Editas Medicine, Inc. (NASDAQ:EDIT) is a clinical-stage biotechnology company at the forefront of the gene editing revolution. The company is dedicated to developing potentially transformative genomic medicines to treat a broad range of serious diseases. With a robust pipeline, strategic partnerships, and a strong intellectual property portfolio, Editas is well-positioned to capitalize on the immense potential of gene editing technologies.
Business Overview
Editas' lead program, renizgamglogene autogedtemcel (reni-cel), formally known as EDIT-301, is an experimental ex vivo gene-edited medicine to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The company is investigating reni-cel in a single Phase 1/2/3 clinical trial, referred to as the RUBY trial, for the treatment of severe SCD. In 2022, Editas dosed the first patient in the RUBY trial, and after completing sequential dosing of the first two patients, the company commenced concurrent patient dosing in the first quarter of 2023. Editas has completed enrollment of the adult cohort and continues to dose patients in the RUBY trial. The company has also enrolled multiple patients in the adolescent cohort in the trial.
In December 2021, the U.S. Food and Drug Administration (FDA) cleared Editas' Investigational New Drug (IND) application for a Phase 1/2 clinical trial of reni-cel for the treatment of TDT, which the company refers to as the EdiTHAL trial. Editas dosed the first patient in this trial in the first quarter of 2023 and commenced concurrent patient dosing in the second quarter of 2023. The company continues to enroll and dose patients in the trial.
In December 2023, Editas presented new safety and efficacy data in 17 patients treated with reni-cel in both the RUBY and EdiTHAL trials. This clinical data, which remained consistent with and further confirmed earlier clinical results, supports the company's belief that reni-cel can be a clinically differentiated, one-time, durable medicine that can provide life-changing clinical benefits to patients with SCD and TDT, specifically driving early and robust correction of anemia and sustained increases in fetal hemoglobin. Editas remains on track to present additional clinical data from both the RUBY and EdiTHAL trials in mid-2024 and further data by year-end 2024.
In addition to its ex vivo hemoglobinopathy program, Editas is pursuing the development of next-generation in vivo administered gene editing medicines. The company is initially focused on editing hematopoietic stem cells (HSCs) through targeted delivery of its AsCas12a enzyme to the clinically validated HBG1 and HBG2 promotor site. Editas' internal development efforts leverage the indel CRISPR technology used to upregulate gamma globin expression in its ex vivo reni-cel program. The company's in vivo approach is aimed at functional upregulation of gene expression in genetic diseases, starting with rare and orphan disease patient populations, with plans to expand to more common disease populations.
Editas is evaluating lipid nanoparticles for delivery of gene editing cargo into multiple tissue types with multiple companies, and is also evaluating additional, next-generation delivery technologies. The company is on track to establish in vivo preclinical proof-of-concept for an undisclosed indication by year-end 2024.
Strategic Partnerships
Editas' strategy also involves leveraging strategic partnerships and collaborations to accelerate its drug discovery and clinical execution objectives. In December 2023, the company entered into a license agreement with Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX), under which Vertex obtained a non-exclusive license for Editas' Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Vertex's CASGEVY™ (exagamglogene autotemcel). Editas received a $50.0 million upfront cash payment in the fourth quarter of 2023 and the 2024 annual license fee of $10.0 million in the first quarter of 2024. The company is also eligible to receive an additional $50.0 million contingent upfront payment, as well as further annual license fees, ranging from $10.0 million to $40.0 million annually, inclusive of certain sales-based annual license fee increases, through 2034.
In the cellular therapy space, Editas is advancing alpha-beta T-cell experimental medicines for the treatment of solid and liquid tumors in collaboration with Bristol Myers Squibb Company (NYSE: BMY) through its wholly owned subsidiary, Juno Therapeutics, Inc. This collaboration, which leverages Editas' Cas9 and AsCas12a platform technologies, has resulted in 13 total programs. In March 2024, Editas entered into an amendment to extend the collaboration to November 2026, with options to extend the collaboration for up to an additional two years, and provided Bristol Myers Squibb the ability to select up to three new gene targets for research.
Financials
Editas' financial performance in the first quarter of 2024 reflects the company's continued investment in its clinical programs and pipeline development. For the quarter, the company reported a net loss of $62.0 million, compared to a net loss of $49.0 million in the same period of 2023. Collaboration and other research and development revenues decreased to $1.1 million in the first quarter of 2024, down from $9.9 million in the same period of the prior year, primarily due to the sale of the company's oncology assets and related licenses in January 2023.
Research and development expenses increased to $48.8 million in the first quarter of 2024, up from $37.8 million in the same period of 2023, driven by additional clinical and manufacturing costs to support the continued progression of the reni-cel program, as well as one-time payments related to sublicense and license obligations. General and administrative expenses decreased to $19.3 million in the first quarter of 2024, down from $23.0 million in the same period of the prior year, primarily due to a reduction in one-time professional service expenses and lower legal and patent costs.
Liquidity
As of March 31, 2024, Editas held $376.8 million in cash, cash equivalents, and marketable securities, down from $427.1 million at the end of 2023. The company expects its existing cash, cash equivalents, and marketable securities, together with the near-term annual license fees and the contingent upfront payment payable under its license agreement with Vertex, to fund its operating expenses and capital expenditure requirements into 2026.
Intellectual Property
Editas' robust intellectual property portfolio, including exclusive licenses to important CRISPR patents from the Broad Institute and Harvard, enhances the company's capacity to build and market new gene-editing treatments. The company's ongoing legal proceedings related to CRISPR patents have been a focus, with oral arguments held before the U.S. Court of Appeals for the Federal Circuit in March 2024 regarding an appeal of the Patent Trial and Appeal Board's (PTAB) previous decision favoring the Broad Institute. Editas expects a decision on the case in the second half of 2024.
Conclusion
In summary, Editas Medicine is a clinical-stage gene editing company with a promising pipeline, strategic partnerships, and a strong intellectual property position. The company's lead program, reni-cel, is advancing through late-stage clinical trials for SCD and TDT, with the potential to provide life-changing benefits to patients. Editas is also making significant progress in developing its in vivo gene editing capabilities, positioning the company to address a wide range of genetically determined diseases. With a robust balance sheet and a clear strategic focus, Editas is well-equipped to continue its transformation into a commercial-stage gene editing leader and deliver on its mission of developing transformative genomic medicines.