Tag: Ocugen

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

Research News and Market Data on OCGN

October 17, 2024

MALVERN, Pa., Oct. 17, 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 a conference call and live webcast to discuss the Company’s third quarter 2024 financial results and provide a business update at 8:30 a.m. ET on Friday, November 8, 2024.

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

Research News and Market Data on OCGN

October 9, 2024

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MALVERN, Pa., Oct. 09, 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 U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the investigational new drug application for the Phase 1 clinical trial evaluating OCU200, a recombinant fusion protein consisting of tumstatin and transferrin, for treating diabetic macular edema (DME).

“We are excited to launch the Phase 1 clinical trial for OCU200, which is designed to treat patients with DME,” said Dr. Arun Upadhyay, Chief Scientific Officer and Head of Research & Development at Ocugen. “Approximately 30 to 40% of DME patients are refractive to current anti-VEGF therapies. OCU200 targets the underlying disease mechanisms through the integrin pathway and holds promise to provide benefits to all DME patients, including non-responders to currently approved therapy.”

OCU200 possesses unique features that potentially enable it to treat vascular complications of DME. Tumstatin is the active component of OCU200 and binds to integrin receptors, which play a crucial role in disease pathogenesis. Transferrin is expected to facilitate the targeted delivery of tumstatin into the retina and choroid and potentially help increase the interaction between tumstatin and integrin receptors.

“I look forward to bringing OCU200 into the clinic and advancing this important candidate in Ocugen’s portfolio for treating blindness diseases,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “DME is becoming more prevalent as the number of people with diabetes in the U.S. rises, making the condition even more imperative to address.”

This is a multicenter, open-label, dose-ranging study with 3 cohorts in the dose-escalation portion assessing safety of OCU200 and in the fourth cohort a combination of OCU200 (MTD) with anti-VEGF following sequential intravitreal administration.

DME is one of the most common vision-threatening diseases occurring in people with diabetes and includes blurriness in vision and progressive vision loss as the disease progresses. Approximately 746,000 people in the United States are affected with DME.

The Company intends to pursue additional indications for OCU200 to potentially treat diabetic retinopathy and wet age-related macular degeneration, which combined affect nearly nine million Americans.

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, 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

September 5, 2024

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MALVERN, Pa., Sept. 05, 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, and vaccines, today announced that Arun Upadhyay, PhD, Ocugen’s Chief Scientific Officer, Head of Research & Development, will be among the featured speakers at the 5^th Annual Gene Therapy for Ophthalmic Disorders conference, which is being held September 10-12, 2024 in Boston, Mass.

“There is tremendous potential for gene therapy to treat both rare retinal diseases and ophthalmic diseases affecting millions,” said Dr. Upadhyay. “I look forward to sharing the development and clinical progress of Ocugen’s modifier gene therapy platform with my peers and learning about the latest advancements in the field from industry experts.”

Details regarding Dr. Upadhyay’s participation are as follows:

Workshop Moderator
Topic: Navigating Regulatory Pathways for Ophthalmic Gene Therapies
Date: Tuesday, September 10, 2024
Time: 1-4 p.m. ET

Presentation
Topic: Advancements in Gene Therapy for Opthalmic Disorders: Ocugen’s Clinical Program Updates
Date: Thursday, September 12, 2024
Time: 11:30 a.m.-noon

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.

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 
Head of Communications 
Tiffany.Hamilton@ocugen.com

Research News and Market Data on OCGN

August 28, 2024

PDF Version

MALVERN, Pa., Aug. 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 and vaccines, today announced that dosing is complete in the third cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease. Stargardt disease affects approximately 100,000 people in the United States (U.S.) and Europe.

“With all patients dosed in cohort 3 (high dose), Phase 1 of the dose-escalation portion of the trial is complete,” said Dr. Huma Qamar, Chief Medical Officer of Ocugen. “We will continue to advance the trial as efficiently as possible, and work toward fulfilling an unmet medical need for Stargardt patients.”

Three subjects received a single subretinal injection of the highest dose (2.25×10¹¹ vg/mL) being tested. The GARDian clinical trial is being performed at six leading retinal surgery centers across the U.S.

“OCU410ST is a novel modifier gene therapy that has the potential to be a one-time treatment given by subretinal injection in the operating room,” said Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas, lead investigator in the study. “The safety and tolerability profile of OCU410ST remains encouraging as the clinical trial progresses and brings hope to patients with Stargardt disease, who have no FDA-approved treatment options.”

The GARDian clinical trial will assess the safety and efficacy of unilateral subretinal administration of OCU410ST in subjects with Stargardt disease and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (3.75×10¹⁰ vg/mL), medium dose (7.5×10¹⁰ vg/mL), and high dose (2.25×10¹¹ vg/mL)].

Ocugen remains committed to advancing treatments for blindness, focusing on innovative gene therapy solutions that aim to provide lasting benefits to patients.

The Company will continue to provide clinical updates on a periodic basis.

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, compliment 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 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.

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
Head of Communications
Tiffany.Hamilton@ocugen.com

Research News and Market Data on OCGN

August 26, 2024

PDF Version

MALVERN, Pa., Aug. 26, 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 and vaccines, today announced that Health Canada provided a “No Objection Letter” to initiate the OCU400 Phase 3 liMeliGhT (pronounced “limelight”) clinical trial in Canada. OCU400 is a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP).

“Expanding the clinical trial to Canada is significant as it will provide an opportunity to reach a broader patient population encompassing many gene mutations associated with RP,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. “The Health Canada trial will run in parallel with the U.S. FDA trial, expediting the ability to potentially provide a gene-agnostic treatment option to approximately 110,000 patients in the United States (U.S.) and Canada.”

Currently there are approximately 10,000 patients in Canada with RP and 1.6 million patients globally. The Phase 3 study in Canada will enroll up to 50 subjects across a maximum of 5 sites for the liMeliGhT clinical trial.

Over 200 mutations in more than 100 genes have been linked to RP. The Phase 3 study, spanning one year, will enroll 150 participants divided into two study arms: 75 participants with RHO gene mutations and 75 participants who are gene agnostic. In each arm, participants will be randomized in a 2:1 ratio to receive either treatment (2.5 x 10¹⁰ vg/eye of OCU400) or remain in an untreated control group, respectively. The liMeliGhT study is recruiting patients aged eight and older, covering the full spectrum from early to late stages of RP progression.

An enhanced sensitive and specific measurement of functional vision test—Luminance Dependent Navigation Assessment (LDNA)—is the primary endpoint for the study. Specifically, the primary endpoint is a measurement of the change in functional vision from baseline to week 52 as measured by the ability of a study participant to navigate through a maze (the LDNA). Those who demonstrate an improved ability to navigate the maze in dimmer light (i.e., by ≥2 Lux levels) compared to baseline will be classified as “responders” to the therapy. The liMeliGhT study will focus on the proportion of responders in both the treated and untreated eyes.

“Establishing clinical sites in Canada may expedite recruitment and open doors for broader commercialization with the U.S. and Europe,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “With only one currently approved treatment targeting a single mutation associated with RP, there remains a significant unmet medical need, and patients worldwide are eager for new therapeutic options. It is highly rewarding to extend our efforts into a new region and offer hope to Canadian patients with RP.”

Ocugen previously announced that OCU400 has received orphan drug and RMAT designations from the FDA. OCU400 remains on track for the 2026 BLA and MAA approval targets.

About OCU400

OCU400 is the Company’s modifier gene therapy product based on a nuclear hormone receptor 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 RP

RP is a group of rare genetic disorders that cause a breakdown in the cells of the retina, leading to vision loss and blindness. RP is associated with mutations in more than 100 genes.

There are no approved treatment options that slow or stop the progression of multiple forms of RP. Proposed treatments for RP include gene replacement therapy, retinal implant devices, retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. Current gene replacement therapies are promising but are limited to treating just a single mutation. In addition, while gene therapies may provide a new functional gene, they do not necessarily eliminate the underlying genetic defect, which may still cause stress and toxic effects leading to retinal degeneration. Therefore, the development of gene-specific replacement therapy will not address all forms of RP, especially when multiple and unknown genes are involved. Thus, novel therapeutic approaches targeting the broader RP disease in a gene-agnostic manner offer greater hope for patients.

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.

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
Head of Communications
Tiffany.Hamilton@ocugen.com

News Research and Market Data on OCGN

August 8, 2024

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

• Actively dosing patients in OCU400 Phase 3 liMeliGhT clinical trial
• OCU410 preliminary safety and efficacy data expected later this year
• Expanded access program approved for OCU400
• $32.6 million net cash from underwritten public offering of common stock

MALVERN, Pa., Aug. 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 second quarter 2024 financial results along with a business update.

“The first half of 2024 has been marked with significant accomplishments for our modifier gene therapy platform—including dosing patients in the OCU400 Phase 3 clinical trial for retinitis pigmentosa (RP) and progressing into Phase 2 of the OCU410 ArMaDa clinical trial for the treatment of geographic atrophy (GA),” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “These meaningful milestones bring us closer to providing a potential one-time therapy for life for patients living with RP (300,000 in the U.S. and Europe) and GA (2-3 million in the U.S. and Europe) who desperately need effective treatment options. Thanks to our Ocugen team for their tireless efforts to keep these and all our clinical trials on track.”

The OCU400 Phase 3 trial has a sample size of 150 participants: one arm has 75 participants with RHO gene mutations, and the other arm has 75 participants with mutations in any of several other genes associated with RP. The Luminance Dependent Navigation Assessment (LDNA) is the primary endpoint for the study. In this assessment, a participant navigates an obstacle course that constitutes a more sensitive and specific measurement of visual function than the mobility measurement used in previous Phase 3 clinical trials. The Phase 3 liMeliGhT trial will focus on the proportion of responders, in treated and untreated groups, who achieve an improvement of at least 2 Lux (light) levels from baseline in the study eyes. More than 60% of the intent-to-treat patients from the Phase 1/2 clinical trial, including patients with the RHO mutation, meet the responder criteria established for Phase 3. The Phase 3 mobility test responder rate for the only FDA-approved product to treat one mutation in RP was 52%. The Phase 3 trial is powered greater than 95% assuming a 50% responder rate.  

Recently, the FDA approved the OCU400 expanded access program (EAP) for the treatment of adult patients, aged 18 and older, with RP. This is the first ever gene therapy candidate to treat patients with RP, regardless of mutation, approved for an EAP and the EAP further supports the gene-agnostic mechanism of action for this novel modifier gene therapy.

Novel modifier gene therapy has the potential to address multiple inherited retinal diseases as well as multifactorial causes of blindness that affect millions of patients, like dry age-related macular degeneration (dAMD). OCU410 and OCU410ST aim to treat geographic atrophy secondary to dAMD and Stargardt disease, respectively. These modifier gene therapies leverage a nuclear hormone receptor gene called RORA (RAR-related orphan receptor A) as a potential one-time therapy for life with a single sub-retinal injection.

OCU410 is specifically designed to address multiple pathways implicated in the pathogenesis of dAMD and offers a distinct advantage over current treatment options that target only one pathway—the complement system—and require frequent intravitreal injections (about 6-12 doses per year), accompanied by various safety concerns, such as roughly 12% of patients progressing to wet AMD. OCU410 has the potential to regulate all four pathways related to disease progression—lipid metabolism, inflammation, oxidative stress, and the complement system—with a one-time sub-retinal injection.

OCU410ST has received an Orphan Drug Designation from the FDA for the treatment of Stargardt disease, which has no approved treatment and affects approximately 100,000 people in the U.S. and Europe combined. The third cohort of the clinical trial is currently receiving the high dose. OCU410ST has the potential to be the first one-time gene therapy for Stargardt disease.

Ocugen continues to pursue strategic partnerships that will drive long-term strategy, and most importantly, will help patients access these novel modifier gene therapies globally. During the 2024 BIO International Convention, Ocugen engaged with potential partners and pharmaceutical executives to explore opportunities for the Company’s dynamic pipeline.

“Ocugen’s inclusion in the Russell Index in June further bolsters the value of our pipeline and recognizes the Company’s robust growth strategy,” said Dr. Musunuri. “This ranking supports our efforts to enable long-term shareholder value, garner significant visibility for Ocugen within the investment community, and broaden our shareholder base. I look forward to the second half of 2024 as we continue to solidify Ocugen’s position as a biotechnology leader.”

Subsequent to June 30, 2024, the Company closed a public offering of common stock with net proceeds of $32.6 million—extending its expected cash runway into the third quarter of 2025. The offering was led by a large premier mutual fund, along with participation from leading life sciences investors.

Ophthalmic Gene Therapies—First-in-Class

OCU400 – Ocugen is actively dosing subjects in the OCU400 Phase 3 liMeliGhT trial for the treatment of RP. With dosing of the Phase 3 trial underway, OCU400 remains on track for the 2026 BLA and MAA approval targets.

OCU410 – In July 2024, Ocugen announced the completion of dosing in the third cohort of the OCU410 Phase 1/2 ArMaDa clinical trial for the treatment of GA. To date, nine patients with GA have been dosed in the Phase 1/2 clinical trial (with low, medium, and high doses). Phase 2 of the clinical trial has been initiated and will assess the safety and efficacy of OCU410 in a larger group of patients who will be randomized into either of two treatment groups (medium or high dose) or a control group.

OCU410ST – Currently dosing the high dose of OCU410ST in the dose-escalation phase of the study.

Regenerative Cell Therapies—First-in-class

NeoCart^® – Ocugen intends to initiate the Phase 3 trial contingent on the availability of adequate funding.
Vaccines Portfolio—First-in-class

Inhaled Mucosal Vaccine Platform – NIAID plans to submit an IND to initiate the OCU500 (COVID-19) Phase 1 clinical trial this year. Ocugen is continuing discussions with relevant government agencies as well as strategic partners regarding funding for the development of the OCU510 and OCU520 platforms.

Ophthalmic Biologic Product

OCU200 – Ocugen continues to work with the FDA to lift the clinical hold.

Second Quarter 2024 Financial Results

• Received $32.6 million net cash from underwritten public offering of common stock that closed on August 2, 2024.
• The Company’s cash, cash equivalents, and restricted cash totaled $16.0 million as of June 30, 2024, compared to $39.5 million as of December 31, 2023. The Company had 257.4 million shares of common stock outstanding as of June 30, 2024.
• Total operating expenses for the three months ended June 30, 2024 were $16.6 million and included research and development expenses of $8.9 million and general and administrative expenses of $7.7 million. This compares to total operating expenses for the three months ended June 30, 2023 of $24.0 million that included research and development expenses of $14.5 million and general and administrative expenses of $9.5 million.
• Ocugen reported a $0.04 net loss per common share for the three months ended June 30, 2024 compared to a $0.10 net loss per common share for the three months ended June 30, 2023.

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 also will be a question-and-answer session following the prepared remarks.

Attendees are invited to participate on the call or webcast:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 7453742
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 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, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; Ocugen’s financial condition and expected cash runway into the third quarter of 2025, 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com 

View full release HERE.

Research News and Market Data on OCGN

July 31, 2024

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MALVERN, Pa., July 31, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (Nasdaq: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that it has commenced an underwritten public offering (the “offering”) of its common stock. In addition, Ocugen expects to grant the underwriter a 30-day option to purchase up to an additional 15% of the aggregate number of shares of its common stock to be sold in the public offering at the public offering price, less the underwriting discounts and commissions. All of the shares of common stock in the offering are to be sold by Ocugen. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

Ocugen intends to use the net proceeds of the proposed offering for general corporate purposes, capital expenditures, working capital, and general and administrative expenses.

Titan Partners Group, a division of American Capital Partners, is acting as the sole book-running manager for the proposed offering.

The offering is being made by Ocugen pursuant to a shelf registration statement on Form S-3 (File No. 333-278774) previously filed with the Securities and Exchange Commission (the “SEC”) on April 18, 2024, which became effective on May 1, 2024. The securities may be offered only by means of a prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement and the accompanying base prospectus relating to and describing the proposed terms of the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Copies of the preliminary prospectus supplement and accompanying base prospectus relating to this offering may be obtained, when available, from Titan Partners Group LLC, a division of American Capital Partners, LLC, 4 World Trade Center, 29th Floor, New York, NY 10007, by phone at (929) 833-1246 or by email at prospectus@titanpartnersgrp.com.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such state or jurisdiction.

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.

Cautionary Statement Regarding 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. Such forward-looking statements within this press release include, without limitation, statements regarding market conditions, completion, timing, size and use of proceeds of the proposed offering and our intention to grant the underwriter a 30-day option to purchase additional shares. There can be no assurance that we will be able to complete the proposed public offering on the anticipated terms, or at all. 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, such as market and other conditions. These and other risks and uncertainties are more fully described in our periodic filings with the 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 applicable law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, changed circumstances or otherwise, after the date of this press release.

Ocugen Contact:	Titan Partners Contact:
Tiffany Hamilton	(929) 833-1246
Head of Communications	prospectus@titanpartnersgrp.com
Tiffany.Hamilton@Ocugen.com

Research News and Market Data on OCGN

July 29, 2024

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MALVERN, Pa., July 29, 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 and vaccines, today announced that it will host a conference call and live webcast to discuss the Company’s second quarter 2024 financial results and provide a business update at 8:30 a.m. ET on Thursday, August 8, 2024.

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: 7453742
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 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. 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