Editas Medicine (EDIT) Announces Positive Initial Clinical Data From Ongoing Phase 1/2 BRILLIANCE Clinical Trial of EDIT-101 for LCA10

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Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced positive initial clinical data from the ongoing, open-label phase 1/2 BRILLIANCE clinical trial of ‘EDIT-101. EDIT-101 is under development for the treatment of blindness due to congenital Leber 10 amaurosis (LCA10), a CEP290– degenerative disease of the related retina. The data, including preliminary patient safety and efficacy assessments relating to potential clinical benefits, are presented today in an oral presentation at the 19th International Symposium on Retinal Degeneration (RD2021) by Dr. Mark Pennesi, MD, Ph.D.

“The preliminary results shared today support our belief that EDIT-101 has the potential to bring significant benefits to people living with it. CEP290– linked retinal degeneration or LCA10. A positive safety profile has been observed for up to 15 months, with mostly mild adverse events primarily related to the retinal injection procedure. The safety profile allowed us to begin recruiting and treating subjects in both the adult high dose cohort and the pediatric medium dose cohort. The first observations of individuals who were treated in the medium dose cohort show clinical evidence that gene editing has occurred, demonstrated by visual improvements, as measured by the light sensitivity threshold test. (FST) full field, best corrected visual acuity (BCVA) or improvement in their ability to navigate in standardized navigation courses with different levels of difficulty. We will continue to follow trial participants prospectively and collect clinical metrics to enable us to determine the extent of continuous and lasting improvements, ”said Lisa Michaels, MD, executive vice president and medical director of Editas Medicine. “I would like to thank the participants, clinicians and collaborating institutions who continue to contribute to this landmark clinical trial of gene editing medicine. ”

“I am encouraged by these early results, which indicate that this experimental gene-editing treatment has been well tolerated by participants in this trial so far and may also help improve eyesight for people with mutations in the CEP290 uncomfortable. Being able to edit genes inside the human body is incredibly deep, and I hope to be able to offer my ACL patients new treatment options involving gene editing in the future, ”said Mark Pennesi, MD, Ph.D., Professor of Molecular Biology and Medical Genetics, Full Professor of Ophthalmology Kenneth C. Swan, Head of Division of Ophthalmic Genetics Paul H. Casey, Casey Eye Institute, Oregon Health & Science University, and Principal Investigator by BRILLIANCE.

Dr Michaels added: “These encouraging results provide proof of concept on our in vivo gene editing platform and increase Editas’ confidence in the vast potential of our gene editing technology to treat other serious diseases.

Preliminary resultsPreliminary results include safety and efficacy data from the first two cohorts, the low dose adult cohort (6×1011 vg / ml) and the medium dose adult cohort (1.1×1012 vg / ml). The BCVA eligibility criteria for the low dose adult cohort (n = 2) are light perception (LP), black-white discrimination, and white field projection. For mid-dose adults and all other cohorts, the first subject should have a light perception of BCVA of 1.6 logMAR (20/800 Snellen). The BCVA criteria for all of the following subjects are LP to 0.4 logMAR (20/50 Snellen).

SecuritySafety data were reported for all six subjects treated in the low dose (n = 2) and medium dose (n = 4) cohorts. Most of the adverse events (AEs) were mild and mainly resulted from the surgery and subretinal injection. There was no dose-limiting toxicity (DLT) defined as vision-threatening toxicity or severe non-ocular AE that occurs before or during the week 4 visit and is assessed by the investigator to be related to EDIT-101 and not to the administration procedure. Mild inflammation of the anterior chamber has been observed and adequately controlled with oral steroids. No Cas9 specific antibody or T cell response was detected. To date, no treatment-related cataracts, edema or retinal thinning have been observed.

Efficiency Efficacy was assessed based on available data from five subjects treated in the low-dose adult cohort (n = 2) and the medium-dose adult cohort (n = 3), who had at least three months of follow-up. post-processing, focusing on those measurements that have been shown to be consistent and reproducible in subjects CEP290 retinal degeneration, including: BCVA, FST and Visual Function Navigation (VNC ™, developed by Ora, Inc.).

Two of three subjects in the mid-dose cohort followed for up to six months showed signs of efficacy suggesting productive editing and providing initial support for clinical benefits, including improvements in BCVA, FST, and / or navigation in mobility. BCVA is assessed using the Diabetic Retinopathy Early Treatment Study (ETDRS) eye chart, or the Berkeley rudimentary vision test (BRVT), or the Lea Symbols 15-line pediatric eye chart. The FST measures the sensitivity of the entire visual field by testing the lowest luminance flash that causes visual sensation. Patients are given blue, red, and white stimuli, and based on the blue-red difference, the experimenter can determine the sensitivity of the perceptions mediated by the rods and cones. Sailing abilities were assessed using standardized mobility tests with four different sailing routes, designed with different levels of difficulty.

Mid-Dose Cohort Subject 1 showed an improvement in BCVA of approximately 0.7 logMAR at month 1.5 which was maintained at month 6 of follow-up. In addition, there was a positive trend for improved retinal sensitivity by FST in the study eye compared to the untreated eye. The subject also demonstrated a 5-level improvement in mobility at month 6, as assessed with the VNC.

Mid-dose cohort Subject 2 showed improvement at month 3 with stable BCVA and noticeable improvement in retinal sensitivity in the study eye compared to the non-FST-treated eye, detectable at month 1, 5 which continued to improve until month 3.

In June, the Independent Data Monitoring Committee (IDMC) approved the continuation of the first pediatric cohort based on a review of clinical safety data from the low-dose adult and mid-dose adult cohorts. Treatment in the adult high dose cohort continues and the pediatric mid dose cohort begins.

Event information for investors and webcastEditas Medicine will host a live webcast today, Wednesday, September 29, 2021, at 11:00 a.m.ET to review the data presented. To participate in the webcast, please visit this connect or visit the Events and presentations page in the Investors section of the Company’s website. A replay of the webcast will be available on the Editas Medicine website for 30 days after the call.

About EDIT-101 EDIT-101 is an investigational CRISPR-based drug under investigation for the treatment of congenital Leber 10 amaurosis (LCA10). EDIT-101 is administered via subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.

About BRILLANCEThe BRILLIANCE Phase 1/2 clinical trial of EDIT-101 for the treatment of congenital Leber 10 amaurosis (LCA10), a CEP290-degenerative disease of the related retina, is designed to evaluate the safety, tolerability and efficacy of EDIT-101 in up to 18 patients with this disease. Clinical trial sites are recruiting up to five cohorts to test up to three dose levels in this open, multi-center study. Adult and pediatric patients (ages 3 to 17) 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 an additional two years thereafter. Further details are available at www.clinicaltrials.gov (NCT # 03872479).

About Leber’s congenital amaurosisLeber’s congenital amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of hereditary childhood blindness, with an incidence of about three per 100,000 live births worldwide. Symptoms of stroke appear in the first few years of life, leading to significant vision loss and potentially blindness. The most common form of the disease, LCA10 or a CEP290degenerative retinal related disease, is a monogenic disease caused by mutations in the CEP290 gene and is the cause of disease in about 20 to 30 percent of all patients with ACL.


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