Cytokinetics Receives Orphan Drug Designation from FDA for CK-274 in the Treatment of Hypertrophic Cardiomyopathy

Strategic Insights -

Cytokinetics, a late-stage biopharmaceutical company, announced that the U.S. Food and Drug Administration has granted orphan drug designation to CK-274, a next-generation cardiac myosin inhibitor, for the treatment of symptomatic hypertrophic cardiomyopathy (HCM). This designation provides Cytokinetics with benefits such as 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. CK-274 is currently being investigated in the Phase 2 clinical trial, REDWOOD-HCM, which aims to evaluate the safety and efficacy of the treatment in patients with symptomatic obstructive HCM.

Key Takeaways:

  • Cytokinetics' CK-274 has received orphan drug designation from the FDA for the treatment of symptomatic hypertrophic cardiomyopathy (HCM).
  • The FDA 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 benefits such as 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.
  • CK-274 is currently being investigated in the Phase 2 clinical trial, REDWOOD-HCM, which aims to evaluate the safety and efficacy of the treatment in patients with symptomatic obstructive HCM.
  • REDWOOD-HCM is a multi-center, randomized, placebo-controlled, double-blind, dose finding clinical trial of CK-274 in patients with symptomatic obstructive HCM.
  • The primary objective of the trial is to determine the safety and tolerability of CK-274, while secondary objectives include describing 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.
  • Interim analysis of data from Cohort 1 of REDWOOD-HCM showed patients experienced substantial reductions in the average resting left ventricular outflow tract gradient (LVOT-G) as well as the post-Valsalva LVOT-G.

Statistics:

  • Approximately 20 million Americans suffer from HCM, a condition that can lead to heart failure, stroke, and death.
  • HCM affects approximately 1 in 500 people in the United States.
  • CK-274 is a next-generation cardiac myosin inhibitor being developed for the potential treatment of HCM.
  • REDWOOD-HCM is expected to enroll patients in approximately 20 investigative sites in North America and Europe.
  • Enrollment in Cohort 2 of REDWOOD-HCM is expected to complete in Q1 2021.

Sources:

  • Cytokinetics, Incorporated. (2021, January 11). UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934.
  • Cytokinetics. (2021). REDWOOD-HCM: Clinical Trial Design.
  • Cytokinetics. (2021). CK-274: A Novel, Oral, Small Molecule Cardiac Myosin Inhibitor.
  • Food and Drug Administration. (2021). Orphan Drug Designation.