|View printer-friendly version|
EDIT-101 for LCA10 set to be first ever in vivo CRISPR medicine administered to patients with dosing expected in second half of 2019
Advancing potentially best-in-class engineered cell medicine for
sickle cell disease and beta-thalassemia
and named Collins as interim Chief Executive Officer
Year-end cash, cash equivalents, and marketable securities of
“2018 was a year of significant achievements for
Recent Achievements and Outlook
- EDIT-101 Investigational New Drug (IND) application accepted upon initial review with patient dosing planned for second half of 2019.
The U.S. Food and Drug Administrationaccepted the IND application in November 2018following its initial 30-day review. As a result of the IND acceptance, Editas Medicinereceived a $25 millionmilestone payment from Allergan Pharmaceuticals International Limited( Allergan) as part of the alliance between the companies to discover and develop ocular medicines targeting serious, vision-threatening diseases. Editas Medicineand Allerganexpect to enroll 10-20 patients in a Phase 1/2 open label, dose escalation study to evaluate the safety, tolerability, and efficacy of EDIT-101 as a treatment for Leber congenital amaurosis 10 (LCA10). EDIT-101 is expected to be the first in vivo, or editing inside the body, CRISPR medicine administered to patients in history.
- Advancing research program to address Usher syndrome 2A (USH2A). The Company and collaborators from Massachusetts Eye and Ear plan to present in vivo proof-of-concept data for a CRISPR gene editing approach for USH2A at the 22nd Annual Meeting of the
American Society of Gene & Cell Therapy.
- Potential for best-in-class engineered cell medicine for sickle cell disease and beta-thalassemia.
Editas Medicineexpects to begin IND-enabling activities for an experimental medicine candidate for sickle cell disease and beta-thalassemia in 2019. At the 60th Annual Meeting of the American Society of Hematology, the Company presented data on its differentiated approach to developing a durable genomic medicine for sickle cell disease and beta-thalassemia. In these pre-clinical mouse data, Editas Medicineshowed that editing the beta-globin locus upregulated fetal hemoglobin with superior repopulation of red blood cell precursors as compared to editing the BCL11A erythroid enhancer.
- Expanding investment in engineered cell medicines for cancer. In addition to the Company’s collaboration with Juno Therapeutics, a
Celgenecompany and wholly-owned subsidiary of Celgene Corporation(Juno Therapeutics), to develop engineered T cell medicines to treat cancer, Editas Medicineis also advancing wholly-owned research programs to develop allogeneic engineered natural killer cell medicines in oncology.
- Preparing organization for the next phase of growth.
Cynthia Collins, a recognized leader with more than 30 years of experience in cell and gene therapy, molecular diagnostics, life sciences tools, and therapeutics, was appointed to the Board of Directors (Board) in December 2018and named interim Chief Executive Officer in January 2019. The Board has retained a leading executive search firm to assist in identifying a permanent successor. In addition, David Scadden, M.D., professor of medicine at Harvard Universityand director of the Center for Regenerative Medicineat Massachusetts General Hospital, was appointed to the Board in February 2019.
- Strong balance sheet to fund business through multiple important milestones. The Company held cash, cash equivalents, and marketable securities of
$369 millionas of December 31, 2018, providing at least 24 months of funding for operating expenses and capital expenditures.
Cowen & Company39th Annual Health Care Conference, March 11, 4:50 p.m. ET, Boston; Barclays Global Healthcare Conference, March 14, 11:15 a.m. ET, Miami
- 6th Annual Retinal Cell and Gene Therapy Innovation Summit,
April 26, Vancouver;
- 22nd Annual Meeting of the
American Society of Gene & Cell Therapy, April 29-May 2, Washington, DC.
Fourth Quarter and Full Year 2018 Financial Results
Cash, cash equivalents, and marketable securities at
For the three months ended
- Collaboration and other research and development revenues were
$6.1 millionfor the three months ended December 31, 2018, compared to $3.7 millionfor the same period in 2017. The $2.4 millionincrease was primarily due to $1.6 millionin increased revenue recognized pursuant to our strategic alliance with Allerganand $0.8 millionin increased revenue recognized pursuant to our collaboration agreement with Juno Therapeutics.
- Research and development expenses decreased by
$7.2 million, to $19.2 millionfor the three months ended December 31, 2018, from $26.4 millionfor the same period in 2017. The $7.2 milliondecrease was related to $9.5 millionin decreased success payment expenses, $2.7 millionin decreased stock-based compensation expenses and $0.9 millionin decreased process and platform development expenses, partially offset by $3.8 millionin increased sublicense payment expenses, $1.3 millionin increased employee related expenses and $0.8 millionin increased other expenses including facility-related expenses.
- General and administrative expenses decreased by
$0.5 million to $13.2 millionfor the three months ended December 31, 2018, from $13.7 millionfor the same period in 2017. The $0.5 milliondecrease was related to $2.5 millionin decreased intellectual property and patent related fees, partially offset by $0.9 millionin increased stock-based compensation expenses, $0.6 millionin increased employee related expenses and $0.4 millionin increased other expenses including facility-related expenses and professional service expenses.
For the full year 2018, net loss attributable to common stockholders was
- Collaboration and other research and development revenues were
$31.9 millionfor 2018, compared to $13.7 millionfor 2017. The increase of $18.2 millionwas primarily due to $12.7 millionin increased revenue recognized related to our strategic alliance with Allergan, $4.0 millionin revenue recognized pursuant to a license agreement with Beam Therapeutics, Inc.and $1.5 millionin increased revenue recognized pursuant to our collaboration agreement with Juno Therapeutics.
- Research and development expenses were
$90.7 millionfor 2018, compared to $83.2 millionfor 2017. The increase of $7.5 millionwas due to $8.3 millionin increased process and platform development expenses, $5.4 millionin increased employee related expenses and $2.0 millionin increased other expenses including facility-related expenses, partially offset by $5.9 millionin decreased licensing and sublicensing payment expenses, $2.0 millionin decreased success payment expenses and $0.4 millionin decreased stock-based compensation expenses.
- General and administrative expenses were
$55.0 millionfor 2018, compared to $50.5 millionfor 2017. The increase of $4.5 millionwas due to $3.6 millionin increased stock-based compensation expenses, $2.6 millionin increased employee related expenses, $0.9 millionin increased other expenses including facility-related expenses and $0.9 millionin increased professional service expenses, partially offset by $3.5 millionin decreased intellectual property and patent related fees.
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.
About Leber Congenital Amaurosis
Leber congenital amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 20‑30 percent of all LCA patients.
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 Company’s EM22 goals and plans to dose patients with EDIT-101 in the second half of 2019. 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 preclinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from preclinical 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 Quarterly Report on Form 10-Q, which is on file with the
|EDITAS MEDICINE, INC.|
|Consolidated Statement of Operations|
|(amounts in thousands, except share and per share data)|
|Three Months Ended||Twelve Months Ended|
|December 31,||December 31,|
|Collaboration and other research|
|and development revenues||$||6,119||$||3,667||$||31,937||$||13,728|
|Research and development||19,195||26,424||90,654||83,159|
|General and administrative||13,177||13,685||55,010||50,502|
|Total operating expenses||32,372||40,109||145,664||133,661|
|Other income (expense), net:|
|Other (expense) income, net||(3||)||129||328||587|
|Interest income (expense), net||1,202||124||3,445||(978||)|
|Total other income (expense), net||1,199||253||3,773||(391||)|
|Net loss per share attributable|
|to common stockholders,|
|basic and diluted||$||(0.52||)||$||(0.84||)||$||(2.33||)||$||(2.98||)|
|Weighted-average common shares outstanding, basic and|
|EDITAS MEDICINE, INC.
|Selected Consolidated Balance Sheet Items
|(amounts in thousands)
|Cash, cash equivalents, and marketable securities||$||368,955||$||329,139|
|Deferred revenue, net of current portion||115,614||94,725|
|Construction financing lease obligation, net of|
|Total stockholders' equity||236,162||208,080|
Source: Editas Medicine, Inc.