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“This was a quarter of continued steady progress in advancing our pipeline of CRISPR medicines toward the clinic,” said
Advancing product candidates and platform to deliver a sustainable pipeline of transformative products
EDIT-101 for Leber Congenital Amaurosis type 10 (LCA10) remains on track for a mid-2018 IND filing
- Demonstrated dose-dependent editing with EDIT-101 that was durable through six months in a pre-clinical, transgenic mouse model. In a study of transgenic mice carrying a copy of the mutated human gene (CEP290) targeted by our clinical candidate, EDIT-101, we demonstrated efficient transduction and gene editing in the retinal photoreceptor cells, the relevant cell type affected in LCA10 patients. Significant CEP290 editing was rapidly achieved and sustained through six months of observation. In addition, both Cas9 messenger RNA and guide RNA levels correlated with editing. These data were presented recently at the
European Society of Gene and Cell Therapycongress and further support advancing EDIT-101 towards clinical development.
- Initiated clinical natural history study to facilitate interventional trial design and enrollment. This study will prospectively evaluate patients with LCA10 across a broad range of ages and disease severity to assess the course of the disease and to pilot potential clinical trial endpoints and designs. The results of the study will inform interventional clinical trial design and enrollment for EDIT-101.
Editas Medicineplans to enroll approximately 40 patients in this study, ages three and above, at multiple sites in the U.S. and Europe. Patients will be evaluated and followed for at least one year. Massachusetts Eye and Ear Institute, an international center for treatment and research and a teaching hospital of Harvard Medical School, is the first site enrolling patients for this study.
- Received Orphan Medicinal Product Designation from the
European Medicines Agency for EDIT-101. Orphan Medicinal Product Designation is intended to support the development of medicines in debilitating disease indications with low prevalence and no satisfactory existing therapies.
Made important progress in engineered cell therapy programs
- Established compelling pre-clinical data for two additional gene targets in our collaboration with
Juno Therapeutics, Inc.( Juno Therapeutics), to develop next-generation engineered T cells for cancer. Improving the ability of T cells to overcome the tumor microenvironment may expand the range of cancers that can be addressed by engineered T cells. In data to be presented by Juno Therapeuticsand Editas Medicinescientists at the 32nd Annual Meeting of the Society for Immunotherapy of Cancer, T cells edited to knock out the CBLB gene will be shown to more efficiently kill antigen presenting target tumors cells, have enhanced cytokine production, and have greater proliferative capacity and survival rates in response to antigen stimulation, relative to unedited controls. In data to be presented at the 59th American Society of Hematology(ASH) Annual Meeting, Juno Therapeuticsand Editas Medicinescientists will report knockout of the TGFBR2 gene using CRISPR gene editing in BCMA-specific CAR-T cells for multiple myeloma prevented the development of the TGF beta-induced gene expression phenotype.
- Advanced exploration of a potentially superior therapy for sickle cell disease and beta thalassemia. In data to be presented at ASH, we will demonstrate high levels of gene disruption of adult human hematopoietic stem cells with CRISPR/Cpf1 and efficient targeted integration at the beta-hemoglobin locus with CRISPR/Cas9. We believe our data demonstrates multiple opportunities to develop best-in-class therapies for hemoglobinopathies.
Developing an outstanding organization
- Expanded capabilities that we believe will be critical to our long-term success. We added key talent in regulatory affairs, manufacturing, and ophthalmology research. This expertise is critical to the continued advancement of our platform and pipeline.
- Barclays Gene Editing & Gene Therapy Summit,
November 30, New York City; and
- Oppenheimer Rare/Orphan Disease Day,
December 5, New York City.
- 32nd Annual Meeting of the
Society for Immunotherapy of Cancer( SITC), November 8-12, National Harbor, MD; and
American Society of HematologyAnnual Meeting (ASH), December 9-12, Atlanta, GA.
Third Quarter 2017 Financial Results
Cash, cash equivalents, and marketable securities at
For the third quarter ended
- Collaboration and other research and development revenues were
$6.3 millionfor the quarter ended September 30, 2017, compared to $1.0 millionfor the same period in 2016. The $5.3 millionincrease was primarily attributable to a $3.2 millionincrease in revenue recognized pursuant to our strategic alliance with Allerganand a $2.5 millionmilestone recognized pursuant to our collaboration with Juno Therapeutics, Inc., partially offset by a $0.4 milliondecrease in reimbursable research and development expenses.
- Research and development expenses were
$20.4 millionfor the quarter ended September 30, 2017, compared to $10.8 millionfor the same period in 2016. The $9.6 million increase was primarily attributable to a $7.2 millionincrease in sublicensing payment expenses, a $1.6 millionincrease in process and platform development costs, and a $1.5 millionincrease in employee related expenses. This increase was partially offset by a $0.3 milliondecrease in facility related costs, a $0.3 milliondecrease in other expenses, and a $0.2 milliondecrease in stock-based compensation expenses.
- General and administrative expenses were
$12.6 millionfor the quarter ended September 30, 2017, compared to $11.3 millionfor the same period in 2016. The $1.3 million increase was primarily attributable to a $1.0 millionincrease in stock-based compensation expenses, a $0.3 millionincrease in intellectual property legal and patent-related fees, and a $0.2 millionincrease in employee related expenses. This increase was partially offset by a $0.2 milliondecrease in other expenses.
Editas Medicine is a leading genome editing company dedicated to treating patients with genetically-defined diseases by correcting their disease-causing genes. The Company was founded by world leaders in genome editing, and its mission is to translate the promise of genome editing science into a broad class of transformative genomic medicines to benefit the greatest number of 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 goal of submitting an IND for EDIT-101 by mid-2018. 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.|
|Condensed Consolidated Statements of Operations|
|(amounts in thousands, except per share and share data)|
|Three Months Ended|
|Collaboration and other research and development revenues||$||6,282||$||962|
|Research and development||20,396||10,832|
|General and administrative||12,635||11,295|
|Total operating expenses||33,031||22,127|
|Other income, net:|
|Other income, net||196||3|
|Interest income (expense), net||(46||)||142|
|Total other income, net||150||145|
|Net loss attributable to common stockholders||$||(26,599||)||$||(21,020||)|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.64||)||$||(0.59||)|
|Weighted-average common shares outstanding, basic and diluted||41,307,092||35,505,429|
|Editas Medicine, Inc.|
|Selected Condensed Consolidated Balance Sheet Items|
|(amounts in thousands)|
|September 30,||December 31,|
|Cash, cash equivalents, and marketable securities||$||295,691||$||185,323|
|Deferred revenue, net of current portion||97,851||26,000|
|Construction financing lease obligation, net of current portion||33,667||35,096|
|Total stockholders’ equity||177,788||134,607|