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EDIT-101 for Leber Congenital Amaurosis type 10 (LCA10) on track for mid-2018 Investigational New Drug (IND) filing
Expecting at least five clinical-stage programs by end of 2022 as part of EM22 five-year goals
Year-end cash, cash equivalents, and marketable securities of
“Our accomplishments in 2017 provide strong momentum into 2018 and beyond as we work to bring transformative medicines to patients,” said
Recent Achievements and Anticipated Progress
Bringing transformative medicines to patients
- EDIT-101 for LCA10 remains on track for a mid-2018 IND filing. Over the past year, the Company has reported on its robust package of preclinical pharmacology data that supports this IND filing at multiple scientific and medical congresses. In addition,
Editas Medicinepresented data at the Keystone Symposium on Genome Engineering with Programmable Nucleases demonstrating low prevalence of pre-existing antibodies in humans to Streptococcus pyogenes Cas9 and Staphylococcus aureus Cas9. A clinical natural history study of LCA10 patients is underway to inform human interventional clinical trial design and facilitate enrollment.
- Broader ocular pipeline emerging with programs for the treatment of recurrent ocular herpes simplex virus type 1 (HSV-1) infection and Usher Syndrome type 2a (USH2a). For the recurrent ocular HSV-1 program, the Company will present in vivo proof-of-concept data at the
Association for Research in Vision & Ophthalmologyin late April. For the USH2a program, the Company and collaborators at Massachusetts Eye and Ear plan to present results that validate a potential gene editing approach in the first half of the year.
- First oncology candidate from collaboration with
Juno Therapeutics, Inc.( Juno Therapeutics) progressing towards clinical trials. Juno Therapeuticsplans to begin IND-enabling studies this year and aims to initiate human clinical trials next year for a T cell medicine, with Editas Medicine'sproprietary gene edits, to treat human papillomavirus (HPV)-associated solid tumors.
- Exploring development of a superior medicine for sickle cell disease and beta-thalassemia. The Company is pursuing multiple approaches including gene disruption to durably induce high levels of fetal hemoglobin and gene insertion to restore adult hemoglobin while simultaneously eliminating sickle hemoglobin. The Company expects to present its latest progress on its work to induce high levels of fetal hemoglobin in the first half of the year.
Advancing organizational excellence
- Strengthened Board of Directors with appointment of
Jessica Hopfield, Ph.D. Dr. Hopfield is a former Partner of McKinsey & Companywith more than 20 years of experience in the medical and healthcare fields.
Building a sustainable and valued business
- Acquired certain assets and capabilities from i2 Pharmaceuticals’ and certain of its affiliated companies for guide RNA engineering and manufacturing. This acquisition brings world-class RNA chemistry capabilities and proprietary classes of guide RNAs with distinct intellectual property and exemplifies our continued commitment to build an unparalleled genome editing platform to develop best-in-class CRISPR medicines.
- Strengthened balance sheet to fund business through multiple value inflection points. The Company held cash, cash equivalents, and marketable securities of
$329 millionas of December 31, 2017, providing at least 24 months of funding for operating expenses and capital expenditures. In addition, the Company raised approximately $50 millionin gross proceeds in the first quarter of 2018 through its at-the-market facility.
- Submit IND for LCA10 program by mid-2018;
- Report preclinical proof-of-concept from additional programs;
- Advance manufacturing capabilities to enable additional IND(s) in 2019;
- Establish additional important strategic alliances; and
- Continue to build a best-in-class organization and culture.
Editas Medicine’s EM22 Vision and Goals
By the end of 2022,
- At least three experimental medicines in early-stage clinical trials;
- At least two experimental medicines in or ready for late-stage clinical trials; and
- A best-in-class platform, pipeline, and organizational culture for developing genomic medicines.
These goals build on Editas Medicine’s current success and on the breadth of its platform to make genome editing medicines. The EM22 goals include delivering at least two experimental medicines in ophthalmology and at least one from the collaboration with
Cowen & Company38th Annual Health Care Conference, March 14, 8:00 a.m. ET, Boston; Barclays Global Healthcare Conference, March 15, 8:30 a.m. ET, Miami;
- Morgan Stanley Healthcare Corporate Access Day,
March 20, Boston; and
- Oppenheimer 28th Annual Healthcare Conference,
March 21, 9:45 a.m. ET, New York. Association for Research in Vision & Ophthalmology, April 29-May 3, Honolulu; and American Society of Gene & Cell Therapy, May 16-19, Chicago.
Fourth Quarter and Full Year 2017 Financial Results
Cash, cash equivalents, and marketable securities at
For the three months ended
- Collaboration and other research and development revenues were
$3.7 millionfor the three months ended December 31, 2017, compared to $0.9 millionfor the same period in 2016. The $2.8 millionincrease was primarily due to a $3.2 millionincrease in revenue recognized pursuant to our strategic alliance with Allergan, partially offset by a $0.4 milliondecrease in reimbursable research and development expenses.
- Research and development expenses decreased by
$0.4 million, to $26.4 millionfor the three months ended December 31, 2017, from $26.8 millionfor the same period in 2016. The $0.4 milliondecrease was primarily related to a $16.5 milliondecrease in license fees primarily related to payments due under certain licensing agreements that were executed in 2016, and a $0.2 milliondecrease in other expenses including facility-related expenses, partially offset by a $9.5 millionincrease in success payments due to triggering multiple success payments under the previously mentioned license agreements, a $3.4 millionincrease in stock-based compensation expense, a $2.6 millionincrease in process and platform development costs, and a $0.8 millionincrease in employee related expenses.
- General and administrative expenses increased by
$0.7 million to $13.7 millionfor the three months ended December 31, 2017, from $13.0 millionfor the same period in 2016. The $0.7 millionincrease was primarily related to a $0.9 millionincrease in stock-based compensation, a $0.4 millionincrease in other expenses including facility-related expenses, and a $0.4 millionincrease in employee related expenses, partially offset by a $1.0 milliondecrease in patent related expenses.
For the full year 2017, net loss attributable to common stockholders was
- Collaboration and other research and development revenues were
$13.7 millionfor 2017, compared to $6.1 millionfor 2016. The increase of $7.6 millionwas due to a $8.8 millionincrease in revenue recognized related to our strategic alliance with Allergan, partially offset by a $1.2 milliondecrease in reimbursable research and development expenses.
- Research and development expenses for 2017 were
$83.2 million, compared to $57.0 millionfor 2016. The increase of $26.2 millionwas due to a $14.5 millionincrease in success payments due to triggering multiple success payments under certain licensing agreements during 2017, a $7.5 millionin increase in process and platform development expenses, a $5.3 millionincrease in employee related expenses, a $2.5 millionincrease in stock-based compensation, and a $0.2 millionincrease in other expenses including facility-related expenses, partially offset by a decrease of $3.8 millionin license fees primarily related to payments due under certain licensing agreements that were executed in 2016.
- General and administrative expenses were
$50.5 millionfor 2017, compared to $46.3 millionfor 2016. The increase of $4.2 millionwas due to a $4.0 millionincrease in stock-based compensation, a $2.0 millionincrease in employee related expenses, a $0.7 millionincrease in other expenses including facility-related expenses, and a $0.5 millionincrease in professional service expenses, partially offset by a $3.0 milliondecrease in intellectual property and patent related fees associated with patents and patent applications, which was primarily due to the fact that the Company’s in-licensors had additional legal costs during the year ended December 31, 2016.
- Other income (expense), net for 2017 was
$(0.4) million, compared to $5 thousandfor 2016. The decrease was primarily attributable to interest expense on certain promissory notes, incurring a full year of interest expense on our construction financing lease obligation, and amortization of premiums associated with marketable securities, partially offset by rental income from our subtenant, interest income, and accretion of discounts associated with marketable securities.
As a leading genome editing company,
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,’’ “goals,” ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ “track,” ‘‘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 2018 goals, including, without limitation, submitting an IND for the LCA10 program by mid-2018, the Company’s EM22 goals, including, without limitation, having five experimental medicines in the clinic by 2022, and establishing additional alliances. 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 Quarterly Report on Form 10-Q, which is on file with the
|EDITAS MEDICINE, INC.
|Consolidated Statement of Operations
|(amounts in thousands, except share and per share data)
|Three Months Ended
||Twelve Months Ended
|Collaboration and other research|
|and development revenues||$||3,667||$||898||$||13,728||$||6,053|
|Research and development||26,424||26,835||83,159||56,979|
|General and administrative||13,685||13,047||50,502||46,262|
|Total operating expenses||40,109||39,882||133,661||103,241|
|Other income (expense), net:|
|Other income (expense), net||129||(35)||587||(57)|
|Interest income (expense), net||124||(357)||(978)||62|
|Total other income (expense), net||253||(392)||(391)||5|
|Accretion of redeemable|
|convertible preferred stock to|
|Net loss attributable to common stockholders|
|Net loss per share attributable|
|to common stockholders,|
|basic and diluted||$||(0.84)||$||(1.10)||$||(2.98)||$||(3.02)|
|Weighted-average common shares outstanding, basic and|
|EDITAS MEDICINE, INC.
|Selected Consolidated Balance Sheet Items
|(amounts in thousands)
|Cash, cash equivalents, and marketable securities||$||329,139||$||185,323|
|Deferred revenue, net of current portion||94,725||26,000|
|Construction financing lease obligation, net of|
|Total stockholders' equity||208,080||134,607|
Source: Editas Medicine, Inc.