Editas Medicine Announces First Quarter 2021 Results and Update
Strengthening Leadership by Adding
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
“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
Recent Achievements and Outlook
- EDIT-101 for Leber Congenital Amaurosis 10 (LCA10)
Initial clinical data expected by year-end
Editas Medicineis 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.
- Other Ocular Programs
Preclinical data for USH2A and
RP4presented at ARVO Editas Medicinepresented preclinical data at the Association for Research in Vision and Ophthalmology(ARVO) 2021 Annual Meeting for Usher Syndrome 2A (USH2A) and retinitis pigmentosa 4 ( RP4). For USH2A, the Company demonstrated that gene editing restored protein expression and rescued deficits in photoreceptor morphology in human retinal organoids. For RP4, the Company demonstrated that a dual adeno-associated virus editing system, which simultaneously knocks out and replaces the rhodopsin mutation of RP4, produced clinically relevant editing levels with no off-target editing. The Company expects to declare a development candidate for its RP4program by the end of 2021.
- EDIT-301 for Sickle Cell Disease
On track to dose first patient in RUBY trial by year-end
Editas Medicineis developing EDIT-301 using Cas12a (Cpf1), a proprietary enzyme, as a potentially best-in-class medicine to treat sickle cell disease. The Phase 1/2 RUBY trial for the treatment of sickle cell disease is active and recruitment of patients has started. The Company remains on track to begin patient dosing in the RUBY trial by the end of 2021.
- EDIT-301 for Beta-Thalassemia
On track to file IND by year-end
Editas Medicineis 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.
- Edited iPSC NK (iNK) Cell Medicines to Treat Solid Tumors
Preclinical data presented at AACR
Editas Medicinepresented preclinical data at the American Association for Cancer Research(AACR) 2021 Annual Meeting for its iNK program to treat solid tumors. The data showed that iPSC-derived natural killer (NK) cells, edited with CRISPR/Cas12a to knockout CISH and TGFβR2, demonstrated superior cytotoxicity and enhanced metabolic function in a tumor microenvironment, as well as improved serial killing capacity of certain tumors. These data confirm the expectation of the CISH and TGFβR2 double knockout as a potential superior treatment for solid tumors.
- Alpha-Beta T Cells for Oncology
Bristol Myers Squibb opting into additional program
Editas Medicineand 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.
Today, the Company announced
Mark S. Shearman, Ph.D., will be joining Editas as Chief Scientific Officer, in June 2021. Dr. Shearmanhas more than 30 years of experience in drug discovery and development across multiple therapeutic modalities, and his expertise in neurology, ophthalmology, and immunology will strengthen the Company’s existing pipeline. Dr. Shearmanwill join Editas Medicinefrom Applied Genetic Technologies Corporation (AGTC), where he is currently serving as Chief Scientific Officer and is responsible for leading the company's product candidate selection process, pre-clinical and translational research, and long-term research and development planning. Prior to AGTC, Dr. Shearmanserved as Senior Vice President of Research & Early Developmentat EMD Serono, Inc., the U.S.and Canadian subsidiary of Merck KGaA. Previously, Dr. Shearmanwas Executive Director of Merck & Co. Research Laboratories in Boston, and Senior Director at the Merck Sharp & Dohme Research Laboratories Neuroscience Research Centrein the United Kingdom. Dr. Shearmanearned a B.Sc. from the University of Bristol, U.K., and a Ph.D. from the University of Nottingham, U.K.He also conducted academic research at institutes in Japanand Germany.
Editas Medicinecontinues to advance internal and external manufacturing capabilities for the Company’s portfolio of in vivo gene edited medicines, ex vivo gene edited cell medicines, and cell therapy medicines. The Company is finalizing the preparation of internal manufacturing capabilities for EDIT-301. This capability will provide early phase product for the RUBY trial. Concurrently, the Company has initiated the technology transfer process of EDIT-301 to its strategic CDMO partner Catalent for the later stages of the RUBY trial.
- Offering of Common Stock
Strengthened balance sheet with net proceeds of approximately
$250 million Editas Medicineclosed an underwritten offering of 4,025,000 shares of its common stock at a public offering price of $66.00per share, before deducting underwriter discounts and commissions and estimated offering expenses. This included 525,000 shares issued upon exercise in full by the underwriters of their option to purchase additional shares. All shares in the offering were sold by Editas Medicine.
- Balance Sheet
The Company expects that its existing cash, cash equivalents and marketable securities of
$723.2 millionas 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
For the three months ended
- Collaboration and other research and development revenues were
$6.5 millionfor the three months ended March 31, 2021, compared to $5.7 millionfor 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.
- Research and development expenses increased by
$7.3 million, to $41.9 millionfor the three months ended March 31, 2021, from $34.6 millionfor the same period in 2020. The $7.3 millionincrease was primarily attributable to sublicense and license fees primarily related to success payments under certain of our license agreements, as well as increased manufacturing and clinical related costs, during the first quarter of 2021.
- General and administrative expenses increased by
$3.6 millionto $21.4 millionfor the three months ended March 31, 2021, from $17.8 millionfor the same period in 2020. The $3.6 millionincrease was primarily attributable to increased employee-related expenses.
- 24th Annual Meeting of the
American Society of Gene & Cell Therapy(ASGCT), May 11-14, Virtual
World Medical Innovation Forum, May 20, Virtual European Hematology Association(EHA) 2021 Virtual Congress, June 11, Virtual
Raymond James Human Health Innovation Conference, June 22, Virtual
As a leading genome editing company,
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.
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. Additional details are available on www.clinicaltrials.gov (NCT#03872479).
EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) that targets the HBG1 and HBG2 promoters in the beta-globin locus where naturally occurring fetal hemoglobin (HbF) inducing mutations reside. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in HbF production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.
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).
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 beginning patient dosing in the RUBY trial by the end of 2021, the timing for the Company’s receipt and presentation of data from its clinical trials and preclinical studies, including presenting data from the first two cohorts of the BRILLIANCE trial by the end of 2021, and the timing or likelihood of regulatory filings and approvals, including filing an IND for EDIT-301 for the treatment of beta-thalassemia by the end of 2021. 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 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
|Condensed Consolidated Statements of Operations|
|(amounts in thousands, except per share and share data)|
|Three Months Ended|
|Collaboration and other research and development revenues||$||6,499||$||5,723|
|Research and development||41,937||34,570|
|General and administrative||21,445||17,769|
|Total operating expenses||63,382||52,339|
|Other income, net:|
|Other income (expense), net||21||7,333|
|Interest income, net||134||1,559|
|Total other income, net||155||8,892|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.86||)||$||(0.69||)|
|Weighted-average common shares outstanding, basic and diluted||65,992,395||54,590,194|
|Selected Condensed Consolidated Balance Sheet Items|
|(amounts in thousands)|
|Cash, cash equivalents, and marketable securities||$||723,223||$||511,774|
|Deferred revenue, net of current portion||56,667||73,984|
|Total stockholders’ equity||637,996||393,586|
Source: Editas Medicine, Inc.