Allergan plc (NYSE: AGN), a leading global pharmaceutical company,
and Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing
company, announced initial data from the ongoing natural history
study to evaluate patients with Leber congenital amaurosis 10
(LCA10), a rare form of blindness caused by mutations in the CEP290
gene. The Companies reported these data on Friday, April 26, in an
oral presentation at the 6th Annual Retinal Cell and Gene Therapy
(RCGT) Innovation Summit in Vancouver.
The Companies initiated the natural history study to
prospectively evaluate and confirm the course of disease in
patients and determine the reproducibility and stability of
ophthalmic assessments over time. This knowledge has informed
the planned Phase 1/2 interventional clinical trial design for
AGN-151587 (EDIT-101), an experimental CRISPR genome editing
medicine being investigated for the treatment of LCA10.
Massachusetts Eye and Ear, an international center for treatment
and research and a teaching hospital of Harvard Medical School, is
one of seven sites enrolling patients in this natural history
study.
“Better understanding the course of disease for patients with
CEP290-associated retinal degeneration is a key step towards the
interventional clinical trial for a CRISPR-based experimental
medicine to treat this disease,” said Eric A. Pierce, M.D., Ph.D.,
Director of the Ocular Genomics Institute and William F. Chatlos
Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard
Medical School, and Principal Investigator for the natural history
study. “We are pleased to be working at the forefront of this
research with Allergan and Editas Medicine and look forward to
testing genome editing for the treatment of CEP290-associated
retinal disease in the interventional trial.”
Twenty-one patients with LCA10-IVS26 mutations in CEP290 were
included in this analysis of baseline characteristics and key
clinical assessments, including visual acuity and full-field
threshold sensitivity changes. Initial data from the natural
history study suggest multiple clinical assessments should prove
informative in the planned Phase 1/2 interventional trial.
AGN-151587 is set to be the first in vivo, or inside the body,
CRISPR-based medicine administered to people anywhere in the world.
In the planned Phase 1/2 clinical trial, Allergan and Editas
Medicine intend to initiate patient screening mid-year and begin
patient dosing in the second half of 2019, enrolling 10-20 patients
in the U.S. and Europe.
About AGN-151587 (EDIT-101)AGN-151587
(EDIT-101) is a CRISPR-based experimental medicine under
investigation for the treatment of Leber congenital amaurosis 10
(LCA10). AGN-151587 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 the Editas Medicine-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 AGN-151587
(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. In August 2018, Allergan exercised its
option to develop and commercialize AGN-151587 globally for the
treatment of LCA10. Additionally, Editas Medicine exercised its
option to co-develop and share equally in the profits and losses
from AGN-151587 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. 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).
About Allergan plcAllergan plc (NYSE: AGN),
headquartered in Dublin, Ireland, is a bold, global pharmaceutical
leader. Allergan is focused on developing, manufacturing and
commercializing branded pharmaceutical, device, biologic, surgical
and regenerative medicine products for patients around the
world.
Allergan markets a portfolio of leading brands and best-in-class
products primarily focused on four key therapeutic areas including
medical aesthetics, eye care, central nervous system and
gastroenterology.
Allergan is an industry leader in Open Science, a model of
research and development, which defines our approach to identifying
and developing game-changing ideas and innovation for better
patient care. With this approach, Allergan has built one of the
broadest development pipelines in the pharmaceutical industry.
Allergan’s success is powered by our global colleagues’
commitment to being Bold for Life. Together, we build bridges,
power ideas, act fast and drive results for our customers and
patients around the world by always doing what is right.
With commercial operations in approximately 100 countries,
Allergan is committed to working with physicians, healthcare
providers and patients to deliver innovative and meaningful
treatments that help people around the world live longer, healthier
lives every day.
For more information, visit Allergan’s website
at www.Allergan.com.
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.
Allergan Forward-Looking StatementsStatements
contained in this press release that refer to future events or
other non-historical facts are forward-looking statements that
reflect Allergan’s current perspective on existing trends and
information as of the date of this release. Actual results may
differ materially from Allergan’s current expectations depending
upon a number of factors affecting Allergan’s business. These
factors include, among others, the difficulty of predicting the
timing or outcome of FDA approvals or actions, if any; the impact
of competitive products and pricing; market acceptance of and
continued demand for Allergan’s products; the impact of uncertainty
around timing of generic entry related to key products, including
RESTASIS®, on our financial results; risks associated with
divestitures, acquisitions, mergers and joint ventures; risks
related to impairments; uncertainty associated with financial
projections, projected cost reductions, projected debt reduction,
projected synergies, restructurings, increased costs, and adverse
tax consequences; difficulties or delays in manufacturing; and
other risks and uncertainties detailed in Allergan’s periodic
public filings with the Securities and Exchange Commission,
including but not limited to Allergan's Annual Report on Form 10-K
for the year ended December 31, 2018. Except as expressly required
by law, Allergan disclaims any intent or obligation to update these
forward-looking statements.
Editas Medicine Forward-Looking StatementsThis
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
Companies’ plans with respect to the planned Phase 1/2 clinical
trial for AGN-151587 (EDIT-101), including initiating patient
screening by mid-year and dosing patients in the second half of
2019. Editas Medicine 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 Editas Medicine’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 Editas Medicine’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 Editas
Medicine’s most recent Annual Report on Form 10-K, which is on file
with the Securities and Exchange Commission, and in other filings
that Editas Medicine 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 Editas Medicine expressly disclaims any obligation to update
any forward-looking statements, whether because of new information,
future events or otherwise.
Contacts:Allergan: Investors:Manisha
Narasimhan, Ph.D.(862)
261-7162manisha.narasimhan@allergan.com |
Editas
Medicine:Investors:Mark Mullikin(617)
401-9083mark.mullikin@editasmed.com |
Media:Fran DeSena(862) 261
8820frances.desena@allergan.com |
Media:Cristi Barnett(617)
401-0113cristi.barnett@editasmed.com |
Editas Medicine (NASDAQ:EDIT)
Historical Stock Chart
From Mar 2024 to Apr 2024
Editas Medicine (NASDAQ:EDIT)
Historical Stock Chart
From Apr 2023 to Apr 2024