Positive early safety, efficacy data reported for Leber congenital amaurosis gene therapy
Key takeaways:
- No serious treatment-emergent adverse events were seen at 12 months.
- Patients who received a high dose of ATSN-101 experienced an improvement in best corrected visual acuity.
Atsena Therapeutics reported positive 12-month safety and efficacy data from a phase 1/2 trial of ATSN-101 gene therapy in GUCY2D-associated Leber congenital amaurosis.
Fifteen patients received unilateral subretinal injections of ATSN-101. The trial included three adult cohorts (three patients each) that received ascending doses. One adult cohort and one pediatric cohort (three patients each) subsequently received a high dose of the therapy, with nine patients in total receiving the high dose.
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There were no serious treatment-emergent adverse events at 12 months, according to a press release, and ocular inflammation was mild and reversible with steroid treatment.
In patients who received the high dose, the mean change from baseline in dark-adapted full-field stimulus testing was greater in the treatment eye than the untreated eye at six follow-up visits. Some patients had 10,000-fold improvement in retinal sensitivity. These high-dose patients also demonstrated statistically significant improvement in best corrected visual acuity, the release said.
“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,” Kenji Fujita, MD, chief medical officer of Atsena, said in the release. “We believe the 12-month findings provide solid proof of concept that ATSN-101 will exceed the requirements set by the U.S. Food and Drug Administration for ultimate approval. We are exploring partnering and out-licensing options to advance ATSN-101 into a pivotal trial.”
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