Strengthening Leadership by Adding Mark S. Shearman, Ph.D., as Chief Scientific Officer
Advancing BRILLIANCE trial of EDIT-101 for LCA10; clinical data expected by year-end
RUBY trial of EDIT-301 for sickle cell disease active and recruiting
Preclinical ocular data presented at ARVO supports in vivo gene editing
Cash, cash equivalents, and marketable securities of $723 million as of March 31, 2021
CAMBRIDGE, Mass., May 05, 2021 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, today reported business highlights and financial results for the first quarter of 2021.
"Our team is making tremendous progress towards discovering, developing, and manufacturing novel genome editing medicines, including excellent forward momentum this quarter for our two clinical-stage medicines," said James C. Mullen, Chairman, President and Chief Executive Officer, Editas Medicine. "We are pleased to announce Dr. Mark Shearman will be joining Editas as Chief Scientific Officer in June 2021. Mark has an outstanding track record of drug discovery and development, and we are confident that his expertise will help us achieve our near-term milestones and continue to realize the promise of bringing transformative medicines to patients."
Mr. Mullen continued, "In ocular, we advanced the BRILLIANCE trial of EDIT-101 for LCA10 and have line of sight for initiating the next cohort. We also presented preclinical data at ARVO that further validates our in vivo gene editing platform in USH2A and RP4. In ex vivo, we advanced the RUBY trial of EDIT-301 for sickle cell disease, including activating sites for recruitment and finalizing preparations to produce study drug material for dosing patients in the trial. We look forward to maintaining the momentum through the remainder of the year and expect to share Editas' first clinical data for EDIT-101 by year-end."
Recent Achievements and Outlook
In Vivo Gene Edited Medicines
- EDIT-101 for Leber Congenital Amaurosis 10 (LCA10)
Initial clinical data expected by year-end
Editas Medicine is progressing the Phase 1/2 BRILLIANCE trial for the treatment of LCA10. The Company is continuing to dose patients in the second cohort, the adult mid-dose, and results of this cohort will inform the path to initiate dosing of pediatric patients. Clinical data from the first two cohorts is expected to be presented by the end of 2021.
Ex Vivo Gene Edited Medicines
- EDIT-301 for Beta-Thalassemia
On track to file IND by year-end
Editas Medicine is confirming the sustained effects of EDIT-301 on cells derived from beta-thalassemia patients. The Company remains on track to file an investigational new drug application for EDIT-301 for the treatment of beta-thalassemia by the end of 2021.
Cellular Therapy
- Alpha-Beta T Cells for Oncology
Bristol Myers Squibb opting into additional program
Editas Medicine and Bristol Myers Squibb continue to advance alpha-beta T cell medicines for the treatment of solid and liquid tumors. Bristol Myers Squibb recently opted into an additional gene editing program, further validating the Company's technology and expertise in engineered cell medicines.
Corporate
- Balance Sheet
The Company expects that its existing cash, cash equivalents and marketable securities of $723.2 million as of March 31, 2021, and anticipated interest income will enable it to fund its operating expenses and capital expenditures well into 2023.
First Quarter for 2021 Financial Results
Cash, cash equivalents, and marketable securities as of March 31, 2021, were $723.2 million, compared to $511.8 million as of December 31, 2020.
For the three months ended March 31, 2021, net loss attributable to common stockholders was $56.7 million, or $0.86 per share, compared to $37.7 million, or $0.69 per share, for the same period in 2020.
- Collaboration and other research and development revenues were $6.5 million for the three months ended March 31, 2021, compared to $5.7 million for the same period in 2020. The majority of the revenue recognized this quarter was attributable to the Company's strategic alliance with Bristol Myers Squibb.
Upcoming Events
Editas Medicine plans to participate in the following scientific and medical conferences:
- 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), May 11-14, Virtual
- 2021 World Medical Innovation Forum, May 20, Virtual
- European Hematology Association (EHA) 2021 Virtual Congress, June 11, Virtual
Editas Medicine plans to participate in the following investor event:
- Raymond James Human Health Innovation Conference, June 20, Virtual
About EDIT-101
EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.
About RUBY
The RUBY Trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in patients with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301. Additional details are available on www.clinicaltrials.gov (NCT#04853576).
Contacts:
Media
Cristi Barnett
(617) 401-0113
[email protected]
Investors
Ron Moldaver
(617) 401-9052
[email protected]
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