Cytokinetics Granted Orphan Drug Designation for CK-3773274 for the Treatment of Hypertrophic Cardiomyopathy

Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CK-3773274 (CK-274) for the treatment of symptomatic hypertrophic cardiomyopathy (HCM). CK-274 is a next-generation cardiac myosin inhibitor in development for the potential treatment of HCM.

The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation provides a drug developer with certain benefits and incentives, including a seven-year period of U.S. marketing exclusivity from the date of marketing authorization, waiver of FDA user fees, and tax credits for clinical research.

“We’re pleased that CK-274 received orphan drug designation from the FDA for symptomatic HCM encompassing both obstructive and non-obstructive presentations.” said Fady I. Malik, M.D., Ph.D., Cytokinetics’ Executive Vice President of Research & Development. “It’s a promising time to develop investigational medicines for patients with HCM who currently have no FDA approved medical therapy available to treat the underlying hypercontractility associated with their disease. We believe treatment with CK-274 may represent a novel approach that may significantly impact patient function and quality of life. We look forward to results from REDWOOD-HCM, our ongoing Phase 2 clinical trial, expected mid-year.”

About CK-274

CK-274 is a novel, oral, small molecule cardiac myosin inhibitor arising from an extensive chemical optimization program conducted with careful attention to therapeutic index and pharmacokinetic properties that may translate into next-in-class potential in clinical development. CK-274 was designed to reduce the hypercontractility that is associated with hypertrophic cardiomyopathy (HCM). In preclinical models, CK-274 reduces myocardial contractility by binding directly to cardiac myosin at a distinct and selective allosteric binding site, thereby preventing myosin from entering a force producing state. CK-274 reduces the number of active actin-myosin cross bridges during each cardiac cycle and consequently reduces myocardial contractility. This mechanism of action may be therapeutically effective in conditions characterized by excessive hypercontractility, such as HCM.

In preclinical models of cardiac function, CK-274 reduced cardiac contractility in a predictable dose and exposure dependent fashion. In preclinical models of disease, CK-274 reduced compensatory cardiac hypertrophy and cardiac fibrosis. The preclinical pharmacokinetics of CK-274 were characterized, evaluated and optimized for potential ease of titration in the clinical setting.

About REDWOOD-HCM: Clinical Trial Design

REDWOOD-HCM is a multi-center, randomized, placebo-controlled, double-blind, dose finding clinical trial of CK-274 in patients with symptomatic obstructive HCM (oHCM). The primary objective of the trial is to determine the safety and tolerability of CK-274. The secondary objectives are to describe the concentration-response relationship of CK-274 on the resting and post-Valsalva left ventricular outflow tract gradient as measured by echocardiography during 10 weeks of treatment, to describe the dose response relationship of CK-274, and to evaluate the plasma concentrations of CK-274 in patients with oHCM.

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