Published On: Sat, Aug 25th, 2018

PTC Therapeutics completes $200m acquisition of Agilis Biotherapeutics

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US biopharma company PTC Therapeutics has completed its $200 million acquisition of Agilis Biotherapeutics, a biotechnology company engaged in the development of gene therapies for rare genetic disorders that affect the central nervous system (CNS).

PTC Therapeutics acquisition of Agilis Biotherapeutics adds an adeno-associated virus (AAV) gene therapy called to the former’s portfolio. GT-AADC has been designed for the of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency, a rare CNS disorder resulting from the mutations in the dopa decarboxylase (DDC) gene.

As of now, there are no treatments for AADC Deficiency that target its underlying cause. The findings from prospective clinical studies of GT-AADC showed that treated patients were found to display substantial gains on motor function and cognitive scales over multiple years after the single gene therapy treatment.

PTC Therapeutics acquisition of Agilis Biotherapeutics has also added a pipeline of gene therapy programs in development – GT-FA, GT-AS, and GT-RLN, for Friedreich Ataxia, and Cognitive Disorders, respectively. The three conditions are associated with various neurodevelopmental and neurodegenerative disorders.

PTC Therapeutics acquires gene therapy company Agilis Biotherapeutics.

PTC Therapeutics acquires gene therapy company Agilis Biotherapeutics. Image courtesy of dream designs at

As per an agreement signed in August 2018, PTC Therapeutics was to pay $50 million upfront and around $150 million worth shares to Agilis Biotherapeutics.

Overall, the deal is likely to fetch the gene therapy company over $945 million, which also covers $60 million in development milestones, up to $535 million in success-based milestones for certain gene therapies, tiered commercial milestones worth $150 million, and also 2-6 % of annual net sales for Friedreich ataxia and Angelman syndrome.

Commenting on PTC Therapeutics acquisition of Agilis Biotherapeutics, Stuart W. Peltz – CEO of PTC Therapeutics, said: “We have added a team with exceptional gene therapy experience, a targeted micro-delivery gene therapy platform that has demonstrated durable clinical benefit, as well as gene therapy manufacturing and R&D capabilities. This acquisition deepens our pipeline and, as a combined organization, we will be able to optimally treat more rare disorders and accelerate the process of bringing important new therapies to patients worldwide.”

Earlier this month, PTC Therapeutics entered into a collaboration with Akcea Therapeutics, an affiliate of to commercialize a couple of the latter’s rare disease drugs in Latin America.

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