SAN DIEGO, Nov. 14, 2014 (GLOBE NEWSWIRE) — Celladon Corporation (Nasdaq:CLDN), a clinical-stage biotechnology company with industry-leading expertise in the development of cardiovascular gene therapy, today announced that Krisztina Zsebo, Ph.D., Chief Executive Officer of Celladon, was selected to present three-year long-term follow up data of MYDICAR in patients with advanced heart failure during the “Best of Circulation Research” Symposium at the American Heart Association (AHA) Scientific Sessions 2014 on Sunday, November 16, 2014 in Chicago.
Each year at the scientific sessions, AHA recognizes the most popular studies that appeared in the organization's peer reviewed journal, Circulation Research, and invites them to present at the conference. This year, the article titled “Long-Term Effects of AAV1/SERCA2a Gene Transfer in Patients With Severe Heart Failure: Analysis of Recurrent Cardiovascular Events and Mortality” was awarded this designation and outlines the three-year follow-up data from the CUPID-1 trial which was originally presented at the AHA Scientific Sessions 2013.
In April 2014 MYDICAR was granted Breakthrough Therapy designation by the Center for Biologics Evaluation and Research (CBER) division of the U.S. Food and Drug Administration (FDA) for reducing hospitalizations for heart failure in NYHA class III or IV chronic heart failure patients who are AAV1 neutralizing antibody negative. Breakthrough therapy designation is intended to expedite the development and review of investigational therapies for serious or life-threatening conditions where initial clinical evidence demonstrates a significant improvement over existing therapies.
“The Best of Circulation Research designation is testament to the importance of the CUPID-1 data in the heart failure community and to the novelty of our approach to treating this very serious disease,” said Krisztina Zsebo, Ph.D. “To build upon the data from the CUPID-1 study, we are conducting a 250 patient, CUPID-2 trial, which is now fully enrolled. We expect to report top-line results from this trial in April 2015.”
Results of the 39-patient, Phase 2a CUPID-1 clinical trial of a single intracoronary infusion of MYDICAR in patients with advanced heart failure showed that the high-dose was safe and well tolerated, reduced heart failure-related hospitalizations, and improved patients' symptoms, quality of life and key markers of cardiac function predictive of survival, such as elevated levels of natriuretic peptides and left ventricular end systolic volume. Long-term follow-up results showed that in the additional two-year follow-up period, the durability of reduced cardiovascular and terminal events previously observed in the MYDICAR high-dose cohort at 12 months was maintained. The risk of recurrent cardiovascular events in the presence of terminal events through three years of follow up was reduced by 82 percent in the high-dose group compared with the placebo group (p=0.048). No safety concerns were noted during the three-year follow-up period.
MYDICAR uses gene therapy to selectively target and restore SERCA2a enzyme levels by transferring the SERCA2a gene directly into cardiac muscle cells, which can improve the heart's ability to pump blood. MYDICAR utilizes a non-pathogenic recombinant adeno-associated virus (AAV) and is delivered directly to the heart in a routine outpatient procedure, similar to an angiogram, in a cardiac catheterization laboratory. In April, 2014 MYDICAR received Breakthrough Therapy designation from the FDA.
Celladon is a clinical-stage biotechnology company applying its leadership position in the field of cardiovascular gene therapy to develop novel therapies for diseases with tremendous unmet medical needs. Our lead programs target SERCA enzymes which are a family of enzymes that play an integral part in the regulation of intra-cellular calcium in all human cells. Calcium dysregulation is implicated in a number of important and complex medical conditions and diseases, such as heart failure, vascular disease, diabetes and neurodegenerative diseases. MYDICAR, the Company's most advanced product candidate, uses gene therapy to target SERCA2a, which is an enzyme that becomes deficient in patients with advanced heart failure. Celladon has completed enrollment of a 250 patient Phase 2b clinical trial evaluating the efficacy of MYDICAR in reducing the frequency of, or delaying heart failure-related hospitalizations. This randomized, double-blind, placebo-controlled, multinational trial is evaluating a single intracoronary infusion of MYDICAR versus placebo added to a maximal, optimized heart failure regimen in patients with New York Heart Association class III or IV symptoms of chronic heart failure due to systolic dysfunction. The Company has received Breakthrough Therapy designation from the FDA for this MYDICAR program and expects to report results from the Phase 2b clinical trial in April 2015. In addition, the Company conducts research and development on its membrane-bound form of the Stem Cell Factor gene therapy program for cardiac diseases. Celladon has also identified a number of potential first-in-class compounds addressing novel targets in diabetes and neurodegenerative diseases with its small molecule platform of SERCA2b modulators. For more information, please visit www.celladon.com.
Forward Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, references to the expected timing of CUPID-2 data, the potential impact of Breakthrough Therapy designation for MYDICAR, and statements regarding Celladon's research and development of its product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon Celladon's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with the process of conducting product development activities and clinical trials and obtaining regulatory approval to commercialize product candidates, our reliance on third parties, the need to raise additional funding when needed in order to conduct our business, and the degree of market acceptance of MYDICAR by physicians, patients, third-party payors and others in the medical community. These and other risks and uncertainties are described more fully in Celladon's filings with the Securities and Exchange Commission, including without limitation its Form 10-Q for the quarter ended September 30, 2014. All forward-looking statements contained in this press release speak only as of the date on which they were made. Celladon undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
CONTACT: Fredrik Wiklund
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and Investor Relations