PTC Dives Into Gene Therapy with $50M Agilis Biotherapeutics Deal

Xconomy New York — 

PTC Therapeutics is looking to gene therapy in a bid to broaden its pipeline, scooping up privately held Agilis Biotherapeutics in a cash and stock deal.

South Plainfield, NJ-based PTC (NASDAQ: PTCT) will pay $50 million up front, plus $150 million in PTC shares, for Agilis. Agilis, based in Cambridge, MA, could get up to $595 million more depending on the progress of its three most advanced gene therapy programs, which target rare central nervous system disorders.

For those payments to kick in, Agilis will need to win FDA approvals for each of those three gene therapies, and get a priority review voucher as well. The FDA awards these vouchers to companies that develop drugs for neglected diseases and rare pediatric disorders. They ensure a speedier review from the agency once a company files for approval. But these vouchers have become valuable commodities themselves, bought and sold at prices topping $100 million.

PTC already focuses on rare disease treatments, and Duchenne muscular dystrophy in particular. The company sells two drugs for Duchenne, ataluren (Translarna) and deflazacort (Emflaza). Ataluren is only approved in Europe, however. PTC has failed three times to win FDA approval. Combined, the two drugs generated $174 million in sales in 2017.

Agilis’ lead therapeutic candidate, GT-AADC, is an experimental treatment for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency, an enzyme deficiency that leads to the inability to develop motor control. AADC patients develop problems breathing and swallowing, and need life-long care. PTC says based on “long-term evidence of durable clinical benefit” from Agilis’ clinical studies, it plans to file for FDA approval next year.

Three additional gene therapy candidates will come to PTC in the Agilis acquisition. AGIL-FA, an experimental treatment for the rare neurodegenerative disease Friedreich’s ataxia, could start clinical trials next year. AGIL-AS, a potential gene therapy for the rare neurological disorder Angelman syndrome, and AGIL-RLN, a potential treatment for unspecified cognitive disorders, are still in preclinical development.

If the gene therapies for Friedreich’s ataxia and Angelman syndrome reach the market, Agilis shareholders would get another $150 million, plus 2 to 6 percent of the net sales of the drugs, according to the agreement.

PTC and Agilis expect the acquisition to close later this quarter. The deal has already been approved by the boards of both companies.

Photo by Flickr user Caroline Davis2010 via a Creative Commons license