Editas Medicine Announces Third Quarter 2024 Results and Business Updates
EDIT 11.04.2024
About Gravity Analytica
Recent News
- 01.15.2025 - 43rd Annual J.P. Morgan Healthcare Conference
- 01.13.2025 - Editas Medicine Highlights New In Vivo Preclinical Proof of Concept Data, Anticipated 2025 Key Milestones, and Three-year Strategic Priorities
- 01.06.2025 - Editas Medicine to Present at the 43rd Annual J.P. Morgan Healthcare Conference
Recent Filings
Achievedin vivopreclinical proof of concept of HBG1/2 editing in hematopoietic stem and progenitor cells (HSPCs) using Editas Medicine’s proprietary targeted LNP in a key step to developing a novelin vivotreatment for sickle cell disease and beta thalassemia
On track to share additional clinical and patient reported outcomes data from 28 patients in the RUBY trial for sickle cell disease at the
Company to provide an update on itsin vivoprogress and pipeline development in 1Q 2025
Company expects the existing cash, cash equivalents, and marketable securities, together with the upfront cash payment from DRI and the retained portions of the payments payable under the license agreement with Vertex, to fund operating expenses and capital expenditures into thesecond quarterof2026
“Achieving preclinical proof of concept of HBG1/2 editing in HSPCs using our proprietary targeted LNP, puts us on a clear path to develop a potentially first- and best-in-classin vivogene edited medicine for the treatment of sickle cell disease and beta thalassemia,” commented Gilmore O’Neill, M.B.,
“I am proud of the Editas team’s work and our advancement in 2024 as we move closer to achieving our vision of becoming a leader inin vivoprogrammable gene editing medicine,” added Dr. O’Neill.
Recent Achievements and Outlook
Ex VivoHemoglobinopathies
- Reni-cel (renizgamglogene autogedtemcel, previously EDIT-301) Partnering/Out-licensing
- The Company has engaged
Moelis & Company LLC , a leading global independent investment bank, to lead the global process to partner or out-license reni-cel.
- The Company has engaged
- Reni-cel for Severe Sickle Cell Disease (SCD)
- The Company will present a substantive RUBY clinical trial update of 28 patients with considerable clinical follow-up at the
American Society of Hematology (ASH) Annual Meeting and Exposition,December 7-10, 2024 .- Dataset will include safety data and efficacy data, including hematology parameters, vaso-occlusive events (VOEs), and patient reported outcomes (PROs).
- The Company continues to dose adult patients in the RUBY trial and has dosed 28 patients to date.
- The Company continues to manufacture drug product for the initial adolescent cohort patients and schedule dosing.
- As previously announced, the Company has completed enrollment of the adult and adolescent cohorts of the Phase 1/2/3 RUBY trial for SCD.
- The Company will present a substantive RUBY clinical trial update of 28 patients with considerable clinical follow-up at the
- Reni-cel for Transfusion-dependent Beta Thalassemia (TDT)
- The Company is on-track to present additional clinical data from the EdiTHAL trial by year-end 2024.
- As previously announced, the Company completed enrollment of the adult cohort of the EdiTHAL trial for TDT and continues to dose patients.
In VivoMedicines
- In vivoPreclinical Proof of Concept Achieved: Hematopoietic Stem and Progenitor Cell (HSPC) Editing
- As disclosed on
October 22 , the Company establishedin vivopreclinical proof of concept for the development of anin vivomedicine for sickle cell disease and beta thalassemia by demonstratingin vivohematopoietic stem and progenitor cell (HSPC) editing of HBG1/2 promoter utilizing a novel, Editas-proprietary targeted lipid nanoparticle (tLNP) for extrahepatic tissue delivery in a humanized mouse model (mice engrafted with human hematopoietic stem cells). - The Company will provide an update on itsin vivoprogress and pipeline development in 1Q 2025.
- The Company continues to pursue anin vivostrategy and approach aimed at driving functional upregulation of gene expression to address loss of function or deleterious mutations.
- As disclosed on
- Additionalin vivoTargets
Editas Medicine and Genevant Sciences entered into a collaboration and nonexclusive license agreement to combine Editas’ CRISPR Cas12a genome editing systems with Genevant’s proprietary LNP technology in the development ofin vivogene editing medicines directed to functional upregulation of two liver targets.
Business Development
- On
October 3, 2024 ,Editas Medicine announced the sale of certain future license fees and other payments owed to the Company under its Cas9 license agreement with Vertex Pharmaceuticals to a wholly owned subsidiary of DRI Healthcare Trust (DRI) for an upfront cash payment of$57 million . The upfront cash payment brings non-dilutive capital toEditas Medicine , helping enable further pipeline development and related strategic priorities. - The Company ended the third quarter 2024 with approximately
$265 .1 million of cash, cash equivalents, and marketable securities, or approximately$322.1 million following receipt of the upfront cash payment from DRI.
Third Quarter 2024 Financial ResultsCash, cash equivalents, and marketable securities as of
Third Quarter 2024
- For the three months ended
September 30, 2024 , net loss attributable to common stockholders was$62 .1 million, or$0.75 per share, compared to net loss of$45 .0 million, or$0.55 per share, for the same period in 2023. - Collaboration and other research and development revenues decreased to
$0 .1 million for the three months endedSeptember 30, 2024 , compared to$5 .3 million for the same period in 2023. The decrease is primarily attributable to the receipt in the 2023 period of the upfront payment for the non-exclusive license toVor Bio . - Research and development expenses increased by
$7 .1 million to$47 .6 million for the three months endedSeptember 30, 2024 , compared to$40 .5 million for the same period in 2023. The increase is primarily related to clinical and manufacturing costs related to the accelerated progression of the Company’s reni-cel program as well as costs attributable toin vivoresearch and discovery. - General and administrative expenses increased by
$3 .1 million to$18 .1 million for the three months endedSeptember 30, 2024 , compared to$15 .0 million for the same period in 2023. The increase is primarily attributable to increased employee-related expenses related to increased headcount to support business operations due to the progression of reni-cel program.
Upcoming Events
American Society of Hematology (ASH) Annual Meeting and ExpositionDecember 7-10, 2024 San Diego, CA
- Guggenheim's
Inaugural Healthcare Innovation Conference November 12, 2024 Boston, MA
- Stifel 2024 Healthcare Conference
November 19, 2024 New York, NY
- 7th Annual
Evercore ISI HealthCONx Conference December 3, 2024 Coral Gables, FL
No 3Q Conference CallThe Company is not hosting a conference call this quarter given it recently held a Strategic Update Webinar on
About
Forward-Looking StatementsThis 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 Company’s intent to partner or out-license reni-cel and any benefits resulting therefrom, the initiation, timing, progress and results of the Company’s preclinical and clinical studies and its research and development programs, the timing for the Company’s receipt and presentation of data from its clinical trials and preclinical studies, including providing an update on itsin vivoprogress and pipeline development in the first quarter of 2025 and presenting additional clinical data from the RUBY trial at the ASH Annual Meeting and Exposition and from the EdiTHAL trial by year-end 2024, the potential of, and expectations for, the Company’s product candidates, including anyin vivogene edited medicines the Company may develop, 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 important factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials, including the RUBY and EdiTHAL trials, and clinical development of the Company’s product candidates, including reni-cel; 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
This press release contains hyperlinks to information that is not deemed to be incorporated by reference in this press release.
| Three Months Ended | Nine Months Ended | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Collaboration and other research and development revenues | $ | 61 | $ | 5,336 | $ | 1,710 | $ | 18,074 | |||||||
| Operating expenses: | |||||||||||||||
| Research and development | 47,639 | 40,512 | 150,636 | 108,095 | |||||||||||
| General and administrative | 18,088 | 14,987 | 55,633 | 55,198 | |||||||||||
| Total operating expenses | 65,727 | 55,499 | 206,269 | 163,293 | |||||||||||
| Operating loss | (65,666 | ) | (50,163 | ) | (204,559 | ) | (145,219 | ) | |||||||
| Other income, net: | |||||||||||||||
| Other income (expense), net | (5 | ) | — | — | (1,590 | ) | |||||||||
| Interest income, net | 3,530 | 5,144 | 12,861 | 12,464 | |||||||||||
| Total other income, net | 3,525 | 5,144 | 12,861 | 10,874 | |||||||||||
| Net loss | $ | (62,141 | ) | $ | (45,019 | ) | $ | (191,698 | ) | $ | (134,345 | ) | |||
| Net loss per share, basic and diluted | (0.75 | ) | (0.55 | ) | (2.33 | ) | (1.81 | ) | |||||||
| Weighted-average common shares outstanding, basic and diluted | 82,485,199 | 81,648,250 | 82,245,679 | 74,029,645 | |||||||||||
| 2024 | 2023 | ||||
| Cash, cash equivalents, and marketable securities | $ | 265,088 | $ | 427,135 | |
| Working capital | 198,786 | 277,612 | |||
| Total assets | 327,567 | 499,153 | |||
| Deferred revenue, net of current portion | 54,204 | 60,667 | |||
| Total stockholders' equity | 175,634 | 349,097 | |||
Media and Investor Contact:Cristi Barnett (617) 401-0113cristi.barnett@editasmed.com
Source: Editas Medicine, Inc.