PTC inks $200M Agilis takeover to snag late-phase gene therapy

PTC Therapeutics is paying $200 million upfront to acquire Agilis Biotherapeutics. The deal gives PTC ownership of a gene therapy treatment for rare neurotransmitter disease AADC deficiency that is set to go before the FDA next year.

Researchers have spent the past eight years tracking the progress of patients treated with the gene therapy, which is designed to reverse severe health problems that arise from a shortage of the enzyme aromatic l-amino acid decarboxylase by correcting the underlying molecular defect. Data on 18 children treated with the adeno-associated virus therapy suggest it helps them to hit motor milestones such as full head control and unassisted standing.

Having licensed the gene therapy from National Taiwan, China University, Agilis was gearing up to file for FDA approval but has now opted to sell up before submitting the BLA. PTC persuaded Agilis to change course by offering $50 million in cash and $150 million in stock upfront. If PTC files the BLA and the program clears other milestones over the next two years, it will hand over up to $60 million more.

More milestones are tied to Agilis’ other pipeline prospects. The most advanced of these prospects is a gene therapy targeting Friedreich ataxia that is due to enter clinical development next year. If Agilis’ three most advanced programs are successful, PTC will pay out up to $535 million tied to regulatory milestones and the receipt of a priority review voucher. The agreement also features $150 million in commercial milestones.

Agilis factored PTC’s ability to make successes of the gene therapies and thereby hit the milestones into its evaluation of the merits of the deal.

"PTC provides a global infrastructure and proven capabilities, which we believe will enable our goal of providing therapy to patients suffering from rare CNS disorders," Agilis CEO Mark Pykett, Ph.D., said in a statement. "I look forward to joining PTC and supporting the advancement of the programs to provide value to patients."

New Jersey-based PTC’s infrastructure hasn’t always translated into success, though. The FDA again turned down PTC’s Duchenne muscular dystrophy drug last year, leaving it needing to generate data on dystrophin production before taking another run on the agency.