Scientific Data for Novel Adeno-Associated Virus Gene Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa P...
August 22 2018 - 7:30AM
Business Wire
- Preclinical Proof-of-Concept Results in a
Large Animal Model Published Online Ahead of Print in PNAS -
Ophthotech Corporation (NASDAQ: OPHT) announced today that
scientists at the University of Pennsylvania (Penn) and University
of Florida published proof-of-concept study results on an
adeno-associated virus (AAV) gene therapy product candidate for the
treatment of rhodopsin-mediated autosomal dominant retinitis
pigmentosa (RHO-adRP) licensed by Ophthotech. Their work conducted
in a naturally occurring canine model of the disease was published
online ahead of print in the journal Proceedings of the National
Academy of Sciences of the USA (PNAS). In June 2018, Ophthotech
announced that it entered into an exclusive global license
agreement with the University of Florida Research Foundation and
Penn for rights to develop and commercialize this novel AAV gene
therapy product candidate for the treatment of RHO-adRP. In
addition to the exclusive license agreement, Ophthotech and Penn
have also entered into a master sponsored research agreement,
facilitated by the Penn Center for Innovation, pursuant to which
Ophthotech and Penn plan to conduct additional preclinical studies
of the RHO-adRP product candidate and a natural history study in
RHO-adRP patients.
This publication is entitled: “Mutation-independent Rhodopsin
Gene Therapy by Knockdown and Replacement with a Single AAV vector”
by Artur V. Cideciyan, Raghavi Sudharsan, Valérie L. Dufour,
Michael T. Massengill, Simone Iwabe, Malgorzata Swider, Brianna
Lisi, Alexander Sumaroka, Luis Felipe Marinho, Tatyana Appelbaum,
Brian Rossmiller, William W. Hauswirth, Samuel G. Jacobson, Alfred
S. Lewin, Gustavo D. Aguirre, and William A. Beltran. PNAS
2018.
RHO-adRP is an orphan monogenic inherited retinal disease that
is characterized by progressive and severe loss of vision, and is
caused by more than 150 different mutations in the RHO gene. The
construct for the RHO-adRP product candidate combines a transgene
expressing a highly efficient, novel short hairpin RNA (shRNA)
designed to target and knock-down endogenous rhodopsin (RHO) in a
mutation-independent manner with a human RHO replacement transgene
made resistant to RNA interference, in a single AAV2/5 vector. This
construct was tested in a naturally-occurring canine disease model
of RHO-adRP by investigators at Penn, resulting in a complete
suppression of the endogenous RHO RNA while the human RHO
replacement transgene resulted in up to 30% of normal RHO protein
levels. Long term (over 8 months) anatomic and functional
preservation was demonstrated with retinal imaging and
electrophysiology. Ophthotech believes these results further
confirm the therapeutic benefit of a similar knock-down and
replacement approach that was tested in mice by investigators at
the University of Florida, the results of which were previously
published in Human Gene Therapy in 2012.
“Our prior research indicated that the canine animal model may
provide useful predictive data when developing treatment strategies
for patients with degenerative retinal diseases,” stated Professor
William A. Beltran, DVM, PhD, Director of the Division of
Experimental Retinal Therapies, Department of Clinical Sciences
& Advanced Medicine, School of Veterinary Medicine, University
of Pennsylvania. “We are honored to have our preclinical research
in AAV therapy for RHO-adRP published in PNAS.”
“We are excited to collaborate with prominent scientists in the
field of degenerative retinal diseases at the University of
Pennsylvania and University of Florida and congratulate them on the
strength and elegance of this publication,” stated Kourous A.
Rezaei, M.D., Chief Medical Officer of Ophthotech. “We are
intrigued by these proof-of-concept results in the canine disease
model which further support the potential therapeutic impact of
this AAV gene therapy product in patients with RHO-adRP. We are
commencing IND-enabling activities, and based on current timelines
and subject to regulatory review, we expect to initiate a Phase 1/2
clinical trial for the treatment of RHO-adRP in 2020.”
About RHO-adRP
RHO-adRP is an orphan monogenic inherited retinal disease that
is characterized by progressive and severe loss of vision.
Ophthotech estimates that there are approximately 11,000
individuals in the United States and the five major European
markets with RHO-adRP. There is currently no U.S. Food and Drug
Administration or European Medicines Agency approved therapy to
treat this orphan inherited retinal disease.
About Ophthotech Corporation
Ophthotech is a science-driven biopharmaceutical company
specializing in the development of novel therapies to treat
ophthalmic diseases, with a focus on age-related and orphan retinal
diseases. 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 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/20180822005074/en/
InvestorsOphthotech CorporationKathy Galante,
212-845-8231Vice President, Investor Relations and Corporate
Communicationskathy.galante@ophthotech.comorMediaSmithSolve
LLC on behalf of Ophthotech CorporationAlex Van Rees,
973-442-1555 ext. 111alex.vanrees@smithsolve.com
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