FDA clears Atsena Therapeutics' IND application for a Phase I/II clinical trial of ATSN-201

FDA has cleared Atsena Therapeutics' Investigational New Drug (IND) application for a Phase I/II clinical trial of ATSN-201 in patients with X-linked retinoschisis (XLRS). (Image Credit: Adobe Stock/NicoElNino)

Atsena Therapeutics today announced the FDA has cleared the company’s Investigational New Drug (IND) application for a Phase I/II clinical trial of ATSN-201 in patients with X-linked retinoschisis (XLRS).

According to the company, ATSN-201 leverages a novel spreading capsids, AAV.SPR, to overcome the challenges associated with intravitreally delivered AAVs in the treatment of XLRS.

Shannon Boye, PhD, founder and director of Atsena Therapeutics, pointed out in a news release the intravitreally delivered AAVs have limitations, as they do not drive sufficient gene expression in photoreceptors to confer therapy and can lead to vision-compromising inflammation.

“AAV.SPR is well-suited for use in XLRS as it can drive therapeutic levels of gene expression in photoreceptors while avoiding the surgical risks of foveal detachment, which is important because XLRS patients have fragile retinas due to the presence of schisis lesions,” Boye said. “Building on decades of research, we’re excited to progress our novel gene therapy for patients with XLRS who currently lack an approved treatment option.”

Kenji Fujita, chief medical officer of Atsena Therapeutics, pointed out that with this clearance, the company is preparing its first program utilizing AAV.SPR into the clinic for the treatment of XLRS in mid-2023.

“We look forward to evaluating ATSN-201 and addressing the unmet need for a treatment to improve or restore vision in patients with XLRS,” Fujita said.

The Lighthouse Study, a Phase I/II, open-label, dose-escalation clinical trial, will evaluate subretinal injection of ATSN-201 in male patients ages 6-65 with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1.