Cytokinetics announces initiation of phase 1 study of aficamten in healthy Japanese participants.

“We are conducting this Phase I bridging study to characterize the pharmacokinetics of aficamten in healthy Japanese adults and to gather evidence that we believe will be required for potential approval in Japan,” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “In parallel, we are continuing to execute on our later-stage global clinical development program for aficamten alongside preparing regulatory submissions in the U.S. and Europe which we expect to submit this year.”

Phase I Clinical Trial Design; The primary objective of this Phase I double-blind, randomized, placebo-controlled study is to evaluate the pharmacokinetics of aficamten following administration of single ascending doses and multiple doses in 70 healthy Japanese and Caucasian participants. The secondary objective is to evaluate the safety and tolerability of aficamten in healthy Japanese and Caucasian participants. The study will enroll four cohorts including three single-ascending cohorts and one multiple dose cohort. Cohorts 1, 2 and 3 will enroll 10 Japanese participants and 10 Caucasian participants each, randomized on an 8:2 basis to receive single-ascending doses of aficamten (5 mg, 10 mg and 20 mg, respectively) or placebo. Enrollment of Cohort 2 and Cohort 3 will commence upon evaluation of the safety of the preceding Cohort. Following the completion of the single ascending dose cohorts, Cohort 4 will enroll 10 healthy Japanese participants randomized on an 8:2 basis to receive single doses of aficamten (5 mg) or placebo, once daily for 14 days.

Aficamten was evaluated in SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), a positive pivotal Phase III clinical trial in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Aficamten received Breakthrough Therapy Designation for the treatment of symptomatic obstructive HCM from the FDA as well as the National Medical Products Administration (NMPA) in China. Cytokinetics expects to submit a New Drug Application (NDA) to the FDA in Q3 2024 and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in Q4 2024.

Aficamten is also currently being evaluated in MAPLE-HCM, a Phase III clinical trial of aficamten as monotherapy compared to metoprolol as monotherapy in patients with obstructive HCM, ACACIA-HCM, a Phase III clinical trial of aficamten in patients with non-obstructive HCM, and CEDAR-HCM, a clinical trial of aficamten in a pediatric population with obstructive HCM, and FOREST-HCM, an open-label extension clinical study of aficamten in patients with HCM.