Dive Brief:

• PTC Therapeutics announced an agreement on Thursday to acquire gene therapy start-up Agilis Biotherapeutics, adding gene therapies to its pipeline of RNA-based drugs and small molecule therapeutics.
• The lead gene therapy, AGIL-AADC, is expected to be submitted for approval next year.
• The transaction is expected to close in the third quarter of 2018, and no financial details have been disclosed. PTC's share price rose just over 4% by market's close on Thursday, and climbed an additional 8% premarket on Friday.

Dive Insight:

The Massachusetts biotech Agilis Biotherapeutics is focused on building gene therapies for rare monogenic diseases that affect the central nervous system (CNS), using its gene therapy platform.

The company's lead gene therapy AGIL-AADC is in a Phase 2b trial for aromatic L-amino acid decarboxylase (AADC) deficiency, with a Biologics License Application in preparation for 2019. The next step for PTC Therapeutics will be to submit an investigational New Drug Application for a gene therapy for Friedreich ataxia, and to move treatments for Angelman syndrome and cognitive disorders into the clinic.

AADC deficiency affects the brain's ability to produce dopamine and serotonin, leading to movement and mood disorders. It has no specific approved treatment, and approaches include a variety of symptomatic treatments. Agilis appears to be developing the most advanced treatment for the disease.

"The addition of the gene therapy platform transforms PTC and aligns with our vision of being a leader in the treatment of rare disorders. 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," said Stuart Peltz, CEO of PTC Therapeutics, in a statement.

There are a number of factors behind Agilis' focus on rare CNS disorders and its route of administration. The CNS has a naturally low immune response, and direct administration potentially avoids immune-mediated adverse events, as well as allows for lower doses. Additionally, rare CNS diseases can receive orphan drug incentives from the Food and Drug Administration.