Ocugen Receives Fourth FDA Orphan Drug Designation for the Same Product, OCU400 (AAV-NR2E3) Gene Therapy, for the Treatment o...
August 10 2020 - 7:30AM
Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on
discovering, developing, and commercializing transformative
therapies to cure blindness diseases, today announced the U.S. Food
and Drug Administration (FDA) granted the fourth Orphan Drug
Designation (ODD) for OCU400 in the treatment of PDE6B gene
mutation-associated retinal diseases. Retinitis Pigmentosa (RP)
caused by PDE6B mutation is an inherited retinal dystrophy that
leads to blindness by midlife and is characterized by the
progressive loss of photoreceptors, with or without the loss of
retinal pigment epithelium cells. At least one mutation in the
PDE6B gene has been found to cause autosomal dominant congenital
stationary night blindness, which is characterized by the inability
to see in low light.
Ocugen’s Modifier Gene Therapy Platform offers a unique approach
in ophthalmology by addressing multiple diseases with a single
product. A novel gene therapy product candidate, OCU400 has the
potential to be broadly effective in restoring retinal integrity
and function across a range of genetically diverse inherited
retinal diseases. It consists of a functional copy of a nuclear
hormone receptor (NHR) gene, NR2E3, delivered to target cells in
the retina using an adeno-associated viral vector. As a
potent modifier gene, expression of NR2E3 within the retina may
help reset retinal homeostasis and potentially offer longer
benefit, stabilizing cells and rescuing photoreceptor degeneration
and vision loss. In pre-clinical studies, OCU400 has demonstrated
improved vision signals in the retina where Electroretinogram
response reveals rescue under both Scotopic (dim-lit) as well as
Photopic (well-lit) conditions. The Company believes targeting
multiple diseases with one product could also offer a smoother
regulatory pathway and the ability to recover development costs
over multiple therapeutic indications. Ocugen is planning to
initiate two parallel Phase I/II clinical trials next year
targeting two unique IRDs.
Following up on recent announcement of an ODD for RHO
mutation-associated retinal degeneration and previous ODDs for both
NR2E3 and CEP290 mutation-associated retinal degeneration, the ODD
for PDE6B gene mutation-associated retinal degeneration continues
to support Ocugen’s breakthrough modifier gene therapy platform’s
potential to treat multiple blindness diseases with a single
product. RP is a group of heterogenic inherited retinal
diseases associated with over 150 gene mutations, affecting over
1.5 million individuals worldwide. In addition, ~40% of RP patients
cannot be genetically diagnosed, confounding the ability to develop
personalized RP therapies. Traditional gene therapy or gene
editing approaches may require more than 150 products to rescue
these patients from vision loss. OCU400, a single product
candidate, has potential to address broad-spectrum RP.
“As principal investigator of numerous major clinical trials
developing new medical and surgical treatments for retinal
disorders, I have been on the cutting-edge of many new
ophthalmology treatments. I am very encouraged by the potential for
OCU400 given the uniqueness of Ocugen’s Modifier Gene Therapy
Platform and the fact that FDA has issued four ODDs for this
product. I look forward to Ocugen commencing clinical trials for
OCU400 next year and the potential of helping patients by restoring
retinal integrity and function across a range of genetically
diverse inherited retinal diseases including broad-spectrum RP,”
said Carl D. Regillo, M.D.,F.A.C.S., member of Ocugen’s Retina
Scientific Advisory Board and Professor of Ophthalmology at the
Sidney Kimmel Medical College at Thomas Jefferson University, Chief
of the Retina Service at Wills Eye Hospital and founder and former
director of the Wills Eye Clinical Retina Research Unit in
Philadelphia.
“I am thrilled to announce our fourth ODD for OCU400 from the
FDA after announcing our third ODD for RHO mutation-associated
retinal degeneration just a few days ago. With no approved
treatments that slow or stop the progression of RP, we are
dedicated to driving the development of our Modifier Gene Therapy
Platform forward and potentially addressing the unmet need of
multiple gene mutations, including mutations in the PDE6B gene with
only one product,” said Dr. Shankar Musunuri, Chairman, Chief
Executive Officer and Co-Founder of Ocugen.
The FDA Office of Orphan Products Development grants orphan
designation for novel drugs or biologics that treat a rare disease
or condition affecting fewer than 200,000 patients in the U.S.
Orphan designation qualifies the sponsor of the drug for various
development incentives of the Orphan Drug Act, including a
seven-year period of U.S. marketing exclusivity, tax credits for
clinical research costs, clinical research trial design assistance,
the ability to apply for annual grant funding and waiver of
Prescription Drug User Fee Act filing fees.
About OCU400OCU400 (AAV-hNR2E3) 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 inherited retinal diseases. It
consists of a functional copy of a nuclear hormone receptor gene,
NR2E3, delivered to target cells in the retina using an
adeno-associated viral vector. As a potent modifier gene,
expression of NR2E3 within the retina may help reset retinal
homeostasis, potentially stabilizing cells and rescuing
photoreceptor degeneration and vision loss.
About Ocugen, Inc.Ocugen, Inc. is a
biopharmaceutical company focused on discovering, developing, and
commercializing transformative therapies to cure blindness
diseases. 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. For more information, please visit
https://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 (the “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.
Corporate Contact:Ocugen, Inc.Sanjay
SubramanianChief Financial OfficerIR@Ocugen.com
Media Contact:LaVoieHealthScienceEmmie
Twomblyetwombly@lavoiehealthscience.com+1 857-389-6042
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