Celladon Corporation and SIRO Clinpharm Announce Presentation at "Statisticians in the Pharmaceutical
Industry International Conference".
Analysis of Recurrent Heart Failure Hospitalizations in Presence of Informative Censoring – Application of Joint
Mumbai, India, May 10, 2013 -
Celladon Corporation, a biopharmaceutical company focused on the discovery and development of innovative treatments for
cardiovascular diseases, announced today that it will present analysis methods for the MYDICAR development program at an upcoming
The conference "Statisticians in the Pharmaceutical Industry International Conference" is being held in Glasgow, UK, on 12-15 May,
"Analysis of Recurrent Heart Failure Hospitalizations in Presence of Informative Censoring" – Application of Joint Frailty Model
by Alex Yaroshinsky (Celladon Corporation); Ladislav Pecen (SIRO Clinpharm); and Alena Cernikova (SIRO Clinpharm)
"This conference presentation represents additional recognition of Celladon's innovative approach to the MYDICAR development
program, and has many benefits over traditional analysis methods including reduced sample size relative to other heart failure
trials," said Krisztina Zsebo Ph.D., President and CEO of Celladon Corp. "It is the first time the U.S Food and Drug
Administration has accepted the use of this statistical analysis method as part of a primary endpoint in a clinical trial for the
treatment of heart failure," continued Dr. Zsebo.
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,
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
II/IV symptoms of HF by reducing the frequency and/or delaying HF-related hospitalizations compared to placebo-treated
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.
"SIRO has been in the clinical research space for close to two decades, and one thing that separates us from competition is our
ability to continuously innovate. Delivering 'solutions simplified' by partnering with our clients is what we are all about." said
Priya Pawar, Global Head, Business Development, SIRO Clinpharm.
About SIRO Clinpharm
SIRO Clinpharm, winner of Frost & Sullivan 'Clinical Research Organization of the year' India Healthcare Award 2012 & 2011, is a
drug development solutions provider to the global healthcare industry. Our subject expertise gives us an edge in clinical trial
management, clinical data management, medical writing, biostatistics and statistical programming pharmacovigilance and clinical
trial supplies management. We offer flexible business models across service verticals based on client needs.