In a major strategic move, US biopharmaceutical company PTC Therapeutics has finalized its acquisition of Agilis Biotherapeutics, a biotech firm specializing in the development of gene therapies for rare genetic disorders affecting the central nervous system (CNS). The acquisition, valued at $200 million, includes a mix of $50 million in cash and approximately $150 million in PTC Therapeutics shares, underscoring a significant investment in the future of gene therapy.

The highlight of this acquisition is the addition of Agilis Biotherapeutics’ adeno-associated virus (AAV) gene therapy, GT-AADC, designed to treat Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency—a rare CNS disorder caused by mutations in the DDC gene. Currently, there are no treatments targeting the underlying cause of AADC Deficiency. However, clinical studies of GT-AADC have shown promising results, with patients experiencing significant improvements in motor and cognitive functions following the treatment.

Beyond GT-AADC, the acquisition also brings a pipeline of innovative gene therapies under PTC Therapeutics’ umbrella, targeting conditions such as Friedreich Ataxia, Angelman Syndrome, and various cognitive disorders. These programs are in various stages of development and hold potential for addressing a range of neurodevelopmental and neurodegenerative disorders.

The agreement also outlines potential future earnings for Agilis Biotherapeutics, which could exceed $945 million. This sum includes $60 million in development milestones, up to $535 million in success-based milestones for specific gene therapies, and commercial milestones worth $150 million. Additionally, Agilis stands to earn between 2-6% of annual net sales for therapies targeting Friedreich ataxia and Angelman syndrome.

Stuart W. Peltz, CEO of PTC Therapeutics, emphasized the acquisition’s strategic importance, noting the addition of a seasoned gene therapy team, advanced micro-delivery platforms, and enhanced 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,” said Peltz.

Earlier this month, PTC Therapeutics also announced a collaboration with Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, to commercialize two rare disease drugs in Latin America, further expanding its global reach and therapeutic impact.

The acquisition of Agilis Biotherapeutics by PTC Therapeutics represents a transformative step in the field of gene therapy, particularly for rare CNS disorders. By integrating Agilis’ advanced gene therapies and platforms, PTC Therapeutics is poised to enhance its research capabilities and accelerate the development of potentially life-altering treatments.

  AADC Deficiency, Agilis Biotherapeutics, Aromatic L-Amino Acid Decarboxylase Deficiency, Biopharmaceutical Industry, biopharmaceutical news, CNS disorders, CNS genetic disorders, gene therapy, gene therapy acquisition, GT-AADC, PTC Therapeutics, Stuart W. Peltz, USA