San Francisco-based biotech company Cytokinetics announced positive results this week from a closely watched late-stage clinical trial of its experimental heart disease drug aficamten. The company’s shares surged over 80% to a 19-year high on the news, adding billions in market value as investors cheered the potential new treatment option for patients.
The phase 3 trial evaluated aficamten in patients with obstructive hypertrophic cardiomyopathy (oHCM), a chronic heart condition where abnormal thickening of heart muscle obstructs blood flow. The main goal was improvement in exercise capacity as measured by peak oxygen uptake.
After 24 weeks, patients receiving aficamten showed a statistically significant 1.74 mL/min/kg increase compared to placebo. This robust efficacy signal exceeded the benchmark set by Bristol Myers Squibb’s recently approved drug Camzyos, which demonstrated a 1.4 mL/min/kg benefit in its phase 3 trial.
Safety was also favorable, with no treatment interruptions due to the drug negatively impacting heart function. Taken together, analysts called it a “home run” result that positions aficamten as potentially best-in-class for this patient population with few options.
Cytokinetics now plans to file for regulatory approval in the second half of 2024 in the US and Europe. With blockbuster sales projections near $2 billion, the company and drug’s success seem linked at this pivotal juncture. The strong data may also draw buyout interest from major pharma companies, as reported by Bloomberg in October.
“We spent considerable time with analysts and investors discussing expectations, and the results met these high bars,” said CEO Robert Blum. “I’m thrilled we can deliver this promising new medicine to oHCM patients with such high unmet need.”
By demonstrating robust safety and efficacy compared to available therapies, aficamten appears poised to become an important new targeted treatment for heart disease if approved. Cytokinetics stands to benefit greatly from their perseverance through years of research and development to help this underserved patient population.
