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Leber Congenital Amaurosis type 10 (LCA10) product candidate EDIT-101 made significant progress toward clinical trials
Achieved research milestone in
“This quarter, we made critical progress in advancing our LCA10 product candidate toward the clinic, as well as in our broader pipeline,” said
Advancing product candidates and platform to deliver a sustainable pipeline of transformative products
• LCA10 program made significant progress toward the clinic and remains on track for an IND filing in mid-2018.
- Received institutional review board approval for the first site in the Company's LCA10 clinical natural history study. This study will prospectively evaluate patients with LCA10 to assess the course of the disease and to pilot potential clinical trial endpoints and designs. This knowledge will inform the interventional clinical trial design for EDIT-101,
Editas Medicine'spre-clinical product candidate to treat LCA10. The Company is finalizing logistical details with trial sites and then will proceed to patient enrollment.
- Received Advanced Therapy Medicinal Product (ATMP) designation for EDIT-101. ATMPs are medicines for human use that are based on genes or cells and that offer groundbreaking new opportunities for the treatment of disease and injury. The classification provides companies with scientific regulatory guidance from the
European Medicines Agency, as well as incentives to develop ATMPs in the European Union.
• Achieved second technical milestone in
• Demonstrated progress in translating the promise of CRISPR technology into a broad class of genomic medicines through more than 20 presentations and posters at multiple scientific and medical conferences.
- Presented results at the 20th Annual Meeting of the
American Society of Cell and Gene Therapyfrom multiple programs, including data demonstrating:
— Potentially therapeutically relevant levels of editing of non-human primate photoreceptors in the Company’s LCA10 program. The study indicated that the projected productive editing rate may be as high as 50 percent of alleles in photoreceptor cells, which
Editas Medicinebelieves is well above the level needed to have a therapeutic effect in patients.
— A substantial increase in fetal hemoglobin protein with a novel genome editing strategy that has the potential to be more potent than other previously reported approaches treating sickle cell disease and beta-thalassemia.
- Reported potentially therapeutic levels of editing in human retinal explants using EDIT-101, the Company's pre-clinical product candidate to treat LCA10 (
Cold Spring Harbor Genome Engineering Conference).
- Presented data demonstrating that multiple Cpf1 variants show efficient and robust editing across multiple genetic loci and cell types, including in T cells and adult hematopoietic stem cells (CRISPR 2017
Building the business for the long term by assembling the capabilities to fully develop and commercialize important medicines
• Strengthened unmatched intellectual property position with issuance of first patent for CRISPR/Cpf1. In May, the European Patent Office issued the first patent for CRISPR/Cpf1, which is exclusively licensed to
Developing an outstanding organization
• Grew organization to more than one-hundred team members. Key people joined the company in several critical functions, including pharmacology, manufacturing, and operations.
• Expanded our Board with the addition of
Morgan Stanley Global Healthcare Unplugged Conference, September 11-13, New York City; Chardan Gene Therapy Conference, October 10, New York City; and
- Jefferies Gene Therapy/Editing Summit,
October 12, New York City. European Society of Gene & Cell Therapy, October 17-20, Berlin.
Second Quarter 2017 Financial Results
Cash, cash equivalents, and marketable securities at
For the quarter ended
- Collaboration and other research and development revenues were
$3.1 millionfor the quarter ended June 30, 2017, compared to $3.4 millionfor the same period in 2016. The $0.3 milliondecrease was primarily attributable to a $2.7 milliondecrease in revenue recognized pursuant to our collaboration with Juno Therapeutics, Inc., of which $2.5 millionrelates to the achievement of a milestone in the period ending June 30, 2016, partially offset by a $2.4 millionincrease in revenue recognized pursuant to our strategic alliance with Allerganin the period ending June 30, 2017.
- Research and development expenses were
$17.3 millionfor the quarter ended June 30, 2017, compared to $10.4 millionfor the same period in 2016. The $6.9 millionincrease was primarily attributable to $5.1 millionin additional sublicense fees that were owed to certain of our licensors in connection with receiving the Allerganupfront payment, an increase of $1.5 millionin employee related expenses, and an increase of $1.5 millionin process and platform development costs, partially offset by a decrease of $0.9 millionin stock-based compensation expense resulting from a decline in the valuation of non-employee restricted stock and options at June 30, 2017, and other expenses.
- General and administrative expenses were
$11.9 millionfor the quarter ended June 30, 2017, compared to $12.2 millionfor the same period in 2016. The $0.3 milliondecrease was primarily attributable to approximately $2.2 millionin decreased external intellectual property legal and patent-related fees associated with patents and patent applications licensed to us, which was partially offset by an increase of $0.7 millionin stock-based compensation expenses, an increase of $0.4 millionin employee related expenses, an increase of $0.4 millionin professional service fees, and an increase of $0.4 millionin other expenses including facility-related 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. 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||$||3,097||$||3,388|
|Research and development||17,318||10,430|
|General and administrative||11,894||12,158|
|Total operating expenses||29,212||22,588|
|Other income (expense), net:|
|Other income, net||122||5|
|Interest income (expense), net||(446||)||153|
|Total other income (expense), net||(324||)||158|
|Net loss attributable to common stockholders||$||(26,439||)||$||(19,042||)|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.65||)||$||(0.54||)|
|Weighted-average common shares outstanding, basic and diluted||40,830,161||35,286,719|
|Editas Medicine, Inc.|
|Selected Condensed Consolidated Balance Sheet Items|
|(amounts in thousands)|
|June 30,||December 31,|
|Cash, cash equivalents, and marketable securities||$||324,795||$||185,323|
|Deferred revenue, net of current portion||100,953||26,000|
|Construction financing lease obligation, net of current portion||33,878||35,096|
|Total stockholders’ equity||185,004||134,607|
Cristi Barnett Editas Medicine, Inc.(617) 401-0113 email@example.com Investor Contact Mark Mullikin Editas Medicine, Inc.(617) 401-9083 firstname.lastname@example.org