Rare Daily Staff

PTC Therapeutics said it has agreed to acquire Agilis Biotherapeutics, a developer of gene therapies that target CNS disease, for $200 million in stock and cash plus potential milestones.

Agilis is advancing a gene therapy platform for rare monogenic diseases that affect the central nervous system. The boards of both companies have approved the transaction.

Agilis’ lead gene therapy candidate, GT-AADC, has clinical data in treating Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency. AADC Deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase gene. In its severe forms, AADC Deficiency causes the inability to develop any motor control resulting in breathing, feeding, and swallowing problems, frequent hospitalizations, and the need for life-long care. Many patients die in the first decade of life due to profound motor dysfunction and secondary complications such as choking, hypoxia, and pneumonia. Currently, no treatment options are available for the underlying cause of the disorder, and care is limited to palliative options with significant burden on caregivers.

The product pipeline also includes a preclinical gene therapy asset targeting Friedreich ataxia, a rare and life-shortening neurodegenerative disease caused by a single defect in the FXN gene which causes reduced production of the frataxin protein. The company expects to file an application in 2019 to begin clinical trials for the gene therapy candidate. Additionally, the acquisition brings two other gene therapy programs targeting CNS disorders, including Angelman syndrome, a rare, genetic, neurological disorder characterized by severe developmental delays.

The transaction is expected to close in the third quarter of 2018. On completion, PTC plans a transition of operations and intigration Agilis’ employees.

“The addition of the gene therapy platform transforms PTC and aligns with our vision of being a leader in the treatment of rare disorders,” said Stuart Peltz, CEO of PTC Therapeutics. “We are impressed with the clinical results shown by the AADC program and are excited with the potential to quickly bring this therapy to patients. We look forward to advancing the Friedreich ataxia and Angelman syndrome programs into the clinic in the next two years.”

Under the terms of the merger agreement, PTC will pay $50 million in cash and approximately $150 million in PTC common stock, subject to an estimated maximum 9.34 million share limit. Any shortfall will be made whole with additional cash consideration.

In addition to the upfront payments, potential future consideration includes $60 million in development milestones to be paid over the next two years which includes the acceptance of an application for marketing approval.

July 19, 2018
Photo: Stuart Peltz, CEO of PTC Therapeutics

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