Editas Medicine Announces First Quarter 2020 Results and Update
Patient dosing initiated in BRILLIANCE Phase 1/2 trial of EDIT-101 (AGN-151587) for LCA10
On track to file IND for EDIT-301 for sickle cell disease by end of 2020
Initiated IND-enabling studies for EDIT-201 allogeneic NK cell medicine for solid tumors
Cash, cash equivalents, and marketable securities of
“The COVID-19 pandemic has given rise to unprecedented circumstances for Editas and the broader life sciences community,” said
Recent Achievements and Outlook
- COVID-19 Impact
Potential business interruptions due to the COVID-19 outbreak
Editas Medicine(Company) continues to evaluate the impact of the global COVID-19 pandemic on the continuity of the Company’s research, clinical trial activity, and business outlook. The Company has taken numerous steps to protect its employees, patients, and partners, while working to ensure the productivity of its business operations. As of March 12, the Company transitioned to remote working for office-based personnel. In addition, essential laboratory, manufacturing, and related support activities have been subject to precautionary measures to ensure the safety of our workforce.
In Vivo CRISPR Medicines
- EDIT-101 for LCA10
First in vivo CRISPR medicine administered to a patient
Editas Medicineand Allergan announced the treatment of the first patient in the BRILLIANCE Phase 1/2 clinical trial of EDIT-101 at Oregon Health & Science University Casey Eye Institute, a world-recognized academic eye center. The study has been cleared to continue based on a review of safety data on the first patient. The Company plans to complete dosing of the adult low-dose cohort and to dose at least one patient of the adult mid-dose cohort by the end of 2020.
- EDIT-102 for Usher Syndrome 2A
Ready for IND-enabling studies pending Allergan option exercise
Under the terms of its 2017 alliance agreement with Allergan,
Editas Medicinehas delivered a preclinical data package to Allergan for potential licensing. EDIT-102 is ready for IND-enabling studies pending Allergan’s option exercise. A decision is expected by the third quarter of 2020.
- Autosomal Dominant Retinitis Pigmentosa 4
Nomination of development candidate delayed to 2021
Due to interruptions from the COVID-19 outbreak,
Editas Medicineis delaying to 2021 the declaration of a development candidate for an experimental medicine to treat autosomal dominant retinitis pigmentosa 4.
Engineered Cell Medicines
- EDIT-301 for Sickle Cell Disease and Beta-Thalassemia
IND filing expected by end of 2020
Editas Medicineis developing EDIT-301 using Cas12a (Cpf1), a proprietary enzyme, as a potentially best-in-class medicine to treat sickle cell disease and beta-thalassemia. Preclinical in vivo toxicology studies are in progress and the Company expects to file an IND for sickle cell disease by the end of 2020.
- EDIT-201 to Treat Solid Tumors
Declared candidate and initiated IND-enabling studies for allogeneic NK cell medicine
EDIT-201 is an allogeneic healthy-donor natural killer (NK) cell medicine for the treatment of solid tumors.
Editas Medicineplans to present preclinical data on EDIT-201 at a scientific conference in the second half of 2020.
The Company has strengthened its executive leadership team with the appointment of
Clare Carmichaelas Chief Human Resources Officer (CHRO). Clare brings more than 30 years of human resources experience and joins Editas Medicinefrom Wave Life Sciences, a clinical-stage genetics medicine company, where she served as CHRO.
- Intellectual Property
June 24, 2019, the U.S. Patent and Trademark Officedeclared an interference 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. Oral arguments have been scheduled for May 18, 2020. The Broad patents remain valid and in force. Foundational claims covering the use of CRISPR/Cas9 for gene editing in eukaryotic cells have issued and continue to issue to Broad as patents in the United States, Europe, Japan, and other jurisdictions.
- Balance Sheet
The Company expects that its existing cash, cash equivalents and marketable securities of
$415.0 millionat March 31, 2020, and anticipated interest income will enable it to fund its operating expenses and capital expenditures for at least the next 24 months. The Company remains committed to diligently managing expenses to maintain a strong balance sheet moving forward.
First Quarter for 2020 Financial Results
Cash, cash equivalents, and marketable securities at
For the three months ended
- Collaboration and other research and development revenues were
$5.7 millionfor the three months ended March 31, 2020, compared to $2.1 millionfor the same period in 2019. The $3.7 millionincrease was primarily attributable to an increase in revenue recognized pursuant to our strategic alliance with Allergan.
- Research and development expenses increased by
$18.7 million, to $34.6 millionfor the three months ended March 31, 2020, from $15.8 millionfor the same period in 2019. The $18.7 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 Allergan in the United Statesfor EDIT-101, and expenses incurred related to an in-license arrangement entered into during the first quarter of 2020.
- General and administrative expenses increased by
$0.3 millionto $17.8 millionfor the three months ended March 31, 2020, from $17.5 millionfor the same period in 2019. The $0.3 millionincrease was primarily attributable to increased employee related expenses.
American Society of Gene & Cell TherapyAnnual Meeting, May 12-15. Raymond James Human Health Innovation Conference, June 18, 9:00 a.m. ET.
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.
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) and filing an IND for EDIT-301 by the end of 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. Those risks and uncertainties include, among other things, the uncertainties related to the impact of the COVID-19 pandemic to the Company’s business, operations, strategy and goals, that data from the Company's development programs may not support registration or further development of its potential medicines due to safety, efficacy or other reasons, and other risks listed under “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the
|Condensed Consolidated Statements of Operations|
|(amounts in thousands, except per share and share data)|
|Three Months Ended|
|Collaboration and other research and development revenues||$||5,723||$||2,069|
|Research and development||34,570||15,842|
|General and administrative||17,769||17,489|
|Total operating expenses||52,339||33,331|
|Other income, net:|
|Other income (expense), net||7,333||(44)|
|Interest income, net||1,559||2,057|
|Total other income, net||8,892||2,013|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.69)||$||(0.60)|
|Weighted-average common shares outstanding, basic and diluted||54,590,194||48,838,229|
|Selected Condensed Consolidated Balance Sheet Items|
|(amounts in thousands)|
|Cash, cash equivalents, and marketable securities||$||414,993||$||457,140|
|Deferred revenue, net of current portion||139,372||163,207|
|Total stockholders’ equity||234,567||262,437|
Source: Editas Medicine, Inc.