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Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A) | ||
By: Nasdaq / GlobeNewswire - 14 Feb 2019 | Back to overview list |
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-- Trial to assess efficacy on neurodevelopmental status of MPS IIIA patients -- CAMBRIDGE, Mass. and PARIS, Feb. 14, 2019 (GLOBE NEWSWIRE) -- Lysogene (FR0013233475 – LYS), a pioneering biopharmaceutical company specializing in gene therapy targeting central nervous system (CNS) diseases, and Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that the first patient has been dosed in AAVance, a global Phase 2-3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA). AAVance is a single-arm trial aimed at evaluating the effectiveness of a one-time delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase (SGSH) gene. MPS IIIA is caused by mutations in the SGSH gene, which is involved in producing an enzyme necessary for the breakdown and disposal of long chain sugar molecules. LAF-SAF302 is intended to deliver a functional copy of the SGSH gene and allow the brain to secrete the missing enzyme. The goal of the trial is to show improved or stabilized neurodevelopmental status of MPS IIIA patients. The trial will enroll 20 patients at eight sites in the U.S. and Europe. More information can be found on www.clinicaltrials.gov. “The first patient dosed in the AAVance trial is an important step in addressing this relentlessly progressing neurodegenerative disease. Our aim is to stabilize or improve the clinical status of patients with MPS IIIA by providing a permanent source of functional enzyme in the brain,” said Karen Aiach, Founder and Chief Executive Officer of Lysogene. “MPS IIIA is a lethal neurological disease with debilitating symptoms for which there is currently no approved treatment,” said Principal Investigator Dr. Ronald Crystal, Chairman of the Department of Genetic Medicine and the Bruce Webster Professor of Internal Medicine at Weill Cornell Medicine, and a physician at NewYork-Presbyterian/Weill Cornell Medical Center, who is a paid consultant for Lysogene. “It is a great motivation to know that the work we are doing here has the potential to make a life-changing difference to so many unfortunate children, and I am very much looking forward to further advancing this innovative therapy.” “Every day is an opportunity to make progress in bringing transformative treatments to patients and today’s milestone is an important advancement toward that goal,” said Doug Ingram, President and Chief Executive Officer, Sarepta. “Sarepta is committed to working with Lysogene to advance this program with the greatest urgency on behalf of patients.” In accordance with the Worldwide License and Collaboration Agreement signed between Lysogene and Sarepta, the dosing of the first patient with LYS-SAF302 in a Phase 2-3 clinical trial triggers milestone payments of USD18 million from Sarepta to Lysogene. About MPS IIIA About LYS-SAF302 About Sarepta Therapeutics About Lysogene Sarepta Forward-Looking Statements These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: the expected benefits and opportunities related to the agreement with Lysogene may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development; in particular, the agreement may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement, the results of research may not be consistent with past results or may not be positive or may otherwise fail to meet regulatory approval requirements for the safety and efficacy of product candidates, possible limitations of Company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sarepta’s product candidates; and even if commercialization of any product under the agreement is achieved, this may not result in any significant revenues to the parties; if the actual number of patients suffering from MPS IIIA is smaller than estimated, Sarepta’s revenue and ability to achieve profitability may be adversely affected; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2017 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's 2017 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the SEC as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us. Source: Sarepta Therapeutics, Inc. Contacts Sarepta Therapeutics, Inc.: Investors: Lysogene: |
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