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PTC Therapeutics Acquires Agilis Biotherapeutics in $200 Million Rare Disease Buyout

The acquisition was largely motivated by Agilis’ lead candidate, GT-AADC, a gene therapy designed to treat Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency, an ultra-rare nervous system disease.

PTC Therapeutics Acquires Agilis Biotherapeutics in $200 Million Rare Disease Buyout

By: Sarah Hand, M.Sc.

Posted on: in News | Pharmaceutical News

Rare disease drug developer PTC Therapeutics has announced their intention to acquire biotech firm Agilis Biotherapeutics in a deal worth $200 million. The acquisition was largely motivated by Agilis’ lead candidate, GT-AADC, a gene therapy designed to treat Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency, an ultra-rare nervous system disease.

The agreement includes a combination of cash and stock, with PTC offering $50 million in an upfront payment and $150 million in PTC shares. Future payments totalling up to $745 will be payed out to Agilis based on successful achievement of development, regulatory and commercial milestones.

“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 Dr. Stuart W. Peltz, Chief Executive Officer of PTC Therapeutics, Inc. “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.”

Patients with AADC Deficiency carry mutations in the dopa decarboxylase (DDC) gene which reduce levels of the AADC enzyme. The disease impacts the development of motor control which means patients suffer breathing, eating and swallowing issues and often results in death within the first ten years of life.

According to the NIH, only 100 individuals in the world have been diagnosed with AADC Deficiency, with about 20 percent of those patients being Taiwanese. While this is an extremely limited patient population, rare disease treatments tend to be higher priced to recoup development costs.

Two small clinical trials involving 18 patients with AADC Deficiency were used to demonstrate the potential of GT-AADC. Those patients treated with the gene therapy showed de novo dopamine production and improvements in motor development multiple years after receiving a single treatment.

“I am proud of the accomplishments achieved by Agilis culminating with this value-creating transaction,” said Dr. Mark Pykett, President and Chief Executive Officer of Agilis Biotherapeutics, Inc. “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. I look forward to joining PTC and supporting the advancement of the programs to provide value to patients.”

The companies expect that the deal will close later this year. A Biologics License Application (BLA) for GT-AADC is set to be submitted in 2019, with successful acceptance of this BLA coming with a $60 million development milestone payment.


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