PTC to acquire Agilis Biotherapeutics advancing its commitment to gene therapy

By Maggie Lynch

- Last updated on GMT


Related tags Gene therapy Rare disease Clinical trial Partnership

PTC Therapeutics will acquire Agilis Biotherapeutics to continue work on gene therapy for rare monogenic diseases affecting the central nervous system.

“The acquisition of Agilis aligns with PTC’s mission to bring enhanced therapies to patients with rare diseases with limited or no treatment options for the underlying cause of disease,” ​PTC Therapeutics representative, Jane Baj, told us.

PTC will pay $50m upfront and $150m in PTC common stock to Agilis. Upon reaching development milestone, including acceptance of biologics license application (BLA). PTC may pay $60m over the next two years after completion of success-based milestones.

This transaction between Agilis and PTC is set to be complete in the third quarter of 2018.

PTC’s pipeline

“The acquisition enhances PTC’s pipeline by adding a gene therapy platform with early and late-stage products,” ​according to Baj. 

Agilis’ lead gene therapy candidate, GT-AADC treating Aromatic L-Amino Acid Decarboxylase Deficiency (AADC), has shown positive clinical trial data. AADC, a rare CNS disorder, causes an inability to develop motor control, causing breathing, feeding and swallowing problems. Many patients see the first signs of AADC​ within the first year of life.

Subjects in prospective GT-AADC trials have shown progress in motor development​ and motor function over multiple years following the gene therapy treatment. PTC has plans to submit a BLA to the FDA in 2019.  

Two prospective studies of GT-AADC therapy have enrolled 18 patients between the ages of 21 to 102 weeks. The first trial conducted has a median age of 54 weeks, and the second a median age of 34 weeks. Only 100 people have been noted to have AADC​, and 20% of these individuals are from Taiwan. According to Baj, the study took place in Taiwan but patients were international.

After the partnership, the product pipeline will also include gene therapy targeting Freidreich ataxia​, a genetic and neurodegenerative disease, and therapy programs for Angelman syndrome​, a rare genetic neurological disorder.

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