EDIT-101 Phase 1/2 BRILLIANCE trial enrolling mid-dose pediatric cohort; clinical data update expected in 2H 2022
EDIT-301 remains on track to dose first sickle cell disease patient in 1H 2022 and first TDT patient in 2022
EDIT-103 for RHO-adRP and EDIT-202 for solid tumors advancing towards IND-enabling studies
CAMBRIDGE, Mass., Feb. 24, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today reported business highlights and financial results for the fourth quarter and full year 2021.
“We’re entering 2022 with an expanded pipeline of clinical programs and new preclinical candidates, enabled by our best-in-class gene editing technologies,” said James C. Mullen, Chairman, President, and Chief Executive Officer, Editas Medicine. “In 2021, we demonstrated the ability to edit in vivo with EDIT-101, supporting continued development and additional ocular opportunities such as EDIT-103 for retinitis pigmentosa, as well as opportunities in other organs. In 2022, we expect to release additional data for EDIT-101, show initial clinical findings for EDIT-301 in sickle cell disease, and begin patient dosing in the beta thalassemia trial. We also expect to begin IND-enabling studies for EDIT-202, the first for our iNK programs.”
Recent Achievements and Outlook
In Vivo Gene Edited Medicines
Ex Vivo Gene Edited Medicines
Cellular Therapy
Corporate
Fourth Quarter and Full Year 2021 Financial Results
Cash, cash equivalents, and marketable securities as of December 31, 2021, were $619.9 million, compared to $657.0 million as of September 30, 2021, and $511.8 million as of December 31, 2020. The Company expects that its existing cash, cash equivalents and marketable securities will enable it to fund its operating expenses and capital expenditures through 2023.
Fourth Quarter 2021
Full Year 2021
Upcoming Events
Editas Medicine plans to participate in the following scientific and medical conferences:
Editas Medicine plans to participate in the following investor events:
Conference Call
The Editas Medicine management team will host a conference call and webcast today at 8:00 a.m. ET to provide and discuss a corporate update and financial results for the fourth quarter and full year 2021. To access the call, please dial 877-407-0989 (domestic) or 201-389-0921 (international) and ask for the Editas Medicine earnings call. A live webcast of the call will also be available on the Investors section of the Editas Medicine website at www.editasmedicine.com, and a replay will be available approximately two hours after its completion.
About Editas Medicine
As a leading 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 treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit www.editasmedicine.com.
About EDIT-101
EDIT-101 is a CRISPR/Cas9-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells. EDIT-101 has been granted Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) and Orphan Designation from the European Medicines Agency (EMA).
About BRILLIANCE
The BRILLIANCE Phase 1/2 clinical trial of EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10) is designed to assess the safety, tolerability, and efficacy of EDIT-101 in up to 18 patients with this disorder. Clinical trial sites are enrolling up to five cohorts testing up to three dose levels in this open label, multi-center study. Both 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 via 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 on www.clinicaltrials.gov (NCT#03872479).
About EDIT-301
EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by a highly specific and efficient proprietary engineered AsCas12a nuclease. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
EDIT-301 is currently being investigated in a clinical study in patients with severe SCD (RUBY trial, NCT04853576). Editas will initiate a new Phase 1/2 study of EDIT-301 in patients with TDT in 2022.
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).
About SLEEK Gene Editing
SLEEK (SeLection by Essential-gene Exon Knock-in) gene editing is an optimized approach to developing the next generation of cell therapy medicines for cancer and other serious diseases. Utilizing Editas Medicine’s proprietary engineered AsCas12a nuclease, SLEEK enables high efficiency, multi-transgene knock-in of induced pluripotent stem cells (iPSCs), T cells, and natural killer (NK) cells while ensuring robust, transgene expression. Editas Medicine is currently leveraging SLEEK technology in its oncology programs.
Forward-Looking Statements
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the initiation, timing, progress and results of the Company’s preclinical and clinical studies and its research and development programs, including completing dosing of the pediatric mid-dose cohort in the first half of 2022, initiating dosing of the pediatric high-dose cohort in the BRILLIANCE trial in 2022, and establishing registrational trial criteria by year-end 2022, dosing the first patient in the RUBY trial in the first half of 2022, dosing the first TDT patient with EDIT-301 in 2022, and declaring an additional new development candidate for an in vivo ocular indication in 2022, the timing for the Company’s receipt and presentation of data from its clinical trials and preclinical studies, including a clinical update on the BRILLIANCE trial in the second half of 2022 and the initial clinical data from the RUBY trial by year-end 2022, the timing or likelihood of regulatory filings and approvals, and the Company’s expectations regarding cash runway. 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 these 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 factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials, including the BRILLIANCE and RUBY trials, and clinical development of the Company’s product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater 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 that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent Company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.
EDITAS MEDICINE, INC.
Consolidated Statement of Operations
(amounts in thousands, except share and per share data)
(Unaudited)
Three Months Ended | Twelve Months Ended | ||||||||||||||||||||||
December 31, | December 31, | ||||||||||||||||||||||
2021 | 2020 | 2021 | 2020 | ||||||||||||||||||||
Collaboration and other research | |||||||||||||||||||||||
and development revenues | $ | 12,469 | $ | 11,419 | $ | 25,544 | $ | 90,732 | |||||||||||||||
Operating expenses: | |||||||||||||||||||||||
Research and development | 37,552 | 61,505 | 142,507 | 157,996 | |||||||||||||||||||
General and administrative | 16,526 | 15,788 | 76,183 | 67,576 | |||||||||||||||||||
Total operating expenses | 54,078 | 77,293 | 218,690 | 225,572 | |||||||||||||||||||
Operating loss | (41,609) | (65,874) | (193,146) | (134,840) | |||||||||||||||||||
Other income, net: | |||||||||||||||||||||||
Other income(expense), net | (325) | 2,853 | (1,698) | 16,259 | |||||||||||||||||||
Interest income, net | 499 | 522 | 2,342 | 2,605 | |||||||||||||||||||
Total other income, net | 174 | 3,375 | 644 | 18,864 | |||||||||||||||||||
Net loss | $ | (41,436) | $ | (62,499) | $ | (192,502) | $ | (115,976) | |||||||||||||||
Net loss per share attributable | |||||||||||||||||||||||
to common stockholders, | |||||||||||||||||||||||
basic and diluted | $ | (0.61) | $ | (1.00) | $ | (2.85) | $ | (1.98) | |||||||||||||||
Weighted-average common | |||||||||||||||||||||||
shares outstanding, basic and | |||||||||||||||||||||||
diluted | 68,355,723 | 62,278,035 | 67,619,388 | 58,609,389 |
EDITAS MEDICINE, INC.
Selected Consolidated Balance Sheet Items
(amounts in thousands)
(Unaudited)
December 31, | December 31, | |||||
2021 | 2020 | |||||
Cash, cash equivalents, and marketable securities | $ | 619,915 | $ | 511,774 | ||
Working capital | 460,426 | 360,879 | ||||
Total assets | 677,483 | 572,602 | ||||
Deferred revenue, net of current portion | 60,888 | 73,984 | ||||
Total stockholders' equity | 553,642 | 393,586 |
Media Contact:
Cristi Barnett
(617) 401-0113
[email protected]
Investor Contact:
Ron Moldaver
(617) 401-9052
[email protected]
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