Axonis is developing first-in-class oral small molecules that potentiate KCC2, a CNS chloride transporter crucial for inhibitory neurotransmission. Its lead candidate, AXN-027, is designed to restore functional inhibition and is being advanced into clinical proof-of-concept in patients with epilepsy and pain. The company plans to use its proprietary KCC2 discovery engine to develop next-generation compounds for additional indications including psychiatric and neurodevelopmental disorders. Axonis was spun out from Boston Children’s Hospital, Harvard and Université Laval and is headquartered in Boston, MA. Leadership includes co-founder and CEO Joanna Stanicka, Ph.D., CSO Shane Hegarty, Ph.D., and new appointments for Chief Medical Officer Donald Manning, M.D., Ph.D., and COO Jeff Imbaro. The company has received grant support from multiple public and non-profit sources and will use the recent financing to fund clinical development and pipeline expansion. AXONIS Therapeutics is an emerging biotechnology company based in Cambridge, MA, advancing neuromodulating therapies for central nervous system disorders. Its pipeline focuses on neuron-reviving approaches that enable CNS neurons to resist degeneration, restore excitation/inhibition balance, and regenerate. The company’s primary program centers on upmodulation of the KCC2 protein and has completed proof-of-concept preclinical studies showing reactivation of spared neural tissue at sites of spinal cord injury. Targeted indications include spinal cord injury and epilepsy, with potential applicability to chronic pain, Rett syndrome and broader neurodegenerative conditions. AXONIS has licensed three technologies, including recently discovered methods to prevent neurodegeneration, and is investigating additional methodologies to revive neurons. Financially, the company secured $4 million in seed funding at its operational launch in early 2020, has received non-dilutive awards from the Massachusetts Life Sciences Center and NIH SBIR and two Golden Tickets from Boehringer Ingelheim and Astellas, and just raised additional financing to support clinical advancement and IND-enabling activities. AXONIS Therapeutics is advancing a novel KCC2 upmodulation therapy to reactivate spared neural tissue after spinal cord injury and is also investigating PTEN inhibition to regenerate injured nerve connections. The company has licensed or optioned two technologies from Boston Children’s Hospital to support its preclinical programs. Proceeds from the current financing will enable AXONIS to conduct preclinical studies and expand its team as it moves toward proof-of-concept milestones. AXONIS reports promising proof-of-concept results to date and expects to deliver additional proof-of-concept data in approximately one year. The company has received state and federal non-dilutive funding and other seed investment, and won a Boehringer Ingelheim Golden Ticket that provides one year of free lab space at LabCentral. Leadership includes CEO Joanna Stanicka, PhD, Executive Chair and co-founder Corey Goodman, PhD, and founder Bob Yant; the company is based in Cambridge, MA.

Total raised

$124M

Funding rounds

3

Latest round

Series A

Latest activity

Oct 2024