Tag: OCGN

Research News and Market Data on OCGN

January 16, 2025

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MALVERN, Pa., Jan. 16, 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 the first patient has been 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 (DR), and wet age-related macular degeneration (wet AMD) with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors that play a crucial role in disease pathogenesis,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. “OCU200 holds the promise to benefit all DME patients, including the 30-40% of patients who do not respond to current anti-VEGF therapies.”

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 a total of two intravitreal injections of OCU200 six weeks apart. Patient follow-up will take place up to three months after the last injection.

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

“I am seeing an increasing rate of vision-threatening diseases associated with diabetes at my clinic and am eager to provide a new therapeutic option to these patients,” said Dr. David Almedia, Vitreoretinal Surgeon and Clinician Scientist, President and CEO of Erie Retina Research, and Founder and President of Case X Global in Erie, Pennsylvania. “There remains a considerable unmet medical need for DME and DR patients with currently available anti-VEGF treatments.”

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.

“We are enthusiastic about getting patients started in the OCU200 Phase 1 clinical trial and sharing not only safety but preliminary efficacy data as the study progresses,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “OCU200 brings an innovative biologic candidate to Ocugen’s ophthalmology portfolio targeting blindness diseases.”

The Company intends to pursue approval to use OCU200 as a first-line therapy for DME, DR, and wet AMD.

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
Head of Communications
Tiffany.Hamilton@ocugen.com

Research News and Market Data on OCGN

January 13, 2025

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• 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

Research News and Market Data on OCGN

November 20, 2024

MALVERN, Pa., Nov. 20, 2024 (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 the European Medicines Agency (EMA) has granted orphan medicinal product designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3).

“We are deeply honored to receive orphan medicinal product designation from the EMA for OCU410ST. This recognition brings us one step closer to providing a much-needed option for Stargardt patients who currently have no therapies available,” said Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen. “We are committed to advancing this treatment with urgency and dedication, with the hope of making a meaningful impact on the lives of those affected by this challenging disease.”  

The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation to OCU410ST in April 2023. Stargardt disease affects approximately 100,000 people in the U.S. and Europe combined.

Orphan medicinal product designation in Europe offers certain benefits to drug developers while they develop drugs intended for safe and effective treatment, diagnosis, or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Benefits include protocol assistance, reduced regulatory fees, research grants, and 10 years of market exclusivity following regulatory approval.

Dosing in the first phase of the Phase 1/2 OCU410ST GARDian trial for Stargardt disease is complete and the Data and Safety Monitoring Board (DSMB) has recommended moving forward with Phase 2. To date, the safety and tolerability profile of OCU410ST appears to be very favorable.

Preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410ST GARDian clinical trial was recently presented at Ocugen’s Clinical Showcase. Data from evaluable subjects at six months demonstrated a remarkable 84% reduction in atrophic lesion growth in treated eyes versus untreated fellow eyes.

“We are encouraged by the preliminary efficacy data showing stabilization or improvement in visual function and retinal structure outcomes in OCU410ST treated eyes,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “These positive clinical and regulatory milestones continue to support the potential for OCU410ST to address inherited retinal diseases with a one-time therapy for life.”

OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene and further represents the impact of Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks.

Ocugen intends to pursue an accelerated marketing authorization application (MAA) for OCU410ST.

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 thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; 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
Tiffany.Hamilton@ocugen.com

Research News and Market Data on OCGN

November 19, 2024

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MALVERN, Pa., Nov. 19, 2024 (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 positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD). Key findings include: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA).

Currently, there are approximately three million people living with GA in the United States (U.S.) and Europe combined. Patients in the U.S. have only one option available, anti-complement therapy, which requires multiple injections and only addresses one aspect of the disease. There remains no treatment option for GA in Europe.

The OCU410 Phase 1 trial is evaluating nine patients in three dose cohorts (low, medium, and high). The following data was observed for the three patients in the low dose cohort at six months:

• Considerably slower lesion growth (21.4%) from baseline in treated vs. untreated fellow eyes that followed the natural history of the disease. This result is favorable when compared to published data on pegcetacoplan injected every month or every other month over six months.
• OCU410 treatment showed increasing preservation of retinal tissue around the GA lesions of treated eyes over six months, which also compared favorably to published data on pegcetacoplan given monthly and every other month.
• 100% of the OCU410 treated eyes showed stabilization of visual function demonstrating treatment benefit as measured by LLVA.

“Currently approved treatments for GA have not shown significant benefit in visual function. More importantly, we often do not realize the logistical challenge and emotional burden both patients and their caregivers must endure for every month or every other month visits,” said Syed M. Shah, MD, FACS, Director of Retina Service, Vice Chair for Research & Digital Health at Emplify Health – La Crosse, Wisconsin. “Based on the science and preliminary data, OCU410 has the potential to improve structural as well as functional outcomes. This ‘one-and-done’ treatment paradigm can be a gamechanger for how we treat patients with GA.”  

“OCU410 addresses multiple aspects of the disease beyond the complement pathway,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “The latest OCU410 data emphasizes the potential of novel modifier gene therapy as a one-time treatment for dAMD. We remain very encouraged by the latest safety and efficacy data and positive patient outcomes.”

Ocugen also announced promising data from the Phase 1/2 OCU410ST GARDian clinical trial for Stargardt disease and data on Leber congenital amaurosis (LCA) from the Phase 1/2 OCU400 clinical trial. All these findings, as well as commentary from study investigators and patient perspectives, were shared at the Company’s recent Clinical Showcase. The data affirms the potential for modifier gene therapy to address both rare inherited retinal diseases and blindness diseases affecting millions.

A full replay of the showcase is available on the Events section of the Ocugen website. For more information about Ocugen’s ongoing clinical trials, please contact clinical.request@ocugen.com

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 thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; 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
Tiffany.Hamilton@ocugen.com

Research News and Market Data on OCGN

November 8, 2024

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

• Subsequent to quarter-end, closed $30 million in debt financing
• OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) on track to complete enrollment in 1H2025
• OCU410 is currently in Phase 2 of the Phase 1/2 ArMaDa clinical trial
• Data and Safety Monitoring Board (DSMB) for the OCU410ST GARDian clinical trial approved enrollment for the second phase of the Phase 1/2 clinical trial
• New data on Phase 1/2 clinical trials for OCU410, OCU410ST and OCU400 to be presented at upcoming Clinical Showcase

MALVERN, Pa., Nov. 08, 2024 (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 reported third quarter 2024 financial results along with a general business update.

“I am very encouraged by the progress of our gene therapy programs and the clinical and regulatory milestones achieved in the third quarter of 2024, including the expansion of the OCU400 Phase 3 liMeliGhT clinical trial into Canada,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “With the recent equity and debt financings, we have sufficient cash-on-hand to continue supporting our robust ophthalmology pipeline and extend our cash runway into 1Q2026.”

As OCU400 is advancing through Phase 3 clinical development, the expanded access program (EAP) for adult patients with early to advanced RP makes it possible to reach a greater segment of the RP patient population—300,000 in the U.S., Canada, and Europe combined. Additionally, including Canadian patients in the OCU400 Phase 3 liMeliGhT trial may allow for broader commercialization with the U.S. and Europe. These accomplishments and consistent trial enrollment are bringing the Company even closer to providing a potential one-time treatment for life to patients living with RP.

Phase 2 of the OCU410 Phase 1/2 ArMaDa clinical trial is underway and will assess the safety and efficacy of OCU410 in a larger group of patients who are randomized into either of two treatment groups (medium- or high-dose) or a control group. OCU410 is being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). GA affects approximately 2-3 million people in the U.S. & EU. Current FDA-approved treatments address only the complement system and require approximately 6-12 intravitreal injections per year, whereas OCU410 addresses all four pathways linked with dAMD pathophysiology and is delivered through a single subretinal injection. There remains no approved product to treat GA in the EU.

Over a series of conferences during the third quarter 2024, Ocugen had the opportunity to provide an update on its three clinical-stage modifier gene therapies to significant investor audiences as well as industry decision-makers during meetings like the Cell & Gene Meeting on the Mesa hosted by the Alliance for Regenerative Medicine.

“It is imperative to continue educating our key stakeholders about the differentiated mechanism of action of our gene-agnostic modifier gene therapy platform,” said Dr. Musunuri. “Unlike other product candidates in development to treat blindness diseases, our approach leverages master gene regulators that reset the functional network—rather than targeting a single mutation—and restore overall health to the retina. Our data continues to support the potential to treat multiple disease mutations with a one-time therapy for life.”

While gene therapy remains the primary focus for the Company, Ocugen continues to pursue funding opportunities across the portfolio to ensure that its innovative platforms reach the people who need them.

A clinical showcase, providing updates from Ocugen’s ongoing gene therapy trials, will be held on November 12, 2024, and will include preliminary safety and efficacy data from the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy and Phase 1/2 OCU410ST GARDian clinical trial for Stargardt disease, along with RP and LCA data updates from the OCU400 Phase 1/2 clinical trial.

Ophthalmic Gene Therapies—First-in-class

• OCU400 – Enrollment continues in the Phase 3 liMeliGhT clinical trial and Health Canada approved enrollment across a maximum of 5 sites in Canada. FDA approved EAP for the treatment of adult patients with RP who may benefit from the mechanism of action of OCU400.
• OCU410 – Actively recruiting patients in Phase 2 of the Phase 1/2 ArMaDa clinical trial. Preliminary safety and efficacy update on OCU410 Phase 1/2 ArMaDa clinical trial will be shared at upcoming clinical showcase.
• OCU410ST – DSMB approved proceeding to Phase 2 of the Phase 1/2 GARDian clinical trial. Preliminary safety and efficacy update will be shared at upcoming clinical showcase.

Ophthalmic Biologic Product

• OCU200 – FDA cleared the investigational new drug application for the Phase 1 clinical trial evaluating OCU200. The Company is planning to initiate the OCU200 Phase I clinical trial this quarter.

Third Quarter 2024 Financial Results

• With the recent $30 million debt financing and $35 million equity financing in the third quarter, the cash runway now extends into 1Q2026.
• The Company’s cash and restricted cash totaled $39.0 million as of September 30, 2024, compared to $39.5 million as of December 31, 2023.
• Total operating expenses for the three months ended September 30, 2024 were $14.4 million and included research and development expenses of $8.1 million and general and administrative expenses of $6.3 million. This compares to total operating expenses for the three months ended September 30, 2023 of $16.1 million that included research and development expenses of $7.0 million and general and administrative expenses of $9.1 million.

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: 9923172
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 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 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
Tiffany.Hamilton@ocugen.com 

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Research News and Market Data on OCGN

November 7, 2024

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MALVERN, Pa., Nov. 07, 2024 (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 on November 6, 2024, the Company entered into a new $30 million credit facility with Avenue Venture Opportunities Fund, L.P., a fund of Avenue Capital Group. Proceeds from the facility are intended for general corporate purposes, capital expenditures, working capital, and general and administrative expenses.

The credit facility, which has a term of 4 years, provided $30 million fully funded on the closing date.

“We are pleased to enter into this relationship with Avenue Capital Group that provides what we believe is a shareholder-friendly financing for the Company,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-founder of Ocugen. “This additional working capital will support the clinical development of our three, first-in-class modifier gene therapies and provide adequate funding to near completion of the OCU400 Phase 3 liMeliGhT clinical trial and prepare for the BLA and MAA submissions.”

This most recent financing is part of Ocugen’s diversified strategy to fund the business and appropriately allocate resources across the portfolio.

“We are pleased to partner with Ocugen with this financing as the Company drives its next chapter of growth, based on its novel scientific platforms and dedication to fighting blindness diseases,” said Chad Norman, Senior Portfolio Manager, Avenue Capital.

With net proceeds from this facility and current cash, cash equivalents, and restricted cash, the Company’s expected cash runway extends into the first quarter of 2026.

Chardan and Titan Partners Group, a division of American Capital Partners, acted as financial advisors to Ocugen on the transaction.

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.

About Avenue Venture Opportunities
The Avenue Venture Debt Funds seek to provide creative financing solutions to high-growth, venture capital-backed technology and life science companies, focusing generally on companies within the underserved segment of the market created by the widening financing gap between commercial banks and larger debt funds. The Avenue Venture Debt funds are part of the larger group of funds of Avenue Capital Group. For additional information on Avenue Capital Group, which is a global investment firm with assets under management of approximately $12.2 billion, visit www.avenuecapital.com.

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; expectations regarding the satisfaction of closing conditions, timing of the funding and the anticipated use of proceeds; Ocugen’s financial condition and expected cash runway into the first quarter of 2026; and 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
Tiffany.Hamilton@ocugen.com

Research News and Market Data on OCGN

October 28, 2024

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• Progress on OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and new data from Phase 1/2
• Preliminary safety and efficacy update on OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy (GA)
• Clinical update on OCU410ST Phase 1/2 GARDian clinical trial for Stargardt disease
• Featuring patients, investigators and thought leaders

MALVERN, Pa., Oct. 28, 2024 (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 an in-person Clinical Showcase on Tuesday, November 12, 2024. The event will take place from 10 a.m.-noon ET at the Nasdaq MarketSite in Times Square, New York City.

The event will focus on encouraging updates from Ocugen’s ongoing gene therapy trials, including:

• Clinical update on Phase 3 liMeliGhT clinical trial for retinitis pigmentosa along with data updates from Phase 1/2 RP and Leber congenital amaurosis (LCA)
• Preliminary safety and efficacy data from Phase 1/2 OCU410 ArMaDa clinical trial for GA
• Clinical trial progress from Phase 1/2 OCU410ST GARDian study for Stargardt disease

In addition, background on Ocugen’s biologic candidate, OCU200 for diabetic macular edema, will be presented. The Company is planning to initiate the OCU200 Phase I clinical trial this quarter.

Ocugen presenters will include:

• Dr. Shankar Musunuri, Chairman, CEO & Co-founder
• Dr. Huma Qamar, Chief Medical Officer
• Dr. Arun Upadhyay, Chief Scientific Officer & Head of R&D
• Mike Shine, Senior Vice President, Commercial

Joining the Ocugen team will be study investigators:

• Dr. Benjamin Bakall, Director of Clinical Research at Associated Retina Consultants (ARC) and Clinical Assistant Professor at University of Arizona, College of Medicine – Phoenix
• Dr. Lejla Vajzovic, Professor of Ophthalmology, Pediatrics, and Biomedical Engineering with Tenure at Duke Eye Center and Duke University School of Medicine
• Dr. Syed M. Shah, Vice Chair for Research and Digital Health, Director of Retina Service at Gundersen Health – La Crosse, Wisconsin

The program will conclude with a patient panel representing participants in Ocugen’s ongoing clinical trials.

Advance registration is required and can be done by contacting Tiffany Hamilton at Tiffany.Hamilton@ocugen.com.

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 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 thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; 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
Tiffany.Hamilton@ocugen.com 

Research News and Market Data On OCGN

October 22, 2024

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• Determined the high dose of OCU410ST to be the maximum tolerated dose
• No serious adverse events have been reported
• Approved proceeding to Phase 2 using high and medium doses of OCU410ST

MALVERN, Pa., Oct. 22, 2024 (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 the Data and Safety Monitoring Board (DSMB) for the OCU410ST GARDian clinical trial recently convened and approved enrollment for the second phase of the Phase 1/2 clinical trial. OCU410ST (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for Stargardt disease.

“The DSMB has recommended moving forward with Phase 2 enrollment, as safety data indicates that OCU410ST appears to be safe and well-tolerated to date,” said Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas & Retina Consultants of America, and a lead study investigator. “The safety and tolerability profile of OCU410ST remains encouraging as the clinical trial has progressed and continues to bring hope to patients with Stargardt disease, which still has no FDA-approved treatments.”

The first phase of the Phase 1/2 clinical trial was an open-label, dose-ranging study that enrolled nine patients to receive either a low (3.75 x 10¹⁰ vg/mL), medium (7.5 x 10¹⁰ vg/mL), or high (2.25 x 10¹¹ vg/mL) dose of OCU410ST administered via subretinal injection. No serious adverse events (SAEs) have been reported, and the DSMB determined the high dose to be the maximum tolerated dose (MTD).

Stargardt disease is the most common form of inherited macular dystrophy. Symptoms of bilateral central vision loss typically begin in childhood and gradually worsen over time.

“We are enthusiastic about the potential of OCU410ST to be the first one-time novel modifier gene therapy for Stargardt disease,” said Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen. “We are encouraged by the prospect of addressing a substantial unmet medical need for the estimated 100,000 Stargardt patients in the U.S. and Europe.”

The GARDian clinical trial is currently being performed at 6 leading retinal surgery centers across the U.S.

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 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 pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.

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 thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; 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
Tiffany.Hamilton@ocugen.com