Ophthotech Obtains Exclusive Global License to AAV Gene Therapy Program for BEST1 Related Retinal Diseases
April 11 2019 - 4:05PM
Business Wire
Ophthotech Corporation (NASDAQ: OPHT) announced today that the
Company has converted its option and entered into an exclusive
global license agreement with the University of Pennsylvania
(Penn), including the Perelman School of Medicine at the University
of Pennsylvania and the University of Pennsylvania School of
Veterinary Medicine, and the University of Florida Research
Foundation (UFRF) for rights to develop and commercialize novel
adeno-associated virus (AAV) gene therapy product candidates for
the treatment of Best vitelliform macular dystrophy, also known as
Best disease and other bestrophinopathies, which are diseases
related to mutations to the BEST1 gene.
Best disease, which generally affects individuals in both eyes,
is an orphan inherited retinal disease. In October 2018, Ophthotech
entered into an exclusive option agreement with Penn and UFRF for
rights to negotiate to acquire a license for novel AAV serotype 2
based gene therapy product candidates for the treatment of Best
disease. Since that time, Ophthotech has sponsored research at
Penn, facilitated by the Penn Center for Innovation (PCI), to
conduct preclinical and natural history studies for Best disease
and commenced IND-enabling activities. Based on current timelines
and subject to regulatory review, the Company expects to
initiate a Phase 1/2 clinical trial in the first half of 2021.
Ophthotech estimates that approximately 10,000 individuals in
the United States and the five major European markets on a combined
basis have Best disease. Patients with Best disease develop an egg
yolk-like vitelliform lesion in their macular region, which over
time leads to macular atrophy and permanent loss of central vision.
There is currently no FDA or EMA approved therapy to treat this
orphan inherited degenerative retinal disease.
Proof-of-concept studies demonstrating that the AAV-BEST1 gene
therapy product candidate restored the anatomy between
photoreceptors and retinal pigment epithelial (RPE) cells in the
naturally occurring canine disease model with distinct phenotypic
similarities to human bestrophinopathies were published in March
2018 in the journal Proceedings of the National Academy of Sciences
(PNAS), titled: “BEST1 gene therapy corrects a diffuse retina-wide
microdetachment modulated by light exposure.”
About Ophthotech Corporation
Ophthotech is a biopharmaceutical company specializing in the
development of transformative gene therapies and novel therapeutics
to treat retinal diseases, with a focus on orphan and age related
indications. For more information, please visit
www.ophthotech.com.
Forward-looking Statements
Any statements in this press release about Ophthotech’s future
expectations, plans and prospects constitute forward-looking
statements for purposes of the safe harbor provisions under the
Private Securities Litigation Reform Act of 1995. Forward-looking
statements include any statements about Ophthotech’s strategy,
future operations and future expectations and plans and prospects
for Ophthotech, and any other statements containing the words
“anticipate,” “believe,” “estimate,” “expect,” “intend”, “goal,”
“may”, “might,” “plan,” “predict,” “project,” “target,”
“potential,” “will,” “would,” “could,” “should,” “continue,” and
similar expressions. In this press release, Ophthotech’s
forward-looking statements include statements about the
implementation of its strategic plan, the timing, progress and
results of clinical trials and other research and development
activities and the potential utility of its product candidates.
Such forward-looking statements involve substantial risks and
uncertainties that could cause Ophthotech’s preclinical and
clinical development programs, future results, performance or
achievements to differ significantly from those expressed or
implied by the forward-looking statements. Such risks and
uncertainties include, among others, those related to the
initiation and the conduct and design of research programs and
clinical trials, availability of data from these programs,
expectations for regulatory matters, need for additional financing
and negotiation and consummation of in-license and/or acquisition
transactions and other factors discussed in the “Risk Factors”
section contained in the quarterly and annual reports that
Ophthotech files with the Securities and Exchange Commission. Any
forward-looking statements represent Ophthotech’s views only as of
the date of this press release. Ophthotech anticipates that
subsequent events and developments will cause its views to change.
While Ophthotech may elect to update these forward-looking
statements at some point in the future, Ophthotech specifically
disclaims any obligation to do so except as required by law.
OPHT-G
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version on businesswire.com: https://www.businesswire.com/news/home/20190411005845/en/
InvestorsKathy GalanteOphthotech CorporationVice
President, Investor Relations and Corporate
Communications212-845-8231kathy.galante@ophthotech.comMediaAlex
Van Rees, 973-442-1555 ext. 111SmithSolve LLC on behalf
of Ophthotech Corporationalex.vanrees@smithsolve.com
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