Full Press Release Details
Ocugen Provides Business Update with Second
Quarter 2025 Financial Results
Conference Call and Webcast Today at 8:30 a.m.
dosing in OCU410ST Phase 2/3 GARDian3 pivotal confirmatory clinical trial
dosing patients in OCU400 Phase 3 liMeliGhT clinical trial and on track for 2026 BLA filing
reverse merger intended to unlock the value of NeoCart/regenerative cell therapies and enable the Company to focus capital on modifier
gene therapy platform
binding term sheet for exclusive Korean rights to OCU400 with upfront fees and near-term development milestone payments totaling up to
MALVERN, Pa., Aug. 01, 2025 (GLOBE NEWSWIRE) - Ocugen, Inc.
(Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today reported second
quarter 2025 financial results along with a business update.
"While our modifier gene therapy clinical trials advance-now with two in late-stage-we are securing strategic partnerships
and evolving the business to support three successful Biologics License Application (BLA) filings over the next three years," said
Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. "We have also made important appointments to our Board of Directors,
Retina Scientific Advisory Board, and Leadership Team to provide the Company with scientific and strategic know-how to bring us closer
to delivering paradigm-changing gene therapies to millions of people with blindness diseases."
In June, the Company announced a proposed reverse merger with OrthoCellix,
a wholly-owned subsidiary, and Carisma Therapeutics, Inc., to create a Nasdaq-listed, late clinical-stage regenerative cell therapy company
with a first-in-class technology platform, focused on orthopedic diseases. The combined company will focus on the development of OrthoCellix's
NeoCart technology for the treatment of articular knee cartilage defects. Previously, NeoCart received Regenerative Medicine
Advanced Therapy (RMAT) designation and concurrence from the U.S. Food and Drug Administration (FDA) on a single, confirmatory Phase 3
clinical trial to enable submission of a BLA.
Aligned with Ocugen's business development strategy to pursue
regional partnerships for OCU400, the Company signed a binding term sheet to negotiate and enter into a licensing agreement with a well-established
leader in the pharmaceutical and healthcare sector in Korea for exclusive Korean rights to OCU400. Pursuant to the term sheet, under the
license agreement, in addition to the upfront and milestone fees, the Company will be entitled to sales milestones of $1 million for every
$15 million of net sales in Korea in addition to a royalty of 25% on net sales of OCU400 generated by Ocugen's partner. Ocugen will
manufacture commercial supply of OCU400 under terms of a supply agreement. A regional approach preserves Ocugen's rights to larger
geographies to maximize total patient reach while also generating return for shareholders.
Following the FDA's agreement to proceed with a Phase 2/3 GARDian3
pivotal confirmatory trial for OCU410ST for Stargardt disease, the agency granted Rare Pediatric Disease Designation (RPDD) to OCU410ST
in May. This designation underscores the urgent need to address Stargardt disease, which remains a significant unmet medical need. Stargardt
disease is an inherited retinal disorder that typically presents in childhood and affects approximately 100,000 people in the U.S. and
Europe combined, and approximately 1 million globally. Currently, there is no FDA-approved treatment available for Stargardt disease.
The OCU410ST Phase 2/3 GARDian3 clinical trial is progressing
well with the first patient dosed in July after FDA clearance in June. The GARDian3 clinical trial builds upon encouraging results
and positive data from the Phase 1 GARDian trial, which demonstrated 48% slower lesion growth at 12-month follow-up in evaluable
treated eyes compared to untreated eyes. Additionally, evaluable treated eyes showed a statistically significant (p=0.031) and
clinically meaningful improvement of nearly 2-line/9-letter gain in best corrected visual acuity (BCVA) at 12-month follow-up when
compared to untreated eyes.
Positive preliminary efficacy and safety data from the OCU410 Phase
1 ArMaDa clinical trial at 12 months demonstrated no drug-related serious adverse events (SAEs), 23% slower geographic atrophy (GA) lesion
growth in treated eyes versus fellow eyes after a single injection, and 2-line/10-letter gain in visual acuity in treated eyes when compared
to untreated fellow eyes. Preliminary results from ongoing Phase 2 clinical trial (N=31), 6-month interim analysis, demonstrated a 27% slower
lesion growth and preservation of retinal tissue. These data support the potential for OCU410 to provide a one-time treatment for life
for the 2-3 million people in the U.S. & EU combined who suffer from GA.
Patients are actively being recruited in the United States and Canada
for the OCU400 Phase 3 liMeliGhT clinical trial, which remains on track for BLA and MAA submissions in 2026. This is the only broad retinitis
pigmentosa (RP) gene-agnostic trial to address multiple genetic mutations with a single therapeutic approach. In addition, the European
Medicines Agency has granted eligibility to submit the OCU400 Marketing Authorization Application (MAA) through the centralized procedure,
based on the current study design and statistical analysis plan.
Regarding the Company's inhaled vaccines portfolio, the National Institute of Allergy and Infectious Diseases (NIAID) intends to
initiate the Phase 1 clinical trial for OCU500 in the third quarter of 2025.
In addition to the notable leadership appointments, Ocugen welcomed
the National Security Commission on Emerging Biotechnology (NSCEB) and U.S. Rep. Chrissy Houlahan to its manufacturing facility as part
of the NSCEB's Biotech Across America events, highlighting biotech innovation in Pennsylvania. Rep. Houlahan subsequently announced
the bipartisan BIOTech Caucus to build greater awareness and understanding of biotechnology among lawmakers and support transformative
advances in healthcare. Dr. Musunuri supports the formation of this very important bipartisan BIOTech Caucus that includes senior congressional
leaders such as Rep. Pete Sessions in addition to local leaders, which will prioritize biotechnology at the national level to ensure U.S.
leadership globally.
"The meaningful progress Ocugen is making across its novel modifier
gene therapy platform, along with strategic leadership changes and significant external alliances are evidence of a strong first half
of 2025," said Dr. Musunuri. "We look forward to providing critical program updates and data in the coming months."
Modifier Gene Therapy Platform-a Novel First-in-Class Platform
Ophthalmic Biologic Product
Second Quarter 2025 Financial Results
Conference Call and Webcast Details
Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET
today to discuss the financial results and recent business highlights. Ocugen's executive leadership team will host the call, which
will be open to all listeners. There also will be a question-and-answer session following the prepared remarks.
Attendees are invited to participate on the call or webcast:
Dial-in Numbers: (800) 715-9871 for U.S. callers and (646)
307-1963 for international callers
Conference ID: 9627149
Webcast: Available on the events section
of the Ocugen investor site
A replay of the call and archived webcast will be available for approximately
45 days following the event on the Ocugen investor site.
Ocugen, Inc. is a pioneering biotechnology leader in gene therapies
for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for
large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen's modifier
gene therapies address the entire disease-complex diseases that are potentially caused by imbalances in multiple gene networks.
Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe,
including retinitis pigmentosa, Stargardt disease, and geographic atrophy-late stage dry age-related macular degeneration. Discover
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, strategy, business plans and
objectives for Ocugen's clinical programs, plans and timelines for the preclinical and clinical development of Ocugen's
product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing,
success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs;
Ocugen's financial condition and expected cash runway into the first quarter of 2026, statements regarding qualitative
assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and
anticipated development timelines, and Ocugen's negotiations regarding the license agreement with a Korean partner and
Ocugen's potential merger transaction regarding the OrthoCellix business, 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