Cytokinetics Announces Start of Phase II Clinical Trial

Cytokinetics, Inc. recently announced the start of a randomized, double-blind, placebo-controlled, two-period crossover clinical trial designed to assess the effect of CK-2127107 (CK-107) on physical function in patients with chronic obstructive pulmonary disease (COPD). CK-107 is a novel fast skeletal muscle troponin activator, which is being developed as a potential treatment for people living with SMA, COPD, and certain other debilitating diseases and conditions associated with muscular weakness and/or fatigue. Astellas is conducting this Phase II clinical trial in collaboration with Cytokinetics.

“Patients with COPD suffer from significant exercise intolerance due to metabolic abnormalities that produce weakness in limb muscles, as well as an associated switch from slow to fast muscle fiber predominance,” said Fady I. Malik, Cytokinetics’ Executive Vice President and Head of Research & Development. “Given CK-107’s selectivity for fast skeletal muscle fibers, we are enthusiastic about exploring this novel therapeutic strategy in this patient population.”

The trial is expected to enroll approximately 40 patients with COPD in the US and is designed to assess the effect of CK-107 compared to placebo on exercise tolerance. Additionally, the trial will assess cardiopulmonary and neuromuscular effects of CK-107 relative to placebo and the effect of CK-107 on resting spirometry relative to placebo. In addition, the safety, tolerability, and pharmacokinetics of CK-107 will be assessed. Additional information on the trial can be found at clinicaltrials.gov.

COPD is a progressive obstructive lung disease that typically includes emphysema and chronic bronchitis. The disease is increasingly common and is the third leading cause of death in the US behind cancer and heart disease. While it is estimated that more than 14 million US adults have been diagnosed with COPD, it is widely underdiagnosed and up to 24 million Americans have evidence of impaired lung function. Current estimates suggest that COPD costs the nation almost $50 billion annually in both direct and indirect health expenditures. Therapeutic strategies for the treatment of COPD have primarily focused to improving lung function and addressing airflow limitations caused by bronchial obstructions. A substantial unmet need exists for disease management strategies related to improved exercise tolerance.

In preclinical studies, CK-107 has been shown to slow the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibers, thus increasing the sensitivity of the skeletal muscle to calcium, which results in an increase in skeletal muscle force production in response to neuronal input and which also delays the onset and reduces the magnitude of fatigue during repetitive muscle stimulation. Thus, CK-107 may improve muscle function and physical performance in people with COPD. In collaboration with Astellas, Cytokinetics is conducting a Phase II clinical trial of CK-107, which is designed to assess the effect of CK-107 on 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.

Cytokinetics is a late-stage biopharmaceutical company focused on discovering, developing, and commercializing first-in-class muscle activators as potential treatments for debilitating diseases in which muscle performance is compromised and/or declining. As a leader in muscle biology and the mechanics of muscle performance, the company is developing small molecule drug candidates specifically engineered to increase muscle function and contractility. Cytokinetics’ lead drug candidate is tirasemtiv, a fast skeletal muscle troponin activator, for the potential treatment of ALS. Tirasemtiv has been granted orphan drug designation and fast track status by the US FDA and orphan medicinal product designation by the European Medicines Agency for the potential treatment of ALS. Cytokinetics retains the right to develop and commercialize tirasemtiv. Cytokinetics is collaborating with Amgen Inc. to develop omecamtiv mecarbil, a novel cardiac muscle activator, for the potential treatment of heart failure. Cytokinetics is collaborating with Astellas Pharma Inc. to develop CK-2127107, a fast skeletal muscle activator, for the potential treatment of spinal muscular atrophy and chronic obstructive pulmonary disease. Amgen holds an exclusive license worldwide to develop and commercialize omecamtiv mecarbil and Astellas holds an exclusive license worldwide to develop and commercialize CK-2127107. Both licenses are subject to Cytokinetics’ specified development and commercialization participation rights. For more information, visit www.cytokinetics.com.