Cytokinetics, Incorporated announced results from its phase-I, randomized, double-blind, placebo-controlled, multiple-dose clinical trial of oral CK-2017357. The primary objective of this clinical trial was to determine the safety and tolerability of CK-2017357 after multiple oral doses to steady state in healthy male volunteers. The secondary objective was to evaluate the pharmacokinetic profile of CK-2017357 after multiple oral doses to steady state.

In this phase-I clinical trial, two cohorts, each comprised of 12 healthy males, were randomized 2:1 to receive daily morning doses of oral CK-2017357 versus matching placebo for seven days. The CK-2017357 dose was 250 mg in Cohort 1 and 375 mg in Cohort 2. At steady state, which was achieved at both dose levels by the sixth day of treatment, both the maximum CK-2017357 plasma concentration (Cmax) and the area under the CK-2017357 plasma concentration versus time curve from dosing until 24 hours after dosing (AUC24h) were generally dose proportional and exhibited only modest accumulation compared to the values measured after the first dose. In general, systemic exposure to CK-2017357 in this trial was high and inter-subject variability was low. In addition, these multiple dose regimens of CK-2017357 were well tolerated, and there were no serious adverse events.

“These results, in combination with the single dose phase-I data presented earlier this month, are encouraging because they demonstrate that with continued daily oral dosing, CK-2017357 plasma concentrations can be achieved and maintained in a range that has been shown to increase skeletal muscle function in humans,” stated Andrew A Wolff, FACC, Cytokinetics’ senior vice president of Clinical Research and Development and Chief Medical Officer. “The low inter-subject variability and modest accumulation we observed during multiple dosing in healthy volunteers in this study suggests that during chronic outpatient administration, plasma concentrations of CK-2017357 may be unlikely to exceed those that have been well-tolerated by healthy volunteers after single doses up to 2000 mg. We believe these data support movement into phase-II Evidence of Effect trials in patients with neuromuscular diseases and other conditions that may limit mobility.”

CK-2017357 is a fast skeletal muscle troponin activator and is the lead drug candidate from the company’s skeletal sarcomere activator programme.

Cytokinetics is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapeutics that modulate muscle function for the potential treatment of serious diseases and medical conditions.