European Commission Grants Ocugen Orphan Medicinal Product Designation for Gene Therapy Product Candidate, OCU400, For the Tr...
23 February 2021 - 11:30PM
Ocugen, Inc., (NASDAQ: OCGN), a biopharmaceutical company focused
on discovering, developing, and commercializing gene therapies to
cure blindness diseases and developing a vaccine to save lives from
COVID-19, today announced that on the recommendation of the
European Medicines Agency (EMA), the European Commission has
granted orphan medicinal product designation for OCU400
(AAV5-hNR2E3), for the treatment of both retinitis pigmentosa (RP)
and Leber Congenital amaurosis (LCA).
The prevalence of RP in Europe is estimated at approximately
165,000 patients and the prevalence of LCA in Europe is estimated
at approximately 40,000 patients. Globally, the number of people
suffering from RP and LCA is estimated to be around 2.0 million and
0.2 million, respectively.
“We believe the granting of this designation by the European
Commission validates the potential of our modifier gene therapy
platform to treat many inherited retinal diseases (IRDs). IRDs
associated with RP and LCA diseases are caused by mutations in over
175 genes, and it is impractical to develop therapies that are
specific to each gene. OCU400 has the remarkable potential to
address a significant number of patients globally who are in
desperate need of rescue from these blindness diseases and we are
working diligently to move this program to clinic,” said Dr.
Shankar Musunuri, Chairman of the Board, Chief Executive Officer,
and Co-founder of Ocugen.
“RP and LCA are chronically debilitating groups of IRDs
characterized by severe impairment in visual functions starting as
young as infancy, often progressing into night blindness and tunnel
vision and eventually causing total blindness as early as the
patient’s mid-40s. Since the existing approved therapy only
addresses a small percentage of this population, there is an unmet
need for new treatment options addressing a wider population of
patients with IRDs,” said Dr. Mohamed Genead, Chair of Retina
Scientific Advisory Board and Acting Chief Medical Officer of
Ocugen.
Nuclear Hormone Receptors such as NR2E3 are important modulators
of retinal development and function acting as “master genes” in the
retina. NR2E3 is delivered to target cells in the retina using an
adeno-associated viral (AAV) vector. As a potent modifier gene,
expression of NR2E3 within the retina may help reset retinal
homeostasis, potentially stabilizing cells and rescuing
photoreceptor degeneration. Preclinical results published in Nature
Gene Therapy demonstrate the potency of modifier gene therapy to
elicit broad-spectrum therapeutic benefits in early and advanced
stages of RP including vision rescue in early and advanced stages
of the disease.
Orphan medicinal product designation in Europe offers certain
benefits to drug developers while they develop drugs intended for
safe and effective treatment, diagnosis, or prevention of rare
diseases or conditions that impact fewer than 5 in 10,000 patients
in the European Union. Benefits include protocol assistance,
reduced regulatory fees, research grants, and 10 years of market
exclusivity following regulatory approval.
About Retinitis PigmentosaRetinitis pigmentosa
is a clinically and genetically heterogeneous group of IRDs
characterized by diffuse progressive dysfunction of predominantly
rod photoreceptors, with subsequent degeneration of cone
photoreceptors, and retinal pigment epithelium (RPE). Visual
impairment usually manifests as night blindness and progressive
visual field loss. Its prevalence is 1 in 3,000 to 1 in 5,000. RP
may be seen in isolation (typical RP) or in association with
systemic disease. Over 150 gene mutations have been associated with
RP and this number represents only 60% of the RP population. The
remaining 40% of RP patients cannot be genetically diagnosed,
making it difficult to develop individual treatments.
About Leber Congenital AmaurosisLeber
congenital amaurosis is a family of congenital retinal dystrophies
that results in severe vision loss at an early age. Patients
present usually with nystagmus, sluggish or near-absent pupillary
responses, severely decreased visual acuity, photophobia and high
hyperopia. It is the most severe retinal dystrophy causing
blindness by the age of 1 year. This dystrophy is a genetically
heterogeneous recessive disease affecting 1 in 30,000 to 1 in
81,000 subjects. Mutations in one of more than two dozen genes can
cause LCA.
About OCU400OCU400 is a novel gene therapy
product candidate with the potential to be broadly effective in
restoring retinal integrity and function across a range of
genetically diverse IRDs. OCU400 is the first program that Ocugen
is advancing based on its breakthrough modifier gene therapy
platform developed by Dr. Neena Haider, Associate Professor of
Ophthalmology at Harvard Medical School and Associate Scientist at
the Schepens Eye Research Institute (SERI) of Massachusetts Eye and
Ear. Ocugen obtained an exclusive worldwide license from SERI to
develop and commercialize ophthalmology products based on specified
nuclear hormone receptor genes, including NR2E3. Consisting of a
functional copy of the nuclear hormone receptor gene NR2E3, OCU400
is delivered to target cells in the retina using an AAV vector. As
a potent modifier gene, expression of NR2E3 within the retina may
help reset retinal homeostasis, stabilizing cells and potentially
rescuing photoreceptors from degeneration.
About Ocugen, Inc.Ocugen, Inc. is a
biopharmaceutical company focused on discovering, developing, and
commercializing gene therapies to cure blindness diseases and
developing a vaccine to save lives from COVID-19. Our breakthrough
modifier gene therapy platform has the potential to treat multiple
retinal diseases with one drug – “one to many” and our novel
biologic product candidate aims to offer better therapy to patients
with underserved diseases such as wet age-related macular
degeneration, diabetic macular edema, and diabetic retinopathy. We
are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for
COVID-19 in the U.S. market. For more information, please visit
www.ocugen.com.
Cautionary Note on Forward-Looking
StatementsThis press release contains forward-looking
statements within the meaning of The Private Securities Litigation
Reform Act of 1995, which are subject to risks and uncertainties.
We may, in some cases, use terms such as “predicts,” “believes,”
“potential,” “proposed,” “continue,” “estimates,” “anticipates,”
“expects,” “plans,” “intends,” “may,” “could,” “might,” “will,”
“should” or other words that convey uncertainty of future events or
outcomes to identify these forward-looking statements. Such
statements are subject to numerous important factors, risks and
uncertainties that may cause actual events or results to differ
materially from our current expectations. These and other risks and
uncertainties are more fully described in our periodic filings with
the Securities and Exchange Commission (SEC), including the risk
factors described in the section entitled “Risk Factors” in the
quarterly and annual reports that we file with the SEC. Any
forward-looking statements that we make in this press release speak
only as of the date of this press release. Except as required by
law, we assume no obligation to update forward-looking statements
contained in this press release whether as a result of new
information, future events or otherwise, after the date of this
press release.
Ocugen Contact:Ocugen, Inc.Sanjay
SubramanianChief Financial Officer and Head of Corporate
Development ir@ocugen.comMedia Contact:For
Ocugen:LaVoieHealthScienceEmmie
Twomblyetwombly@lavoiehealthscience.com+1 857-389-6042
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