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- Building on focus in oncology through newly-formed partnership with BlueRock Therapeutics to develop universal allogeneic cell medicines for cancer -
- EDIT-101 (AGN-151587) for LCA10 on track for first patient dosing in second half of 2019 -
- Initiated IND-enabling activities for potentially best-in-class medicine for sickle cell disease and beta-thalassemia -
"2019 is off to a strong start with tangible progress on multiple fronts," said
Recent Achievements and Outlook
- Advancing universal allogeneic cell medicines for cancer through partnership with BlueRock Therapeutics.
Editas Medicineand BlueRock Therapeutics will combine their respective CRISPR genome editing and induced pluripotent stem cell (iPSC) platforms to develop universal allogeneic cell medicines to treat cancer and other serious diseases. Gene-edited, iPSC-derived, allogeneic cell medicines represent universal, off-the-shelf treatments that may be mass produced with superior quality, greater scale, and lower cost than autologous or donor-derived therapies. The Company believes that the combination will enable the high level of multiplexed editing needed to realize the full potential of cell medicine, particularly in solid tumors where the greatest unmet need exists.
- EDIT-101 on track for first patient dosing in second half of 2019.
Editas Medicineand Allergan Pharmaceuticals International Limitedexpect to enroll approximately 18 patients, aged 3 years and above, in a Phase 1/2 open label, dose escalation study to evaluate the safety, tolerability, and efficacy of EDIT-101 as a treatment for Leber congenital amaurosis 10 (LCA10). In April, initial data from the ongoing enLIGHTen natural history study of LCA10 patients were presented at the Retinal Cell and Gene Therapy Innovation Summit by Principal Investigator Eric A. Pierce, M.D., Ph.D., Director of the Ocular Genomics Instituteand William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Earand Harvard Medical School.
- Initiated IND-enabling activities for a potentially best-in-class medicine for sickle cell disease and beta-thalassemia.
Editas Medicineis developing an experimental medicine to directly upregulate fetal hemoglobin by editing the beta-globin locus. The Company has previously presented data demonstrating potential advantages of its approach as compared to indirectly upregulating fetal hemoglobin by editing the BCL11A erythroid enhancer. Editas Medicinewill present additional data next month at the 24th Congressof the European Hematology Association.
Progresstowards an experimental medicine for Usher syndrome 2A (USH2A). Pre-clinical in vivo proof-of-concept data presented at the 22nd Annual Meeting of the American Society of Gene & Cell Therapydemonstrated CRISPR/Cas9 gene editing of human USH2A exon 13 can rescue the retinal phenotype and restore auditory function in mice. Based on these data, the Company is optimizing a lead candidate to be ready for IND-enabling studies by the end of 2019.
- Strong balance sheet to advance the business. The Company held cash, cash equivalents, and marketable securities of
$342 millionas of March 31, 2019, providing at least 24 months of funding for operating expenses and capital expenditures.
- 5th Annual SunTrust Robinson Humphrey Life Sciences Summit,
May 8, New York City; RBC Capital Markets2019 Global Healthcare Conference, May 21, 8:00 a.m. ET, New York City; and Raymond James2019 Life Sciences and MedTech Conference, June 18-19, New York City.
Congressof the European Hematology Association, June 13-16, Amsterdam; and TIDES Oligonucleotide and Peptide Therapeutics Conference, May 20-23, San Diego.
First Quarter 2019 Financial Results
Cash, cash equivalents, and marketable securities at
For the three months ended
- Collaboration and other research and development revenues were
$2.1 millionfor the three months ended March 31, 2019, compared to $3.9 millionfor the same period in 2018. The $1.9 milliondecrease was primarily attributable to a $1.0 milliondecrease in revenue recognized pursuant to our collaboration agreement with Juno Therapeutics, a Celgenecompany that is a wholly-owned subsidiary of Celgene Corporation, and a $0.9 milliondecrease in revenue recognized pursuant to our strategic alliance with Allergan Pharmaceuticals International Limited, partially offset by $0.1 millionin revenue recognized in the first quarter of 2019 pursuant to an out-license agreement.
- Research and development expenses decreased by
$5.5 million, to $15.8 millionfor the three months ended March 31, 2019, from $21.3 millionfor the same period in 2018. The $5.5 milliondecrease was primarily attributable to $5.8 millionin decreased process and platform development expenses, mostly relating to the acquisition of certain non-capitalizable intangible assets during the first quarter of 2018, and $0.5 millionin decreased stock-based compensation expenses, partially offset by $0.6 millionin increased other expenses including facility-related expenses and $0.3 millionin increased employee related expenses.
- General and administrative expenses increased by
$3.3 million to $17.5 millionfor the three months ended March 31, 2019, from $14.2 millionfor the same period in 2018. The $3.3 millionincrease was primarily attributable to $1.9 millionin increased stock-based compensation expenses, $2.1 millionin increased professional service expenses and $0.7 millionin increased employee related expenses, partially offset by $1.4 millionin decreased intellectual property and patent related fees.
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 planned Phase 1/2 clinical trial for EDIT-101 (AGN-151587), including dosing patients in the second half of 2019. 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 Annual Report on Form 10-K, 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||$||2,069||$||3,927|
|Research and development||15,842||21,300|
|General and administrative||17,489||14,186|
|Total operating expenses||33,331||35,486|
|Other income, net:|
|Other (expense) income, net||(44||)||182|
|Interest income, net||2,057||438|
|Total other income, net||2,013||620|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.60||)||$||(0.67||)|
|Weighted-average common shares outstanding, basic and diluted||48,838,229||45,992,008|
|Editas Medicine, Inc.
Selected Condensed Consolidated Balance Sheet Items
(amounts in thousands)
|Cash, cash equivalents, and marketable securities||$||342,065||$||368,955
|Deferred revenue, net of current portion||105,865||115,614|
|Construction financing lease obligation, net of current portion||-
|Total stockholders’ equity||217,162||236,162|
Mark Mullikin(617) 401-9083 firstname.lastname@example.org Media Contact Cristi Barnett(617) 401-0113 email@example.com
Source: Editas Medicine, Inc.