EDIT-101 demonstrates a favorable safety profile across all dose cohorts
Preliminary efficacy signals of steady improvement in BCVA plus additional efficacy endpoints seen in homozygous patients
Realization of a proof of concept and identification of a population of stakeholders
Given the low population, the Company will suspend recruitment in the BRILLIANCE trial and seek to identify a collaborative partner to pursue the development of EDIT-101
The company will 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 gene editing company, today announced clinical data from the trial of phase 1/2 BRILLIANCE of EDIT-101, a Direct CRISPR/Cas9 genome-editing drug in a company-sponsored webinar. EDIT-101 is being developed for the treatment of blindness due to Leber congenital amaurosis 10 (LCA10, a CEP290retinal degenerative disease) and is designed to repair the mutant IVS26 CEP290 allele that affects approximately 1,500 LCA10 patients in the United States. There is currently no effective treatment for this serious and rare disease. The BRILLIANCE update includes safety and efficacy data from 14 patients treated in the study to date, including 12 adult patients and two pediatric patients.
Three 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 testing (FST), vision course navigation function (VFN), or visual function quality of life (VFQ).
A review of the baseline characteristics of treatment responder patients revealed that two of the three responders were homozygous for the IVS26 mutation (2/2; 100% of treated homozygous patients). No other baseline characteristics that could preselect a responder patient population were identified in the BRILLIANCE dataset.
EDIT-101 was tolerated with no serious ocular adverse effects or dose-limiting toxicity observed. Most adverse events were mild and expected for subretinal delivery.
Given that LCA10 patients homozygous for the CEP290 IVS26 mutation represent an estimated population of approximately 300 in the United States, the Company will not advance this program independently and will seek to identify a collaborative partner to pursue the development of EDIT -101. Consequently, Editas Medicine is suspending recruitment in the BRILLIANCE trial and will continue the long-term follow-up of all patients treated to date.
“The results of the BRILLIANCE trial provide important proof of concept and lessons for our inherited retinal disease programs. We have demonstrated that we can safely deliver CRISPR-based gene-editing therapy to the retina and achieve clinically meaningful results,” said Gilmore O’Neill, MB, MMSc., President and CEO of ‘Editas Medicine. “While we are not progressing through EDIT-101 alone and have made the decision to suspend enrollment, we have the patient community in mind and are seeking a collaborative partner to move this agenda forward.”
Editas Medicine will host a webinar today, Thursday, November 17, at 8:00 a.m. ET to present the data. The live and archived webcast of the presentation will be accessible via this webcast link or via the Events and Presentations page in the “Investors” section of the Company’s website. The presentation will also be available for download shortly after the webinar.
EDIT-101 is an investigational CRISPR/Cas9-based drug being investigated for the treatment of Leber congenital amaurosis 10 (LCA10), by deleting the mutant IVS26 CEP290 allele. EDIT-101 is administered via a subretinal injection to reach and deliver gene editing machinery directly to photoreceptor cells. EDIT-101 has received Rare Pediatric Disease and Orphan Drug Designations from the United States Food and Drug Administration (FDA) and Orphan Medical Product Designation from the European Medicines Agency (EMA).
The BRILLIANCE Phase 1/2 clinical trial of EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290related retinal degenerative disease, is designed to evaluate the safety, tolerability and efficacy of EDIT-101 in up to 34 patients with this disease. Clinical trial sites are recruiting up to five cohorts testing up to three dose levels in this open-label, multicenter study. Adult and pediatric patients (3-17 years old) with a range of baseline visual acuity assessments are eligible for enrollment. Patients receive a single administration of EDIT-101 by subretinal injection into one eye. Patients are monitored every three months for one year after administration and less frequently for two additional years thereafter. Additional details are available at www.clinicaltrials.gov (NCT#03872479).
About Leber’s congenital amaurosis
Leber congenital amaurosis, or ACL, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of approximately three per 100,000 live births worldwide. Symptoms of ACL appear during the first years of life, leading to significant vision loss and potentially blindness. The most common form of the disease, LCA10 or a CEP290related retinal degenerative disease, is a monogenic disease caused by mutations in the CEP290 gene and is the cause of the disease in approximately 20-30% of all patients with ACL.
As a clinical-stage genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome-editing systems into a robust portfolio of treatments for sufferers. serious illnesses around the world. Editas Medicine aims to discover, develop, manufacture and commercialize transformative, sustainable and precision genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of the Broad Institute and Harvard University Cas9 patent domains and the Broad Institute Cas12a patent domain for human medicines. For the latest scientific information and presentations, please visit www.editasmedicine.com.
This press release contains forward-looking statements and information within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s plans to seek a collaborative partner to further develop EDIT-101. The words “anticipate”, “believe”, “continue”, “could”, “estimate”, “expect”, “intend”, “may”, “plan”, “potential”, ” predict”, “project”, “target”, “should”, “should” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain such identifying words. The Company may not actually achieve the plans, intentions or expectations disclosed in such forward-looking statements, and you should not place undue reliance on such forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements due to various important factors, including: the uncertainties inherent in the initiation and completion of preclinical studies and clinical trials, including the BRILLIANCE trial, and the clinical development of the Company’s product candidates; the ability to establish and maintain a collaboration on favorable terms, if any, and the success of any such collaboration entered into by the Company; the availability and timing of results from preclinical studies and clinical trials; whether the interim results of a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations regarding regulatory approvals to conduct trials or to commercialize products and the availability of sufficient funding for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure needs. These and other risks are described in greater detail under the heading “Risk Factors” included in the company’s most recent Annual Report on Form 10-K, which is filed with the Securities and Exchange Commission, as updated. by the Company’s subsequent filings with the Securities and Exchange Commission, and in other documents that the Company may file with the Securities and Exchange Commission in the future. All forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or other.
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