HF Gene-Therapy Trial CUPID-2 Fails to Meet Primary End Point: Top-Line Results

April 29, 2015

SAN DIEGO, CA — Despite earlier promise in a small dose-finding study, a novel gene-based, single-dose treatment for heart failure failed to make a significant difference in a primary end point reflecting recurrent HF events in a placebo-controlled phase 2b trial with several hundred participants, the sponsor, Celladon, has announced[1].

The study, CUPID-2b, explored intracoronary delivery of a gene tied to myocardial contraction and relaxation through regulation of intracellular calcium, AAV1/SERCA2a (Mydicar), in patients with NYHA 2-4 systolic heart failure despite optimal drug and device therapy.

The therapy seemed to improve symptoms, functional status, and ventricular volumes in the 39-patient first-in-human CUPID study, presented at Heart Failure Congress 2010 and covered then by heartwire from Medscape.

In the CUPID 2b 243-patient intention-to-treat population, the hazard ratio on active therapy for the primary end point, a composite of HF hospitalization or ambulatory treatment for worsening heart failure at 12 months, was 0.93 (95% CI 0.53-1.65, P=0.81).

There were no significant effects for secondary end points including death from any cause, need for a mechanical circulatory support device or heart transplant, and improvement in NYHA class, 6-minute walk test, or quality of life. "No safety issues were noted," according to the company announcement.

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