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 European patient in its ongoing international Phase 2b clinical trial of MYDICAR, Celladon's first in class therapeutic for the treatment of advanced heart failure (HF).
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 first patient was dosed in August, 2012 in the United States and the trial will enroll approximately 200 patients in up to 50 international sites.
The first European patient was dosed at The Heart Centre in Rigshospitalet University Hospital in Copenhagen, Denmark by Professor Jens Kastrup MD, DMSc, FESC. “The development of both ischemic and non-ischemic heart failure in patients is an increasing medical problem and there has been no major breakthrough in medication therapy for several years. Therefore, we are constantly looking for new treatment modalities for these patients. The established collaboration with Celladon and the investigation of the new innovative therapy MYDICAR for these patients with serious heart failure is very promising,” noted Dr. Kastrup.
“We are pleased to dose our first patient with MYDICAR outside of the United States and hereby initiating an enduring and imperative collaboration with our European clinical investigators. This is an important step in our development program which aims to provide a novel, safe, and highly effective therapeutic option to advanced heart failure patients worldwide,” said Krisztina Zsebo Ph.D., President and CEO of Celladon Corp.
Celladon Corporation has received investment capital supporting the ongoing clinical trial from an international investor syndicate including Lundbeckfond Ventures, the venture investment arm of the Lundbeck Foundation in Copenhagen, Denmark.
About the CUPID Phase 2b Trial
The CUPID Phase 2b trial was initiated in August 2012 and 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. A recent Phase 2 clinical trial demonstrated sustained improvement at one year in cardiac function parameters and quality of life. A 200 patient Phase 2b study of MYDICAR was initiated in August, 2012
Celladon is a privately held biotechnology company founded with the goal of becoming the leader in developing molecular therapies for the treatment of heart failure and cardiac diseases. The company's lead product, MYDICAR, targets the key enzyme deficiency in advanced heart failure, SERCA2a, which regulates calcium cycling and contractility in heart muscle cells. Celladon also conducts pre-clinical research on a proprietary platform of small molecule activators of SERCA enzymes for the treatment of metabolic and cardiovascular diseases. Further information can be found at www.celladon.net
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