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Celladon Corporation Announces Publication of Data From Its Stem Cell Factor Gene Therapy Program in Circulation Heart Failure

SAN DIEGO, Oct. 27, 2014 (GLOBE NEWSWIRE) -- Celladon Corporation (Nasdaq:CLDN), a biotechnology industry leader in the field of cardiovascular gene therapy, today announced together with researchers at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai, the results of pre-clinical work on the membrane-bound form of the Stem Cell Factor (mSCF) gene therapy product candidate in the September 2014 online issue of Circulation Heart Failure. The paper is entitled "Stem Cell Factor Gene Therapy Improves Cardiac Function after Myocardial Infarction in Swine".

Stem cell factor (SCF) is a ligand of the c-kit receptor, and is a critical cytokine which contributes to cell migration, proliferation, and survival. It has been shown that SCF expression naturally increases after myocardial infarction (MI) and may be involved in cardiac repair. The aim of this study was to determine whether mSCF gene therapy improves cardiac function in a large animal model of MI-induced heart failure. The study demonstrated clear therapeutic efficacy in the animal model, where mSCF gene therapy, delivered at the ischemic border area in the heart, improved cardiac function, decreased cardiac cell death, and increased the density of blood vessels in the heart for up to three months post-treatment. These results extend the previously reported efficacy data of stem cell factor treatment in rodents to a more clinically relevant model, supporting the potential translation of this gene therapy approach to a functional cardiac regenerative therapy.

"This important research further expands our understanding of the multiple ways mSCF may help improve cardiac function. We are excited by the potential of mSCF gene transfer as a new modality in treating cardiac disease and the possibility of testing this gene therapy in patients," said Dr. Krisztina Zsebo, Chief Executive Officer of Celladon Corporation and author of the study.

"The study results showed increased cardiac performance at the chronic stages of ischemic heart failure and enhance the potential of mSCF gene therapy as a future treatment option for heart failure," said the study's senior investigator Roger J. Hajjar, M.D., Director of the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai and the Arthur & Janet C. Ross Professor of Medicine at Mount Sinai.

About Stem Cells and mSCF Gene Therapy

Stem cell research has demonstrated potential to treat a variety of diseases. Stem Cell Factor exists in both soluble and membrane bound forms. Celladon's current approach with the membrane bound form of Stem Cell Factor gene therapy is to express the cytokine locally at sites of injury and to recruit and expand resident stem cells, in diseases in which stem cells have shown promise in clinical and preclinical testing. In preclinical cardiac applications, mSCF gene therapy has demonstrated a regenerative response characterized by an enhancement in hemodynamic function after injury, an improvement in survival, a reduction in fibrosis, infarct size and cell death, and an increase in cardiac c-kit+ progenitor cells recruitment to the injured area.

About Celladon

Celladon is a clinical-stage biotechnology 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 mSCF 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, statements regarding the future possibility to test mSCF in patients, the potential therapeutic utility in humans based on the animal data and the potential for mSCF to be a future treatment option for heart failure. 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 June 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 Vice President, Corporate Development and Investor Relations +1- 858-432-7215 fwiklund@celladon.com

Source:Celladon Corporation