Tag: Ocugen

Research News and Market Data on OCGN

May 31, 2024

• Established Medium Dose as Safe and Tolerable Dose in Current OCU410 Clinical Trial
• DSMB Recommends Continuing with High-Dose Cohort Dosing with Concurrent Phase 2 Dosing

MALVERN, Pa., May 31, 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 a positive outcome of the Data and Safety Monitoring Board (DSMB) Review for its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV5-hRORA)—a modifier gene therapy candidate 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. and Europe combined.

Six subjects with GA were dosed in the Phase 1/2 clinical trial to date—three subjects were dosed with the low dose and three subjects were dosed with the medium dose. An additional three patients will be dosed with the high dose of OCU410 in the dose-escalation phase.

“The DSMB has recommended to proceed with dosing subsequent GA subjects with the high dose of OCU410 in the dose-expansion phase of the study and concurrently initiate Phase 2 dosing,” said Dr. Peter Chang, MD, FACS, DSMB Chair for the OCU410 clinical trial. “No serious adverse events (SAEs) related to OCU410 have been reported to date in both low- and medium-dose cohorts. I believe that this marks a critical next step towards determining the maximum tolerated dose for OCU410 and is an important milestone for its clinical development.”

“We are delighted to report a second positive DSMB recommendation for the treatment of GA, which significantly builds on the favorable safety and tolerability profile exhibited by OCU410,” said Huma Qamar, MD, MPH, CMI, Chief Medical Officer of Ocugen. “We are very enthusiastic about the potential of OCU410 as a potential one-time treatment for GA with a single sub-retinal injection. The currently approved treatments for GA target only the complement pathway and require approximately 6-12 intravitreal injections annually. OCU410 addresses multiple pathways causing dAMD, including complement, lipid metabolism, inflammation, and oxidative stress, providing long-term benefit to patients.”

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

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

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

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

Research News and Market Data on OCGN

May 15, 2024

MALVERN, Pa., May 15, 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 second cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease as a one-time treatment for life.

“The completion of dosing for Cohort 2 participants signifies an important clinical milestone for our pioneering modifier gene therapy,” said Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen. “We are encouraged by the ongoing positive safety and tolerability profile demonstrated by OCU410ST, enabling us to consider higher doses in patients as we progress with the dose-escalation study. We look forward to sharing preliminary safety and efficacy data from Phase 1 of the clinical trial.”

Six patients with Stargardt disease have been dosed in the Phase 1/2 clinical trial to date. An additional three patients will be dosed with the high dose (Cohort 3) of OCU410ST in the dose-escalation phase.

“There remains a great unmet medical need for patients with Stargardt disease, which is the most common inherited retinal disease affecting the center of the vision and does not have any FDA-approved treatment options. OCU410ST is a novel modifier gene therapy that provides hope to these patients,” said Benjamin Bakall, MD, PhD, Director of Clinical Research at Associated Retina Consultants and Clinical Assistant Professor at University of Arizona, College of Medicine – Phoenix. “I am excited that we completed dosing of the last patient in Cohort 2, who received medium dose of this novel therapeutic leveraging a gene-agnostic approach, at Associated Retina Consultants (ARC) in Phoenix, AZ with the surgical team led by Dr. Mark Kwong, Medical Director of ARC.”

A Data and Safety Monitoring Board meeting will convene next month to review the 4-week safety data of the medium dose cohort before proceeding with Cohort 3 (high dose), which is the final dose in the Phase 1 dose-escalation study.

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)]. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which adult and pediatric subjects will be randomized in a 1:1:1 ratio to either one of two OCU410ST dose groups or to an untreated group.

Ocugen is committed to finding solutions for people with inherited retinal disease for whom no effective treatment options exist. While an orphan disease, Stargardt affects approximately 100,000 people in the United States and Europe combined.

The Company expects to provide a clinical trial update for OCU410ST in the third quarter of 2024.

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

May 14, 2024

PDF Version

CONFERENCE CALL AND WEBCAST TODAY AT 8:30 A.M. ET

• All three first-in-class modifier gene therapy product candidates currently in the clinic with OCU400 Phase 3 in progress
• OCU400 on track to meet 2026 Biologics License Application (BLA) and Market Authorization Application (MAA) approval targets

MALVERN, Pa., May 14, 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 reported first quarter 2024 financial results along with a general business update.

“We’ve experienced several important clinical and regulatory milestones since the beginning of 2024 that we believe are leading the way to a new treatment paradigm for patients with blindness diseases,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “It’s very encouraging to have FDA clearance and EMA support for the Phase 3 clinical trial of our lead modifier gene therapy candidate that offers a potential one-time treatment for life.”

With FDA clearance to begin the Phase 3 liMeliGhT clinical trial, OCU400 becomes the first gene therapy to progress to late-stage trials with a broad retinitis pigmentosa (RP) indication. Until now, there has been only one marketed product to treat one of the 100 gene mutations associated with RP. The gene-agnostic mechanism of action for OCU400 provides hope for a much larger RP patient population. In the U.S. and Europe combined, RP affects nearly 300,000 people.

Ocugen expects to begin dosing patients in the Phase 3 liMeliGhT clinical trial in the second quarter of 2024. The Phase 3 trial will have a sample size of 150 participants—one arm of 75 participants with the RHO gene mutation and the other arm with 75 participants that are gene-agnostic. Luminance Dependent Navigation Assessment (LDNA) is the primary endpoint for the study and focuses on the proportion of responders, in treated and untreated groups, achieving an improvement of at least 2 Lux levels from baseline in the study eyes.

Leveraging a dual-track strategy, the Company plans to expand the Phase 3 OCU400 clinical trial in the second half of 2024 to include patients with Leber congenital amaurosis (LCA), contingent on favorable results from the Phase 1/2 study.

Modifier gene therapy has the potential to treat inherited retinal diseases as well as multifactorial blindness diseases affecting millions of patients. Leveraging the nuclear receptor gene RAR-related orphan receptor A (RORA), OCU410 is designed to regulate all four pathways involved with dry age-related macular degeneration (dAMD)—including lipid metabolism, inflammation, oxidative stress, and membrane attack complex (complement). Ocugen is developing OCU410 as a one-time gene therapy for the treatment of geographic atrophy (GA), an advanced stage of dAMD, affecting 2-3 million people in the U.S. and Europe combined. OCU410ST is being developed as a one-time gene therapy for the treatment of Stargardt disease, affecting approximately 100,000 people in the U.S. and Europe combined.

In April, dosing was completed in the second cohort (medium dose) of the Phase 1/2 ArMaDa clinical trial for OCU410. Dosing in the first cohort (low dose) of the Phase 1/2 GARDian trial for OCU410ST was completed earlier in the first quarter and in April 2024, the Data Safety and Monitoring Board approved the continuation to cohort 2 (medium dose).

“Our efforts in the first quarter of the year evidence the importance of our gene therapy programs and the need to operate the business to ensure their success,” said Dr. Musunuri. “We are opportunistic about Ocugen’s cell therapy and vaccine platforms, knowing that these technologies have great therapeutic and financial potential and are pursuing partnerships to support our entire pipeline.”

Ophthalmic Gene Therapies —First-in-class

• OCU400—Received FDA clearance of IND amendment to initiate OCU400 Phase 3 liMeliGhT clinical trial in RP. EMA provided acceptability of the U.S.-based trial for submission of Marketing Authorization Application (MAA). Currently, the multi-center Phase 3 clinical trial is in progress.
• OCU410 – Currently in Phase 1/2 stage of clinical development with active patient enrollment. Dosing is complete in the second cohort (medium dose) in the dose-escalation phase of the study. Once the third cohort (high dose) is complete, the Company will move into the Phase 2 clinical trial—the expansion phase—in the third quarter of 2024.
• OCU410ST – Currently in Phase 1/2 stage of clinical development with active patient enrollment. Dosing is complete for cohort 1 (low dose). Initiated enrollment in cohort 2 (medium dose) in the dose-escalation phase of the trial.

Regenerative Cell Therapies—First-in-class

• NeoCart® – Completed renovating an existing facility into a current Good Manufacturing Practice (“GMP”) facility in accordance with the FDA’s regulations. Intend to initiate the Phase 3 trial, contingent on adequate availability of funding.

Vaccines Portfolio—First-in-class

• Mucosal Vaccine Platform – NIAID is collaborating with Ocugen on clinical development of OCU500. Planning to submit IND by mid-2024 to initiate Phase 1 clinical trial.

Biologics

• OCU200—Continue to work with FDA to address comments to lift the clinical hold.

First Quarter 2024 Financial Results

• The Company’s cash and cash equivalents totaled $26.4 million as of March 31, 2024, compared to $39.5 million as of December 31, 2023. The Company had 257.3 million shares of common stock outstanding as of March 31, 2024.
• Total operating expenses for the three months ended March 31, 2024 were $13.2 million and included research and development expenses of $6.8 million and general and administrative expenses of $6.4 million. This compares to total operating expenses for the three months ended March 31, 2023 of $18.5 million that included research and development expenses of $10.2 million and general and administrative expenses of $8.3 million.
• Ocugen reported a $0.05 net loss per common share for the three months ended March 31, 2024 compared to a $0.08 net loss per common share for the three months ended March 31, 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 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: 8699924
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, 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, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

Contact:
Tiffany Hamilton
Head of Communications
IR@ocugen.com

View full release here.

Research News and Market Data on OCGN

May 2, 2024

MALVERN, Pa., May 02, 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, Chief Scientific Officer, Head of Research & Development, Ocugen will speak at the Retina World Congress (RWC) being held May 9-12, 2024 in Ft. Lauderdale, FL.

“This is a terrific opportunity to share the most recent developments in modifier gene therapy with my peers and learn more about their discoveries to fight retinal disease,” said Dr. Upadhyay. “Opportunities like these are critical as we work together to address unmet medical needs for patients with blindness diseases.”

RWC is one of the largest retina meetings in the world and is leading the advancement of science in retina. This annual meeting brings together retina specialists from every continent to achieve a global scientific and clinical exchange in retinal health. 

Details on Dr. Upadhyay’s presentation are as follows:

RWC Unplugged Panel Session: Gene Therapy, Pharmacotherapy and Optogenetics for IRD
Date: Thursday, May 9, 2024
Time: 9:05 – 9:35 a.m. (ET)
Location: Marriott Harbor Beach Resort

Additionally, Ocugen’s abstract, “Safety and Efficacy Results from a Phase 1/2 Clinical Trial of OCU400 Modifier Gene Therapy for Treatment of Retinitis Pigmentosa,” has been accepted as an e-poster/video presentation and will be showcased in a video display during the conference.

The OCU400 Phase 3 liMeliGhT clinical trial is currently underway and on track to meet the Company’s 2026 BLA and MAA approval targets. OCU400 is the first gene therapy program to enter Phase 3 with a broad retinitis pigmentosa (RP) indication.

About OCU400
OCU400 is the Company’s gene-agnostic modifier gene therapy product based on nuclear hormone receptor (NHR) gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with RP. Between the U.S. and EU, nearly 300,000 people are affected by RP.

About Modifier Gene Therapy
Modifier gene therapy is designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, Leber congenital amaurosis 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 between the U.S. and EU and Stargardt disease affects nearly 100,000 people between the U.S. and EU.

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, 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

April 29, 2024

MALVERN, Pa., April 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 Benjamin Bakall, MD, PhD, Director of Clinical Research at Associated Retina Consultants and Clinical Assistant Professor at the University of Arizona, College of Medicine—Phoenix will present data from the OCU400 Phase 1/2 clinical trial at the Retinal Cell and Gene Therapy Innovation Summit being held May 3, 2024 in Seattle, WA.

“I look forward to sharing more about my clinical experience with this novel modifier gene therapy with my peers and industry leadership during the summit,” said Dr. Bakall. “There remains a significant unmet need for patients with retinitis pigmentosa (RP) and I believe that this approach can offer a new therapeutic option to address the disease itself.”

The Retinal Cell and Gene Therapy Innovation Summit 2024 is jointly organized by the Foundation Fighting Blindness and the Oregon Health and Science University Casey Eye Institute. The Summit brings together representatives from the biotech and pharma industries, along with members of the medical and research communities, to discuss rapidly emerging ocular gene and cell therapies and strategize how to move the most advanced retinal disease therapies toward clinical utility.

Details on Dr. Bakall’s presentation are as follows:

Presentation Title: “Nuclear Hormone Receptor-Based Gene Modifier Therapy: Safety and Efficacy from Phase 1/2 Clinical Trials for Retinitis Pigmentosa”
Date: Friday, May 3, 2024
Time: 10:50 – 11:05 a.m. (PT)
Location: Hyatt Regency Seattle

The OCU400 Phase 3 liMeliGhT clinical trial is currently underway and on track to meet the Company’s 2026 BLA and MAA approval targets. Between the U.S. and EU, nearly 300,000 people are affected by RP.

About OCU400
OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with RP.

About RP
RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, leading to vision loss and blindness. Currently, 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 retina degeneration. Therefore, the development of gene-specific replacement therapy is highly challenging, especially when multiple and unknown genes are involved. Thus, novel therapeutic approaches targeting 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, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

Research News and Market Data on OCGN

April 12, 2024

MALVERN, Pa., April 12, 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 Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder will participate in a fireside chat during the Emerging Growth Virtual Healthcare Equity Conference, presented by Noble Capital Markets, taking place from April 17 – 18, 2024.

Details of the fireside chat are as follows:

Date: Wednesday, April 17, 2024

Time: 10 a.m. ET

Location: Presentation Track 1

Webcast: Available in the Events section of Ocugen website

During the fireside chat and one-on-one sessions, Dr. Musunuri will showcase the Company’s recent business and clinical development progress including FDA clearance to initiate a Phase 3 clinical trial for OCU400—a modifier gene therapy for patients battling retinitis pigmentosa (RP). OCU400 is the first gene therapy to enter a late-stage trial with a broad RP indication.

A live webcast of the fireside chat will be available in the Events section of the Company’s website and as part of a complete catalog of presentations available on Channelchek—the investor portal created by Noble. The webcast will be archived on the Company’s website for 90 days following the event. 

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.

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