Mergers and Acquisitions

Date: 2018-07-19

Type of information: Company acquisition

Acquired company: Agilis Biotherapeutics (USA - MA)

Acquiring company: PTC Therapeutics (USA - NJ)

Amount: up to $260 million and success-based milestones


  • • On July 19, 2018, PTC Therapeutics announced that it has entered into an agreement to acquire Agilis Biotherapeutics,, a biotechnology company advancing an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system (CNS). The transaction was approved by the Boards of both companies.
  • Under the terms of the merger agreement, PTC will pay an upfront consideration of $50 million in cash and approximately $150 million in PTC common stock, subject to an estimated maximum 9.34 million share limit (with any shortfall to be made whole with additional cash consideration).
  • In addition to the upfront payments, potential future consideration includes $60 million in development milestones to be paid over the next two years which includes the acceptance of a BLA. Additionally, the transaction includes up to $535 million in success-based milestones in connection with regulatory approvals on the three most advanced programs and receipt of a priority review voucher, as well as tiered commercial milestones of $150 million, and 2-6 % of annual net sales for Friedreich ataxia and Angelman syndrome.
  • The transaction is expected to close in the third quarter of 2018, pending successful fulfillment of all customary closing conditions. On completion, PTC plans a smooth transition of operations and the integration of Agilis' talented and dedicated employees to continue the mission of bringing the pipeline of gene therapies for CNS disorders to patients worldwide.


  • Agilis Biotherapeutics develops gene therapies designed to provide long-term efficacy for patients with rare diseases of the central nervous system (CNS) that are caused by single-gene defects. Its programs are focused on gene therapy for AADC deficiency, Friedreich ataxia, and Angelman syndrome, all rare genetic diseases that include neurological deficits and result in physically debilitating conditions.
  • AADC Deficiency is a rare CNS disorder arising from reductions in the enzyme aromatic L-amino acid decarboxylase (AADC) from mutations in the dopa decarboxylase (DDC) gene.  This deficiency causes the inability to develop any motor strength and control resulting in breathing, feeding, and swallowing problems. Many patients die in the first decade of life due to profound motor dysfunction and secondary complications such as choking, hypoxia, and pneumonia. Currently, no treatment options are available for the underlying cause of the disorder.
  • Agilis' lead gene therapy candidate, GT-AADC, has compelling clinical data in treating AADC Deficiency . The results from two prospective clinical studies enrolling 18 subjects with severe AADC Deficiency indicated that treated subjects with GT-AADC exhibited de novo dopamine production. Additionally, the subjects demonstrated achievement of motor development milestones and substantial gains on motor function and cognitive scales over multiple years following the single gene therapy treatment. Data from trials that began in 2010 have demonstrated long-term evidence of durable clinical benefit. Based on multi-year data from initial clinical studies with the AADC gene therapy treatment candidate, PTC plans to submit a Biologics License Application (BLA) to the FDA in 2019.
  • The product pipeline also includes a gene therapy asset targeting Friedreich ataxia, a rare and life-shortening neurodegenerative disease caused by a single defect in the FXN gene which causes reduced production of the frataxin protein. An IND submission for this program is expected in 2019. Additionally, the acquisition brings two other gene therapy programs targeting CNS disorders, including Angelman syndrome, a rare, genetic, neurological disorder characterized by severe developmental delays.

Related: Rare diseases - CNS Diseases - Gene therapy  

Is general: Yes