Editas Medicine Announces Clinical Data Demonstrating Proof of Concept of EDIT-101 from Phase 1/2 BRILLIANCE Trial
EDIT-101 demonstrates a favorable safety profile across all dose cohorts
Preliminary efficacy signals of consistent improvement in BCVA plus additional efficacy endpoints seen in homozygous patients
Achieved proof of concept and identified a responder population
In view of the small population, the Company will pause enrollment in the BRILLIANCE trial and seek to identify a collaboration partner to continue development of EDIT-101
Company to host a webinar today at 8:00 a.m. ET
CAMBRIDGE, Mass., Nov. 17, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome editing company, today announced clinical data from the Phase 1/2 BRILLIANCE trial of EDIT-101, an in vivo CRISPR/Cas9 genome editing medicine in a Company-sponsored webinar. EDIT-101 is under development for the treatment of blindness due to Leber congenital amaurosis 10 (LCA10, a CEP290-related retinal degenerative disorder) and is designed to repair the IVS26 CEP290 mutant allele that impacts approximately 1,500 LCA10 patients in the U.S. There is no effective treatment currently available for this serious, rare disease. The BRILLIANCE update includes safety and efficacy data from all 14 patients treated in the study to date, which includes 12 adult patients and two pediatric patients.
Three out of 14 treated subjects met a responder threshold having experienced clinically meaningful improvements in best corrected visual acuity (BCVA) (LogMAR >0.3) and demonstrated consistent improvements in two of the following three additional endpoints: full field sensitivity test (FST), visual function navigation course (VFN), or the visual function quality of life (VFQ).
An examination of baseline characteristics of the treatment responder patients revealed that two of the three responders were homozygous for IVS26 mutation (2/2; 100% of the homozygous patients treated). No other baseline characteristics that could pre-select a responder patient population were identified in the BRILLIANCE dataset.
EDIT-101 was tolerated with no ocular serious adverse events or dose-limiting toxicities observed. Most adverse events were mild and expected for subretinal delivery.
Since LCA10 patients homozygous for CEP290 IVS26 mutation represent an estimated population of approximately 300 in the U.S., the Company will not progress this program independently, and will seek to identify a collaboration partner to continue the development of EDIT-101. As a result, Editas Medicine is pausing further enrollment in the BRILLIANCE trial and will continue long term follow-up of all patients who have been treated to date.
“The results from the BRILLIANCE trial provide a proof of concept and important learnings for our inherited retinal disease programs. We’ve demonstrated that we can safely deliver a CRISPR-based gene editing therapeutic to the retina and have clinically meaningful outcomes,” said Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. “While we will not progress EDIT-101 on our own and have made the decision to pause enrollment, we have the patient community top of mind and are looking for a collaboration partner to advance this program.”
About Leber Congenital Amaurosis
About Editas Medicine
Contacts: Media Cristi Barnett (617) 401-0113 [email protected] Investors Ron Moldaver (617) 401-9052 [email protected]
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