OCU400 shows significant long-term improvements in visual function and mobility for retinitis pigmentosa patients, with a strong safety profile.
Quiver AI Summary
Ocugen, Inc. announced positive results from its Phase 1/2 clinical trial of OCU400, a novel gene therapy aimed at treating retinitis pigmentosa (RP). The study showed that 100% of treated subjects experienced improvement or preservation of visual function and stabilization in mobility, with significant statistical results (p=0.01). OCU400 demonstrated meaningful gains in low-luminance visual acuity over two years, indicating its potential as a mutation-agnostic therapy for RP, which affects millions globally. The treatment had a favorable safety profile with no serious adverse events reported. Ocugen's Chief Executive, Shankar Musunuri, highlighted the importance of these findings in advancing therapies for inherited retinal disorders, while the ongoing Phase 3 liMeliGhT trial is set for regulatory submission by mid-2026.
Potential Positives
- 100% of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes at both one and two years.
- Statistically significant improvements in visual function (p=0.01) were observed regardless of mutation at two years, validating the gene-agnostic mechanism of OCU400.
- OCU400 exhibited a favorable long-term safety and tolerability profile with no serious adverse events reported.
- The ongoing Phase 3 OCU400 liMeliGhT clinical trial is on track for BLA submission in the first half of 2026, indicating progress towards commercialization.
Potential Negatives
- Reliance on preliminary clinical data with no guarantees that results will predict future clinical success, indicating a risk of disappointment as trials progress.
- The potential for negative outcomes or adverse effects to arise in the ongoing Phase 3 liMeliGhT clinical trial, which could impact the company's reputation and market value.
- The company emphasizes its mutation-agnostic approach, but this could draw skepticism from the scientific community regarding the true breadth of OCU400's effectiveness across the varied genetic causes of RP.
FAQ
What is OCU400 designed to treat?
OCU400 is intended for the treatment of early to advanced retinitis pigmentosa (RP) in pediatric and adult populations.
What improvements were observed in the OCU400 trial?
100% of treated subjects showed improvement or preservation in visual function and stabilization in mobility testing after two years.
What is the significance of the trial results?
The trial results demonstrated statistically significant improvement in visual function (p=0.01) across all mutations after two years.
What are the safety outcomes associated with OCU400?
OCU400 has a favorable long-term safety and tolerability profile with no serious adverse events reported in the trial.
When is the next phase of the OCU400 trial planned?
The Phase 3 OCU400 liMeliGhT clinical trial is ongoing, with plans for a BLA submission in the first half of 2026.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
$OCGN Insider Trading Activity
$OCGN insiders have traded $OCGN stock on the open market 2 times in the past 6 months. Of those trades, 2 have been purchases and 0 have been sales.
Here’s a breakdown of recent trading of $OCGN stock by insiders over the last 6 months:
- PRABHAVATHI FERNANDES purchased 10,000 shares.
- KIRSTEN CASTILLO purchased 25,000 shares.
To track insider transactions, check out Quiver Quantitative's insider trading dashboard.
$OCGN Hedge Fund Activity
We have seen 63 institutional investors add shares of $OCGN stock to their portfolio, and 38 decrease their positions in their most recent quarter.
Here are some of the largest recent moves:
- GMT CAPITAL CORP added 2,454,958 shares (+inf%) to their portfolio in Q3 2024
- ALTIUM CAPITAL MANAGEMENT LP added 2,350,000 shares (+inf%) to their portfolio in Q3 2024
- NORTHERN TRUST CORP added 1,555,980 shares (+249.4%) to their portfolio in Q3 2024
- VANGUARD GROUP INC added 1,427,852 shares (+9.2%) to their portfolio in Q3 2024
- STATE STREET CORP added 1,047,896 shares (+7.1%) to their portfolio in Q3 2024
- BLACKROCK, INC. added 694,571 shares (+3.9%) to their portfolio in Q3 2024
- WELLINGTON MANAGEMENT GROUP LLP added 459,102 shares (+inf%) to their portfolio in Q3 2024
To track hedge funds' stock portfolios, check out Quiver Quantitative's institutional holdings dashboard.
Full Release
100% (9/9) of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes at both one and two years
100% (9/9) of treated evaluable subjects demonstrated improvement or stabilization in mobility testing, which was only performed up to one year
Improvement in visual function was statistically significant (
p=0.01
, treated
vs
untreated eyes), regardless of mutation at two years
Favorable long-term safety and tolerability profile with no serious adverse events related to OCU400
MALVERN, Pa., Jan. 13, 2025 (GLOBE NEWSWIRE) -- 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 positive 2-year safety and efficacy update for the Phase 1/2 OCU400 clinical trial. OCU400 is intended for the treatment of early to advanced retinitis pigmentosa (RP) in pediatric and adult populations. Currently, about 2 million patients globally (~300,000 in U.S./EU) desperately need rescue, otherwise they can potentially become legally blind at some point in their life. OCU400 is intended to treat all these patients with a one-time therapy.
OCU400 demonstrated meaningful improvement of 2-line gain (10 letters on ETDRS chart) in low-luminance visual acuity (LLVA) in treated eyes when compared to untreated fellow eyes. This treatment effect was statistically significant (
p=0.01
) in all subjects, regardless of mutation at two years, validating the gene-agnostic mechanism of action for OCU400.
This data highlights the unique therapeutic potential of OCU400. To date, Ocugen’s groundbreaking modifier gene therapy platform provides long-term safety and meaningful improvements in visual function for patients suffering from RP—a condition that leads to progressive vision loss.
“We are thrilled to share these compelling results from the Phase 1/2 OCU400 trial,” said Shankar Musunuri, PhD, MBA, Chairman, CEO, and Co-Founder of Ocugen. “These findings represent a significant step forward in our mission to bring life-changing therapies to patients with inherited retinal disorders and confirm the transformative potential of our modifier gene therapy platform.”
"It is truly remarkable to see the significant improvements in visual acuity in patients treated with OCU400 sustained at two years,” said Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin. “The broad spectrum of genes and mutations causing RP presents a unique challenge in developing treatments for this unmet need. This is where the promise of mutation-agnostic therapies becomes particularly compelling. OCU400’s demonstrated effectiveness across multiple mutations not only offers hope to RP patients but also opens new possibilities for treating other retinal diseases."
“Establishing the long-term safety and efficacy of OCU400 demonstrates the durability of this novel gene therapy,” said Huma Qamar, MD, MPH, Chief Medical Officer at Ocugen. “These 2 year low light visual acuity (LLVA) findings, which are the most sensitive measure of visual function, are consistent with the results observed at one year.”
The Phase 3 OCU400 liMeliGhT clinical trial is currently ongoing and on target for BLA submission in the first half of 2026.
About OCU400
OCU400 is the Company’s modifier gene therapy product based on a nuclear hormone receptor (NHR) gene called
NR2E3
. This gene regulates diverse physiological functions within the retina, such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival. Retinal cells in RP patients have a dysfunctional gene network, and OCU400 resets this network to reestablish a healthy cellular homeostasis—which has the potential to improve vision in patients with RP.
About
Modifier
Gene
Therapy
Modifier gene therapy is designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP and Stargardt disease, as well as multifactorial diseases like dry age-related macular degeneration (dAMD). Our modifier gene therapy platform is based on the use of 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 in the clinic: OCU400, OCU410, and OCU410ST. In addition to the OCU400 Phase 3 liMeliGhT clinical trial, the OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy (GA) secondary to dAMD and the OCU410ST Phase 1/2 GARDian clinical trial for Stargardt disease are currently underway. GA affects approximately two to three million people in the U.S. and EU combined, and Stargardt disease affects nearly 100,000 people in the U.S. and EU combined.
About
Ocugen,
Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies 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
X
and
LinkedIn
.
Cautionary
Note
on
Forward-Looking
Statements
This
press
release
contains
forward-looking
statements
within
the
meaning
of
The
Private
Securities
Litigation
Reform
Act
of
1995,
including,
but
not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines,
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, 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;
the ability of OCU400 to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study data;
that
unfavorable
new
clinical
trial
data
may
emerge
in
ongoing
clinical
trials
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 Hamilton
Head of Corporate Communications
Tiffany.Hamilton@ocugen.com
This article was originally published on Quiver News, read the full story.
The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.
