|Atsena Therapeutics Announces Positive Results from Phase I/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-associated Leber Congenital Amaurosis (LCA1)|
|By: GlobenewsWire - 03 Oct 2022||Back to overview list
ATSN-101 demonstrated clinically meaningful improvements in vision with no drug-related serious adverse events
Data presented at the American Academy of Ophthalmology 2022 Annual Meeting
DURHAM, N.C., Oct. 03, 2022 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, announced positive results from the Phase I/II clinical trial of ATSN-101, its lead investigational gene therapy product formerly known as SAR439483, for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1).
The data demonstrated that subretinal delivery of ATSN-101 was well tolerated and patients treated with the highest dose (1.0E11 vg/eye) saw clinically meaningful improvements in vision, as measured by full-field stimulus testing (FST) and multi-luminance mobility testing (MLMT), at more than one-month post treatment.
As of the July 25, 2022, data cut-off date, 15 patients, including three pediatric patients, were treated with ascending doses of ATSN-101. Patients treated with the highest dose (N=9) demonstrated a significantly larger mean change from baseline in retinal sensitivity and a trend toward a larger mean change in best-corrected visual acuity (BCVA) in treated eyes as compared with untreated eyes. In addition, three of four patients tested on MLMT demonstrated at least two-level improvement from baseline light levels. No drug-related serious adverse events were reported, and most treatment-emergent adverse events were mild and transient.
“Patients with LCA1 have profound visual impairment or blindness at birth, but their retinal structure remains intact, which indicates an opportunity to confer meaningful improvements following delivery of a subretinal gene therapy such as ATSN-101,” said Kenji Fujita, MD, Chief Medical Officer of Atsena Therapeutics. “We’re encouraged by these data that demonstrate ATSN-101 improved visual function while maintaining a favorable safety profile. We look forward to launching a pivotal trial for the evaluation of ATSN-101, which will lay the groundwork for successful registration and commercialization. We also look forward to advancing other promising programs in our gene therapy pipeline to reverse or prevent blindness for people with inherited retinal diseases.”
The data were presented on Saturday, Oct. 1, in a Late Breaking Developments session during the Retina Subspecialty Day at the American Academy of Ophthalmology Annual Meeting (AAO 2022) in Chicago, by Christine Nichols Kay, MD, Clinical Ophthalmology Advisor for Atsena.
About GUCY2D-associated Leber congenital amaurosis (LCA1)
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