PTC Expanding Gene Therapy Portfolio by Buying Agilis Biotherapeutics

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by Alice Melão |

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PTC Therapeutics announced it will acquire Agilis Biotherapeutics, a company focused on the development of gene therapies targeting rare diseases that affect the central nervous system (CNS).

The acquisition will represent an expansion of PTC Therapeutics’ portfolio by including Agilis’ lead product candidate AGIL-AADC, for aromatic L-amino acid decarboxylase (AADC) deficiency, but also gene therapies for Friedreich’s ataxia, Angelman syndrome, and cognitive disorders.

The merger is expected to be concluded during the third quarter of 2018, pending the fulfillment of all established conditions. Under the agreement, PTC will pay an upfront amount of $50 million in cash and about $150 million in PTC common stock. Development, success-based, and tiered commercial milestones in coming years amount to $745 million. Annual net sales for Friedreich’s ataxia and Angelman syndrome therapies amount to 2 to 6 percent.

“The addition of the gene therapy platform transforms PTC and aligns with our vision of being a leader in the treatment of rare disorders,” Stuart W. Peltz, PhD, the CEO of PTC Therapeutics, said in a press release.

“We look forward to advancing the Friedreich ataxia and Angelman syndrome programs into the clinic in the next two years,” he said. “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.”

Agilis’ gene therapy candidate AGIL-FA was being developed in collaboration with Intrexon as a potential strategy to restore a functional FXN gene in the central nervous system, and overcome the effects of the genetic mutations that cause Friedreich’s ataxia.

This clinical program was also being developed in close partnership with the Friedreich’s Ataxia Research Alliance (FARA) to guarantee its focus in real patients needs.

In November 2017, the European Medicines Agency granted orphan medicinal product designation to AGIL-FA. This decision was supported by the positive opinion of the Committee for Orphan Medicinal Products (COMP), issued in July 2017. The U.S. Food and Drug Administration also recognized the therapy’s potential by granting AGIL-FA orphan drug status.

These regulatory designations are expected to accelerate the development and review of this investigative gene therapy, and expedite its final approval.

The companies expect to submit an investigational new drug (IND) application with regulatory agencies in 2019 to support the initiation of AGIL-FA clinical studies.

“I am proud of the accomplishments achieved by Agilis culminating with this value-creating transaction,” said Mark Pykett, DVM, PhD, president and CEO of Agilis Biotherapeutics.

“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.”