Celladon announces completion of patient enrollment for Phase 2 CUPID clinical trial

Aug 31 2009

Celladon Corporation and Momentum Research, Inc. today announced completion of enrollment of the phase 2 double-blind portion of the Calcium Up-regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID) trial. Momentum Research is Celladon’s consulting and management organization with clinical expertise in the development of cardiovascular products and trials.

“Strong interest in MYDICAR® from investigators and patients emphasizes the substantial unmet medical need in these very sick patients with few other viable options. Patients will be observed for 6 months, and we expect Interim data results to be available mid-2010,” said Krisztina M. Zsebo, Ph.D., president and chief executive officer. “We remain encouraged by the meaningful improvements in cardiac function and overall condition of patients; findings that we believe demonstrate the return toward normal intracellular calcium cycling and contractility in some of the heart muscle cells.”

“The CUPID trial is designed to rescue a failing heart by replacing an enzyme known to play a critical role in healthy cardiac function,” said Mariell Jessup, M.D., Lead Investigator, who works at the University of Pennsylvania. “The objective of the study is not only to improve the symptoms of heart failure but to reverse the severity of the disease in individual patients. The extraordinary team of clinical investigators, Celladon and Momentum Research have made this challenging concept a reality.”

The randomized, double-blind, placebo-controlled study is designed to examine the effect of MYDICAR® (AAV1/SERCA2a) in the treatment of severe heart failure. MYDICAR® is an enzyme-replacement therapy intended to restore levels of SERCA2a, a protein that is vital in the proper functioning of the heart. The CUPID trial enrolled 37 patients with severe forms of ischemic and dilated cardiomyopathies who had New York Heart Association Class III or IV heart failure, significantly impaired pumping function of their hearts, and less than half the normal ability to transport and utilize oxygen during cardiopulmonary exercise testing. Patients were treated with 1 of 3 doses of MYDICAR® or placebo via a single intracoronary infusion and will be followed for 12 months. Effects of treatment will be assessed by changes in how the heart contracts, a blood test of an important marker of heart failure called NT-proBNP, symptoms of heart failure and ability to exercise.

Zsebo adds, “In addition, we have adequate MYDICAR® product manufactured to complete phase 3 and recently acquired an exclusive license to utilize Adeno-Associated Viral (AAV) vector technology in heart failure. This, combined with our extensive preclinical and clinical investigation of gene-based therapy for heart failure, bodes well for commercial product development of MYDICAR® and is important to potential strategic partners.”

http://www.momentum-research.com/