Cytokinetics Announces Opening of a Phase IIa "Evidence of Effect" Clinical Trial of CK-2017357 in Patients with Myasthenia Gravis
News Jan 04, 2011
CK-2017357 is a fast skeletal muscle troponin activator and is the lead drug candidate that has emerged from the company's skeletal muscle contractility program. CK-2017357 selectively activates the fast skeletal muscle troponin complex and increases its sensitivity to calcium, which increases skeletal muscle force in response to neuronal input and also delays the onset and reduces the degree of muscle fatigue. In July 2010, Cytokinetics was awarded a grant in the amount of $2.8 million by the National Institute of Neurological Disorders and Stroke to support research and development of CK-2017357 in MG.
This Phase IIa EoE clinical trial is a double-blind, randomized, three-period crossover, placebo-controlled, pharmacokinetic (PK) and pharmacodynamic (PD) study of CK-2017357 in patients with generalized MG. At least 36 and up to 78 patients may be enrolled at approximately 15 study centers in the United States. Patients enrolled in the trial will receive single oral doses of placebo, 250 mg, and 500 mg of CK-2017357 in random order. A wash-out period of at least 7 days (to a maximum of 10 days) will be employed between the individual doses for each patient.
The primary objective of this hypothesis-generating clinical trial is to assess the effects of CK-2017357 on measures of muscle strength, muscle fatigue and pulmonary function utilizing the standardized Quantitative MG score, Manual Muscle Test, and MG Composite score. The secondary objectives of this clinical trial are to evaluate and characterize the relationship, if any, between the doses and plasma concentrations of CK-2017357 and its PD effects; to evaluate the safety and tolerability of CK-2017357 administered as single doses to patients with MG; and to evaluate the effect of CK-2017357 on investigator and patient determined global functional assessment and the Modified MG Symptom Score.
"This hypothesis-generating clinical trial is designed to seek evidence of a pharmacodynamic effect of CK-2017357 to increase skeletal muscle performance or to delay the onset and reduce the magnitude of muscle fatigue in patients with generalized myasthenia gravis. Based on non-clinical and clinical results generated to date, we believe that we may be able to demonstrate potentially clinically relevant pharmacodynamic effects of CK-2017357, even after a single dose," stated Fady Malik, MD, PhD, FACC, Cytokinetics' Vice President of Biology and Therapeutics. "Recent data presented from a similarly-designed Phase IIa clinical trial in patients with amyotrophic lateral sclerosis (ALS) provided encouraging signals of potential efficacy in a disease in which neuromuscular signaling is impaired, thereby underscoring a possible role for this drug candidate in patients with other neuromuscular conditions such as myasthenia gravis."