Editas Medicine Announces Second Quarter 2020 Results and Update
Regained full control of ocular medicines under new agreement with AbbVie
BRILLIANCE trial for EDIT-101 on track to dose at least three patients by end of 2020
Plan to file IND for EDIT-301 for sickle cell disease by end of 2020
Strengthened balance sheet through equity offering raising
Cash, cash equivalents, and marketable securities of
“I am extremely proud of our recent progress, as we remain on track with previously shared guidance despite the unpredictable challenges presented by COVID-19,” said
Recent Achievements and Outlook
In Vivo CRISPR Medicines
- Ocular Medicines
Regained full control of ocular medicines
Editas Medicine(Company) has terminated its 2017 agreement with Allergan, now part of AbbVie, and entered a new agreement with AbbVie that returns development and commercialization rights for ocular medicines to the Company. The Company plans to continue to advance ocular medicines including EDIT-101 for Leber congenital amaurosis 10 (LCA10).
- EDIT-101 for LCA10
Continuation of dosing in BRILLIANCE Phase 1/2 clinical trial
Enrollment remains active and sites have reopened for dosing following a brief pause due to COVID-19. The study, which represents the first and only clinical trial to administer an in vivo CRISPR medicine, remains on track to complete dosing of the adult low-dose cohort (two patients) and dose at least one patient in the adult mid-dose cohort by the end of 2020.
Engineered Cell Medicines
- EDIT-301 for Sickle Cell Disease and Beta-Thalassemia
Preclinical data presented at EHA reinforces best-in-class potential
Data presented at the 25th
Congressof the European Hematology Association(EHA) showed that treatment with EDIT-301, which leverages the Company’s proprietary Cas12a (Cpf1) enzyme to edit directly at the HBG1/2 promoter, resulted in long-term in vivo editing with elevated and pan-cellular fetal hemoglobin expression. The Company remains on track to file an investigational new drug application (IND) for EDIT-301 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 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 plans to file an IND for EDIT-201 in the second half of 2021.
- Offering of Common Stock
Strengthened balance sheet with gross proceeds of approximately
$216 million Editas Medicineclosed an underwritten offering of 6,900,000 shares of its common stock at a public offering price of $31.25per share, before deducting underwriter discounts and commissions and estimated offering expenses. This included 900,000 shares issued upon exercise in full by the underwriter of its option to purchase additional shares. All shares in the offering were sold by Editas Medicine.
Editas Medicinehas appointed Gad Berdugoas Chief Business Officer. Mr. Berdugowill lead business and corporate development, alliance management, and strategic planning for the Company. He brings more than 25 years of business and corporate development experience, most recently serving as Chief Executive Officer of EpiVax Oncology, a precision cancer immunotherapy company that he co-founded in 2017.
Editas Medicineestablished manufacturing agreements with Azzur Groupand Catalent to support preclinical and clinical development of the Company’s portfolio of in vivo CRISPR and engineered cell medicines, including EDIT-101, EDIT-201, and EDIT-301. Following the termination of the 2017 agreement with Allergan, the Company has entered into a transition services agreement to transfer certain manufacturing material and processes for EDIT-101 and EDIT-102 from Allergan to Editas Medicine.
- 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 in the interference took place on 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
$598.7 millionat June 30, 2020, and anticipated interest income will enable it to fund its operating expenses and capital expenditures into 2023.
Second 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
$10.7 millionfor the three months ended June 30, 2020, compared to $2.3 millionfor the same period in 2019. The $8.4 millionincrease was primarily attributable to $7.6 millionin cash revenues received in connection with an out-license agreement and $0.8 millionin non-cash revenue earned under our ongoing collaborations.
- Research and development expenses increased by
$4.4 million, to $28.0 millionfor the three months ended June 30, 2020, from $23.6 millionfor the same period in 2019. The $4.4 millionincrease was primarily attributable to fees incurred related to licensing and sublicensing activities, research personnel growth to support our programs as well as our expansion of the development organization and facilities These increases were partially offset by a decrease in process and platform expenses and share-based compensation.
- General and administrative expenses decreased by
$0.3 millionto $14.1 millionfor the three months ended June 30, 2020, from $14.4 millionfor the same period in 2019. The $0.3 milliondecrease was primarily attributable to a decrease in the expense for professional service expenses and patent related fees which was partially offset by an increase is costs related to the hiring of key executives in the second half of 2019 and into 2020.
Society for Immunotherapy of Cancer35th Annual Meeting, November 10-15, Virtual.
- Citi’s 15th Annual
BioPharma Conference, September 9-10, Virtual;
Wells Fargo Healthcare Conference, September 9-10, Virtual; and
- Morgan Stanley 18th Annual Global Healthcare Conference,
September 14-18, Virtual.
As a leading genome editing company,
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 using the proprietary Staphylococcus aureus Cas9 (SaCas9) enzyme, which can be packaged in a single adeno-associated virus (AAV) to deliver the gene editing machinery to photoreceptor cells. EDIT-101 is the first in vivo CRISPR medicine administered to humans.
EDIT-201 is an experimental, allogeneic natural killer (NK) cell medicine under investigation for the treatment of solid tumor cancers. EDIT-201 is comprised of NK cells derived from pooled healthy donor blood and genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP).
EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.
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 timing of dosing in and updates related to the Phase 1/2 BRILLIANCE clinical trial for EDIT-101, filing an IND for EDIT-301 by the end of 2020, and filing an IND for EDIT-201 in the second half of 2021. 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, that the Company's expectations regarding the effects of COVID-19 may be incorrect, 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 Quarterly Report on Form 10-Q, 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||$||10,749||$||2,330|
|Research and development||28,007||23,565|
|General and administrative||14,081||14,414|
|Total operating expenses||42,088||37,979|
|Other income, net:|
|Other income (expense), net||7,175||(68||)|
|Interest income, net||592||1,931|
|Total other income, net||7,767||1,863|
|Net loss per share basic and diluted||$||(0.43||)||$||(0.69||)|
|Weighted-average common shares outstanding, basic and diluted||55,346,052||49,070,574|
|Selected Condensed Consolidated Balance Sheet Items|
|(amounts in thousands)|
|Cash, cash equivalents, and marketable securities||$||598,720||$||457,140|
|Deferred revenue, net of current portion||138,406||163,207|
|Total stockholders’ equity||426,772||262,437|
Source: Editas Medicine, Inc.