Filed IND application for EDIT-101 with FDA in
October
Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing
company, today reported business highlights and financial results
for the third quarter ended September 30, 2018. In addition, the
Company highlighted select corporate milestones and data from its
pipeline of CRISPR medicines under investigation.
“The filing of the IND for EDIT-101 for the treatment of LCA10
marks a significant milestone for Editas and brings us closer to
helping people living with this devastating disease,” said Katrine
Bosley, President and Chief Executive Officer of Editas Medicine.
“We have also made important progress on our engineered cell
medicines and we look forward to presenting data from our novel
program to treat Sickle Cell Disease and Beta-Thalassemia in an
oral presentation at the American Society of Hematology meeting
next month.”
Recent Achievements and
Outlook
- Filed EDIT-101 Investigational New Drug (IND)
application in October with U.S. Food and Drug Administration (FDA)
for treatment of Leber Congenital Amaurosis type 10
(LCA10). In addition, the National Institutes of Health
(NIH) determined that a Recombinant DNA Advisory Committee review
of the protocol for the Phase 1/2 trial of EDIT-101 was not
necessary, and the protocol is now registered with the NIH. The
Company expects to treat 10 to 20 patients in this open label, dose
escalation study in order to evaluate the safety and efficacy of
EDIT-101.
- Allergan and Editas Medicine to co-develop and equally
share profits and losses from EDIT-101 in the U.S. In the
third quarter, Allergan Pharmaceuticals International Limited
(Allergan) exercised its option to develop and commercialize
EDIT-101 under the strategic alliance formed in March 2017.
Allergan paid an optionexercise fee of $15 million, which was
recorded in the third quarter. In addition, Editas Medicine is
eligible to receive a $25 million milestone payment from Allergan
upon clearance of the IND application for EDIT-101. Editas Medicine
also exercised its option in the third quarter to co-develop and
equally share profits and losses in the U.S. for
EDIT-101.
- Novel approach to Sickle Cell Disease and
Beta-Thalassemia to be highlighted in an oral presentation at the
60th American Society of Hematology (ASH) Annual Meeting.
Editas Medicine will present pre-clinical in vivo data comparing
its novel gene editing strategy to reactivate fetal hemoglobin
(HbF) by editing the beta-globin locus to strategies that edit the
BCL11A erythroid enhancer (BCL11Ae). In this mouse study, editing
the beta-globin locus upregulated HbF with superior repopulation of
red blood cell precursors as compared to editing
BCL11Ae.
- Federal Appeals Court ruling affirms strength of
foundational CRISPR/Cas9 intellectual property. The U.S.
Court of Appeals for the Federal Circuit (Appeals Court) upheld the
U.S. Patent and Trademark Office no interference-in-fact decision
concerning certain CRISPR/Cas9 patents Editas Medicine exclusively
licenses from Broad Institute, Inc. The patents broadly cover
CRISPR/Cas9 gene editing in eukaryotic cells, which the Company
believes is essential to making CRISPR medicines.
- Added key leadership in oncology and engineered cell
medicines. Richard A. Morgan, Ph.D., a leading expert in
gene therapy and oncology, joined Editas Medicine as Senior Vice
President of Immunogenetics. Dr. Morgan brings more than 30 years
of scientific leadership in the life sciences industry.
- Strong balance sheet to advance Editas Medicine through
multiple value inflection points. The Company held cash,
cash equivalents, and marketable securities of $337.5 million as of
September 30, 2018, providing at least 24 months of funding for
operating expenses and capital expenditures without any assumption
of future cash received from milestones or additional
financings.
Upcoming Events
Editas Medicine will participate in the following investor
conferences:
- Credit Suisse 27th Annual Healthcare Conference, November 13,
3:25 p.m. PT, Phoenix;
- Barclays Gene Editing & Gene Therapy Summit, November 29,
1:15 p.m. ET, New York City; and
- J.P. Morgan 37th Annual Healthcare Conference, January 7-10,
2019, San Francisco
Editas Medicine will present pre-clinical data from its novel
program to address Sickle Cell Disease and Beta-Thalassemia in an
oral presentation at the 60th ASH Annual Meeting. Details are as
follows:
|
Oral
Abstract Number: 409 |
|
Title:
Comparative Studies Reveal Robust HbF Induction By Editing of
HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+ Cells
but That BCL11A Erythroid-Enhancer Editing Is Associated with
Selective Reduction in Erythroid Lineage Reconstitution in a
Xenotransplantation Model |
|
Presenter: KaiHsin Chang, Ph.D. |
|
Session:
112. Thalassemia and Globin Gene Regulation: Hemoglobin
Switching |
|
Time:
Sunday, December 2, 4:30 p.m. |
|
Location:
San Diego Convention Center, Room 25B |
Third Quarter 2018 Financial Results
Cash, cash equivalents, and marketable securities at September
30, 2018, were $337.5 million, compared to $329.1 million at
December 31, 2017.
For the third quarter ended September 30, 2018, net loss
attributable to common stockholders was $15.2 million, or $0.32 per
share, compared to $26.6 million, or $0.64 per share, for the same
period in 2017.
- Collaboration and other research and development revenues were
$14.5 million for the quarter ended September 30, 2018, compared to
$6.3 million for the same period in 2017. The $8.2 million increase
was primarily attributable to a $10.6 million increase in revenue
recognized pursuant to our strategic alliance with Allergan and
$0.1 million in revenue recognized pursuant to a license agreement
with Beam Therapeutics, Inc., partially offset by a $2.4 million
decrease in revenue recognized pursuant to our collaboration
agreement with Juno Therapeutics, Inc., a Celgene company and
wholly-owned subsidiary of Celgene Corporation.
- Research and development expenses were $17.4 million for the
quarter ended September 30, 2018, compared to $20.4 million for the
same period in 2017. The $3.0 million decrease was primarily
attributable to $4.9 million in decreased sublicensing expenses and
$1.0 million in decreased process and platform development
expenses, mostly related to reimbursable expenses related to the
Allergan profit-sharing arrangement. These decreases were partially
offset by $1.3 million in increased employee related expenses, $1.1
million in increased stock-based compensation expenses, $0.3
million in increased facility-related expenses and $0.3 million in
increased other expenses.
- General and administrative expenses were $13.3 million for the
quarter ended September 30, 2018, compared to $12.6 million for the
same period in 2017. The $0.7 million increase was primarily
attributable to $1.2 million in increased stock-based compensation
expenses, $1.1 million in increased employee related expenses, $0.7
million in increased professional service expenses and $0.2 million
in increased other expenses, partially offset by $2.4 million in
decreased intellectual property and patent related fees.
Conference Call The Editas Medicine management
team will host a conference call and webcast today, November 7,
2018, at 5:00pm ET. To access the call, please dial 844-348-3801
(domestic) or 213-358-0955 (international) and provide the passcode
8397886. A live webcast of the call will be available on the
Investors & Media section of the Editas Medicine website at
www.editasmedicine.com and a replay will be available approximately
two hours after its completion.
About Editas Medicine As a leading genome
editing company, Editas Medicine is focused on translating the
power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known
as Cas12a) genome editing systems into a robust pipeline of
treatments for people living with serious diseases around the
world. Editas Medicine aims to discover, develop,
manufacture, and commercialize transformative, durable, precision
genomic medicines for a broad class of diseases. For the
latest information and scientific presentations, please visit
www.editasmedicine.com.
About EDIT-101EDIT-101 is a CRISPR-based
experimental medicine under investigation for the treatment of
Leber Congenital Amaurosis type 10 (LCA10). EDIT-101 is an
AAV5 construct and is administered via a subretinal injection to
reach and deliver the gene editing machinery directly to
photoreceptor cells.
About Leber Congenital AmaurosisLeber
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.
About Allergan AllianceIn March 2017, Editas
Medicine and Allergan Pharmaceuticals International Limited
(Allergan) entered a strategic alliance and option agreement under
which Allergan received exclusive access and the option to license
up to five of Editas Medicine’s genome editing programs for ocular
diseases, including EDIT-101. Under the terms of the
agreement, Allergan is responsible for development and
commercialization of optioned products, subject to Editas
Medicine’s option to co-develop and share equally in the profits
and losses of two optioned products in the United States.
Editas Medicine is also eligible to receive development and
commercial milestones, as well as royalty payments on a per-program
basis where the parties are not sharing profits and losses.
The agreement covers a range of first-in-class ocular
programs targeting serious, vision-threatening diseases based on
Editas Medicine’s unparalleled CRISPR genome editing platform,
including CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a).
Forward-Looking Statements This press release
contains forward-looking statements and information within the
meaning of The Private Securities Litigation Reform Act of 1995.
The words “aim,” ‘‘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 planning to present data and the Company
developing and bringing transformative medicines to patients.
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 Securities and Exchange Commission,
and in other filings that the Company may make with the Securities
and Exchange Commission in the future. Any forward-looking
statements contained in this press release speak only as of the
date hereof, and the Company expressly disclaims any obligation to
update any forward-looking statements, whether as a result of new
information, future events or otherwise.
Editas Medicine, Inc. |
|
|
Condensed Consolidated Statements of
Operations |
|
|
(unaudited) |
|
|
(amounts in thousands, except per share and
share data) |
|
|
|
|
|
|
|
Three Months
Ended |
|
|
|
September 30, |
|
|
|
2018 |
|
|
2017 |
|
Collaboration and
other research and development revenues |
|
$ |
14,519 |
|
|
$ |
6,282 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research
and development |
|
|
17,443 |
|
|
|
20,396 |
|
General
and administrative |
|
|
13,334 |
|
|
|
12,635 |
|
Total
operating expenses |
|
|
30,777 |
|
|
|
33,031 |
|
Operating loss |
|
|
(16,258 |
) |
|
|
(26,749 |
) |
Other income, net: |
|
|
|
|
|
|
|
|
Other
(expense) income, net |
|
|
(4 |
) |
|
|
196 |
|
Interest
income (expense), net |
|
|
1,024 |
|
|
|
(46 |
) |
Total
other income, net |
|
|
1,020 |
|
|
|
150 |
|
Net loss |
|
$ |
(15,238 |
) |
|
$ |
(26,599 |
) |
Net loss per share
attributable to common stockholders, basic and diluted |
|
$ |
(0.32 |
) |
|
$ |
(0.64 |
) |
Weighted-average common
shares outstanding, basic and diluted |
|
|
47,414,271 |
|
|
|
41,307,092 |
|
|
|
|
|
|
|
|
|
|
Editas Medicine,
Inc. |
Selected Condensed Consolidated Balance Sheet
Items |
(unaudited) |
(amounts in thousands) |
|
|
|
|
|
|
|
|
|
|
|
September 30, |
|
December 31, |
|
|
|
2018 |
|
2017 |
|
|
|
|
|
|
|
|
Cash, cash
equivalents, and marketable securities |
|
$ |
337,492 |
|
$ |
329,139 |
Working
capital |
|
|
316,467 |
|
|
295,492 |
Total
assets |
|
|
387,630 |
|
|
373,260 |
Deferred revenue,
net of current portion |
|
|
|
104,100 |
|
|
94,725 |
Construction financing
lease obligation, net of current portion |
|
|
|
32,694 |
|
|
33,431 |
Total stockholders’
equity |
|
|
|
224,449 |
|
|
208,080 |
|
|
|
|
|
|
|
|
Media ContactCristi BarnettEditas Medicine,
Inc.(617) 401-0113 cristi.barnett@editasmed.com
Investor ContactMark MullikinEditas Medicine,
Inc.(617) 401-9083mark.mullikin@editasmed.com
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