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Amicus Therapeutics Acquires Gene Therapy Portfolio of Ten Clinical and Pre-Clinical Stage AAV Programs in Neurologic Lysosomal Storage DisordersLicense with Nationwide Children’s Hospital (NCH) through the Acquisition of Celenex (NCH Spinout) Leverages Amicus Therapeutics Expertise in Global Development and Delivery of Novel Therapies for Rare Metabolic Disorders with Nationwide Children’s AAV Gene Therapy Technologies Conference Call and Webcast Scheduled for Today, September 20, 2018 at 8:00am ET CRANBURY, N.J., Sept. 20, 2018 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced the signing of a definitive agreement in which Amicus Therapeutics will receive worldwide development and commercial rights for ten gene therapy programs developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University. The ten programs are licensed to Amicus from Nationwide Children’s Hospital through the acquisition of Celenex, a private, clinical stage gene therapy company. The lead programs in CLN6, CLN3, and CLN8 Batten disease are potential first-to-market curative therapies for these rare, devastating diseases. Batten disease, also known as Neuronal Ceroid Lipofuscinosis (NCL), is a family of rare disorders that can be life-threatening and debilitating, with high unmet medical need. More detail about these programs will be presented on this morning’s conference call. The slides for this conference call are available on our website. “The in-licensing and acquisition of these gene therapy programs provides an extraordinary opportunity to transform the lives of thousands of children living with some of the most devastating forms of lysosomal storage disorders, for which there are virtually no treatment options today,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics. “The groundbreaking work of Drs. Brian Kaspar and Kathrin Meyer at Nationwide Children’s Hospital, along with collaborator, Arthur Burghes, Ph.D., professor at The Ohio State University, on these programs has led to remarkably strong and consistent pre-clinical results and now, in CLN6 Batten disease, encouraging early results in children. This is science and biotechnology at its best. And it has at its core the love, drive and passion of two remarkable parents, Gordon and Kristen Gray, who moved heaven and earth to partner with these researchers to advance these potentially life-saving medicines for their daughters and now for many thousands more. I am honored that they and their research team have chosen to entrust these ten programs to the passionate team of scientists and entrepreneurs at Amicus. I cannot think of a better foundation for Amicus’ entry into gene therapies.” All acquired programs leverage intrathecal delivery, using the same AAV vector approach utilized successfully in clinical trials across other rare CNS indications, such as SMA. This approach and technology are considered to be a clinically validated gene delivery platform for diseases of the central nervous system (CNS). Brian Kaspar, Ph.D., co-founder of Celenex, and Kathrin Meyer, Ph.D., a Principal Investigator at Nationwide Children’s Hospital Center for Gene Therapy, will continue to support these programs as scientific advisors to Amicus Therapeutics. “The preclinical proof-of-concept we have seen to date in CLN6, CLN3, and CLN8 further support the applicability of the AAV vector we developed at Nationwide Children’s in gentic disease of the CNS,” said Kathrin Meyer, Ph.D. “I firmly believe that Amicus is the optimal scientific and clinical partner to advance these programs and look forward to actively collaborating with the Amicus team on the development of these important potential therapies and getting them to as many children as quickly as possible. They truly have the potential to transform lives.” Deal Terms Celenex shareholders are also eligible for up to $15 million in development milestones and $262 million in BLA/MAA submission and approval milestones across multiple programs. Amicus expects to pay no more than $75 million over the next 4 years in these milestones. No royalties are owed to Celenex for any of these programs. Celenex shareholders may also be eligible for up to $75 million in tiered sales ($500 million/$750 million) milestone payments. The acquisition and several years of related development costs for all of these programs will be financed through a new $150 million debt facility provided by BioPharma Credit PLC, an investment fund managed by Pharmakon Advisors, L.P. Skadden, Arps, Slate, Meagher & Flom LLP acted as legal counsel to Amicus Therapeutics on the transaction. RBC Capital Markets acted as exclusive financial advisor and Fenwick & West LLP acted as exclusive legal counsel to Celenex on the transaction. The transaction was approved by the Board of Directors of both companies and closed immediately. Debt Facility Conference Call and Webcast An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/, and will be archived for 30 days. Web participants are encouraged to go to the website 15 minutes prior to the start of the call to register, download, and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET on September 20, 2018. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); conference ID: 7084639. About Batten Disease About Amicus Therapeutics Forward Looking Statement CONTACTS: Investors: Media / Multimedia Assets: FOLD–G |