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Filed IND application for EDIT-101 with
Favorable Appeals Court ruling affirms strength of foundational intellectual property
Oral presentation at ASH Annual Meeting to highlight novel Sickle Cell Disease and Beta-Thalassemia program
Strong balance sheet with
“The filing of the IND for EDIT-101 for the treatment of LCA10 marks a significant milestone for Editas and brings us closer to helping people living with this devastating disease,” said
Recent Achievements and Outlook
- Filed EDIT-101 Investigational New Drug (IND) application in October with
U.S. Food and Drug Administration( FDA) for treatment of Leber Congenital Amaurosis type 10 (LCA10). In addition, the National Institutes of Health(NIH) determined that a Recombinant DNA Advisory Committeereview of the protocol for the Phase 1/2 trial of EDIT-101 was not necessary, and the protocol is now registered with the NIH. The Company expects to treat 10 to 20 patients in this open label, dose escalation study in order to evaluate the safety and efficacy of EDIT-101. Allerganand Editas Medicineto co-develop and equally share profits and losses from EDIT-101 in the U.S. In the third quarter, Allergan Pharmaceuticals International Limited( Allergan) exercised its option to develop and commercialize EDIT-101 under the strategic alliance formed in March 2017. Allerganpaid an optionexercise fee of $15 million, which was recorded in the third quarter. In addition, Editas Medicineis eligible to receive a $25 millionmilestone payment from Allerganupon clearance of the IND application for EDIT-101. Editas Medicinealso exercised its option in the third quarter to co-develop and equally share profits and losses in the U.S. for EDIT-101.
- Novel approach to Sickle Cell Disease and Beta-Thalassemia to be highlighted in an oral presentation at the 60th
American Society of Hematology(ASH) Annual Meeting. Editas Medicinewill present pre-clinical in vivo data comparing its novel gene editing strategy to reactivate fetal hemoglobin (HbF) by editing the beta-globin locus to strategies that edit the BCL11A erythroid enhancer (BCL11Ae). In this mouse study, editing the beta-globin locus upregulated HbF with superior repopulation of red blood cell precursors as compared to editing BCL11Ae.
- Federal Appeals Court ruling affirms strength of foundational CRISPR/Cas9 intellectual property.
The U.S. Court of Appeals for the Federal Circuit(Appeals Court) upheld the U.S. Patent and Trademark Office no interference-in-fact decision concerning certain CRISPR/Cas9 patents Editas Medicineexclusively licenses from Broad Institute, Inc.The patents broadly cover CRISPR/Cas9 gene editing in eukaryotic cells, which the Company believes is essential to making CRISPR medicines.
- Added key leadership in oncology and engineered cell medicines.
Richard A. Morgan, Ph.D., a leading expert in gene therapy and oncology, joined Editas Medicineas Senior Vice President of Immunogenetics. Dr. Morgan brings more than 30 years of scientific leadership in the life sciences industry.
- Strong balance sheet to advance
Editas Medicinethrough multiple value inflection points. The Company held cash, cash equivalents, and marketable securities of $337.5 millionas of September 30, 2018, providing at least 24 months of funding for operating expenses and capital expenditures without any assumption of future cash received from milestones or additional financings.
Credit Suisse27th Annual Healthcare Conference, November 13, 3:25 p.m. PT, Phoenix;
- Barclays Gene Editing & Gene Therapy Summit,
November 29, 1:15 p.m. ET, New York City; and
- J.P. Morgan 37th Annual Healthcare Conference,
January 7-10, 2019, San Francisco
|Oral Abstract Number: 409|
|Title: Comparative Studies Reveal Robust HbF Induction By Editing of HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+ Cells but That BCL11A Erythroid-Enhancer Editing Is Associated with Selective Reduction in Erythroid Lineage Reconstitution in a Xenotransplantation Model|
|Presenter: KaiHsin Chang, Ph.D.|
|Session: 112. Thalassemia and Globin Gene Regulation: Hemoglobin Switching|
|Time: Sunday, December 2, 4:30 p.m.|
|Location: San Diego Convention Center, Room 25B|
Third Quarter 2018 Financial Results
Cash, cash equivalents, and marketable securities at
For the third quarter ended
- Collaboration and other research and development revenues were
$14.5 millionfor the quarter ended September 30, 2018, compared to $6.3 millionfor the same period in 2017. The $8.2 millionincrease was primarily attributable to a $10.6 millionincrease in revenue recognized pursuant to our strategic alliance with Allerganand $0.1 millionin revenue recognized pursuant to a license agreement with Beam Therapeutics, Inc., partially offset by a $2.4 milliondecrease in revenue recognized pursuant to our collaboration agreement with Juno Therapeutics, Inc., a Celgenecompany and wholly-owned subsidiary of Celgene Corporation.
- Research and development expenses were
$17.4 millionfor the quarter ended September 30, 2018, compared to $20.4 millionfor the same period in 2017. The $3.0 milliondecrease was primarily attributable to $4.9 millionin decreased sublicensing expenses and $1.0 millionin decreased process and platform development expenses, mostly related to reimbursable expenses related to the Allerganprofit-sharing arrangement. These decreases were partially offset by $1.3 millionin increased employee related expenses, $1.1 millionin increased stock-based compensation expenses, $0.3 millionin increased facility-related expenses and $0.3 millionin increased other expenses.
- General and administrative expenses were
$13.3 millionfor the quarter ended September 30, 2018, compared to $12.6 millionfor the same period in 2017. The $0.7 millionincrease was primarily attributable to $1.2 millionin increased stock-based compensation expenses, $1.1 millionin increased employee related expenses, $0.7 millionin increased professional service expenses and $0.2 millionin increased other expenses, partially offset by $2.4 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 type 10 (LCA10). EDIT-101 is an AAV5 construct and 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 “aim,” ‘‘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 planning to present data and the Company developing and bringing transformative medicines to patients. 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||$||14,519||$||6,282|
|Research and development||17,443||20,396|
|General and administrative||13,334||12,635|
|Total operating expenses||30,777||33,031|
|Other income, net:|
|Other (expense) income, net||(4||)||196|
|Interest income (expense), net||1,024||(46||)|
|Total other income, net||1,020||150|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.32||)||$||(0.64||)|
|Weighted-average common shares outstanding, basic and diluted||47,414,271||41,307,092|
|Editas Medicine, Inc.|
|Selected Condensed Consolidated Balance Sheet Items|
|(amounts in thousands)|
|September 30,||December 31,|
|Cash, cash equivalents, and marketable securities||$||337,492||$||329,139|
|Deferred revenue, net of current portion||104,100||94,725|
|Construction financing lease obligation, net of current portion||32,694||33,431|
|Total stockholders’ equity||224,449||208,080|
Source: Editas Medicine, Inc.