Axonis Therapeutics: $115 Million (Series A) Raised To Develop Novel Neuromedicines

By Amit Chowdhry • Nov 8, 2024

Axonis Therapeutics, a biotechnology company focused on the development of novel neuromedicines, announced the completion of an oversubscribed $115 million Series A financing. The proceeds from the financing will be used to advance Axonis’ lead development candidate, AXN-027, through clinical proof-of-concept in patients. Cormorant Asset Management and venBio Partners co-led the financing with significant investments from Sofinnova Investments, MRL Ventures Fund, Perceptive Advisors, Lumira Ventures and Solasta Ventures. Eos Bioinnovation, Hatteras Venture Partners, SCI Ventures, Alexandria Venture Investments, Tachyon Ventures, BoxOne, Iaso Ventures and Pathway Bioventures also participated in the financing.

In connection with this funding, Axonis named Raymond J. Kelleher, M.D., Ph.D., Managing Director at Cormorant Asset Management and Jonathan Leff, M.D., Executive Partner, Private Equity, from Sofinnova Investments to its Board of Directors.

AXN-027 is an oral small molecule designed to potentiate the function of KCC2, a major CNS chloride transporter essential for inhibitory neurotransmission, for the treatment of epilepsy and pain. Proceeds will also enable the development of next generation compounds targeting KCC2 in these and other indications, including psychiatric and neurodevelopmental disorders.

Even though the approval of several drugs for epilepsy and pain, many patients do not tolerate or respond to current first-line therapies due to impaired synaptic inhibition, which can contribute to drug resistance. And Axonis’ oral KCC2 potentiators are designed to restore functional inhibition and overcome drug resistance in patients.

Axonis is headed by Joanna Stanicka, Ph.D., co-founder and co-inventor of Axonis’ underlying IP. And she is a Harvard-trained scientist, drug hunter, and entrepreneur, and a recipient of the Massachusetts Next Generation Award, a prize in leadership for woman-CEO in life sciences.

Shane Hegarty, Ph.D., is the company’s Chief Scientific Officer, co-founder, and IP co-inventor. And he was previously a Research Fellow at Harvard Medical School and Boston Children’s Hospital, faculty member in Neuroscience Department of University College Cork, and recipient of the Neuroscience Ireland Investigator Award.

In connection with the funding, Axonis also announces the appointments of Donald Manning, M.D., Ph.D., as Chief Medical Officer and Jeff Imbaro, M.B.A., as Chief Operating Officer. Dr. Manning is a clinician-scientist with comprehensive experience in acute and chronic clinical practice, research and management. And he brings to Axonis over 25 years of executive experience, having overseen the filings of seven INDs and five NDAs that have led to nine approved products. Mr. Imbaro brings over 20 years of company building expertise, having led Business Development and General and Administrative functions of several successful biotech and pharmaceutical companies. He was a member of the founding team of Semma Therapeutics, which was later acquired by Vertex Pharmaceuticals in 2019 for nearly $1 billion. 

KEY QUOTES:

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. As we head into the clinic later this year, this financing is a significant milestone for Axonis as we translate our exciting science into efficacious and well-tolerated treatments for patients suffering from neurological disorders.”

– Joanna Stanicka, Ph.D., Chief Executive Officer, President, and co-founder of Axonis

“We’re delighted that this accomplished group of investors share our mission to translate Axonis’ science into impactful medicines. I’m pleased to see how far the science has progressed at Axonis; what the team has accomplished is remarkable. We are now well positioned to bring our lead therapeutic program through clinical proof of concept and expand into related arenas and build a leading biotechnology company.”

– Corey Goodman, Ph.D., Executive Chair and co-founder of Axonis

“I’m enthusiastic about the potential of Axonis’ novel precision approach to re-shape how we treat patients with neurological disorders, particularly epilepsy and pain where modulation of synaptic inhibition has great therapeutic promise. Axonis’ scientists have done amazing work. We’re very excited about what this program could mean for patients and their families.”

– Raymond J. Kelleher, M.D., Ph.D., Managing Director at Cormorant