EDIT-101 demonstrates an adequate safety profile across all dosage groups
Primary efficacy signals continuous improvement in BCVA as well as additional efficacy endpoints observed in homozygous patients
Achieve a proof of concept and identify the group of respondents
Due to population shortages, the Company will pause enrollment in the BRILLIANCE trial and will seek to identify a collaboration partner to further develop EDIT-101.
The company is hosting a webinar today at 8:00 AM ET
CAMBRIDGE, MA, Nov. 17, 2022 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported clinical data from the Phase 1/2 BRILLIANCE trial of EDIT-101, and in vivo CRISPR/Cas9 genome-editing medicine at a company-sponsored webinar. EDIT-101 is under development for the treatment of blindness due to Leber congenital disease 10 (LCA10, a CEP290associated with the retinal degenerative disorder) and is designed to repair the mutated IVS26 CEP290 allele affecting approximately 1,500 LCA10 patients in the United States. There is no effective treatment currently available for this rare, serious 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 of the 14 treated subjects met the responder threshold after experiencing clinically meaningful improvements in best-corrected visual acuity (BCVA) (LogMAR > 0.3) and showed consistent improvements in two of the following three additional endpoints: full-field sensitivity test (FST), Visual Functional Navigation Course (VFN), or Visual Quality of Life (VFQ).
Examination of baseline characteristics of patients responding to treatment revealed that two of the three responders were homozygous for the IVS26 mutation (2/2; 100% homozygous patients treated). No other essential characteristics that could pre-select a cohort of respondent patients were identified in the BRILLIANCE data set.
EDIT-101 was tolerated with no serious ocular adverse events or dose-limiting toxicities. Most of the adverse events were mild and expected for a subretinal birth.
Because LCA10 patients homozygous for the CEP290 IVS26 mutation represent an estimated 300 people in the United States, the Company will not offer this program independently, and will seek to identify a collaboration partner to further develop EDIT-101. As a result, Editas Medicine has paused further enrollment in the BRILLIANCE trial and will continue long-term follow-up of all patients treated to date.
“The results of the BRILLIANCE trial provide proof of concept and important learning for our inherited retinal disease programs. We’ve shown that we can safely deliver CRISPR-based gene-editing therapy to the retina with clinically beneficial outcomes,” said Gilmore O’Neill, MB, MMSc., President and CEO of Editas Medicine. “While we will not be advancing 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.”
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 can be accessed through this Webcast linkor through Events and presentations Page of the “Investors” section on the company’s website. The presentation will also be available for download shortly after the webinar.
EDIT-101 is an experimental CRISPR/Cas9-based drug under investigation for the treatment of congenital Leber’s disease 10 (LCA10), by deletion of the IVS26 CEP290 mutant allele. EDIT-101 is given by subretinal injection to access the gene-editing machinery and deliver it directly to photoreceptor cells. EDIT-101 has been awarded the Rare Pediatric Disease and Orphan Drug designations by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) Orphan Medicinal Products designation.
Brilliance Phase 1/2 Clinical Trial of EDIT-101 for the Treatment of Leber Congenital Disease Type 10 (LCA10), A CEP290Associated Degenerative Retinal Disorder, and was designed to evaluate the safety, tolerability, and efficacy of EDIT-101 in up to 34 patients with this disorder. Clinical trial sites are enrolling up to five groups to test up to three dose levels in this open-label, multicenter study. Both adult and pediatric patients (3-17 years of age) with a range of baseline visual acuity assessments are eligible for enrolment. Patients receive a single administration of EDIT-101 by subretinal injection in one eye. Patients are monitored every three months for a year after dosing and less frequently for an additional two years thereafter. Additional details are available at www.clinicaltrials.gov (NCT# 03872479).
About Leber Congenital Amaurosis
Leber Congenital Amaurosis, or LCA, is a group of inherited degenerative disorders of the retina caused by mutations in at least 18 different genes. It is the most common cause of inherited infant blindness, with an incidence of approximately three per 100,000 live births worldwide. Symptoms of LCA appear during the first years of life, leading to significant vision loss and possibly blindness. The most common form of the disease, LCA10 or a CEP290Associated retinal degenerative disorder is a monogenic disorder caused by mutations in the retina CEP290 The gene is the cause of the disease in approximately 20-30 percent of all LCA patients.
about eMedicine Dietas
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 pipeline of therapies for people with critical illnesses around the world. Editas Medicine aims to discover, develop, manufacture and commercialize transformative, robust and precise genomic medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of the Broad Institute and Harvard University’s Cas9 patent drug and holder of the Broad Institute’s Cas12a patent drug for human medicine. For the latest information and scientific 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 cooperation partner to further develop EDIT-101. The words “anticipate,” “believe,” “communicate,” “could,” “estimate,” “anticipate,” “intend,” “may,” “plan,” “possibility,” “anticipate,” and “project,” aim “target,” “should,” “would” and other similar expressions identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these 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 as a result of various important factors, including: uncertainty inherent in initiating and completing preclinical studies and clinical trials, including the BRILLIANCE trial, and clinical development of product candidates company; the ability to establish and maintain cooperation on favorable terms, if any, and the success of any such cooperation into which the Company enters; availability and timing of results from preclinical studies and clinical trials; whether the interim results of the clinical trial are predictive of the final results of the trial or the outcome of future trials; Expectations of regulatory approvals to conduct trials or to market products, availability of adequate funding for the company’s anticipated and unforeseen operating expenses, and capital expenditure requirements. These and other risks are described in more detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, as updated by the Company’s subsequent filings with the Securities and Exchange Commission, and in Other filings the company may file with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date of this release, and the Company expressly disclaims any obligation to update any forward-looking statements, whether due to new information, future events or otherwise.
CONTACT: Contacts: Media Cristi Barnett (617) 401-0113 email@example.com Investors Ron Moldaver (617) 401-9052 firstname.lastname@example.org