Amicus Therapeutics, headquartered in Cranbury, New Jersey, inked a definitive agreement for 10 gene therapy programs licensed from Nationwide Children’s Hospital through the acquisition of Celenex.
Amicus Therapeutics, headquartered in Cranbury, New Jersey, inked a definitive agreement for 10 gene therapy programs licensed from Nationwide Children’s Hospital through the acquisition of Celenex. The programs were developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University. Celenex was a spinout of Nationwide Children’s Hospital.
Under the terms of the deal, Amicus will pay $100 million up front in cash. Celenex shareholders are eligible for up to $15 million in development milestones and $262 million in regulatory submission and approval milestones. Amicus indicates it doesn’t expect to pay more than $75 million over the next four years in milestones payments.
All of the programs are in neurologic lysosomal storage disorders. The three lead programs are CLN6, CLN3 and CLN8 for Batten disease. Batten disease is also called Neuronal Ceroid Lipofuscinosis (NCL), a rare and life-threatening group of disorders.
“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, Amicus’ chairman and chief executive officer, in a statement. “The groundbreaking work of Drs. Brian Kaspar and Kathrin Meyer at Nationwide Children’s Hospital, along with collaborator, Arthur Burghes, PhD, 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.”
Two of the 10 programs have already entered human clinical trials. Amicus markets Galafold to treat Fabry disease and is currently developing a second therapeutic to treat Pompe disease. This acquisition expands the company’s pipeline significantly, shifting the focus from drugs that stabilize but don’t cure lysosomal storage disorders to potential cures.
STAT writes, “One of those newly acquired gene therapies targets the CLN6 mutation form of Batten disease. Gordon Gray, a Hollywood film producer, and his wife Kristen have two daughters born with this form of Batten. The Grays formed a scientific foundation and later co-founded Celenex as a way to find a treatment for their daughters and others with the disease.”
“Gordon and Kristen Gray 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,” stated Crowley. “I am honored that they and their research team have chosen to entrust these 10 programs to the passionate team of scientists and entrepreneurs at Amicus. I cannot think of a better foundation for Amicus’ entry into gene therapies.”
Gordon Gray is best known for his work on the films The Rookie, Secretariat and Invincible.
BioMarin Pharmaceutical offers Brineura, a drug to treat the CLN2 type of Batten disease in children. Abeona Therapeutics is developing a gene therapy for the CLN1 type of Batten.
All 10 programs acquired in the deal leverage intrathecal delivery. This approach utilizes AAV vector technology that has been successful in other rare CNS indications, such as spinal muscular atrophy (SMA).
“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 Kathrin Meyer, in a statement. “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.”
