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Celladon Corporation Announces In-License of Stem Cell Factor Development Program

21 / 07 / 2014

SAN DIEGO, July [21], 2014 (GLOBE NEWSWIRE) -- Celladon Corporation (Nasdaq:CLDN), a clinical-stage biotechnology company applying its leadership position in the field of gene therapy and calcium dysregulation today announced an exclusive, global license from Enterprise Partners Venture Capital for gene therapy applications of the membrane-bound form of the Stem Cell Factor gene (mSCF) for treatment of cardiac ischemia. Stem Cell Factor is a critical cytokine which contributes to cell migration, proliferation, and survival of cardiac stem cells.


Researchers at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai have

successfully tested mSCF gene therapy to reverse heart damage following myocardial infarction in animal models. These results were recently published in Circulation Research and are available on the Company’s website "mSCF gene therapy promoted a regenerative response in damaged hearts similar to that observed with stem cell therapy and may be one of the first non-cell therapies to increase cardiac muscle precursors in the heart," 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. "We are excited by the future possibility of testing this gene therapy in patients."


Celladon plans to commence further preclinical work, building on available data from Mount Sinai in myocardial infarction. “We believe mSCF gene therapy has the potential to be a powerful therapeutic approach, harnessing the potency of stem cell therapy without the associated complications of developing cells as products,” said Krisztina Zsebo, Ph.D., Chief Executive Officer of Celladon. “We're delighted that this promising program will be taken forward by Celladon, leveraging our extensive gene therapy research and product development experience. Our initial focus will be to generate clinically acceptable gene therapy vectors in support of potentially conducting a future clinical trial in patients who have suffered cardiac damage, as well as exploration for potential other applications.”


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 planned 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 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 company applying its leadership position in the field of gene therapy and calcium dysregulation 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 heart failure. Celladon recently completed enrollment of a 250 patient Phase 2b 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 trial in April 2015. In addition, Celladon has 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.

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Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forwardlooking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements regarding mSCF’s potential as one of the first non-cell therapies to increase cardiac muscle precursors in the heart, the future possibility to test mSCF in patients, references to mSCF’s potential therapeutic effect and believed benefits over other approaches, as well as statements regarding Celladon’s current plans for the program. 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 ourbusiness, 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 March 31, 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.


For further information, please contact:

Fredrik Wiklund

Vice President, Corporate Development and Investor Relations

+1- 858-432-7215