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Initiated Brilliance Phase 1/2 clinical trial of EDIT-101(AGN-151587) for LCA10
Patient dosing in Brilliance trial on track for 2H19
EDIT-301 for hemoglobinopathies pre-clinical in vivo data to be presented by YE19
Cash, cash equivalents, and marketable securities of
"I am pleased and honored to be appointed CEO at this exciting time for the company," said
Recent Achievements and Outlook
In Vivo CRISPR Medicines
- EDIT-101 for LCA10
Patient screening initiated with dosing planned for 2H19
Editas Medicineand its partner, Allergan, expect to enroll approximately 18 patients, aged 3 years and above, in the Brilliance Phase 1/2 clinical trial. The Brilliance clinical trial is a multi-center, open label, dose escalation study to evaluate the safety, tolerability, and efficacy of EDIT-101 as a treatment for Leber congenital amaurosis 10 (LCA10). It is the first ever clinical trial of an in vivo CRISPR medicine.
- Usher Syndrome 2A
Ready for IND-enabling studies by YE19
In vivo proof-of-concept data was presented at the 2019
American Society of Gene & Cell TherapyAnnual Meeting. The Company plans to present in vitro cellular data demonstrating predicted therapeutically relevant and specific editing with its lead molecule at a 2019 medical conference.
Engineered Cell Medicines
- EDIT-301 for Sickle Cell Disease and Beta-thalassemia
IND-enabling activities under way for a potentially best-in-class medicine
Editas Medicineis developing EDIT-301 to directly upregulate fetal hemoglobin by editing the HBG1/2 promoter in the beta-globin locus. The Company plans to present pre-clinical in vivo data at a 2019 medical conference demonstrating robust and durable induction of fetal hemoglobin with EDIT-301.
The Company is advancing engineered cell medicines for cancer including engineered T cells, in collaboration with
Juno Therapeutics, Inc., a Celgenecompany ( Celgene), as well as wholly owned engineered NK cell programs. Editas Medicineis developing universal allogeneic medicines derived from induced pluripotent stem cells (iPSCs) to create off-the-shelf oncology treatments that are highly engineered for areas of unmet need, such as solid tumors. Toward that end, the Company has successfully generated iPSCs from fibroblasts and peripheral blood cells, achieved efficient CRISPR gene editing of iPSCs, and differentiated iPSCs into functional NK cells.
The Company announced today the appointment of
Cynthia Collinsas President and Chief Executive Officer of Editas Medicine. Ms. Collins has served as a Director of the Company since December 2018, and as interim Chief Executive Officer since January 2019. Ms. Collins has more than 30 years of experience as an executive leading and growing gene and cell medicine companies.
- Intellectual Property
June 24, 2019, the U.S. Patent and Trademark Office declared an interference (#106115) between certain CRISPR/Cas9 patent applications submitted by the University of California, the University of Vienna, and Emmanuelle Charpentierand certain patents issued to the Broad Institute, Inc.(Broad) that have been licensed to Editas Medicine. The Broad patents continue to be valid and in force. Foundational claims covering the use of CRISPR/Cas9 for gene editing in eukaryotic cells have also issued to Broad as patents in each of the United States, Europe, Japan, and other jurisdictions.
Editas Medicineis building a Good Manufacturing Practice facility in Boulder, Colorado, to supply guide RNA and ribonucleoprotein in support of its EDIT-301 experimental medicine for sickle cell disease and beta-thalassemia as well as engineered cell medicines to treat cancer. The Company expects the facility to be commissioned in 2020.
- Balance Sheet
The Company held cash, cash equivalents, and marketable securities of
$317.9 millionas of June 30, 2019, providing at least 24 months of funding for operating expenses and capital expenditures.
- Citi 14th Annual
Biotech Conference, September 4-5, Boston; Morgan Stanley17th Annual Global Healthcare Conference, Fireside Chat, September 9, 2:15 p.m. ET, New York City; and
- Chardan 3rd Annual
Genetic Medicines Conference, October 7-8, New York City.
- Cold Spring Harbor Genome Engineering,
October 10-13, Cold Spring Harbor; and
- 27th Annual
Congressof the European Society of Gene & Cell Therapy, October 22-25, Barcelona.
Second Quarter 2019 Financial Results
Cash, cash equivalents, and marketable securities at
For the three months ended
- Collaboration and other research and development revenues were
$2.3 millionfor the three months ended June 30, 2019, compared to $7.4 millionfor the same period in 2018. The $5.0 milliondecrease was primarily attributable to a decrease in revenue recognized pursuant to our collaboration agreement with Celgeneand an out-license arrangement entered into during the second quarter of 2018, partially offset by an increase in revenue recognized pursuant to our strategic alliance with Allergan.
- Research and development expenses decreased by
$9.2 million, to $23.6 millionfor the three months ended June 30, 2019, from $32.7 millionfor the same period in 2018. The decrease was primarily attributable to decreased sublicensing and success payment expenses due to additional expenses incurred during the second quarter of 2018.
- General and administrative expenses increased by
$0.1 million to $14.4 millionfor the three months ended June 30, 2019, from $14.3 millionfor the same period in 2018.
As a leading genome editing company,
About EDIT-101 (AGN-151587)
EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.
About Leber Congenital Amaurosis
Leber congenital amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 20‑30 percent of all LCA patients.
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Company’s plans with respect to the Brilliance Phase 1/2 clinical trial for EDIT-101 (AGN-151587), including dosing patients in the second half of 2019, and its expectations of completing a manufacturing facility in
|Editas Medicine, Inc.|
|Condensed Consolidated Statements of Operations|
|(amounts in thousands, except per share and share data)|
|Three Months Ended|
|Collaboration and other research and development revenues||$||2,330||$||7,372|
|Research and development||23,565||32,718|
|General and administrative||14,414||14,311|
|Total operating expenses||37,979||47,029|
|Other income, net:|
|Other (expense) income, net||(68)||154|
|Interest income, net||1,931||780|
|Total other income, net||1,863||934|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.69)||$||(0.82)|
|Weighted-average common shares outstanding, basic and diluted||49,070,574||46,952,059|
|Editas Medicine, Inc.|
|Selected Condensed Consolidated Balance Sheet Items|
|(amounts in thousands)|
|June 30,||December 31,|
|Cash, cash equivalents, and marketable securities||$||317,930||$||368,955|
|Deferred revenue, net of current portion||75,911||115,614|
|Construction financing lease obligation, net of current portion||—||32,417|
|Total stockholders’ equity||193,062||236,162|
Source: Editas Medicine, Inc.