Axonis secures $115M for epilepsy and neuropathic pain drug trials

Boston-based biotech firm Axonis Therapeutics has successfully raised $115 million in Series A funding to advance its investigational oral drug, AXN-027, aimed at treating focal epilepsy and diabetic neuropathic pain. This financing, announced on Wednesday morning, is expected to facilitate the company’s entry into clinical trials by the end of the year. Around the same time, Ovid Therapeutics plans to initiate human trials using a similar approach based on a platform licensed from AstraZeneca. Both companies are focusing on KCC2, a central nervous system chloride transporter crucial for electrical signaling in the brain.

KCC2 is essential for neuronal GABA signaling, a pathway targeted by various drugmakers, including Sage Therapeutics with their postpartum depression medication, Zurzuvae. However, according to CEO Joanna Stanicka, no biotech firms have yet entered clinical trials focusing on KCC2. Stanicka explained to Endpoints News that traditional GABA treatments often result in global inhibition, leading to cognitive impairment and sedation. She believes that a more targeted inhibition approach could avoid these side effects.

The company, which has a team of 13 employees, also announced two significant appointments on Wednesday. Donald Manning has been named the new Chief Medical Officer, having previously served in similar roles at Travecta, Pacira BioSciences, and Adynxx. Jeff Imbaro has taken on the role of Chief Operating Officer, with past experience as the operating chief at stroke-focused Elevian and as a founding member of Vertex-acquired Semma Therapeutics.

Axonis was established around 15 years ago by Bob Yant, a spinal cord injury patient advocate who also serves as a board observer. However, the company did not become fully operational until 2019, according to Stanicka. Before securing the Series A funding, Axonis raised approximately $34 million in seed funding and an additional $9 million from government and foundation grants. The company’s investors include Cormorant Asset Management, venBio, Sofinnova Investments, and the corporate venture arm of Merck, among others.

Stanicka mentioned that the Series A funding will support the development of AXN-027 through Phase 2 proof-of-concept studies. This funding milestone marks a significant step forward for Axonis Therapeutics as it strives to bring innovative treatments to patients suffering from focal epilepsy and diabetic neuropathic pain.