Pompey, France, and San Diego, CA, USA, March 24, 2015 - Novasep, a leading supplier of services and technologies for the life sciences and chemical industries and Celladon Corporation (Nasdaq:CLDN), a clinical-stage cardiovascular gene therapy company, today announce that they have signed a Development, Manufacturing and Supply Agreement pursuant to which, if supported by upcoming MYDICAR clinical data, Novasep would manufacture MYDICAR drug substance through 2018 with extension options through 2020.
The contract follows an earlier letter agreement concerning the initial process transfer of MYDICAR and pre-validation studies, which was signed in December 2014. The new agreement continues the work necessary for Novasep to achieve GMP production of MYDICAR drug substance.
MYDICAR is an innovative, genetically-targeted enzyme replacement therapy based on AAV1/SERCA2a that is being developed for advanced heart failure and has been granted Breakthrough Therapy Designation by the US Food and Drug Administration (FDA). MYDICAR is currently being tested in human clinical trials, including a Phase IIb study in the USA and Europe referred to as CUPID2, the top-line results of which are expected to be announced in late April 2015.
“We are very pleased to have the option to partner with Novasep to prepare for and commence commercial manufacturing of MYDICAR drug substance,” said Dr. Krisztina Zsebo, CEO of Celladon. “Novasep has demonstrated technical excellence and a real commitment to helping advance the MYDICAR program.”
“This agreement validates the trusted relationship Novasep has established with Celladon,” said Andrew Brennan, Novasep General Manager, US Operations. “The relationship represents a partnership in every sense of the word and, I believe, is a model for future development and commercial supply agreements in the fast growing gene therapy space.”
Under the terms of the agreement, Novasep and Celladon would conduct the process transfer, development, facility retrofitting and scale-up activities necessary to carry out production of MYDICAR drug substance in a 2000L bioreactor at an enhanced bioproduction plant in Seneffe, Belgium. GMP operations are planned to start in Q4 2016 and continue through 2018. An option to extend through 2020 will be determined based on Celladon’s requirements for the MYDICAR drug substance. Celladon has the right to terminate the agreement, exercisable for a specified period of time following the un-blinding of the data from the CUPID2 study, if Celladon concludes in good faith that the CUPID2 data is such that Celladon does not require production of MYDICAR drug substance at the Novasep facility.
“Celladon has impressed us with their strategic approach in building a robust supply chain for a challenging biopharmaceutical. Celladon is preparing for potential fast track development, which makes Novasep’s technical expertise and dynamic business processes an excellent match.” said Michel Spagnol, Chairman of the Board and CEO of Novasep. “We are thrilled to be part of a team that is striving to enable the treatment of many patients suffering serious heart disease by Celladon’s innovative viral vector based therapy.”
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. Celladon’s 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’s CUPID2 trial is 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, known as HFrEF. 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 late 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
Novasep is a global provider of cost-effective and sustainable manufacturing solutions for life sciences molecules and fine chemicals. Novasep's unique offering includes process development services, purification equipment and turnkey processes, contract manufacturing services and complex active molecules to serve pharmaceutical, biopharmaceutical, fine chemical, food and functional ingredients as well as fermentation and chemical commodities industries.
Celladon 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 potential future GMP production and commercial supply of MYDICAR drug substance at the Novasep facility; the expected timing of CUPID2 data; the impact of Breakthrough Therapy designation on MYDICAR development and the potential for a fast-track development schedule; and Celladon’s decision and ability to proceed under the agreement following the un-blinding of CUPID2 data and commit to a long-term supply arrangement with Novasep. 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, Celladon’s reliance on third parties, the need to raise additional funding when needed in order to conduct our business, and the degree of future 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.