Celladon Corporation Announces First European Patient Dosed in CUPID Phase 2b Trial of MYDICAR® for Advanced Heart Failure

Celladon Corporation Announces First European Patient Dosed in CUPID Phase 2b
                 Trial of MYDICAR® for Advanced Heart Failure

  PR Newswire

  SAN DIEGO, Dec. 11, 2012

SAN DIEGO, Dec. 11, 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 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 10^13 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 10^13 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.

About MYDICAR

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

About Celladon

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
.

Website: http://www.celladon.net
Contact: Fredrik Wiklund, Head of Corporate Development, +1-858-432-7215,
fwiklund@celladon.net
 
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