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Cytokinetics Initiates Phase II Trial of Potential SMA Drug

11 January 2016

Cytokinetics and Astellas have begun enrolling SMA patients in a Phase II clinical trial of their muscle-activating drug, CK-2127107.

CK-2127107 is a drug that is able to increase the force generated by skeletal muscle and delay the onset and extent of muscle fatigue. Given this ability, it has been suggested that CK-2127107 may improve muscle function and physical performance in diseases causing neuromuscular dysfunction, weakness and fatigue, such as spinal muscular atrophy.

A very similar, but structurally distinct drug, known as Tirasemtiv, was shown by the Cytokinetics team to improve muscle force and resistance to fatigue in SMA model mice.

Five different Phase I trials of CK-2127107 in healthy volunteers have already been conducted by Cytokinetics in association with Astellas. These studies all showed CK-2127107 to be safe and well-tolerated.

This new Phase II trial is designed to assess the effect of multiple oral doses of CK-2127107 on skeletal muscle function in ambulatory and non-ambulatory patients with SMA types II-IV. The principal objective of the study is to obtain information on the pharmacokinetics, safety, and tolerability of the drug in SMA patients.

The trial will be double-blind, randomised, and placebo-controlled, meaning that patients will be randomly allocated to either a placebo or treatment group, and that this will be concealed from the patients and people administering the treatments.

Further Information

Cytokinetics press release

CK-2127107 clinical trial website 

Background on the development of CK-2127107