Release – Ocugen, Inc. Announces First Patient Dosed In Phase 1/2 Clinical Trial Evaluating The Safety And Efficacy Of OCU410—Modifier Gene Therapy—For Geographic Atrophy Secondary To Dry Age-Related Macular Degeneration

Research News and Market Data on OCGN

December 13, 2023

MALVERN, Pa., Dec. 13, 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 first patient has been dosed in the ArMaDa Phase 1/2 clinical trial of OCU410 (AAV-RORA), a modifier gene therapy product candidate being developed for dry AMD (dAMD).

“OCU410, our first-in-class modifier gene therapy for dAMD, addresses gaps among other therapies available and in development for dAMD as a potential one-time treatment for life,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “We are very pleased to continue advancing our ophthalmic gene therapy pipeline, which remains the Company’s primary focus.”

This Phase 1/2 trial will assess the safety and efficacy of OCU410 for geographic atrophy (GA) secondary to dAMD and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study. Phase 2 is a randomized expansion phase in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 dose groups or to an untreated control group.

OCU410 is a potential curative therapy with a single sub-retinal injection that targets multiple pathways causing dAMD, including lipid metabolism, inflammation, oxidative stress, and complement activation. Currently, the other therapeutic options available target only complement activation and require approximately 6-12 intravitreal injections annually.

“Breaking new ground in the pursuit of vision restoration, our pioneering modifier gene therapy candidate, OCU410, achieves another major milestone by dosing a GA patient in a Phase 1/2 clinical trial,” said Arun Upadhyay, PhD, Chief Scientific Officer, Head of R&D at Ocugen. “OCU410 offers hope for those battling GA that are faced with limited treatment options and the real prospect of ultimately losing their vision.”

“There remains a great unmet need for novel durable and effective treatments for GA, which remains one of the most common causes of vision loss globally,” said Benjamin Bakall, MD, PhD, director of clinical research at Associated Retina Consultants (ARC) and clinical assistant professor at University of Arizona, College of Medicine – Phoenix. “I am excited that we performed the first surgery with this novel therapeutic approach—designed to restore homeostasis and slow disease progression following a single treatment—at ARC in Phoenix, AZ, with the surgical team led by Dr. Mark Kwong, medical director of ARC.”

The first surgery was successful in delivering the new gene underneath the retina; the light sensitive nerve tissue lining the inside of the eye.

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.

GA, an advanced form of dry age-related macular degeneration, affects approximately 1 million people in the United States alone.
About OCU410
OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR 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 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. Currently, the other therapeutic options available target only complement activation and require approximately 6-12 intravitreal injections annually.

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

About Associated Retina Consultants
Associated Retina Consultants is the largest independent retina practice in the state of Arizona, taking part in groundbreaking clinical trials to bring new treatments for the benefit of patients with diseases affecting the retina. In addition to collaborating with Ocugen on the OCU410 clinical trial, in October 2023, Associated Retina Consultants—with a surgical team led by Dr. Mark Kwong—performed gene therapy with OCU400 on a pediatric CEP290 patient. This was the first retinal gene therapy of its kind performed on a child in Arizona.

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

Release – Ocugen CEO To Present at Noblecon19 – Noble Capital Markets’ Nineteenth Annual Emerging Growth Equity Conference

Research News and Market Data on OCGN

November 29, 2023

MALVERN, Pa., Nov. 29, 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, CEO, and Co-Founder will present at NobleCon19 – Noble Capital Markets’ Nineteenth Annual Emerging Growth Equity Conference at Florida Atlantic University, Executive Education Complex, in Boca Raton, FL—taking place December 3 – 5, 2023.

Details regarding the presentation and moderated Q&A are as follows:

Date: Monday, December 4, 2023

Location: Presentation Room 1

Time: 2 p.m. ET  

Following the presentation, there will be an opportunity to meet with the Company’s management team in a breakout session at 2:30 p.m. ET—Breakout Table 1 (located directly across from the presentation room). Also, Ocugen will conduct one-on-one meetings with registered investors to showcase the Company’s business and clinical development strategy across its unique gene therapy, cell therapy, and vaccine platforms, and highlight recent corporate and pipeline-related achievements.

A webcast of the presentation will be available the following day in the Events section of the Company’s website and as part of a complete catalog of presentations available at Noble Capital Markets’ Conference website and on Channelchek—the investor portal created by Noble. The webcast will be archived on the Company’s website, the NobleCon website, and on Channelchek.com 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 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

Release – Ocugen, Inc. Announces First Patient Dosed in Phase 1/2 Clinical Trial Evaluating The Safety And Efficacy Of OCU410ST—Modifier Gene Therapy—For Stargardt Disease

Research News and Market Data on OCGN

November 10, 2023

MALVERN, Pa., Nov. 10, 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 first patient has been dosed in its Phase 1/2 GARDian clinical trial for OCU410ST (AAV5-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease, a rare genetically inherited disease that directly affects the retina, often resulting in slow progressive vision loss in children and adults.

“There is a significant unmet medical need for the approximate 35,000 patients in the U.S. living with Stargardt disease,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “It is critical to our mission to develop innovative treatments for inherited retinal diseases and this milestone is an important step in bringing our novel modifier gene therapies to people who desperately need them.”

This Phase 1/2 trial will assess the safety 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. 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 control group.

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.

“It is important and exciting to pursue novel therapies for untreatable blinding diseases,” said Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas. “Initiation of this trial program investigating a new mechanism of action for the treatment of Stargardt disease is inspiring and brings hope to patients and families.”

About Stargardt Disease

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

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

About Ocugen, Inc. 
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on 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  

Ocugen (OCGN) – 3Q23 Reported With Several Products On Schedule To Reach Clinical Milestones In 2024


Friday, November 10, 2023

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

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

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

Pipeline Is Set For 2024. Ocugen reported 3Q23 loss of $14.2 million or $(0.06) per share, compared with our estimated loss of $18.2 million or $(0.07) per share. Cash balance at the end of the quarter was $53.5 million. During 3Q23, data was presented for OCU400, INDs were approved for OCU410 and OCU410ST, and OCU500 was selected by the NIAID-NIH (National Allergy and Infectious Disease Institute of the National Institutes of Health) to begin clinical testing. NeoCart is also expected to begin a Phase 3 trial in 2H24. We see these pipeline events as important milestones for the company. 

OCU400 is on schedule to finalize the Phase 3 trial design. The Phase 1/2 trial has completed dosing of the RP patients and three LCA patients. The treatment protocols for the Phase 3 trial are expected to be finalized by year-end. Following FDA acceptance, the Phase 3 trial is expected to begin in early 2024 as expected. The final LCA patient data could allow FDA approval to include LCA patients in 2H24.


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This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Release – Ocugen Provides Business Update With Third Quarter 2023 Financial Results

Research News and Market Data on OCGN

November 9, 2023

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

  • OCU400 demonstrated favorable safety and tolerability profile in retinitis pigmentosa (RP) and Leber congenial amaurosis (LCA) subjects
  • Completed dosing of three LCA patients including a pediatric patient
  • OCU400 Phase 1/2 study results suggest stabilization or improvement of Best-Corrected Visual Acuity (BCVA) or Multi-Luminance Mobility Testing (MLMT) or Low-Luminance Visual Acuity (LLVA) in treated eyes of 83% (10/12) subjects
  • Stabilization or improvement in MLMT scores from baseline in 86% (6/7) of RHO subjects demonstrated gene-agnostic property of OCU400 modifier gene therapy and its potential benefits in broader RP and LCA subjects
  • Ocugen’s inhaled mucosal vaccine candidate for COVID-19—OCU500—selected by National Institutes of Health (NIH)/National Institute of Allergy and Infectious Diseases’ (NIAID) Project NextGen for inclusion in clinical trials

MALVERN, Pa., Nov. 09, 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 third quarter 2023 financial results along with a general business update.

“Ocugen has made significant pipeline progress in the third quarter,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “In particular, based on the OCU400 data presented in September, I am as enthusiastic as ever regarding the potential for our modifier gene therapy approach to make an important difference in the lives of people living with blindness diseases. OCU400—our lead candidate—has the potential to address multiple genetic mutations with a single product compared to traditional gene therapies that target one gene at a time.”

This clinical efficacy update provided in September 2023 included data for 12 subjects who have completed a minimum of 6 months follow up. 83%, 83%, and 75% of subjects demonstrated stabilization or improvements in OCU400 treated eyes on BCVA, LLVA, and MLMT scores, respectively from baseline. 86% of subjects with the RHO gene mutation experienced either stabilization or increase in MLMT scores from baseline, including a subset of 29% that demonstrated a three Lux luminance level improvementPreservation of remaining vision, slowing disease progression, or improving the vision can significantly impact patients’ quality of life.

“The improvements in BCVA, LLVA and MLMT in RHO patients—a disease affecting more than 10,000 people in the U.S. alone—supports the gene-agnostic mechanism of action for OCU400,” said Dr. Musunuri.

In October 2023, OCU500, was selected by the NIAID Project NextGen for inclusion in clinical trials. OCU500 will be tested via two different mucosal routes, inhalation into the lungs and as a nasal spray. Currently used injected vaccines, including mRNA vaccines, are not effective in preventing infection and spread although effective against severe infection. Generating mucosal immunity in nasal and respiratory airways could help block the infection at its origin, thus limiting spread.

“NIAID support for our mucosal vaccine platform is the result of many months of hard work and dedicated effort by our Ocugen team and is a first step in potentially expanding the platform to flu and other respiratory viral diseases and infections,” said Dr. Musunuri. “Additionally, this funding makes it possible to focus the majority of Ocugen’s R&D and clinical resources on our first-in-class gene and cell therapies.”

As Ocugen prepares to start Phase 3 for OCU400, begin dosing patients for OCU410 and OCU410ST, and initiate the Phase 1 trial for OCU500 in collaboration with NIAID, the company is making meaningful progress toward its long-term strategy and delivering on each of its scientific platforms in the near-term.

Ophthalmic Gene Therapies—First-in-class

  • OCU400 – Completed dosing adult RP patients in the dose-escalation and dose-expansion portions of the trial; completed dosing three LCA patients including a pediatric patient. Phase 3 clinical trial for the treatment of RP to be initiated in early 2024 following FDA concurrence on study design. Subsequently, the Company is expecting to expand the OCU400 Phase 3 clinical trial for LCA patients in the second half of 2024 based on Phase 1/2 study results in LCA patients and alignment with the FDA.
  • OCU410 and OCU410ST – IND applications to initiate Phase 1/2 trials for both OCU410 and OCU410ST were cleared by the FDA and the Company intends to dose patients in Phase 1/2 trials by the end of 2023.

Ophthalmic Biologic Product

  • OCU200 – Continuing to work on the Company’s response to the FDA regarding the IND application and expect to initiate the Phase 1 clinical trial in the first half of 2024, contingent on the lift of the FDA hold and adequate availability of funding.

Regenerative Cell Therapies—First-in-class

  • NeoCart® – Ocugen’s autologous regenerative cell therapy (using patients’ own cartilage cells) remains on track to begin its Phase 3 clinical trial in the second half of 2024. A cGMP facility for manufacturing NeoCart is expected to be completed at the end of 2023 and qualification is expected in the first half of 2024.  

Vaccines Portfolio—First-in-class

  • Mucosal Vaccine Platform – The Company is collaborating with NIAID to initiate clinical trials of OCU500 in early 2024.

Third Quarter 2023 Financial Results

  • The Company’s cash, cash equivalents, and investments totaled $53.5 million as of September 30, 2023, compared to $90.9 million as of December 31, 2022. The Company had 256.5 million shares of common stock outstanding as of September 30, 2023.
  • Total operating expenses for the three months ended September 30, 2023 were $15.4 million and included research and development expenses of $6.3 million and general and administrative expenses of $9.1 million. This compares to total operating expenses for the three months ended September 30, 2022 of $23.1 million that included research and development expenses of $15.6 million and general and administrative expenses of $7.5 million.
  • Ocugen reported a $0.06 net loss per common share for the three months ended September 30, 2023 compared to a $0.10 net loss per common share for the three months ended September 30, 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: 1787631
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 include, but are not limited to, statements regarding the Company’s clinical development activities and related anticipated timelines; strategy, business plans and objectives for its clinical stage programs; plans and timelines for the preclinical and clinical development of its 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; and Ocugen’s financial condition. 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, as well as discussions of potential risks, uncertainties, and other important factors in Ocugen’s subsequent filings with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. 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 

Release – Ocugen to Host Conference Call on Thursday, November 9 at 8:30 A.M. ET To Discuss Business Updates and Third Quarter 2023 Financial Results

Research News and Market Data on OCGN

November 2, 2023

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MALVERN, Pa., Nov. 02, 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 will host a conference call and live webcast to discuss the Company’s third quarter 2023 financial results and provide a business update at 8:30 a.m. ET on Thursday, November 9, 2023.

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: 1787631
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

Ocugen (OCGN) – OCU500 Nasal COVID-19 Vaccine Chosen By NIH To Start Clinical Trials

Wednesday, October 11, 2023

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

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

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

Good News For A Program Where No News Was Expected. Ocugen announced that its OCU500 nasal vaccine for COVID-19 has been chosen to start Phase 1 clinical trials run by the National Institute for Allergy and Infectious Disease, a division of the National Institutes of Health. We see this as a positive development for the vaccine program since Ocugen had no plans to develop OCU500 without obtaining non-dilutive funding. We have no revenues for the vaccine programs in our models, and we see the selection by NIAID for Phase 1 clinical trials as upside for the company.

We See The Selection By NIAID As A Technology Validation. OCU500 was developed to stimulate immunity in the nasal passages and lungs where the virus enters the body and the infection begins. This is intended to prevent both the initial infection and transmission of the virus to others. We see the OCU500 selection as a validation to the Ocugen inhaled vaccine technology, which currently includes OCU500 for COVID-19, OCU510 for influenza, and OCU520 for both COVID-19 and influenza. The carrier technology can be used to develop other vaccines as well.


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*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Release – Ocugen Mucosal Vaccine Candidate Ocu500 Selected By NIH/NIAID Project Nextgen For Inclusion In Clinical Trials

Research News and Market Data on OCGN

October 10, 2023

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  • NIAID is conducting early phase clinical trials on select next generation vaccine candidates with the intent to identify the most effective platforms and delivery routes
  • OCU500 will be tested as both inhaled and intranasal vaccine candidates
  • Clinical trials scheduled to start in early 2024

MALVERN, Pa., Oct. 10, 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 National Institute of Allergy and Infectious Diseases (NIAID), a part of the National Institutes of Health, will conduct a trial comparing the administration of Ocugen’s mucosal vaccine candidate, OCU500, via two different mucosal routes, inhalation into the lungs and as a nasal spray.

Ocugen is developing a novel anti-viral mucosal vaccine platform initially targeting COVID-19 and influenza (flu). The intent is to provide protection against severe disease, increase durability and prevent transmission of viral threats. OCU500 is based on a novel chimpanzee adenovirus-vectored (ChAd) technology. Earlier clinical studies to prevent COVID-19 employing a similar vector administered via inhalation demonstrated increased mucosal antibodies, systemic antibodies, and durable immune response up to one year using one fifth (1/5) of the dose compared to the same vaccine given via intramuscular administration. Additionally, Ocugen believes that this vaccine can be rapidly scaled-up as new variants emerge.

“We believe our novel mucosal vaccine platform technology has the potential to prevent infection and spread of COVID-19, and improve durability for an annualized vaccine similar to flu,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “This is the first vaccine candidate using our inhaled platform technology, which we hope to expand in order to address multiple respiratory threats, including flu. We have benefited from a strong collaborative relationship with NIAID and BARDA since the start of Project NextGen and we look forward to participating in this initiative.”

NIAID will be conducting clinical trials to evaluate several early stage vaccine candidates. The study involving Ocugen’s vaccine will be administered via both intranasal and inhaled routes and is designed to help answer an important question – does an inhaled COVID-19 vaccine provide greater immune response than the same vaccine administered through a nasal spray. Upon completion of the trial NIAID and Ocugen will assess the results and determine next steps for OCU500.

Project NextGen is a $5 billion multi-government agency initiative to develop the next generation of vaccines and therapeutics to combat the spread of COVID-19. NIAID, with funding from Project NextGen, will cover the full cost of the clinical trials, including operations and related analysis. Ocugen will be responsible for providing clinical trial materials and upon completion will have full right of reference to the findings, which Ocugen believes will provide clinical evidence to support the further development of the Company’s lead mucosal vaccine candidate.

The announced collaboration comes at a time when COVID-19 infection rates are rising with the emergence of new variants. Durability of existing vaccines continues to be of concern with antibody protection waning several months following vaccination while vaccine compliance rates have declined since the initial wave. According to a recent Harris poll, 66% of Americans would prefer to have more vaccine options. The poll also found that 52% of Americans would be more open to getting an intranasal or inhaled, versus injectable COVID-19 vaccine.

Ocugen looks forward to this important next step in the development of its novel mucosal vaccine platform and further supporting the Company’s commitment to advancing public health.

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 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
Tiffany.Hamilton@ocugen.com 

Release – Ocugen To Present at The 2023 Cell & Gene Meeting On The Mesa

Research News and Market Data on OCGN

October 4, 2023

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MALVERN, Pa., Oct. 04, 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 present at the 2023 Cell & Gene Meeting on the Mesa being held October 10-12, 2023 in Carlsbad, CA at the Park Hyatt Aviara Resort.

Dr. Musunuri will provide an overview of Ocugen’s clinical development programs, including the business strategy across its unique gene and cell therapy platforms, anticipated milestones, and more detail about the Company’s recent positive Phase 1/2 OCU400 data results.

Details of the presentation are as follows:

Event:2023 Cell & Gene Meeting on the Mesa
Date & Time:October 11, 2023 at 5:15 PM, PDT
Location:Park Hyatt Aviara Resort, Carlsbad, CA, Aseptic Technologies Ballroom

“The Cell & Gene Meeting on the Mesa convenes leading industry professionals confronting major scientific, logistic, policy, and economic questions in cell and gene therapy,” said Dr. Musunuri. “I am pleased to join this impressive group and share the latest gene therapy news from Ocugen—compelling positive results from our Phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA)—as well as our progress toward initiating the Phase 3 trial for the Company’s novel cell therapy product candidate for cartilage repair in the second half of 2024.”

Virtual attendance is available, which includes a livestream of Ocugen’s presentation and on-demand viewing of all conference sessions. Please visit https://meetingonthemesa.com for full information including registration.

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.

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 

Release – Ocugen, Inc. Announces Connie Collingsworth Joins Business Advisory Board

Research News and Market Data on OCGN

September 21, 2023

MALVERN, Pa., Sept. 21, 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 Connie Collingsworth, former Chief Operating Officer of the Bill and Melinda Gates Foundation, will join the Company’s Business Advisory Board (BAB). The BAB was established in June 2023 to assist in driving public/private partnerships with governments around the world; pursuing business collaborations, partnerships, and licensing opportunities; creating awareness of the Company’s differentiated capabilities; and promoting access to the Company’s therapies around the world.

“Connie brings value-added expertise in all aspects of strategic growth and organizational governance—across a variety of industries—that will offer unique insights to the BAB,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “Connie’s deep financial background at a global level will provide meaningful counsel to Ocugen as we pursue new business development opportunities to support and evolve our clinical programs.”

Ms. Collingsworth has been a primary advisor to Bill Gates, Melinda French Gates, and Warren Buffett regarding governance. During her tenure with the Bill and Melinda Gates Foundation, she designed and implemented the core operational infrastructure and created the intellectual property strategy known as “Global Access,” as well as advising on the creation of a $2.5 billion strategic investment fund.

“I am inspired by Ocugen’s commitment to not only developing novel therapies that address unmet medical needs for patients with serious conditions, but their passion to ensure access to all patients around the world,” said Ms. Collingsworth. “I look forward to sharing my diverse experiences as an executive and a board member so that patients can potentially benefit.”

Ms. Collingsworth joins Senator Pat Toomey, Ambassador Joseph W. Westphal, Ph.D., and Dennis Carey, Ph.D. on the BAB. These advisors will work alongside the Executive Leadership Team to offer guidance to enable the Company to fulfill its mission and achieve its short- and long-term strategy.

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 Corporate Communications
Tiffany.Hamilton@ocugen.com

Source: Ocugen

Release – Ocugen To Participate in Panel At Cantor Fitzgerald Global Healthcare Conference 2023

Research News and Market Data on OCGN

September 20, 2023

PDF Version

MALVERN, Pa., Sept. 20, 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 panel at the annual Cantor Fitzgerald Global Healthcare Conference taking place from September 26-28, 2023 in NYC.

On the panel, Dr. Musunuri will join the CEO of Tarsier Pharma to discuss their companies’ respective work toward developing novel therapies for hard-to-treat ocular diseases. In addition, members of Ocugen’s executive team will conduct one-on-one meetings with registered investors, to showcase the Company’s business and clinical development strategy across its unique gene therapy, cell therapy, and vaccine platforms, and highlight recent corporate achievements and anticipated milestones.

Details of the presentation are as follows:

Date: Tuesday, September 26, 2023
Presentation Time: 1 p.m. ET
Location: Track 1, InterContinental Barclay Hotel, New York City

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 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.

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  

Ocugen (OCGN) – OCU400 Update Shows Improvement In Three Vision Measures


Thursday, September 14, 2023

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

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

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

Data Shows Clinically Meaningful Improvements. Ocugen provided an update to the Phase 1/2 clinical trial data for OCU400 in retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA), two rare diseases in which genetic mutations cause loss of vision and progress to blindness. We see these data as clinically meaningful for these indications as well as providing proof-of-concept for Ocugen’s method of targeting a “master control gene” that has downstream effects on other genes.

We See Proof-of-Concept For The “Master Control Gene” Approach. RP is a group of genetic disorders with over 100 possible genetic mutations that may be present and contributing to loss of vision. This makes the course of disease highly variable and difficult to target with a single therapy. Ocugen has been developing OCU400 to target the RHO and NR2E3 genes that control pathways and regulate other genes that act later in those pathways. The data show that OCU400 can affect several genetic pathways in eye, with improvements in three measures of vision.


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Rocket Shares Soar as FDA Aligns on Danon Disease Gene Therapy Trial

Rocket Pharmaceuticals (NASDAQ: RCKT) announced it has reached alignment with the U.S. Food and Drug Administration (FDA) on the design of a global pivotal Phase 2 trial for its gene therapy RP-A501 in Danon Disease. This marks an important milestone on the path to delivering a potentially transformative treatment for this devastating inherited cardiomyopathy.

Danon Disease is caused by genetic mutations in the LAMP2 gene leading to fatal heart failure. There are currently no approved therapies. The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe, taking the lives of most males by age 20 and females by 40. RP-A501 aims to be the first-ever therapy to change the course of Danon Disease.

The news sent Rocket’s stock price soaring 34% to $20.46 in after-hours trading on Tuesday, as investors welcomed the positive regulatory update.

The planned Phase 2 pivotal trial will be a global, single-arm, open label study enrolling 12 Danon Disease patients. This includes a pediatric safety lead-in of 2 patients. All participants will receive a dose of 6.7 x 1013 GC/kg of RP-A501 delivered through intravenous infusion.

To support potential accelerated approval by the FDA, the co-primary efficacy endpoints at 12 months are LAMP2 protein expression and reduction in left ventricular mass, a key measure of heart damage. Expression of LAMP2, which is deficient in Danon patients, and decreased cardiac hypertrophy would signal RP-A501 is restoring cardiac function at its root genetic cause.

Take a moment to look at Ocugen Inc., a biotechnology company focused on discovering, developing and commercializing novel gene and cell therapies and vaccines.

Secondary trial endpoints consist of the biomarker troponin, heart failure questionnaires, functional classification, event-free survival, and safety. These could support eventual full approval. A concurrent global natural history study will provide an external control arm for comparison.

According to Rocket CEO Dr. Gaurav Shah, “RP-A501 has the potential to restore normal cardiac function and provide a lifetime of benefit to patients with Danon Disease who have no other viable treatment options.” The company believes this pivotal trial design sets the most rapid path to deliver RP-A501 to patients in dire need.

Rocket’s gene therapy approach involves using an engineered virus called AAV9 to deliver a functional LAMP2 gene into patients’ heart cells. The gene insert encodes LAMP2B, a key protein involved in the cellular recycling process called autophagy. Restoring LAMP2B in the heart could potentially halt the accumulation of cellular debris and improve cardiac structure and function.

The company has already manufactured sufficient high-quality RP-A501 drug product at its in-house cGMP facility to supply the full pivotal Phase 2 trial. Qualified potency assays are also in place to support quality control and regulatory compliance.

Looking ahead, Rocket plans to file a Clinical Trial Application in the EU in Q3 2023 to initiate study activities abroad. The company also recently secured an ICD-10 code from the Centers for Medicare and Medicaid Services for LAMP2 deficiency, which will support diagnostic efforts.

This alignment with the FDA represents a major achievement for both Rocket and the Danon Disease community. RP-A501 would be the first-ever approved treatment for this deadly cardiovascular condition. The gene therapy aims to be a one-time curative infusion that could provide transformative and lifelong benefits to affected patients.

Beyond Danon Disease, Rocket believes this program paves the way for developing genetic medicines against other inherited heart diseases. Cardiac gene therapy has long faced hurdles, but the company is forging a new path for treating genetic cardiovascular conditions at their root.

With the pivotal Phase 2 trial design now locked in, Rocket can move full speed ahead on enrolling patients and gathering data to support potential approval. The company expects to release initial results from the trial evaluating RP-A501 in 2024. This could lead to a approved treatment for Danon Disease in the not too distant future—bringing tremendous hope to patients and families affected by this devastating illness.

Tonix Pharmaceuticals announces poster presentation involving TNX-1700 in preclinical colorectal cancer models at the 7th International Cancer Immunotherapy Conference 2023.