The announcement follows an independent Data Monitoring Committee’s review of the therapy’s safety, absorption, distribution, metabolism, and excretion in Cohort 1.
The randomized, double-blind, placebo-controlled, multiple-dose, Phase 2 clinical trial (NCT02644668) will evaluate CK-2127107’s pharmacodynamics in two groups that receive ascending multiple doses of the therapy. The groups will consist of ambulatory and non-ambulatory patients with SMA Types II, III and IV.
The objective of the trial, which is in the patient-recruitment stage, is to see whether CK-2127107 can improve the patients’ skeletal muscle function and muscle fatigue. The study will also look at the safety, tolerability and pharmacokinetics of CK-2127107.
Cohort 1 consisted of 18 ambulatory Type III or IV patients and 18 non-ambulatory Type II or III patients 12 years of age and older. They were randomly assigned to 150 mg of CK-2127107 or a placebo twice a day for eight weeks.
The second cohort also will consist of 18 ambulatory Type III or IV patients and 18 non-ambulatory Type II or III patients 12 and older. They will be randomized to received 450 mg of CK-2127107 or a placebo twice a day, also for eight weeks.
Both cohorts’ skeletal muscle function and fatigue levels will be assessed as part of the trial. The measuring devices will include respiratory evaluations, tests of the strength and functionality of the upper limbs in non-ambulatory patients, and six-minute walk and timed-up-and-go tests for ambulatory patients.
“We are pleased to advance this clinical trial to the next phase of execution, and appreciate the enthusiasm of our investigators to explore the potential of this novel mechanism drug candidate that specifically targets skeletal muscle and potentially improves muscle force, function and stamina,” Fady I. Malik, MD, PhD, Cytokinetics’ executive vice president of research and development, said in a press release.
CK-2127107 is an investigational drug candidate intended to slow the regulatory troponin complex’s rate of calcium release in fast skeletal muscles. The complex consists of three proteins integral to muscle contraction.
Cytokinetics and Astellas Pharma are developing CK-2127107 (CK-107) as a treatment for SMA and other conditions characterized by neuromuscular or non-neuromuscular dysfunction, muscular weakness, or muscle fatigue.