Cytokinetics, Inc. (NASDAQ:CYTK) announced that its first Phase 2 clinical trial of CK-2127107, a novel fast skeletal muscle troponin activator, in patients with spinal muscular atrophy (SMA) has opened to enrollment. The clinical trial is designed to assess the effect of CK-2127107 on multiple measures of muscle function in both ambulatory and non-ambulatory patients with SMA, a severe, genetic neuromuscular disease that leads to debilitating muscle function and progressive, often fatal, muscle weakness. In collaboration with Astellas (Tokyo Stock Exchange: 4503, “Astellas”),Cytokinetics is developing CK-2127107 as a potential treatment for people living with SMA and certain other debilitating neuromuscular and non-neuromuscular diseases and conditions associated with skeletal muscle weakness and/or fatigue.

The primary objective of this double-blind, randomized, placebo-controlled clinical trial is to determine the potential pharmacodynamic effects of a suspension formulation of CK-2127107 following multiple oral doses in patients with Type II, Type III, or Type IV SMA. Secondary objectives are to evaluate the safety, tolerability and pharmacokinetics of CK-2127107. The trial will enroll seventy-two patients in two sequential, ascending dose cohorts (two cohorts of 36 patients each, half ambulatory and half non-ambulatory). Each cohort will be stratified by ambulatory versus non-ambulatory status to receive CK-2127107 dosed twice daily for 8 weeks.

“Initiating this first Phase 2 trial of CK-2127107 represents a major step forward given our interests to serve the many adolescents and adults who are living with SMA, a disorder with few treatment options,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “We look forward to working closely with the investigators and clinical trial sites to evaluate the effects of our next-generation skeletal muscle activator, which we believe holds promise for the potential treatment of patients battling this devastating disease.”

Clinical Trial Design

In this trial, both of the planned ascending dose cohorts will enroll 18 ambulatory (Type III or Type IV) and 18 non-ambulatory patients (Type II or Type III) who are 12 years of age and older and will randomize them 2:1 to receive CK-2127107 or placebo, stratified by ambulatory versus non-ambulatory status.  The first cohort of patients will receive 150 mg of CK-2127107 dosed twice daily for eight weeks; the second cohort of patients will receive 450 mg of CK-2127107 dosed twice daily or a lower dose, depending on the data from the first cohort. At the conclusion of the trial, approximately 24 patients will have been randomized to placebo, approximately 24 patients to 150 mg of CK-2127107 twice daily and approximately 24 patients to 450 mg of CK-2127107 twice daily (or a lower dose, pending the review of data from the first cohort). Multiple assessments of skeletal muscle function and fatigability will be performed including respiratory assessments, upper limb strength and functionality for non-ambulatory patients, as well as six-minute walk and timed-up-and-go for ambulatory patients.  Additional information can be found at (Original Source)

Shares of Cytokinetics closed last Thursday at $10.46, down $0.05 or -0.48%. CYTK has a 1-year high of $13.18 and a 1-year low of $5.50. The stock’s 50-day moving average is $11.29 and its 200-day moving average is $8.20.

On the ratings front, Cytokinetics has been the subject of a number of recent research reports. In a report issued on December 16, Piper Jaffray analyst Charles Duncan reiterated a Buy rating on CYTK, with a price target of $24, which implies an upside of 129.4% from current levels. Separately, on November 9, Roth Capital’s Joseph Pantginis reiterated a Buy rating on the stock and has a price target of $22.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Charles Duncan and Joseph Pantginis have a total average return of 5.0% and -5.2% respectively. Duncan has a success rate of 44.7% and is ranked #753 out of 3648 analysts, while Pantginis has a success rate of 38.2% and is ranked #3571.

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