Home / Business News / PTC Therapeutics to Acquire Agilis Biotherapeutics Expanding its Gene Therapy Pipeline

PTC Therapeutics to Acquire Agilis Biotherapeutics Expanding its Gene Therapy Pipeline

SOUTH PLAINFIELD, N.J., July 19, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that it has entered into an agreement to acquire Agilis Biotherapeutics, Inc., 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.

“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 Stuart W. Peltz, Ph.D., Chief Executive Officer, 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.”

“I am proud of the accomplishments achieved by Agilis culminating with this value-creating transaction,” said Mark Pykett, DVM, Ph.D., 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 lead gene therapy candidate, GT-AADC, has compelling clinical data in treating Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency. AADC Deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase (DDC) gene. In its severe forms, AADC Deficiency causes the inability to develop any motor control (global muscular hypotonia/dystonia) resulting in breathing, feeding, and swallowing problems, frequent hospitalizations, and the need for life-long care. 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, and care is limited to palliative options with significant burden on caregivers.

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.

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.

Transaction Details
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. 

Conference Call and Webcast Details
A conference call will take place on Thursday, July 19 at 4:30 pm ET and can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 8469109. A live, listen-only webcast of the conference call can be accessed at https://edge.media-server.com/m6/p/25h8j9nj. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for two weeks.

About Agilis Biotherapeutics, Inc.
Agilis is advancing innovative gene therapies designed to provide long-term efficacy for patients with debilitating, often fatal, rare genetic diseases that affect the central nervous system. Agilis’ gene therapies are engineered to impart sustainable clinical benefits by inducing persistent expression of a therapeutic gene through precise targeting and restoration of lost gene function to achieve long-term efficacy. Agilis’ rare disease 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.

About PTC Therapeutics
PTC is a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. Founded 20 years ago, PTC Therapeutics has successfully launched two rare disorder products and has a global commercial footprint. This success is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. For more information, please visit our website at www.ptcbio.com.

Check Also

Takeda, Microsoft and EURORDIS Release Report with Action Plan to Help Rare Disease Community Shorten the Diagnostic Odyssey

NEW YORK and BRUSSELS, Feb. 20, 2019 /PRNewswire/ — The Global Commission to End the …