label expansion into non-obstructive HCM (nHCM), a related genetic heart condition with no approved therapies. Detailed late-stage trial data presented at the European Society of Cardiology Congress showed the drug meaningfully improved symptoms, stamina, and heart strain in nHCM patients, with a supplemental U.S. marketing application planned for Q4 2026.
The 517-patient trial delivered encouraging but nuanced results. While aficamten cleared its dual primary endpoints of improving heart failure symptoms and quality of life, it did not significantly improve heart structure or delay cardiovascular events. Notably, about 10% of patients experienced a dangerous drop in cardiac pumping capacity, and 12 heart failure events occurred — attributed by the company's CMO to a rigid trial dosing algorithm rather than the drug itself, noting that real-world physicians will have more flexibility to manage dosing safely.
By the Numbers
Why it matters: With no approved treatments for nHCM, aficamten could fill a critical gap for patients. Analysts also see the nHCM data as a "bridge" validating Cytokinetics' next-generation drug, ulacamten, in the larger heart failure with preserved ejection fraction (HFpEF) market — setting up a multi-front competition with Bristol Myers Squibb.
