NEW YORK – Atsena Therapeutics is looking to advance its investigational gene therapy ATSN-101 into a pivotal trial after an ongoing Phase I/II study yielded positive findings, the company said Tuesday.
The therapy, a recombinant adeno-associated virus (AAV) vector that carries the GUCY2D gene, is designed to treat patients with Leber congenital amaurosis (LCA1). Patients with this rare and inherited monogenic eye disorder, caused by mutations in the GUCY2D gene, have severely impaired vision and can even be blind.
At a medical meeting Tuesday, Durham, North Carolina-based Atsena presented six-month findings from an ongoing Phase I/II trial, in which LCA1 patients received ascending doses of ATSN-101 via subretinal injection. Patients who received ATSN-101 at the highest dose achieved clinically meaningful vision improvements six months after treatment, the company presented at the Association for Research in Vision and Ophthalmology 2023 annual meeting in New Orleans. Among nine patients who received a high-dose treatment in the trial, the mean change from baseline in retinal sensitivity by dark-adapted full-field stimulus testing was significantly greater in eyes treated with ATSN-101 compared to untreated eyes.
"The latest data reinforce our confidence in the potential of ATSN-101 to improve vision in patients with GUCY2D-associated LCA1," Atsena Chief Medical Officer Kenji Fujita said in a statement. "We look forward to reporting 12-month data later this year and are exploring options to advance ATSN-101 into a pivotal trial."
Study participants to date have reported no drug-related serious adverse events. Ocular inflammation has been infrequent and reversible with steroid treatment, according to the company.