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EDIT-101 (AGN-151587) for LCA10 first patient dosing expected by early 2020
EDIT-301 for hemoglobinopathies in vivo pre-clinical data to be presented at ASH
"Our momentum in 2019 remains strong in advancing our pipeline of in vivo CRISPR and engineered cell medicines," said
Recent Achievements and Outlook
In Vivo CRISPR Medicines
- EDIT-101 (AGN-151587) for LCA10
First patient dosing expected by early 2020
Editas Medicineand its partner, Allergan, are conducting the Brilliance Phase 1/2 clinical trial to evaluate the safety, tolerability, and efficacy of EDIT-101 as a treatment for Leber congenital amaurosis 10 (LCA10). The first potential patient has been successfully screened and confirmation of the first surgery to administer EDIT-101 by early 2020 is pending.
- Usher Syndrome 2A
Expect to be ready for IND-enabling studies by YE19
Pre-clinical in vitro data were presented at the 27th Annual
Congressof the European Society of Gene & Cell Therapy. The Company’s lead candidate demonstrated successful knock-out of USH2A exon 13, producing up to 60% corrected gene expression with no off-target activity. These data supplement in vivo pre-clinical data presented earlier in the year supporting further development of the lead candidate to treat Usher syndrome 2A.
- Gene Editing for Neurological Diseases
Formed research collaboration with AskBio for AAV delivery of CRISPR medicines
Editas Medicineand Asklepios BioPharmaceutical, Inc.(AskBio) are working together to develop transformative medicines for neurological disorders by combining AskBio’s adeno-associated virus (AAV) development and manufacturing expertise with Editas Medicine’s leading CRISPR gene editing platform.
Engineered Cell Medicines
Celgenecollaboration to focus on engineered alpha-beta T cell medicines Editas Medicineand Juno Therapeutics, Inc., a Celgenecompany ( Celgene), have amended their collaboration, formed in 2015, to focus on developing autologous and allogeneic engineered alpha-beta T cell medicines for the treatment of cancer and autoimmune diseases. Under the amended collaboration, Editas Medicineis entitled to receive a $70 millionpayment from Celgeneand may be eligible for future milestone and royalty payments. Under the terms of the amended collaboration, Editas Medicinehas expanded its wholly owned portfolio to treat cancer to include non-alpha-beta T cell medicines in addition to programs utilizing natural killer cells derived from both healthy donors and induced pluripotent stem cells.
- EDIT-301 for Sickle Cell Disease and Beta-Thalassemia
In vivo pre-clinical proof-of-concept data to be presented at ASH
Editas Medicineis developing EDIT-301 using CRISPR/Cas12a (Cpf1) as a potentially best-in-class medicine to treat sickle cell disease and beta-thalassemia. The Company will present in vivo pre-clinical data at the 61st American Society of HematologyAnnual Meeting & Exposition (ASH) demonstrating that EDIT-301 induces predicted therapeutically relevant levels of fetal hemoglobin with no off-target activity.
In October, the Company announced the appointment of
Judith R. Abrams, M.D., as Chief Medical Officer. Dr. Abrams is a leading drug development clinician who has brought multiple medicines from clinical stage development to regulatory approval. Dr. Abrams has more than 25 years of experience in leadership roles in the biopharmaceutical industry managing portfolios of products across all phases of global clinical development.
- Balance Sheet
The Company expects that its existing cash, cash equivalents and marketable securities of
$332.6 millionat September 30, 2019, and anticipated interest income will enable it to fund its operating expenses and capital expenditures for at least 24 months from today. Cash, cash equivalents and marketable securities as of September 30, 2019excludes the anticipated $70 millionpayment from Celgenepursuant to the amended collaboration announced today.
Piper Jaffray31st Annual Healthcare Conference, Fireside Chat, December 3, 10:30 a.m. ET, New York City; and
- J.P. Morgan 38th Annual Healthcare Conference,
January 13-16, 2020, San Francisco.
Abstract Number: 4636
Title: EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD
Presenter: Edouard De Dreuzy, Ph.D.
Session: 801. Gene Therapy and Transfer: Poster III
Location: Hall B,
Third Quarter 2019 Financial Results
Cash, cash equivalents, and marketable securities at
For the three months ended
- Collaboration and other research and development revenues were
$3.8 millionfor the three months ended September 30, 2019, compared to $14.5 millionfor the same period in 2018. The $10.7 milliondecrease was primarily attributable to $15.0 millionin revenue recognized during the third quarter of 2018 related to the EDIT-101 option exercise payment pursuant to our strategic alliance with Allergan.
- Research and development expenses increased by
$5.3 million, to $22.7 millionfor the three months ended September 30, 2019, from $17.4 millionfor the same period in 2018. The $5.3 millionincrease was primarily attributable to increased process and platform development expenses driven by increased manufacturing and clinical related costs, including costs under our profit-sharing arrangement with Allerganin the United Statesfor EDIT-101.
- General and administrative expenses increased by
$2.4 million to $15.7 millionfor the three months ended September 30, 2019, from $13.3 millionfor the same period in 2018. The $2.4 millionincrease was primarily attributable to increased professional service expenses.
As a leading genome editing company,
About EDIT-101 (AGN-151587)
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 plans with respect to the Brilliance Phase 1/2 clinical trial for EDIT-101 (AGN-151587), including the Company’s expectations regarding the timing of dosing a patient by early 2020. 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 pre-clinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from pre-clinical 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||$||3,848||$||14,519|
|Research and development||22,702||17,443|
|General and administrative||15,734||13,334|
|Total operating expenses||38,436||30,777|
|Other income, net:|
|Other expense, net||(33||)||(4||)|
|Interest income, net||1,680||1,024|
|Total other income, net||1,647||1,020|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.66||)||$||(0.32||)|
|Weighted-average common shares outstanding, basic and diluted||49,820,455||47,414,271|
|Editas Medicine, Inc.
Selected Condensed Consolidated Balance Sheet Items
(amounts in thousands)
|September 30,||December 31,|
|Cash, cash equivalents, and marketable securities||$||332,616||$||368,955|
|Deferred revenue, net of current portion||82,379||115,614|
|Construction financing lease obligation, net of current portion||—||32,417|
|Total stockholders’ equity||209,990||236,162|
Source: Editas Medicine, Inc.