Editas Medicine Announces First Quarter 2017 Results and Update
Entered strategic R&D alliance with ophthalmology leader
Completed successful follow-on offering of common stock raising
Presented new data at the
“We made outstanding progress this quarter building our business for the long term, including forming a strategic alliance with
Building the business for the long term by assembling the capabilities to fully develop and commercialize important medicines
- Established strategic alliance with
Allergan plc. Allergan Pharmaceuticals International Limited( Allergan), a wholly-owned subsidiary of Allergan plc, and Editas Medicineformed a strategic alliance to research, develop, and commercialize CRISPR genome editing medicines for patients suffering from serious ocular diseases. Under the terms of the agreement, Editas Medicinereceived an upfront payment of $90 millionand granted Allerganan option to license up to five candidate programs. Allergan will be responsible for development and commercialization of any optioned products, subject to Editas Medicine'soption to co-develop and co-promote up to two products in the United States, including the program for Leber Congenital Amaurosis type 10 (LCA10). Editas Medicine is also eligible to receive more than $200 millionin milestone payments per optioned product, with more than half of this value for development, approval, and launch milestones, as well as high-single digit royalties on net sales of optioned products, subject to the co-development and co-promotion arrangement.
- Updated timelines for LCA10 Investigational New Drug (IND) application submission. The target date for filing the IND application for the LCA10 program is being moved to the middle of 2018 to accommodate certain delays in third-party manufacturing of our LCA10 product candidate and to take full advantage of our newly formed alliance with
- Successfully completed common stock offering resulting in
$104 millionin gross proceeds including full exercise of the underwriters' option to purchase additional shares.
- Strengthened Editas Medicine’s unmatched intellectual property position. The U.S. Patent and Trademark Office (USPTO) issued a highly favorable decision in the patent interference between the
University of California, the University of Vienna, Emmanuelle Charpentierand the Broad Institute, Inc.(Broad) regarding certain CRISPR/Cas9 patents the Company exclusively licenses from Broad. The USPTO granted Broad’s Motion for No Interference in Fact, ending the interference before the USPTO and upholding Broad's issued patents.
The European Patent Office also issued a notice of intent to grant the first Cpf1 patent, which is exclusively licensed to
Editas Medicine. Cpf1 offers several potential advantages including expanding the range of genomic sites that can be edited, simplifying manufacturing and delivery, and increasing efficiency and accuracy of some forms of gene insertion or correction. The patent is expected to grant on May 31, 2017.
As the only company with rights to both Cas9 and Cpf1, the two proven CRISPR systems for human medicines,
Editas Medicinebelieves it is in an unparalleled position to continue building a broad and differentiated product pipeline.
Advancing a pipeline strategy to enable successful product development and expand Editas Medicine’s platform in the years ahead
- Presented six posters and oral presentations at the recent
American Society of Gene & Cell Therapy(ASGCT) meeting demonstrating progress in translating the promise of CRISPR technology into a broad class of genomic medicines.
— Demonstrated therapeutically relevant editing of photoreceptors in non-human primates in the Company’s LCA10 program. In this study, two guide RNAs and S. aureus Cas9 expressed under the control of a photoreceptor-specific promoter were delivered in a single, subretinally-injected adeno-associated virus (AAV) at two different doses. Gene editing was shown to be dose and time dependent. For animals treated with the higher dose, the projected productive editing rate may be as high as 50% of alleles in photoreceptor cells, based on directly measured editing of 15% in total genomic DNA and an estimate for the proportion of cells represented by photoreceptors. We believe this is well above the editing rate needed to have a therapeutic effect in patients.
— Demonstrated the induction of therapeutically relevant levels of fetal hemoglobin protein and showed that edited hematopoietic stem cells durably repopulate the bone marrow and blood in vivo in program to treat sickle cell disease and beta-thalassemia. Also, presented data that suggests this proprietary editing approach has the potential to be more potent than other approaches that have been previously reported.
— Released data on new proprietary technologies to potentially enable unprecedented medicines. Showed rigorous new empirical methods for assessing specificity referred to as Uni-directional Targeted Sequencing (UDITAS™), novel compositions and methods for making covalently-coupled dual guide RNAs, the potential for self-inactivating Cas9 to control expression while maintaining efficient gene editing, and targeted insertion of DNA for efficient and specific gene correction.
Joined Duke-Margolis Value-Based Payment Consortium, a multi-disciplinary initiative to define and develop more feasible paths to value-based payment arrangements that support better care and outcomes for patients. One of the focuses of the initiative is in Genomic Medicine and it is led by Mark McClellan, M.D., Ph.D., Director of the Duke-Margolis Center for Health Policy. Dr. McClellan is the former Administrator of the Centers for Medicare and Medicaid Servicesand former Commissioner of the Food and Drug Administration. The advisory group brings together a select team of leaders in the biopharmaceutical and medical device industries, including patient advocates, payers, manufacturers, and providers, as well as experts on regulatory affairs, law, and policy makers to address common legal and regulatory issues as well as to innovate payment models that create value for the healthcare system and for patients.
Bank of America Merrill Lynch2017 Health Care Conference, May 16-18, Las Vegas; UBS Global Healthcare Conference, May 22-24, New York City; JMP Securities2017 Life Sciences Conference, June 20-21, New York City; and
- Goldman Sachs Third Annual Innovation Symposium,
June 27, New York City.
- CRISPR 2017,
June 8-10, Big Sky;
- 2017 Synthetic Biology: Engineering, Evolution & Design (SEED),
June 20-23, Vancouver; and
- Cold Spring Harbor Genome Engineering,
July 21-24, Cold Spring Harbor.
First Quarter 2017 Financial Results
Cash and cash equivalents at
For the quarter ended
- Collaboration and other research and development revenues were
$0.7 millionfor the quarter ended March 31, 2017, compared to $0.8 millionfor the same period in 2016. The $0.1 milliondecrease was due to a decrease in revenue recognized pursuant to our collaboration with Juno Therapeutics, Inc.
- Research and development expenses were
$19.0 millionfor the quarter ended March 31, 2017, compared to $8.9 millionfor the same period in 2016. The $10.1 millionincrease is attributable to the issuance of $5.0 millionnotes payable to the Broad Instituteand Wageningen Universitythat became issuable under one of our licensing agreements during the first quarter of 2017, an increase of $2.3 millionthat became due to certain of our licensors in connection with receiving the Allerganupfront payment during the first quarter of 2017, a $1.4 millionincrease in employee related expenses, excluding stock-based compensation, a $1.1 millionincrease in process and platform development costs, as well as a $0.2 millionincrease in stock-based compensation expense.
- General and administrative expenses were
$12.3 millionfor the quarter ended March 31, 2017, compared to $9.8 millionfor the same period in 2016. The $2.5 millionincrease is attributable to an increase of $1.4 millionin stock-based compensation expense, an increase of $1.0 millionin employee related expenses, an increase of $0.5 millionin contractor consulting fees, and an increase of $0.3 millionin other facility-related expenses, partially offset by a decrease of $0.7 millionin external IP legal and patent-related fees, including expenses associated with the prosecution and maintenance of our patents and patent applications owned by us or licensed to us.
Editas Medicine is a leading genome editing company dedicated to treating patients with genetically-defined diseases by correcting their disease-causing genes. The Company was founded by world leaders in genome editing, and its mission is to translate the promise of genome editing science into a broad class of transformative genomic medicines to benefit the greatest number of patients.
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s goals of submitting of an IND for the LCA10 program by the middle of 2018 and achieving any milestones under the Company’s alliance with
|Editas Medicine, Inc.|
|Condensed Consolidated Statements of Operations and Comprehensive Loss|
|(amounts in thousands, except per share and share data)|
|Three Months Ended|
|Collaboration and other research and development revenues||$||682||$||805|
|Research and development||19,021||8,882|
|General and administrative||12,288||9,762|
|Total operating expenses||31,309||18,644|
|Other income (expense), net:|
|Other income (expense), net||140||(30||)|
|Interest income (expense), net||(610||)||124|
|Total other income (expense), net||(470||)||94|
|Net loss and comprehensive loss||$||(31,097||)||$||(17,745||)|
|Reconciliation of net loss to net loss attributable to common stockholders:|
|Accretion of redeemable convertible preferred stock to redemption value||—||(47||)|
|Net loss attributable to common stockholders||$||(31,097||)||$||(17,792||)|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.85||)||$||(0.80||)|
|Weighted-average common shares outstanding, basic and diluted||36,485,421||22,280,797|
|EDITAS MEDICINE, INC.|
|Selected Condensed Consolidated Balance Sheet Items|
|(amounts in thousands)|
|March 31,||December 31,|
|Cash and cash equivalents||$||351,552||$||185,323|
|Deferred revenue, net of current portion||104,033||26,000|
|Construction financing lease obligation, net of current portion||33,929||35,096|
|Total stockholders’ equity||206,461||134,607|
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