• Amicus will make an upfront cash payment of $100 million
• The company also announced closing a $150 million term loan to help finance the acquisition and development costs
• Lead programs are for three forms of Batten disease

Amicus Therapeutics (Nasdaq: FOLD) announced on 9/20/18 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.

“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.

Under the terms of the agreement, Amicus will pay $100 million in an upfront cash payment to acquire all of these assets.

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.
 
Amicus Therapeutics also announced it has closed a five-year, senior credit facility with BioPharma Credit. The new credit facility consists of a $150 million non-dilutive term loan, which requires interest-only payments through 2022 and matures in 2023. Interest will accrue at a floating rate of LIBOR plus 7.5%, subject to a floor and ceiling on the rate. There are no warrants or any equity conversion features associated with the loan. The proceeds from this financing will be used to support the cost of the acquisition and several years of related development costs.

It is estimated that 2-4 births per 100,000 in the U.S. are affected by Batten disease, though some researchers in the field suggest these numbers are low. There are 14 known forms of Batten disease.