Rare Daily Staff

Amicus Therapeutics agreed to acquire Celenex in a deal that provides it worldwide development and commercial rights for ten gene therapy programs for $100 million upfront and up to $277 million in milestone payments.

Through the acquisition, Amicus gains rights to ten programs developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University.

The lead programs in CLN6, CLN3, and CLN8 Batten disease are potential first-to-market curative therapies for these fatal, genetic diseases. Batten disease is a family of rare disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. In these diseases, a defect in a specific gene triggers a cascade of problems that interferes with a cell’s ability to recycle certain molecules.

The disease has several forms with similar features and symptoms but vary in severity and age of onset. The first symptom is usually progressive vision loss in previously healthy children followed by personality changes, behavioral problems, and slow learning. Seizures appear within 2-4 years of vision loss. Seizures and psychosis can appear at any time. Patients experience progressive loss of motor functions and become wheelchair-bound, are bedridden, and die prematurely.

“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 Crowley, chairman and CEO of Amicus Therapeutics.

The company said the acquired programs leverage intrathecal delivery, using the same vector to carry the gene therapy utilized successfully in clinical trials across other rare neurological indications. This approach and technology are considered to be a clinically validated gene delivery platform for diseases of the central nervous system.

Under the terms of the agreement, Amicus will pay $100 million in an upfront cash payment to acquire all of Celenex’s assets. Celenex shareholders are also eligible for up to $15 million in development milestones and $262 million in 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. Amicus said it will finance the acquisition and several years of related development costs for all of these programs through a new $150 million debt facility provided by BioPharma Credit, an investment fund managed by Pharmakon Advisors.

Brian Kaspar, co-founder of Celenex, and Kathrin Meyer, 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 genetic disease of the CNS,” said Meyer. “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.”

 
September 20, 2018
Photo: John Crowley, CEO of Amicus Therapeutics