SAN DIEGO, Aug. 29, 2012 /PRNewswire/ -- Celladon Corporation, a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, announced today that it has dosed the first patient in a Phase 2b clinical trial of MYDICAR, Celladon's first in class therapeutic for the treatment of advanced heart failure (HF). Chronic HF is a leading cause of hospitalization and resulted in direct and indirect costs of $39.2 billion to the U.S. healthcare system in 2010. Nearly 6 million people in the U.S. have HF and at least 670,000 new cases are diagnosed yearly. Heart failure leads to an estimated 280,000 deaths annually. There is no cure.
The Phase 2b study titled "Calcium Up-Regulation by Percutaneous Administration of Gene Therapy In Cardiac Disease" ("CUPID Phase 2b Trial") is a multinational, multicenter, double-blind, placebo-controlled, randomized study of a single intracoronary administration of 1 x 1013 DRP MYDICAR versus placebo added to an optimal HF regimen. The primary objective is to determine the efficacy of MYDICAR in patients with ischemic or dilated cardiomyopathy and NYHA class III/IV symptoms of HF by reducing the frequency and/or delaying HF-related hospitalizations compared to placebo-treated patients. Secondary objectives will include assessment of the safety of MYDICAR by determining the incidence and severity of adverse events and changes in laboratory parameters. The trial will enroll approximately 200 patients in up to 50 international sites. Results are anticipated in the first half of 2015.
The Chairman of the Executive Clinical Steering Committee of the CUPID Phase 2b Trial is Barry H. Greenberg M.D., FACC, Director, Advanced Heart Failure Treatment Program; Professor of Medicine, University of California, San Diego. Dr. Greenberg stated, "We are enthusiastically looking forward to evaluating MYDICAR in the CUPID Phase 2b Trial for advanced heart failure. There is a major unmet need to provide safe and effective therapies for patients with advanced heart failure such as the ones who we will be studying in this trial."
"With the encouraging results from the preceding CUPID Phase 2 trial and the timely dosing of the first patient in this Phase 2b trial, we are firmly on track with our plan to develop a novel, safe, and effective therapy for patients with advanced heart failure," said Krisztina Zsebo Ph.D., President and CEO of Celladon Corp.
The first patient was dosed at Sharp Memorial Hospital in San Diego, CA by Brian Jaski, M.D., FACC, and Scientific Director of Research at the San Diego Cardiac Center. "We are grateful to be able to collaborate with Celladon for new innovative therapies for patients with serious heart failure. Heart failure is an increasing medical problem with no major breakthrough in medication therapy in over twenty years. Thus, there exists a large unmet need to help these patients." noted Dr. Jaski.
About the CUPID Phase 2b Trial
The CUPID Phase 2b trial will enroll approximately 200 patients in up to 50 sites worldwide. Patients will first be prescreened for the presence of AAV neutralizing antibodies. Those patients with a negative titer will undergo further screening tests and procedures to determine eligibility prior to randomization and enrollment into the study. All patients will be randomized in parallel to MYDICAR or placebo in a ratio of 1:1 (1 x 1013 DRP MYDICAR to placebo).
The primary objective is to determine the efficacy of MYDICAR in patients with ischemic or dilated cardiomyopathy and NYHA class III/IV symptoms of HF by reducing the frequency and/or delaying HF-related hospitalizations compared to placebo-treated patients.
The primary efficacy endpoint is time-to-recurrent HF-related hospitalizations in the presence of terminal events (all-cause death, heart transplant, LVAD implantation). The secondary efficacy endpoint is the time-to-terminal event (all-cause death, heart transplant, LVAD implantation). Exploratory endpoints include change from baseline in NYHA class, 6 minute walk test distance, and quality of life (KCCQ) score.
Secondary objectives will include assessment of the safety of MYDICAR by determining the incidence and severity of adverse events and changes in laboratory parameters. Safety evaluations include the incidence and severity of all adverse events (including procedure-related), summaries of concomitant medications, vital signs, physical exams, implantable cardioverter defibrillator (ICD) interrogations and laboratory parameters, and the time to cardiovascular-related death.
MYDICAR is a genetically targeted enzyme replacement therapy intended to restore levels of SERCA2a, a regulator of calcium cycling in the heart and cardiac contractility. SERCA2a levels decline in all forms of late-stage HF resulting in deficient heart function. With MYDICAR, the SERCA2a gene is delivered using recombinant adeno-associated virus (AAV) as the vector. AAV is a naturally occurring virus not associated with any disease in humans. MYDICAR is delivered in a single dose directly to the heart during a routine outpatient cardiac catheterization procedure, similar to an angiogram. MYDICAR is synergistic and additive across current HF treatments such as ACE inhibitors, beta-blockers, sprinolactone/diuretics, and biventricular pacing devices. No treatment substitution decision is required by the treating physician.
SOURCE Celladon Corporation