Cytokinetics’ Aficamten with breakthrough Phase III results

Cytokinetics Inc’s comparative study MAPLE-HCM provides clear evidence of the superiority of monotherapy with the oral cardioselective myosin inhibitor Aficamten over monotherapy with the beta-blocker metoprolol in terms of all clinically relevant endpoints, as well as its potential benefit as first-line therapy for obstructive hypertrophic cardiomyopathy (oHCM) – a genetic heart disease in which the heart muscle thickens abnormally to the point that it impedes blood flow. The integrated safety analysis showed that treatment with Aficamten was well tolerated and had a similar side effect profile to placebo, indicating its safety for oHCM patients.

In parallel with the MAPLE-HCM study, Cytokinetic is also testing aficamten in a paediatric population with oHCM and in adults with HCM in whom blood flow is not yet impeded by hypertrophy. According to analyses by four different market research firms, the global market for HCM therapeutics was between US$1.3bn and US$1bn in 2024.

European companies that are licensing and marketing partners of Cytokinetics can also rejoice in this success. Bayer AG has secured the exclusive marketing rights for Aficamten for €50 million upfront, €90 million in milestone payments until market approval, and sales-targeted milestones of up to €490 million in Japan, where it is conducting a Phase III study in patients with obstructive HCM. Sanofi has secured exclusive marketing rights for the Greater China region through its acquisition of China’s Corxel Co. Ltd. and can look forward to double-digit royalties upon approval.