Axonis Therapeutics gets $115 million to treat pain

Axonis Therapeutics has received $115 million in an oversubscribed series A financing round. CEO and cofounder Joanna Stanicka calls this a huge milestone and says the money will help fund a Phase 1 clinical trial of the firm’s neuropathic pain treatment that is scheduled to begin at the end of the year.

Stanicka started working on neurobiology as a postdoc in Zhigang He’s laboratory at Boston Children’s Hospital Harvard Medical School. There, she and research fellow Shane Hegarty studied ways to repair central nervous system function in people with spinal cord injuries. The pair cofounded Axonis with Corey Goodman, a cofounder of the venture capital firm venBio Partners, and Bob Yant, who had a severe spinal cord injury in 1981 and has raised millions for spinal cord research.

Stanicka says that when it comes to neuropathic pain, γ-aminobutyric acid (GABA) mediated pathways are “one of the best studied and targeted neural pathways.” But despite the successes, she says, many patients do not respond, or they become refractory to treatment.

Stanicka pins the issues with GABA-based medications on the activity of potassium chloride cotransporter 2 (KCC2), located in the membrane of neurons. “An injury to the nervous system often leads to down regulation of KCC2, which leads to this basically imbalance in excitation and inhibition signals,” she says. This imbalance is caused by irregular chloride gradients; without the proper chloride gradients, medications that target GABA won’t be fully effective. That’s why Stanicka set up a KCC2-targeting pipeline at Axonis.

The company’s lead compound, AXN-027, is an oral potentiator of KCC2 that is aimed at restoring GABA signaling in people with chronic pain, spinal cord injuries, and epilepsy.

Axonis says no drugs targeting KCC2 are currently on the market, which is why the company is racing to get its compound into trials. “We have a really ambitious plan for the next 3 years to achieve the proof of concept,” Stanicka says.