Atsena Therapeutics Announces 12-Month Safety and Efficacy Data from Phase 1/2 Trial of ATSN-101 in Patients with LCA1
Atsena Therapeutics announced positive 12-month safety and efficacy data from the ongoing phase 1/2 trial of ATSN-101, the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). At 12 months post-treatment, ATSN-101 has conferred clinically meaningful improvements in vision at the highest dose with no serious treatment-emergent adverse events, according to Atsena.
“We continue to be encouraged by the data emerging from our phase 1/2 trial of ATSN-101 in patients with GUCY2D-associated LCA1," Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics, said in a company news release. “The durability of clinically meaningful visual improvements in the absence of serious treatment-related adverse events at the 12-month mark underscore the safety, tolerability, and efficacy of our subretinal gene therapy. We believe the 12-month findings provide solid proof of concept that ATSN-101 will exceed the requirements set by the FDA for ultimate approval. We are exploring partnering and out-licensing options to advance ATSN-101 into a pivotal trial.”
In the phase 1/2 trial, 15 patients ages 12 to 76 received unilateral subretinal injections of ATSN-101. Three adult cohorts (N=3 each) were treated with three ascending doses. Subsequently, one adult and one pediatric cohort (N=3 each) were treated at the high dose. In total, 9 patients received the high dose.
At 12 months, there were no serious treatment-emergent adverse events. Ocular inflammation was mild and reversible with steroid treatment. For high-dose patients, the mean (SE) change from baseline in dark-adapted full-field stimulus testing (FST) (white stimulus) was greater in treated eyes compared with untreated eyes at all six follow-up visits, and some patients exhibited over 10,000-fold improvements in retinal sensitivity. Of the six high-dose patients who were tested with multi-luminance mobility testing (MLMT), three improved by ≥2 levels compared to baseline (when available) or to the untreated eye, and all three demonstrated a maximum score of 6 in the treated eye. At 12 months, patients receiving the high-dose demonstrated a statistically significant improvement in best-corrected visual acuity (BCVA).
“The noteworthy improvements in key visual parameters demonstrate the potential of ATSN-101 to make a meaningful impact on the lives of patients affected by GUCY2D-associated LCA1,” said Paul Yang, MD, PhD, Associate Professor of Ophthalmology at OHSU School of Medicine. “As LCA1 causes early and severe vision impairment or blindness and there are no approved treatments, ATSN-101 has the potential to fill a significant unmet need within the LCA community.”
The 12-month data have been accepted for presentation at the 47th Annual Macula Society Meeting, which will be held February 7-10, 2024, in Palm Springs, CA.
The FDA recently granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101. Atsena has also received orphan drug designation from the FDA for ATSN-101 for the treatment of LCA1.