Boston-based Axonis Therapeutics has successfully completed an oversubscribed $115 million Series A financing round, aimed at advancing its pioneering neuromedicines targeting neurological disorders. This substantial investment will support the clinical development of Axonis' lead candidate, AXN-027, an innovative oral small molecule designed to enhance the function of KCC2, a crucial chloride transporter in the central nervous system. This therapy aims to address significant unmet medical needs in conditions such as epilepsy and pain, where current treatments often fall short.

The financing round was co-led by prominent investors Cormorant Asset Management and venBio Partners, with additional backing from Sofinnova Investments, MRL Ventures Fund, Perceptive Advisors, Lumira Ventures, and Solasta Ventures. Notably, the financing attracted a diverse group of investors eager to support Axonis' vision for advancing its therapeutics pipeline.

Addressing Key Challenges in Neurological Treatments

With a growing understanding of the challenges faced by patients with epilepsy and chronic pain, Axonis aims to create solutions that target the underlying issues of impaired synaptic inhibition. Many existing therapies for these conditions are ineffective for a subset of patients who may experience drug resistance due to this inhibition. By developing oral KCC2 potentiators, Axonis hopes to restore normal inhibitory neurotransmission, offering new hope for those who have not responded adequately to standard treatments.

"Despite the availability of several approved drugs for epilepsy and pain, a considerable number of patients do not tolerate or respond to these therapies," said Joanna Stanicka, Ph.D., CEO and co-founder of Axonis. "Our approach aims to address these gaps in treatment options, providing patients with effective, well-tolerated medications that can improve their quality of life."

Progressing Towards Clinical Trials

The funding will facilitate the progression of AXN-027 through clinical proof-of-concept studies, an essential step toward eventual market introduction. Axonis is set to commence clinical trials later this year, marking a significant milestone in the company's trajectory.

"Axonis' vision is to advance a pipeline of oral KCC2 therapeutics to restore functional inhibition in the CNS to treat neurological disorders without disabling side effects," Dr. Stanicka added. "This financing is a significant milestone for Axonis as we translate our exciting science into efficacious and well-tolerated treatments."

Strengthening Leadership and Operations

In conjunction with the financing, Axonis has appointed Dr. Raymond J. Kelleher and Dr. Jonathan Leff to its Board of Directors, both of whom bring extensive experience in the life sciences sector. Dr. Kelleher, a Managing Director at Cormorant Asset Management, and Dr. Leff, an Executive Partner at Sofinnova Investments, will provide valuable insights and guidance as the company advances its development initiatives.

Additionally, Axonis has strengthened its leadership team by appointing Dr. Donald Manning as Chief Medical Officer and Jeff Imbaro as Chief Operating Officer. Dr. Manning's background includes overseeing multiple drug approvals, while Mr. Imbaro has a proven track record of building successful biotech companies.

A Promising Future for Axonis

Axonis Therapeutics is uniquely positioned to transform the treatment landscape for neurological disorders with its KCC2-targeting therapeutics. By building upon its proprietary KCC2 discovery engine, the company aims to establish itself as a leader in this promising area of drug development.

Corey Goodman, Ph.D., Executive Chair and co-founder of Axonis, expressed his optimism for the future, stating, "We're delighted that this accomplished group of investors shares our mission to translate Axonis' science into impactful medicines. We believe we are well-positioned to bring our lead therapeutic program through clinical proof of concept and expand into related arenas."

As Axonis Therapeutics embarks on this pivotal journey to bring innovative treatments for neurological disorders to market, the recent $115 million funding round signifies both the strong investor confidence in its approach and the urgency of addressing significant unmet medical needs in the field. With a clear focus on advancing its KCC2 therapeutics, Axonis is poised to make a meaningful impact on the lives of patients suffering from epilepsy, chronic pain, and beyond.