Ocugen (OCGN) – 1Q25 Reported With All Three Main Clinical Trials On Schedule


Monday, May 12, 2025

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Clinical Programs Meet Or Beat Expected Timeframes. Ocugen reported a 1Q25 loss of $15.4 million or $(0.05) per share, consistent with our estimates. The company’s three clinical trial continue to progress on schedule, with the first BLA application expected in mid-2026. Its fourth product, OCU200, initiated Phase 1 patient treatment during the quarter. Cash balance on March 31 was $38.1 million, projected to fund operations through early FY2026.

OCU400 Trial Continues To Lead The Way. The OCU400 liMeliGhT (pronounced “Limelight”) Phase 3 trial in RP continues to enroll patients, with completion expected around YE2025. The study enrolls patients over 5 years of age with any combination of over 100 mutations that cause RP. These patients are at stages ranging from early to advanced RP.


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Release – Ocugen Provides Business Update with First Quarter 2025 Financial Results

Research News and Market Data on OCGN

May 9, 2025

PDF Version

Conference Call and Webcast Today at 8:30 a.m. ET

  • OCU400 Phase 3 clinical trial for retinitis pigmentosa (RP) is progressing well and on target for potential BLA/MAA filings by mid-2026
  • Anticipate initiating OCU410ST Phase 2/3 pivotal confirmatory clinical trial for Stargardt disease mid- 2025

MALVERN, Pa., May 09, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today reported first quarter 2025 financial results along with a general business update.

“All three of our novel modifier gene therapies are advancing through the clinic and we are on track to meet our goal of three Biologics License Application (BLA)/Marketing Authorization Application (MAA) filings in the next three years,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “We remain steadfast in our mission to provide a one-time therapy for life to address considerable unmet medical needs that exist for millions of patients facing the terrifying prospect of losing their vison.”

In January, the Company announced positive two-year long-term safety and efficacy data from the Phase 1/2 clinical trial of OCU400 for RP that demonstrated a durable, clinically meaningful, and statistically significant (p=0.005) improvement in visual function (LLVA) in all evaluable treated subjects at two years when compared to untreated eyes. Additionally, 100% (10/10) of treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes. This data further supports the gene-agnostic mechanism of action of OCU400, a broad RP treatment not restricted to specific mutations, with durability.

The OCU400 Phase 3 liMeliGhT clinical trial is open to all eligible RP patients—early to advanced stage RP including pediatric subjects age 5+—regardless of gene mutation (syndromic and non-syndromic forms of RP). OCU400 has the potential to treat all 300,000 RP patients in the U.S. and EU.

Alignment was reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory trial for OCU410ST for Stargardt disease, which includes an adaptive design with a masked interim analysis at 8 months. Stargardt disease affects 100,000 people in the U.S. and EU. The Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, injection of OCU410ST (200 μL at a concentration of 1.5 × 10¹¹ vector genomes/mL) in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. The primary endpoint in the clinical trial is change in atrophic lesion size. Key secondary endpoints include visual acuity as measured by best corrected visual acuity (BCVA) and LLVA compared to untreated controls. One-year data will be utilized for the BLA filing. The latest data from the OCU410ST Phase 1 clinical trial demonstrates atrophic lesions grew slower by 54% at six months in evaluable treated eyes when compared to untreated eyes. In BCVA, treated eyes demonstrated statistically significant (p=0.02) improvement in visual function when compared to untreated fellow eyes. Ocugen plans to initiate the Phase 2/3 study by mid-year with a target BLA filing in 2027.

In February, dosing was complete in the Phase 2 portion of the OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). In evaluable subjects, OCU410 12-month data demonstrates a 4-line (23-letter) gain in visual acuity and 41% slower GA lesion growth in treated eyes versus untreated fellow eyes after a single injection. The unique mechanism of action of OCU410 targets multiple pathways associated with dAMD pathogenesis, in contrast to products currently approved or under development that treat only one cause of GA, require multiple injections per year, and have safety considerations. Approximately 2-3 million patients in the U.S. & EU and 8 million globally suffer from GA.

All of Ocugen’s modifier gene therapies—OCU400 for RP, OCU410ST for Stargardt disease, and OCU410 for GA were granted Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency’s (EMA) Committee for Advanced Therapies. ATMP classification is granted to medicines that can offer groundbreaking opportunities for the treatment of disease and accelerates the regulatory review timeline of this potential one-time gene therapy for life. Additionally, this classification allows the Company to interact with the EMA more frequently for scientific advice and protocol assistance.

Relevant to Ocugen’s recent ATMP classifications, Dr. Musunuri participated in a panel addressing the journey of successfully commercializing ATMPs, which focused on the challenges and strategies in achieving commercial success while also ensuring access for the patients who need them, during the 2025 Cell & Gene Meeting on the Mediterranean, hosted by the Alliance for Regenerative Medicine. The Meeting on the Med provided an excellent forum to reach a wide audience about the importance of changing the treatment paradigm by bringing potentially transformative modifier gene therapies to market.

Also in the first quarter of 2025, the first patient was dosed in the Phase 1 clinical trial for OCU200, the Company’s biologic product candidate for diabetic macular edema. Currently, patients are being dosed in the second cohort and Ocugen is planning to complete the Phase 1 clinical trial in the second half of this year. The Company intends to initiate the Phase 3 trial for NeoCart® contingent on adequate availability of funding and/or based on the potential of a future partnership. Finally, the Investigational New Drug application is in effect for OCU500, and the National Institute of Allergy and Infectious Diseases intends to initiate a Phase 1 clinical trial in the second quarter of 2025. Ocugen is continuing discussions with relevant government agencies as well as strategic partners regarding developmental funding for its vaccines technology for flu.

“We have had a strong start to 2025 and are approaching considerable milestones in the next few months,” said Dr. Musunuri. “I am enthusiastic about where we are as a Company, with a clear and precise business strategy to operate efficiently in service of our patients and shareholders.”

Modifier Gene Therapy Platform—a Novel First-in-Class Platform

  • OCU400 for RP – The EMA provided a positive opinion for ATMP classification for OCU400 and granted eligibility to submit the OCU400 MAA via the centralized procedure as an ATMP based on the current study design and statistical analysis plan. Actively recruiting patients in the U.S. and Canada in the Phase 3 liMeliGhT clinical trial and on track to meet BLA/MAA filing targets in mid-2026.
  • OCU410ST for Stargardt Disease  Received FDA alignment to move forward with Phase 2/3 pivotal confirmatory clinical trial, which can be the basis of a BLA submission. ATMP classification granted by the EMA.
  • OCU410 for GA – Announced that dosing was complete, ahead of schedule, in the Phase 2 portion of the Phase 1/2 ArMaDa clinical trial for OCU410. Received ATMP classification from the EMA.

First Quarter 2025 Financial Results

  • The Company’s cash and restricted cash totaled $38.1 million as of March 31, 2025, compared to $58.8 million as of December 31, 2024. The Company had 292.0 million shares of common stock outstanding as of March 31, 2025. The Company expects its cash and restricted cash runway into the first quarter of 2026.
  • Total operating expenses for the three months ended March 31, 2025 were $16.0 million and included research and development expenses of $9.5 million and general and administrative expenses of $6.5 million. This compares to total operating expenses for the three months ended March 31, 2024 of $13.2 million that included research and development expenses of $6.8 million and general and administrative expenses of $6.4 million.
  • Ocugen reported a $0.05 net loss per common share for the three months ended March 31, 2025 compared to a $0.05 net loss per common share for the three months ended March 31, 2024.

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 senior management team will host the call, which will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.

Attendees are invited to participate on the call or webcast using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 1773288
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.

About Ocugen, Inc.
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 more at www.ocugen.com and follow us 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, 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 expected cash runway, 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; 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 annual and 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 Communications
IR@ocugen.com 

View full release here.

Ocugen to Host Conference Call on Friday, May 9 at 8:30 A.M. ET to Discuss Business Updates and First Quarter 2025 Financial Results

Research News and Market Data on Ocugen

MALVERN, Pa., May 02, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that it will host a conference call and live webcast to discuss the Company’s first quarter 2025 financial results and provide a business update at 8:30 a.m. ET on Friday, May 9, 2025.

Ocugen will issue a pre-market earnings announcement on the same day. Attendees are invited to participate on the call using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 1773288
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.

About Ocugen, Inc.
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 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, 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.

Contact:
Tiffany Hamilton
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

Release – Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2025 Annual Meeting and Retina World Congress

Research News and Market Data on Ocugen

April 29, 2025

MALVERN, Pa., April 29, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa (Phase 3 LiMeliGhT clinical trial), OCU410ST for the treatment of Stargardt disease (Phase 2/3 pivotal confirmatory clinical trial), and OCU410 for the treatment of geographic atrophy (Phase 2 ArMaDa clinical trial), at The Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting at the Calvin L. Rampton Salt Palace Convention Center in Salt Lake City, Utah from May 4-8, 2025, and Retina World Congress at the Marriott Harbor Beach Resort in Ft. Lauderdale, Florida from May 8-11, 2025.

“We look forward to sharing more about the potential of our modifier gene therapy platform and the meaningful results we are seeing in the clinic during these two important meetings for the retina community,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Ocugen remains on track to deliver on our commitment to file three Biologics License Applications (BLAs)/Marketing Authorization Applications (MAAs) in the next three years—potentially addressing significant unmet medical need for large patient populations through our gene-agnostic approach.”

The ARVO Annual Meeting is a premiere gathering for eye and vision scientists from across the globe, students, and those in affiliated fields to share the latest research findings and collaborate on innovative solutions. Retina World Congress brings together leading retina specialists from every continent to achieve a global scientific and clinical exchange in retinal health.

Ocugen’s presence in Utah kicks off with the Company Showcase at Eyecelerator, presented by Dr. Huma Qamar, Chief Medical Officer at Ocugen, and continues through presentations and thought leadership engagement at ARVO.

Eyecelerator @ Park City 2025

Session: Retina—Gene Therapy and Novel Mechanisms of Action Showcase
Location: Grand Hyatt Deer Valley, Strawberry Ballroom, Park City, UT
Date: Friday, May 2, 2025
Time: 2:06 p.m. MDT
Presenter: Dr. Huma Qamar

ARVO

Exhibitor Education Forum
Two-Year Follow-Up of a Phase 1/2 Clinical Trial for the Safety and Efficacy of OCU400 Novel Modifier Gene Therapy for Retinitis Pigmentosa
Location: Exhibitor Floor, Section 1037
Date: Monday, May 5, 2025
Time: 2 p.m. MDT 
Presenter: Benjamin Bakall, MD, Ph.D., Assistant Clinical Professor, University of Arizona, College of Medicine–Phoenix, and Director for Clinical Research, Director for The Inherited Retinal Disease and Visual Function Clinic, Associated Retina Consultants

An Evaluation of the Safety and Efficacy of Novel Modifier Gene Therapy OCU410 for the Treatment of Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
Location: Exhibitor Floor, Section 1037
Date: Tuesday, May 6, 2025
Time: 2 p.m. MDT
Presenter: Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin

Safety and Efficacy of OCU410ST: A Phase 1/2 Trial of a Novel Modifier Gene Therapy for Stargardt Disease (GARDian)
Location: Exhibitor Floor, Section 1037
Date: Wednesday, May 7, 2025
Time: 2 p.m. MDT
Presenter: Neena Haider, Ph.D., Faculty Harvard Medical School and Founder, CEO, Shifa Precision

Paper Session
Preliminary Safety and Efficacy of OCU410 for Treatment of Geographic Atrophy: Phase 1/2 OCU410: The Age-related Macular Degeneration (ArMaDa) Study Update
Presentation Number: 3675 
Session Number and Title: 358/AMD Clinical research II 
Location: Ballroom J 
Date: Tuesday, May 6, 2025
Time: 4:15 p.m. MDT
Presenter: Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin

Poster Session
A0513: Safety and Efficacy of OCU410ST for the Treatment of Stargardt Disease: Phase 1/2 Study Update
Location: Hall A-E
Date: Thursday, May 8, 2025
Time: 2 p.m. MDT
Presenter: Ramiro Maldonado, MD, Duke Center for Ophthalmic Genetics, Duke Pediatric Retina, Adult vitreo-Retinal diseases

Dr. Qamar will represent Ocugen at Retina World Congress to share the Company presentation and serve alongside notable retinal surgeons and industry peers during a panel discussion.

Retina World Congress

Retina Unplugged
Inherited and Rare Retinal Diseases Session
Moderators: Rishi P. Singh, MD, FASRS and Kourous A. Rezaei, MD
Location: Grand Ballroom
Date: Thursday, May 8, 2025
Time: 10:35 am – 11:12 a.m. EDT

Ocugen is committed to bringing game-changing therapies to treat inherited retinal diseases as well as blindness diseases affecting millions to market and working even harder to provide access to patients globally.

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 patient’s 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; 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

Release – Data and Safety Monitoring Board Reviews Cohort 1 Safety Data and Approves Dosing Cohort 2 in the Clinical Trial of OCU200—a Novel Fusion Protein for Diabetic Macular Edema

Research News and Market Data on OCGN

March 18, 2025

PDF Version

  • OCU200 has a very favorable safety and tolerability profile
  • No serious adverse events related to the study drug have been reported
  • Dosing of the second cohort has been approved

MALVERN, Pa., March 18, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU200 clinical trial recently convened and reviewed safety data following dosing of the first cohort in the dose-escalation portion of the Phase 1 study and approved continuation of dosing in the second cohort. OCU200 is a novel fusion protein consisting of two human proteins, tumstatin and transferrin, with the potential to treat diabetic macular edema (DME).

“OCU200 is given intravitreally,” said Peter Chang, MD, FACS, Co-President and Partner of the Massachusetts Eye Research and Surgery Institution (MERSI). “No serious adverse events related to OCU200 have been reported to date.”

The OCU200 Phase 1 clinical trial is a multicenter, open-label, dose-escalation study to assess drug safety via intravitreal injection in three cohorts: low dose (0.025 mg), medium dose (0.05 mg), and high dose (0.1 mg). All subjects will receive two doses six weeks apart, and patients will be followed for up to 6 months.

“It is encouraging that we have successfully completed dosing in the low dose cohort for OCU200, a novel biologic that has a very favorable safety and tolerability profile,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “There remains a considerable unmet medical need for the 30% to 40% of DME patients who do not respond to current anti-VEGF therapies. OCU200 holds the promise of potentially benefiting all DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD) patients.”

Approximately 12 million people in the United States and 130 million people worldwide are affected by DME, DR, or wet AMD. Patients affected by them share common symptoms, such as blurriness in vision and progressive vision loss, as the diseases progress. The formation of fragile and leaky new blood vessels leads to fluid accumulation in and around the retina, causing damage to vision.

OCU200 has the potential to change the treatment landscape for DME, DR, and wet AMD with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors on active endothelial cells that play a crucial role in disease pathogenesis.

OCU200 brings an innovative biologic candidate to Ocugen’s ophthalmology portfolio targeting blindness diseases. The Company intends to complete the Phase 1 OCU200 clinical trial in the second half of 2025 and to provide preliminary safety and efficacy updates throughout the year.

About OCU200

OCU200 is a recombinant fusion protein that consists of two parts connected by a linker: tumstatin, the active component, acts as an anti-inflammatory, anti-VEGF agent by binding to integrin receptors; and transferrin, which targets the drug to the choroid and retina by binding transferrin receptors on endothelial cells. These features will potentially enable OCU200 to reduce the vascular permeability, inflammation, and neovascularization that drive the pathophysiology of DME, DR, and wet AMD at a significantly lower dose compared to currently approved therapies.

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 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 OCU200 to perform in humans in a manner consistent with nonclinical or preclinical 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

Ocugen (OCGN) – Ocugen Reports FY2024 With Progress Toward “3 BLA Filings In 3 Years”


Thursday, March 06, 2025

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Clinical Progress Expected To Lead To Filings For Three Product Approvals. Ocugen reported a 4Q24 loss of $13.9 million or $(0.05) per share and FY2024 loss of $54.1 million or $(0.20) per share. The company made significant progress in its clinical trials during the quarter and since the start of FY2025. It has also received regulatory designations that accelerate product approval. The company had $58.5 million in cash on December 31, sufficient to fund operations through 1Q26.

Clinical Trial Advances Point To Three BLAs In 3 Years. Ocugen has made significant progress with three products for three diseases that lead to vision loss. The three ongoing trials are Phase 3 for OCU400, the Phase 2/3 for OCU410ST in Stargardt disease, and the Phase 1/2 trial for GA. These trials are on schedule for filing applications for approval in 2026, 2027, and 2028 respectively. OCU400 and OCU410ST have Orphan Drug designations that can accelerate approval, while GA is a large market of over 10 million patients in the US alone.


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Release – Ocugen Provides Business Update with Fourth Quarter and Full Year 2024 Financial Results

Research News and Market Data on OCGN

March 5, 2025

PDF Version

Conference Call and Webcast Today at 8:30 a.m. ET

  • Reached an alignment with FDA on Phase 2/3 pivotal confirmatory clinical trial for OCU410ST for Stargardt disease potentially expediting clinical development by two to three years
  • Completed dosing in OCU410 Phase 2 ArMaDa clinical trial (N=51) for geographic atrophy (GA). Data and Safety Monitoring Board (DSMB) recently reviewed interim safety data from Phase 2. All subjects from multiple dose levels in Phase 2 to date demonstrated a very favorable safety and tolerability profile.
  • Positive 2-year long-term data across multiple mutations of retinitis pigmentosa (RP) from the Phase 1/2 clinical trial of OCU400 demonstrated a durable and statistically significant (p=0.005) improvement in visual function (LLVA) in all evaluable treated subjects at two years when compared to untreated eyes
  • OCU400 remains on track to meet 1H 2025 recruitment completion and potential BLA/MAA filings by mid-2026

MALVERN, Pa., March 05, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today reported fourth quarter and full year 2024 financial results along with a general business update.

“Ongoing data from all three clinical programs, along with patient testimonials, continue to support the benefit of one-time treatment from our revolutionary modifier gene therapy platform—as we are seeing consistent stabilization or improvement in treated eyes while maintaining a favorable safety and tolerability profile,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “Throughout 2024, the Company continually advanced its clinical programs in line with enrollment and dosing timelines, and we will continue to drive the product pipeline forward in 2025.”

Recently, the Company announced it will proceed with a Phase 2/3 pivotal confirmatory clinical trial of OCU410ST for Stargardt disease after alignment with the FDA. This accelerated regulatory pathway for OCU410ST is driven by the incredible unmet medical need that exists for 100,000 Stargardt patients in the U.S. and Europe who have no treatment option available. Accelerating the clinical timeline of OCU410ST is expected to save significant costs in addressing disease burden even sooner than anticipated.

The OCU410ST Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST at a concentration of 1.5 x 1011 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. One-year data will be utilized for the BLA filing planned for 2027. 6-month data from Phase 1 of the OCU410ST GARDian clinical trial demonstrated considerably slower lesion growth (52%) from baseline in treated eyes versus untreated fellow eyes and clinically meaningful 2-line (10-letter) improvement in visual function (BCVA), which is statistically significant (p=0.02) in treated eyes.

This week, the DSMB for the OCU410 ArMaDa clinical trial for GA convened and reviewed the safety and tolerability profile of an additional 15 subjects from the Phase 2 portion of the study. No serious adverse events related to OCU410 have been reported to date in all 60 subjects, including Phase 1. Unlike currently available treatments for GA, there were no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization, and no adverse events of special interest.

Preliminary 9-month efficacy data of OCU410 in GA patients showed considerably slower lesion growth (44%) from baseline and clinically meaningful 2-line (10-letter) improvement in visual function (LLVA) in treated eyes compared to untreated eyes in the Phase 1 portion of the trial. Furthermore, a single subretinal OCU410 treatment preserves more retinal tissue around GA lesions of treated eyes at 9 months compared to published data on currently available GA therapies. GA, an advanced form of dry age-related macular degeneration, affects 2-3 million people in the U.S. and Europe combined.

Two-year data from the Phase 1/2 OCU400 clinical trial for RP demonstrated a durable and statistically significant (p=0.005) improvement in visual function (LLVA) in all evaluable treated subjects when compared to untreated eyes. 100% (10/10) of treated evaluable subjects showed improvement or preservation in visual function compared to untreated eyes, and OCU400 was observed to have a favorable safety and tolerability profile in all subjects across multiple mutations.

In parallel with clinical milestones, considerable regulatory accomplishments were achieved, including orphan medicinal product designation (OMPD) from the European Medicines Agency (EMA) for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD), and positive opinions from the EMA’s Committee for Advanced Therapies (CAT) for OCU400, OCU410, and OCU410ST Advanced Therapy Medicinal Product (ATMP) classification.

Ocugen’s biologic candidate moved into the clinic and patients are currently being dosed in the OCU200 Phase 1 clinical trial for diabetic macular edema (DME). OCU200 has the potential to change the treatment landscape for DME, diabetic retinopathy, and wet age-related macular degeneration with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors that play a crucial role in disease pathogenesis and holds the promise to benefit all DME patients, including the 30-40% of patients who do not respond to current anti-VEGF therapies.

“I am extremely proud of our team at Ocugen, who are working tirelessly to enroll patients across all four clinical programs in close collaboration with the study centers and investigators,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “It is very gratifying to think about the potential unmet medical need that can be addressed through modifier therapy for patients who currently have no options and are facing the prospect of losing their vision altogether.”

Finally, the Investigational New Drug (IND) application for OCU500—the Company’s inhaled mucosal vaccine for COVID-19 was cleared by the FDA. The National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, is expected to sponsor and conduct the Phase 1 trial to assess the safety, tolerability, and immunogenicity of OCU500 administered via two different routes, inhalation into the lungs and intranasally as a spray.

To extend the cash runway into the first quarter of 2026, and support Ocugen’s clinical trials, the Company secured $65 million in equity/debt financings in the second half of 2024. Further supporting Ocugen’s efforts to enable long-term shareholder value, garner significant visibility within the investment community, and broaden the shareholder base, the Company was included in the Russell Index in May 2024.

“Meaningful progress in 2024 and recent success with FDA brought us closer to our goal of three potential BLAs in the years—2026, 2027, 2028, encompassing RP, Stargardt and GA treatments, respectively,” said Dr. Musunuri. “While this is an ambitious goal, I am confident that we have the strategic and scientific expertise, and an unrelenting commitment to patients, to deliver on our commitment.”

Business Updates

Novel Modifier Gene Therapy Platform—Potentially Targeting Three BLA Filings in the Next Three Years

  • OCU400 –The European Commission (EC) provided a positive opinion from the EMA’s CAT for OCU400 ATMP classification. Actively enrolling patients in the U.S. and Canada in the Phase 3 liMeliGhT clinical trial for the treatment of RP and are on track to complete enrollment in the first half of 2025 and file BLA and MAA submissions in mid-2026.
  • OCU410ST –The EMA granted OMPD for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). The FDA approved a Phase 2/3 pivotal confirmatory clinical trial for BLA filing. Plan to initiate the Phase 2/3 study mid-2025 and targeting BLA submission by 2027. The EC provided a positive opinion from the EMA’s CAT for OCU410ST ATMP classification.
  • OCU410 – Completed dosing in Phase 2 of the OCU410 Phase 1/2 ArMaDa clinical trial ahead of schedule. Intend to complete 1-year follow up in the first quarter of 2026 and targeting a BLA filing in 2028. The EC provided a positive opinion from the EMA’s CAT for OCU410 ATMP classification.

Financial Results

  • Fourth quarter — Research and development expenses for the three months ended December 31, 2024, were $8.3 million compared to $7.8 million for the three months ended December 31, 2023. General and administrative expenses for the three months ended December 31, 2024, were $6.3 million compared to $5.2 million for the three months ended December 31, 2023. Ocugen reported a $0.05 net loss per common share for the three months ended December 31, 2024, compared to a $0.04 net loss per common share for the three months ended December 31, 2023.
  • Full year — Research and development expenses for the year ended December 31, 2024, were $32.1 million compared to $39.6 million for the year ended December 31, 2023. General and administrative expenses for the year ended December 31, 2024, were $26.7 million compared to $32.0 million for the year ended December 31, 2023. Ocugen reported a $0.20 net loss per common share for the year ended December 31, 2024, compared to a $0.26 net loss per common share for the year ended December 31, 2023.
  • Ocugen’s cash and restricted cash, totaled $58.8 million as of December 31, 2024, compared to $39.5 million as of December 31, 2023. The Company estimates that its current cash, cash equivalents, and investments will enable it to fund its operations into the first quarter of 2026. The Company had 291.4 million shares of common stock outstanding as of December 31, 2024.

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 leadership team will host the call, which will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.

Attendees are invited to participate on the call or webcast using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 5045393
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.

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 patient’s 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, 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 and efficacy thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, expected cash runway into the first quarter of 2026, the ability to initiate new clinical programs, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing and future 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; 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 annual and 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com 

View Full Release Here.

Ocugen (OCGN) – OCU410 and OCU410ST Receive ATMP Classification In Europe


Tuesday, March 04, 2025

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Ocugen Now Has Three Products With ATMP Designation. Two Ocugen products, OCU410 and OCU410ST, received Advanced Therapy Medicinal Product (ATMP) designation from the European Medicines Agency Committee for Advanced Therapies (EMA-CAT). These join OCU400, which received this designation for retinitis pigmentosa (RP) in February 2025. The designation is similar to the Breakthrough Therapy designation from the FDA, allowing increased interactions with the regulators and accelerating regulatory review.

OCU410 Has Completed Phase 2 Dosing In GA. The Phase 2 portion of the ArMaDa (pronounced ‘Armada”) trial has completed enrollment for OCU410 in geographic atrophy (GA), a lesion that results from advancing dry Age-related Macular Degeneration (dry AMD). The study has enrolled 51 patients randomized into a high dose arm, medium dose arm, or control. The completion of the dosing phase was ahead of our expected time frame, keeping the company on schedule to conduct Phase 3 in 2026 and potentially file for regulatory approval in 2028.


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Release – Ocugen Announces Positive Opinion of EMA’s Committee for Advanced Therapies for ATMP Classification for Novel Modifier Gene Therapy Candidate OCU410 for Geographic Atrophy and OCU410ST for Stargardt Disease

Research News and Market Data on OCGN

March 3, 2025

MALVERN, Pa., March 03, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the European Commission has provided a positive opinion from the European Medicines Agency’s (EMA) Committee for Advanced Therapies (CAT) for OCU410 and OCU410ST Advanced Therapy Medicinal Product (ATMP) classification. OCU410 is a novel, multifunctional modifier gene therapy candidate being developed for the treatment of patients with vision loss due to geographic atrophy (GA)—an advanced stage of dry age-related macular degeneration (dAMD)—and OCU410ST is being developed for Stargardt disease due to ABCA4-related retinopathies.

GA affects 2-3 million people in the United States (U.S.) and Europe combined. There are two approved therapies in the U.S. that require frequent dosing (every month or every other month), however neither therapy has been approved in Europe. Stargardt disease affects 100,000 people in the U.S. and Europe combined, and there are no approved treatments available globally.

“Receiving ATMP classification for OCU410 and OCU410ST is a critical step to potentially address these severely unmet medical needs in the very near future,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Dosing of Phase 2 in the ongoing OCU410 ArMaDa clinical trial is complete, and we are on track to initiate the Phase 3 clinical trial next year to pursue potential Marketing Authorization Application (MAA) and Biologics License Application (BLA) filings in 2028. Last week, the U.S. Food and Drug Administration (FDA) endorsed Ocugen’s plan to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST, which can be the basis of BLA and potential MAA submissions in 2027.”

ATMP classification is granted to medicines that can offer groundbreaking opportunities for the treatment of disease and accelerates the regulatory review timeline of this potential one-time gene therapy for life. Additionally, this classification allows Ocugen to interact with EMA more frequently for scientific advice and protocol assistance.

Preliminary 9-month data of OCU410 in GA patients demonstrated considerably slower lesion growth (44%) from baseline and clinically meaningful 2-line (10-letter) improvement in visual function (LLVA) in treated eyes compared to untreated eyes in the Phase 1 portion of the trial. Furthermore, a single subretinal OCU410 treatment preserves more retinal tissue around GA lesions of treated eyes at 9 months compared to published data on currently available GA therapies.

6-month data from Phase 1 of the OCU410ST GARDian clinical trial demonstrated considerably slower lesion growth (52%) from baseline in treated eyes versus untreated fellow eyes and clinically meaningful 2-line (10-letter) improvement in visual function (BCVA), which is statistically significant (p=0.02) in treated eyes. The Company plans to initiate the Phase 2/3 pivotal confirmatory clinical trial for OCU410ST by mid-2025.

“The novel modifier gene in OCU410 and OCU410ST targets all four pathways linked with dAMD and Stargardt and is delivered through a single subretinal injection as a one-and-done treatment,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “We are very pleased with the structural and functional outcomes demonstrated by both of these candidates, along with a stellar safety profile.”

OCU410 and OCU410ST utilize an adeno-associated virus (AAV) platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role as well as inhibiting the complement system in both in vitro and in vivo (animal model) studies.

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 patient’s 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 OCU410 and OCU410ST 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

Release – Ocugen, Inc. Announces FDA Alignment on Phase 2/3 Pivotal Confirmatory Clinical Trial for Modifier Gene Therapy Candidate OCU410ST for Stargardt Disease

Research News and Market Data on OCGN

February 27, 2025

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MALVERN, Pa., Feb. 27, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that alignment has been reached with the U.S. Food and Drug Administration (FDA) to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which, if positive, can be the basis of a biologics license application (BLA) submission.

The GARDian trial for OCU410ST demonstrated:

  • A favorable safety and tolerability profile with no serious adverse events related to OCU410ST, including no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization and no adverse events of special interest
  • Considerably slower lesion growth (52%) from baseline in treated eyes versus untreated fellow eyes at 6-month follow-up from the Phase 1 study
  • Clinically meaningful 2-line (10-letter) improvement in visual function (BCVA) at 6-month follow-up from the Phase 1 study, which is statistically significant (p=0.02) in treated eyes

“I am very pleased that the FDA has recognized the promise of Ocugen’s modifier gene therapy for Stargardt disease and accelerated the regulatory pathway for OCU410ST,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “This new development allows us to initiate a pivotal confirmatory trial for this game-changing, one-time treatment for life in the next few months and prepare for a potential BLA filing by 2027. Now patients suffering from Stargardt disease have a new hope where previously none existed. This achievement furthers our mission to cure blindness diseases.”

Stargardt disease affects 100,000 people in the U.S. and Europe combined, and there is no treatment. OCU410ST received orphan drug designations from the FDA and the European Medicines Agency (EMA) in 2023 and 2024, respectively.

“Getting approval for a Phase 2/3 trial is a pivotal milestone, as this approach has never been explored in clinical trials for Stargardt disease. The FDA’s decision underscores the potential of OCU410ST to meet a critical unmet medical need for the approximately 44,000 Stargardt patients in the U.S.,” said Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Professor of Ophthalmology, Pediatrics and Biomedical Engineering with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair of Ocugen.

The Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST at a concentration of 1.5 x 1011 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. The primary endpoint in the clinical trial is change in atrophic lesion size. Secondary endpoints include visual acuity as measured by best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA) compared to untreated controls. One-year data will be utilized for the BLA filing.

“This approval pathway, established in collaboration with the FDA, has made it possible to expedite the clinical development of OCU410ST by two to three years and has aided in bringing an innovative gene therapy to patients desperate for a treatment option,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “Recent data from the OCU410ST clinical trial have shown significant improvements in both structural and functional outcomes. Additionally, OCU410ST has consistently demonstrated a very favorable safety and tolerability profile.”

Accelerating the clinical timeline of OCU410ST will save significant costs in addressing disease burden even sooner than anticipated.

About OCU410ST
OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR-Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.

About Stargardt Disease
Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula.

Decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. Some peripheral vision is usually preserved. Stargardt disease typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in people with Stargardt disease.

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 patient’s 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 OCU410ST 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

Release – Ocugen to Host Conference Call on Wednesday, March 5, 2025 at 8:30 A.M. ET to Discuss Business Updates and Fourth Quarter and Full Year 2024 Financial Results

Research News and Market Data on OCGN

February 21, 2025

PDF Version

MALVERN, Pa., Feb. 21, 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 that it will host a conference call and live webcast to discuss the Company’s fourth quarter and full year 2024 financial results and provide a business update at 8:30 a.m. ET on Wednesday, March 5, 2025.

Ocugen will issue a pre-market earnings announcement on the same day. Attendees are invited to participate on the call using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 5045393
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.

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 patient’s 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, 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.

Contact:
Tiffany Hamilton
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com 

Ocugen (OCGN) – Phase 2 OCU410 Trial Completes Patient Enrollment Ahead Of Schedule


Thursday, February 13, 2025

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

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Ocugen Announced Completion of OCU410 Phase 2 Enrollment. The Phase 2 trial testing OCU410 for Geographic Atrophy in Dry Age-Related Macular Degeneration (GA/dAMD) has completed patient enrollment ahead of schedule. The trial is expected to announce interim data around YE2025 with top-line data in 1H26. We anticipate a Phase 3 trial starting in late 2026, which could allow for a BLA application in 2028. This could become Ocugen’s third BLA in three years.

Trial Design Has Two Stages. The Phase 1/2 ArMaDa trial is treating patients with geographic atrophy (GA), a symptom of advanced dry AMD. The trial completed a Phase 1 dose-escalation stage to determine basic safety. The current Phase 2 is a randomized, dose expansion stage with two doses tested against a control. The study has 17 patients in each arm (21 patients total).


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Release – Ocugen, Inc. Announces Dosing Completion in the Phase 2 ArMaDa Clinical Trial for OCU410—a Multifunctional Modifier Gene Therapy for the Treatment of Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration

Research News and Market Data on OCGN

February 12, 2025

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  • Completed Phase 2 enrollment with randomization of 51 subjects into treatment and control arms
  • Phase 1/2 study (N=60) demonstrated favorable safety and tolerability profile with no serious adverse events related to OCU410, including no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization
  • Subjects showed considerably slower lesion growth (44%) from baseline in treated eyes versus untreated fellow eyes at 9 months in follow-up data from the Phase 1 study
  • Clinically meaningful 2-line (10-letter) improvement in visual function (LLVA) in treated eyes compared to untreated eyes was noted in the Phase 1 portion of the trial
  • Preservation of retinal tissue at 9 months around GA lesions of treated eyes with a single injection of OCU410 in Phase 1 compared favorably to published data on a leading FDA-approved complement inhibitor given monthly or every other month at the same time points

MALVERN, Pa., Feb. 12, 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 that dosing is complete, ahead of schedule in the Phase 2 portion of the Phase 1/2 ArMaDa clinical trial for OCU410—a novel multifunctional modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). Age-related macular degeneration (AMD) affects 1 in 8 people 60 years and older. The global prevalence of dAMD is 266 million worldwide and by 2050 more than 5 million Americans may suffer from this incurable condition. Today, GA – the later stage of dAMD – affects approximately 2-3 million people in the United States (U.S.) and Europe.

There are limited options for patients with dAMD in the U.S. and current therapies involve frequent (monthly or every other month) injections and have unwanted side effects that can affect vision. These therapies are not approved in Europe, leaving approximately 2 million patients with no therapeutic option.

“Dosing completion is a major accomplishment for our OCU410 program,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Based on the multifunctional effect of our modifier gene therapy, the profound unmet medical need, limited treatment options, and the fact that it is designed as a one and done treatment, we believe OCU410 can be a potential blockbuster therapy and the gold standard for treating GA worldwide. The data from this trial will help us design a future pivotal Phase 3 study planned for 2026 and enable our commercial strategy for Biologics License Application (BLA) and Marketing Authorization Application (MAA) filings as soon as 2028.”

“The preliminary efficacy and safety data from the Phase 1/2 study are highly encouraging, demonstrating the potential of OCU410 to improve both structural and functional outcomes,” said Lejla Vajzovic, MD, FASRS, Director of the Duke Surgical Vitreoretinal Fellowship Program and Professor of Ophthalmology, Pediatrics and Biomedical Engineering with Tenure at Duke University Eye Center. “I look forward to the Phase 2 results and believe a one-time gene therapy could reshape the treatment landscape, offering a transformative option for patients.”

GA is a multifactorial disease with a complex etiology that involves genetic and environmental factors. The current treatment options for GA in the U.S. are limited to those targeting a single mechanism—the complement pathway—requiring frequent intravitreal injections, either monthly or every other month. By contrast, OCU410 is a multifunctional modifier gene therapy, which targets multiple pathways associated with GA.

“Given the safety concerns associated with currently approved GA treatments, the encouraging safety and tolerability profile of OCU410 offers a promising treatment option,” said Dr. Huma Qamar, Chief Medical Officer of Ocugen. “With Phase 2 enrollment now complete, OCU410 has the potential to be a one-time treatment, reducing the burden of frequent injections, improving patient compliance, and ultimately enhancing quality of life.”

In the Phase 2 study, the safety and efficacy of OCU410 in patients with GA secondary to dAMD will be assessed. Fifty-one (51) patients were randomized 1:1:1 into either of two treatment groups (medium or high dose) or a control group. In the treatment groups, subjects received a single subretinal 200-µL administration of 5 x 1010 vector genomes (vg)/mL (medium dose) or 1.5 x 1011 vg/mL (high dose), while the control group remained untreated.

The ArMaDa clinical trial for OCU410 is being performed at 14 leading retinal surgery centers across the U.S.

About the Phase 1/2 ArMaDa clinical trial
The ArMaDa Phase 1/2 clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects with GA and will be conducted in two phases. Phase 1 is a multicenter, open label, dose-escalation study consisting of three dose levels [low dose (2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5 ×1011 vg/mL)]. Phase 2 is a randomized, outcome assessor-blinded, dose-expansion study in which subjects were randomized in a 1:1:1 ratio to either the medium dose or high dose OCU410 treatment groups or to an untreated control group.

About dAMD and GA
dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula, the portion of the retina responsible for clear vision in one’s direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function, and central vision impairment. dAMD accounts for 85-90% of all AMD cases.

About OCU410
OCU410 utilizes an adeno-associated virus (AAV) platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role as well as inhibiting the complement system in both in vitro and in vivo (animal model) studies. These results demonstrate the ability of OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA.

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 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 OCU410 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com