Tag: OCGN

Research News and Market Data on OCGN

April 26, 2023

MALVERN, Pa., April 26, 2023 (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 Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen will participate in a fireside chat at the H.C. Wainwright BioConnect Investor Conference on May 2, 2023, at the Nasdaq Market Site in Times Square, New York City.

Details regarding Ocugen’s fireside chat are as follows:

Event: H.C. Wainwright BioConnect Investor Conference

Date: Tuesday, May 2, 2023

Time: 11 – 11:30 a.m. EDT

Location: Nasdaq Market Site, New York City, Room 1 West (10^th Floor)

Dr. Musunuri will highlight Ocugen’s progress across its integrated pipeline including the Company’s positive preliminary safety and efficacy results from its ongoing OCU400 Phase 1/2 trial and modifier gene therapy platform, as well as provide an overview of anticipated short- and long-term milestones. Ocugen’s leadership team will be available for one-on-one meetings with registered investors throughout the event.

A live video webcast of the fireside chat will be available on the events page of 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 Twitter 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

• Favorable safety and tolerability profile related to OCU400 investigational product candidate
• Initial clinical data from low and medium dose cohorts indicates positive trend in Multi-luminance mobility testing and Best-Corrected Visual Acuity scores for OCU400 treated eyes
• 71.4% (5/7) of OCU400 treated eyes in low and medium dose cohorts experienced at least 1 Lux luminance level improvement in mobility test from baseline
• 66.7% (2/3) of OCU400 treated eyes in low dose cohorts at 9-month follow-up experienced at least 2 Lux luminance level improvement in mobility test from baseline
• Ocugen believes these preliminary data supports potential of modifier gene therapy platform in gene-agnostic treatment of complex and heterogenous inherited genetic diseases

MALVERN, Pa., April 14, 2023 (GLOBE NEWSWIRE) —  Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced positive preliminary data among retinitis pigmentosa (RP) participants treated in the first two cohorts of the Phase 1/2 trial to assess the safety and efficacy of OCU400 for RP associated with NR2E3 and Rhodopsin (RHO) mutations and Leber Congenital Amaurosis (LCA) with mutation(s) in the CEP290 gene. These preliminary results provide support that OCU400, Ocugen’s first-in-class therapeutic approach utilizing a proprietary modifier gene therapy platform, has the potential to be a gene-agnostic therapeutic for RP and LCA patients with inherited retinal degeneration.

“It is very gratifying to see these positive preliminary results from our novel modifier gene therapy approach,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “This is the first clinical validation of the platform where patient responses across various genetic mutations support that OCU400 has the potential to transform the lives of many patients who are struggling with debilitating blindness diseases.”

This Phase 1/2 trial is a multicenter, open-label, dose ranging study. A total of 18 RP subjects have been enrolled in this study—10 subjects in the dose escalation and 8 subjects in the expansion phase, respectively. The age of subjects enrolled to date ranges from 18-77 years across RHO and NR2E3 gene mutations. We further expanded this Phase1/2 trial to enroll LCA patients with CEP290 gene mutation and pediatric patients with NR2E3, RHO and CEP290 mutations.

In Cohort 1 and 2 of the clinical trial, 7 participants with severe vision impairment due to RP associated with RHO and NR2E3 gene mutations received a unilateral subretinal injection of either a low dose (1.66 x 10¹⁰ vg/mL) or medium dose (3.33 x 10¹⁰ vg/mL) OCU400, respectively. In the preliminary data analysis, 9-month follow-up data for 3 subjects [Cohort 1], and 6-month follow-up data for 4 subjects [N=1 from Cohort 1 and N=3 from Cohort 2] were evaluated.

Results showed a favorable safety profile and visual improvements after treatment with OCU400 as measured by multi-luminance mobility testing (MLMT) and best corrected visual acuity assessment (BCVA).

Key efficacy outcomes from 7 subjects demonstrated:

• 100% of treated eyes showed a stable or improved MLMT score trend;
• 5 of 7 (71.4%) OCU400 treated eyes demonstrated a 1 or more Lux level improvement in MLMT score compared to 28.6 % of untreated eyes;
• 66.7% (2 of 3) of OCU400 treated eyes in Cohort 1 with 9-month follow-up demonstrated a 2 or more Lux level improvement in MLMT score compared to none of the untreated eyes; and
• 3 of 7 (42.9%) OCU400 treated eyes demonstrated 8-11 letters of improvement in BCVA score compared to none of the untreated eyes.

“I was not expecting such substantial improvements in visual function among the trial participants I have been working with because of the advanced stage of their retinal disease,” said David Birch, PhD, Scientific Director, Retina Foundation of the Southwest, principal investigator of the study. “I am very pleased by the outcomes I have seen in my own clinic and am hopeful that OCU400 could provide a therapeutic solution for RP patients who are not only facing loss of vision, but also challenged with the psychological burden of losing their independence.”

“The early results from patients treated in the Phase 1/2 clinical trial are encouraging and support the paradigm-changing potential of modifier gene therapy technology to address unmet medical needs for patients with RP and LCA,” said Arun Upadhyay, PhD, Chief Scientific Officer and Head of Research, Development and Medical at Ocugen. “With this favorable safety profile and positive trend in efficacy signals, we are very eager to see longer-term data, and to potentially initiate Phase 3 trials in the U.S. and EU.”

Ocugen will continue to monitor long-term safety and efficacy data from the treated patients, and advance development of OCU400 to bring a potential treatment option to RP and LCA patients.

CanSinoBIO, Ocugen’s strategic partner, provided all CMC development and clinical supplies for the Phase 1/2 trial of OCU400.

A webcast and conference call will take place today at 8 a.m. ET:
Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 4898155
Webcast: Available on the events section of the Ocugen investor site

About Modifier Gene Therapy
Modifier gene therapy is designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, LCA, and Stargardt disease, as well as dry AMD. 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 OCU400 (RP, LCA), OCU410 (dry AMD), OCU410ST (Stargardt disease).

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 potential to improve retinal health and function in patients with inherited retinal diseases.

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 Twitter 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, including, but not limited to, statements regarding the development of OCU400 and the interpretation of preliminary clinical trial results. 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 risk that preliminary clinical data may not be indicative of final clinical data or data in later stage clinical trials. 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
IR@ocugen.com

Research News and Market Data on OCGN

March 31, 2023

PDF Version

MALVERN, Pa., March 31, 2023 (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 Company’s Chief Scientific Officer, Arun Upadhyay, PhD, will present at the World Vaccine Congress being held April 3 – 6 in Washington D.C.

“Current COVID-19 vaccines are limited by a lack of durability and inability to stop infection and transmission,” said Dr. Upadhyay. “Inhaled vaccines have the potential to generate rapid mucosal immunity in respiratory pathways, limiting infection and transmission. I look forward to discussing Ocugen’s inhaled vaccine technology—to address COVID-19 and flu—during the World Vaccine Congress.”

Ocugen is currently developing a novel mucosal vaccine platform that includes OCU500, a bivalent COVID-19 inhaled vaccine; OCU510, a seasonal quadrivalent flu inhaled vaccine; and OCU520, a combination quadrivalent seasonal flu and bivalent COVID-19 inhaled vaccine. The OCU500 series grants Ocugen a distinct product candidate profile status that could significantly impact major global health obstacles and maximize the Company’s opportunity to serve broader patient markets. For the 2022 to 2023 flu season, in the United States alone, more than 50% of the population above six months of age received a seasonal flu shot, representing a market size of more than 170 million doses.

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

Presentation Title: “A next generation inhalation-based mucosal vaccine for COVID-19 and Flu: Potential approach to reduce infection and transmission”
Date: Wednesday, April 5, 2023
Time: 5:40 p.m. ET
Location: Walter E. Washington Convention Center, Washington D.C. – Level 2, Room 203AB

Roundtable Discussion Title: “Going beyond existing limitations – Why mucosal vaccines are essential for respiratory diseases”
Date: Tuesday, April 4, 2023
Time: 11:40 – 12:20 p.m. ET (rotation 1); 12:30 – 1:10 p.m. ET (rotation 2)
Location: Walter E. Washington Convention Center, Washington D.C. – Room 203B

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 Twitter 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
IR@ocugen.com

Research News and Markert Data on OCGN

• U.S. Food & Drug Administration (FDA) approves enrolling pediatric patients in the ongoing OCU400 Phase 1/2 trial who have: 1) RP associated with NR2E3 and RHO mutations and 2) LCA associated with CEP290 gene mutations
  
  
• Ocugen has completed enrollment of adult RP patients with NR2E3 and RHO mutations in the Phase 1/2 trial and expanded enrollment in LCA patients with CEP290 mutations

MALVERN, Pa., March 27, 2023 (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 FDA approved enrolling pediatric patients in the ongoing OCU400 Phase 1/2 trial.

“This approval moves us one step closer in our efforts to bring OCU400, a novel gene-agnostic modifier gene therapy, to market as a potential life-changing treatment for children afflicted with inherited retinal diseases, such as RP and LCA,” noted Arun Upadhyay, PhD, Ocugen’s Chief Scientific Officer. “This approval further demonstrates the consistent, positive, and timely progress we are making with the Phase 1/2 trial in adult patients. Since a significant number of individuals in the pediatric age group are diagnosed with RP and LCA, it is very important for us to cover this age group in our clinical trials.”

Enrollment of adult RP patients in the Phase 1/2 trial is complete—per protocol—and enrollment continues among patients with LCA. The Company plans to initiate the Phase 3 trial near the end of 2023.

Unlike single-gene replacement therapies, which only target one genetic mutation, Ocugen believes that its modifier gene therapy platform, through its use of Nuclear Hormone Receptors (NHRs), represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and potentially address complex diseases that are caused by imbalances in multiple gene-networks. While single-gene replacement therapies have shown tremendous promise in rare retinal diseases, they are highly specific and cannot improve a multitude of disease-causing genetic defects. For example, RP and LCA are associated with mutations in more than 100 and in more than 25 genes, respectively. Ocugen is the only company with a gene-agnostic modifier platform that aims to alter this single-gene therapy paradigm through the introduction of a functional gene to modify the expression of multiple genes and gene-networks. We believe that patient prevalence in the United States alone would provide significant long-term value, with RP and LCA affecting 110,000 and 15,000 people, respectively.

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 diverse 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 inherited retinal diseases.

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 Twitter 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
IR@ocugen.com 

Research News and Market Data on OCGN

February 28, 2023

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

• Completed retinitis pigmentosa patient enrollment in OCU400 Phase 1/2 clinical trial
• Continued progress for programs targeting eye diseases with the submission of an IND application for OCU200
• Expanded portfolio now includes inhaled vaccines for COVID-19, seasonal flu, and a combination COVID-19+seasonal flu vaccine

MALVERN, Pa., Feb. 28, 2023 (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 fourth quarter and full year 2022 financial results along with a general business update.

“We continue to grow and advance as a diversified biotechnology organization as reflected in our accomplishments of 2022,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “Our pipeline has been expanded to appropriately address the current challenges and gaps in the fight against COVID-19, application of OCU410 to address Stargardt (a rare eye disease), and our novel approach to address dry age-related macular degeneration (dAMD)—a disease affecting vision in over 266 million people worldwide.

“Following FDA concurrence in the fourth quarter of 2022 on a confirmatory Phase 3 clinical trial design for NeoCart^®, we are developing internal capabilities to move our regenerative medicine asset, NeoCart^®, into the clinic next year.”

“The FDA has granted expanded orphan drug designations to OCU400 for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA),” said Dr. Musunuri. “These broad, gene-agnostic designations are encouraging at this stage in the development of OCU400.”

“During our first decade, we have built a strong foundation for addressing the diseases and conditions we aim to treat. We delivered on our promise to file an OCU200 IND in the first quarter of 2023 and look forward to delivering on important milestones in 2023, especially regarding preliminary efficacy data for gene therapy product OCU400, as we progress toward realizing our long-term vision to address unmet medical needs through courageous innovation,” Dr. Musunuri concluded.

Business Updates

Ophthalmic Gene Therapies

• OCU400 – Established the high dose as the maximum tolerable dose, completed retinitis pigmentosa patient enrollment, and continuing to enroll patients with LCA to receive the high dose. Ocugen intends to initiate a Phase 3 clinical trial near the end of 2023.
• OCU410 and OCU410ST – Executing IND-enabling studies and intend to submit IND applications in the second quarter of 2023 to initiate Phase 1/2 clinical trials in dry AMD (geographic atrophy) and Stargardt disease.

Ophthalmic Biologic Product

• OCU200 – Submitted an IND application on February 27, 2023 to initiate a Phase 1 clinical trial targeting diabetic macular edema.

Regenerative Cell Therapies

• NeoCart^® – Received concurrence from the FDA on the confirmatory Phase 3 clinical trial design. Ocugen intends to initiate the Phase 3 clinical trial in the first half of 2024. Ocugen is renovating its facility to accommodate cGMP manufacturing of NeoCart^® for clinical trials and beyond.

Vaccines Portfolio

• OCU500 Series – Developing a novel mucosal vaccine platform which includes OCU500, a bivalent COVID-19 inhaled vaccine; OCU510, a seasonal quadrivalent flu inhaled vaccine; and OCU520 a combination quadrivalent seasonal flu and bivalent COVID-19 inhaled vaccine.
  
  
• COVAXIN™ (BBV152) – Completed enrollment in Phase 2/3 immuno-bridging and broadening clinical trial in fourth quarter 2022.

Financial Results

• Fourth quarter — Research and development expenses for the three months ended December 31, 2022, were $17.2 million compared to $7.1 million for the three months ended December 31, 2021. General and administrative expenses for the three months ended December 31, 2022, were $6.9 million compared to $7.5 million for the three months ended December 31, 2021. Ocugen reported a $0.10 net loss per common share for the three months ended December 31, 2022, compared to a $0.07 net loss per common share for the three months ended December 31, 2021.
• Full year — Research and development expenses for the year ended December 31, 2022, were $49.8 million compared to $35.1 million for the year ended December 31, 2021. General and administrative expenses for the year ended December 31, 2022, were $35.1 million compared to $22.9 million for the year ended December 31, 2021. Ocugen reported a $0.38 net loss per common share for the year ended December 31, 2022, compared to a $0.30 net loss per common share for the year ended December 31, 2021.
• Ocugen’s cash, cash equivalents, restricted cash, and investments totaled $90.9 million as of December 31, 2022, compared to $95.1 million as of December 31, 2021. The Company estimates that its current cash, cash equivalents, and investments will enable it to fund its operations into the first quarter of 2024. The Company had 221.6 million shares of common stock outstanding as of December 31, 2022.

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: 8912239
Webcast: Available on Ocugen’s 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.

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
IR@ocugen.com

Research News and Market Data on OCGN

February 27, 2023

MALVERN, Pa., Feb. 27, 2023 (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 has submitted an Investigational New Drug application (IND) with the U.S. Food and Drug Administration (FDA) to initiate a Phase 1 clinical trial of OCU200, a fusion protein with a distinct mechanism of action (MOA), for the treatment of diabetic macular edema (DME). This regulatory milestone fulfills the Company’s commitment to file the IND for OCU200 within the first quarter of 2023.

“Today’s achievement is an important step towards fulfilling our mission to bring novel therapeutics to address limitations of the current standard of care or unmet medical needs in hard-to-treat blindness diseases,” said Dr. Arun Upadhyay, Chief Scientific Officer at Ocugen. “We are encouraged by the potential for OCU200 to provide a new treatment option for the significant percentage of people living with DME, including non-responders to the current standard of care.”

The planned Phase 1 clinical study will assess the unilateral intravitreal administration of OCU200 alone or in combination with an approved anti-VEGF therapy in participants with DME. This is a multicenter, open-label, dose-ranging study with 3 cohorts in the dose-escalation portion of the study and 1 cohort in the combination therapy portion of the study.

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 745,000 people in the United States are affected with DME, and this number is expected to further increase as the number of people with diabetes increases.

The Company intends to pursue additional indications for OCU200 to potentially treat diabetic retinopathy and wet age-related macular degeneration, which combined affect nearly 9.0 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 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 Twitter and LinkedIn.

About OCU200
OCU200 is a novel fusion protein consisting of human transferrin linked to human tumstatin. It exerts anti-proliferative, anti-inflammatory, and anti-oxidative effects by selective targeting to the retinal and choroidal tissues. OCU200 potentially showcases better bioavailability and tissue penetrance than tumstatin alone due to transferrin and provides distinct MOA binding through αVβ3 integrin pathways that can potentially reduce the number of injections for patients.

OCU200 can potentially be used for the treatment of diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration. These diseases, combined, account for approximately 10 million cases in the U.S.

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
IR@ocugen.com