Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a
biotechnology company focused on discovering, developing, and
commercializing novel gene and cell therapies, biologics, and
vaccines, today announced a clinical study update for Retinitis
Pigmentosa (RP) participants treated in the Phase 1/2 trial to
assess the safety and efficacy of OCU400 for RP associated with
NR2E3 and Rhodopsin (RHO) mutations and Leber congenital amaurosis
(LCA) with mutation(s) in the CEP290 gene. This clinical study
update is an extension of results provided by Ocugen on April 14,
2023, and includes additional subjects from the high dose group.
The Company believes that OCU400—Ocugen’s therapeutic approach,
utilizing a proprietary modifier gene therapy platform—has the
potential to be a gene-agnostic therapeutic for RP and LCA patients
with inherited retinal degeneration.
“This clinical study update supports our vision to help change
the lives of patients suffering from inherited retinal diseases,”
said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and
Co-Founder of Ocugen. “We remain dedicated to our mission of
pioneering breakthroughs in biotechnology and believe that OCU400
has the potential to have an impact on the future treatment of
patients with RP and LCA.”
This Phase 1/2 trial is a multicenter, open-label, dose ranging
study. A total of 18 subjects with vision impairment due to RP
associated with RHO and NR2E3 gene mutations received a unilateral
subretinal injection of either a low dose (1.66 x 1010 vg/mL),
medium dose (3.33 x 1010 vg/mL), or high dose (1.66 x 1011 vg/mL)
of OCU400. The study profile included a diverse group of subjects
aged 18-77 years old, with varied disease stages, racial and ethnic
profiles, medical histories, and mutation subgroups. Ocugen further
expanded this Phase1/2 trial to enroll LCA patients with CEP290
gene mutation and pediatric patients with NR2E3, RHO and CEP290
mutations.
Inherited retinal diseases (IRDs) such as RP and LCA encompass a
group of genetic disorders that affect the retina, the
light-sensitive tissue at the back of the eye. These diseases often
lead to a gradual loss of vision over time and can ultimately
result in blindness. Stabilization of vision is crucial for
patients with IRDs due to the progressive and degenerative nature
of these conditions.
Preserving remaining vision, slowing disease progression, or
improving the vision can significantly impact patients’ quality of
life. It not only enhances the quality of life for affected
individuals but also provides hope for future treatments that may
ultimately lead to vision restoration. Comprehensive care, early
diagnosis, and access to emerging therapies are essential
components of a strategy to stabilize vision in IRD patients.
“I am gratified to see the progress we have made in our pursuit
of developing a novel gene-agnostic therapy for RP and LCA. Our
team's unwavering dedication to advancing modifier gene therapy
research demonstrated positive preliminary clinical results that
offer renewed hope to patients and their families. We remain
resolute in our mission and vision to bring a bright future to
those with inherited or age-related retinal diseases through
courageous innovation and unwavering determination,” said Dr. Arun
Upadhyay, Chief Scientific Officer, Head of Research, Development
and Medical at Ocugen.
This clinical study update is based on the currently available
data from Phase 1 (dose-escalation: Cohort 1, 2 and 3) and the
Phase 2 (open enrollment) portion of the study. The exploratory
efficacy update includes data for 12 subjects who have completed a
minimum of 6-month follow up. The data set comprised of 2 subjects
[Cohort 1] with 12-month follow-up, 5 subjects [N=2 from Cohort 1
and N=3 from Cohort 2] with 9-month follow-up, and 5 subjects [N=2
from Cohort 3 and N=3 from Open Enrollment/Phase 2] with 6-month
follow-up.
“It is an important steppingstone for Ocugen and its mission to
help the nearly 1.6 million patients affected by RP and LCA
worldwide. For those suffering from these IRDs, this clinical trial
update provides hope. It is encouraging to see a favorable safety
and tolerability profile and positive efficacy readout for OCU400
in RP patients,” said Dr. Lejla Vajzovic, Associate Professor of
Ophthalmology with Tenure, Director of Duke Vitreoretinal
Fellowship Program at Duke Eye Center and Duke University School of
Medicine and leader in gene-therapy research.
Key efficacy outcomes from 12 subjects demonstrated: BCVA:
- 83% (10/12) of subjects demonstrated stabilization or
improvements in treated eyes in BCVA scores from baseline
- 42% (5/12) of OCU400 treated eyes experienced 4-letter
improvement and 33% (4/12) treated eyes experienced 7-letter
improvement in BCVA from baseline
- 57% (4/7) of RHO subjects’ treated eyes experienced 4-letter
improvement and 43% (3/7) treated eyes experienced 7-letter
improvement in BCVA scores from baseline
LLVA:
- 83% (10/12) of subjects demonstrated stabilization or
improvement in treated eyes in LLVA scores from baseline
- 42% (5/12) of OCU400 treated eyes experienced 5-letter
improvement (1 line) in LLVA from baseline, with 25% (3/7)
increasing by 10 letters (2 lines)
- 43% (3/7) of RHO subjects experienced 5-letter improvement (1
line) in treated eyes in LLVA scores from baseline, among which 29%
(2/7) increased by 10 letters (2 lines)
MLMT:
- 75% (9/12) of subjects demonstrated
stabilization or improvement in treated eyes in MLMT scores from
baseline
- 33% (4/12) of subjects in the low, medium, and high dose
cohorts experienced at least 1 Lux luminance level improvement from
baseline in treated eyes, among which 17% (2/12) increased by 3 Lux
luminance levels
- 86% (6/7) of RHO subjects experienced either stabilization or
increases in MLMT scores from baseline, among which 29% (2/7)
improved by 3 lux levels
“The RHO mutation affects more than 10,000 people in the US,”
said Dr. David Birch, Scientific Director, Retina Foundation of the
Southwest and Principal investigator of the study. “In my view, the
clinical study update supports the gene-agnostic mechanism of
action of OCU400 in RHO patients. The improvements in BCVA, LLVA
and MLMT in this patient population are very exciting and
encouraging because stabilization alone could be considered as a
treatment benefit.”
The clinical study update from the Phase 1/2 clinical trial
demonstrated that OCU400 continued to be generally safe and
well-tolerated in subjects across different mutations and dose
levels. There were no serious adverse events (SAEs) related to the
investigational product in the low and medium-dose cohorts. In the
high-dose and open-enrollment cohorts, SAEs were reported for two
subjects. Adverse events were mostly deemed related to the surgical
procedure and resolved within a few days to weeks. “The clinical
study update released by Ocugen appears to have a tangible
biological impact on Retinitis Pigmentosa associated with NR2E3 and
RHO mutations,” said Dr. David Boyer, Clinical Professor of
Ophthalmology USC/Keck School of Medicine Los Angeles, CA and
Partner, Retina Vitreous Associates Medical Group. “These findings
may indicate a huge step forward in the way we approach and treat
this condition. We remain optimistic and eager to continue the
trial and understand the full potential of OCU400.”
Ocugen will continue to monitor long-term safety and efficacy
data from the treated patients and provide additional updates.
A webcast and conference call will take place today at 8:30 a.m.
ET:
Dial-in Numbers: (800) 715-9871 for U.S. callers and (646)
307-1963 for international callersConference ID: 7803227
Webcast: Available on the events section of the Ocugen investor
site
About Modifier
Gene TherapyModifier gene therapy
is designed to fulfill unmet medical needs related to retinal
diseases, including IRDs, such as RP, LCA, and Stargardt disease,
as well as dry AMD. Our modifier gene therapy platform is based on
the use of Nuclear hormone receptors (NHRs), master gene
regulators, which have the potential to restore homeostasis — the
basic biological processes in the retina. Unlike single-gene
replacement therapies, which only target one genetic mutation, we
believe that our modifier gene therapy platform, through its use of
NHRs, represents a novel approach that has the potential to address
multiple retinal diseases caused by mutations in multiple genes
with one product, and to address complex diseases that are
potentially caused by imbalances in multiple gene networks.
Currently Ocugen has three modifier gene therapy programs OCU400
(RP, LCA), OCU410 (dry AMD), OCU410ST (Stargardt disease).
About OCU400OCU400 is the
Company’s gene-agnostic modifier gene therapy product based on NHR
gene, NR2E3. NR2E3 regulates diverse physiological functions within
the retina—such as photoreceptor development and maintenance,
metabolism, phototransduction, inflammation and cell survival
networks. Through its drive functionality, OCU400 resets
altered/affected cellular gene-networks and establishes
homeostasis—a state of balance, which has the potential to improve
retinal health and function in patients with inherited retinal
diseases.
About Ocugen,
Inc.Ocugen, Inc. is a biotechnology company
focused on discovering, developing, and commercializing novel gene
and cell therapies, biologics, and vaccines that improve health and
offer hope for patients across the globe. We are making an impact
on patients’ lives through courageous innovation—forging new
scientific paths that harness our unique intellectual and human
capital. Our breakthrough modifier gene therapy platform has the
potential to treat multiple retinal diseases with a single product,
and we are advancing research in infectious diseases to support
public health and orthopedic diseases to address unmet medical
needs. Discover more at www.ocugen.com and follow us on Twitter and
LinkedIn.
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, including, but not limited to, statements
regarding qualitative assessments of available data, potential
benefits, expectations for ongoing clinical trial results, and
anticipated timing of clinical trial updates and regulatory
interactions. 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,
including, but not limited to, the risks that preliminary, interim
and top-line clinical trial results may not be indicative of, and
may differ from, final clinical data; that unfavorable new clinical
trial data may emerge in the Phase 1/2 clinical trial or through
further analyses of existing clinical trial data; that earlier
non-clinical and clinical data and testing of may not be predictive
of the results or success of later clinical trials; and that that
clinical trial data are subject to differing interpretations and
assessments, including by regulatory authorities. 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.
Contact:Tiffany HamiltonHead of
CommunicationsTiffany.Hamilton@ocugen.com
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