Company Overview of Celladon Corporation

Celladon Corporation Presents at BioCentury Future Leaders in the Biotech Industry 2013, Apr-05-2013 10:00 AM. Venue: Millennium Broadway Hotel & Conference Center, New York, New York, United States. Speakers: Krisztina M. Zsebo, Chief Executive Officer, President and Director.

Celladon Corporation Presents at The BIOCOM 3rd Annual Global Life Science Partnering Conference, Feb-27-2013 . Venue: The Lodge at Torrey Pines, 11480 North Torrey Pines Road, La Jolla, CA 92037, United States.

Celladon Corporation announced 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, the company is 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. Celladon Corporation has received investment capital supporting the ongoing clinical trial from an international investor syndicate including Lundebeckfond Ventures, the venture investment arm of the Lundbeck Foundation in Copenhagen, Denmark. 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.