Stargardt Disease

Progress on OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and new data from Phase 1/2Preliminary safety and efficacy update on OCU410 Phase 1/2 ArMaDa clinical trial for geographic atrophy (GA)Clinical update on OCU410ST Phase 1/2 GARDian clinical trial for Stargardt diseaseFeaturing patients, investigators and thought leaders MALVERN, Pa., Oct. 28, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that it will host an in-person Clinical Showcase on Tuesday, November 12, 2024. The event will take place from 10 a.m.-noon ET at the Nasdaq MarketSite in Times Square, New York City. The event will focus on encouraging updates from Ocugen’s ongoing gene therapy trials, including: Clinical update on Phase 3 liMeliGhT clinical trial for retinitis pigmentosa along with data updates from Phase 1/2 RP and Leber congenital amaurosis (LCA)Preliminary safety and efficacy data from Phase 1/2 OCU410 ArMaDa clinical trial for GAClinical trial progress from Phase 1/2 OCU410ST GARDian study for Stargardt disease In addition, background on Ocugen’s biologic candidate, OCU200 for diabetic macular edema, will be presented. The Company is planning to initiate the OCU200 Phase I clinical trial this quarter. Ocugen presenters will include: Dr. Shankar Musunuri, Chairman, CEO & Co-founderDr. Huma Qamar, Chief Medical OfficerDr. Arun Upadhyay, Chief Scientific Officer & Head of R&DMike Shine, Senior Vice President, Commercial Joining the Ocugen team will be study investigators: Dr. Benjamin Bakall, Director of Clinical Research at Associated Retina Consultants (ARC) and Clinical Assistant Professor at University of Arizona, College of Medicine – PhoenixDr. Lejla Vajzovic, Professor of Ophthalmology, Pediatrics, and Biomedical Engineering with Tenure at Duke Eye Center and Duke University School of MedicineDr. Syed M. Shah, Vice Chair for Research and Digital Health, Director of Retina Service at Gundersen Health – La Crosse, Wisconsin The program will conclude with a patient panel representing participants in Ocugen’s ongoing clinical trials. Advance registration is required and can be done by contacting Tiffany Hamilton at Tiffany.Hamilton@ocugen.com. About Ocugen, Inc.Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn. Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release. Contact:Tiffany HamiltonHead of CommunicationsTiffany.Hamilton@ocugen.com

Oral gildeuretinol demonstrated a statistically significant reduction in the geographic atrophy (GA) lesion growth rate of 15.3% from 6 to 24 months (p=0.047) in a pre-specified analysis. Gildeuretinol is the first investigational oral therapy to demonstrate a functional benefit in low luminance visual acuity (LLVA) over 24 months (p=0.031), a key secondary endpoint. Gildeuretinol showed a clinically meaningful reduction in GA lesion growth rate of 13.4% from baseline to 24 months (p=0.075), the study’s primary endpoint. Results indicate that slowing the formation of toxic vitamin A dimers could help address the high unmet need for oral therapies in multiple retinal diseases. Gildeuretinol demonstrated a favorable safety and tolerability profile. Among participants at risk of developing Choroidal Neovascularization (CNV) or wet AMD, only 11% in the gildeuretinol group experienced these adverse events compared to 44% in the placebo group, as reported by clinical investigators. CAMBRIDGE, Mass., Oct. 23, 2024 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases, today announced that patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) treated with investigational oral therapy gildeuretinol (ALK-001) showed a statistically significant reduction in the GA lesion growth rate of 15.3% vs. placebo from 6 to 24 months (p=0.047). Gildeuretinol also demonstrated a statistically significant visual function improvement, showing 4.4 fewer letters lost (p=0.031) in low luminance visual acuity (LLVA) over 24 months. Gildeuretinol showed a clinically meaningful trend in slowing GA growth rate of 13.4% from baseline to 24 months (p=0.075), the study’s primary endpoint. A trend toward functional benefit in best corrected visual acuity (BCVA) was demonstrated with 3.3 fewer letters lost (p=0.099). Topline results of the SAGA study of gildeuretinol for the treatment of GA secondary to AMD were presented by Alexander Melamud, M.D., M.A., retina specialist and vitreoretinal surgeon with the Retina Group of Washington and a principal investigator in the SAGA study during the 128th Annual Meeting of the American Academy of Ophthalmology (AAO) held in Chicago October 18-21, 2024. “These data indicate a significant slowing of the growth rate of GA lesions, as well as reducing visual function decline,” said Seemi Khan, M.D., M.P.H., M.B.A., Chief Medical Officer of Alkeus Pharmaceuticals. “It takes a couple of months for gildeuretinol to replace vitamin A and achieve a therapeutic effect. Therefore, we are encouraged by the sensitivity analysis measuring the effect starting at six months of treatment. These data represent the first clinical demonstration that slowing vitamin A dimerization could be a target in the treatment of GA secondary to AMD. By slowing dimerization with gildeuretinol’s unique mechanism, we could reduce further damage to the retina at more advanced stages and possibly delay or prevent the onset of GA. We are excited by the potential of gildeuretinol as an oral therapy with functional benefits in GA, in addition to its potential to be the first therapy for Stargardt disease. We would like to thank the patients, investigators and trial sites for their participation in this study, as well as the National Institutes of Health National Eye Institute for providing funding for this study.” SAGA was a 24-month, double-masked, randomized, placebo-controlled trial to investigate the safety, pharmacokinetics, tolerability and efficacy in patients with GA secondary to AMD. The study enrolled 198 patients. The primary efficacy endpoint was the growth rate of GA lesions from baseline to 24 months as assessed by Fundus Autofluorescence (FAF). The first key secondary endpoint was the change in LLVA from baseline to 24 months. A favorable safety and tolerability pro demonstrated by gildeuretinol, consistent with prior clinical studies of gildeuretinol in Stargardt disease. Additionally, among participants at risk of developing Choroidal Neovascularization (CNV) or wet AMD, only 11% in the gildeuretinol group experienced these adverse events compared to 44% in the placebo group, as reported by the retina specialist investigators. “These data are meaningful and changing the conversation in the space about what might be possible with next generation treatments for patients with GA and earlier stages of AMD,” said Charles Wykoff, M.D., Ph.D., retina specialist and Director of Research at Retina Consultants of Texas. “While the primary endpoint was not met, the anatomic efficacy signal appears real, and most interestingly there appears to be a consistent signal of functional preservation with treatment. This patient population could benefit tremendously from a safe and effective systemic treatment option.” GA is a serious, progressive condition that causes irreversible loss of central vision. It is estimated that the median time of progression to severe visual impairment is slightly over six years. There is no oral therapy approved by the FDA to treat GA. The prevalence of GA in the United States is estimated to be over 1 million people, with 160,000 new cases occurring each year. “One of the most exciting aspects of these results is that gildeuretinol is the first oral medication to show a meaningful positive effect on low luminance visual acuity in macular degeneration,” Dr. Melamud said. “One of the first things patients lose in macular degeneration is low light vision, which can have a dramatic impact on daily living. The positive effect demonstrated on LLVA is significant. Based on these results, this therapy would definitely have a place in the overall strategy of treating AMD and slowing the decline observed in our patients. The favorable safety pro warrant treatment as early as possible.” About Gildeuretinol Acetate (ALK-001) Oral gildeuretinol acetate (ALK-001) is a new chemical entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. In preclinical studies, gildeuretinol decreased vitamin A dimerization down to the normal rate and prevented retinal degeneration and loss of visual function in animals with Stargardt disease. A randomized, placebo-controlled, double-masked clinical trial of gildeuretinol in late-stage Stargardt patients (TEASE-1) showed clinically and statistically significant slowing of the growth of retinal lesions over two years of treatment. Additional clinical trials of gildeuretinol in Stargardt disease are ongoing. Gildeuretinol has received breakthrough therapy designation and orphan drug designation for Stargardt disease from the U.S. Food and Drug Administration. A study (SAGA) of gildeuretinol in 198 patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) demonstrated a meaningful trend in the reduction of lesion growth rate and demonstrated a functional benefit in low luminance visual acuity (LLVA). In studies, gildeuretinol demonstrated a favorable safety and tolerability profile. About Alkeus Pharmaceuticals Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical company with headquarters in Cambridge, Mass., backed by institutional investors led by Bain Capital Life Sciences. Founded in 2010, Alkeus is developing therapies for serious diseases of the eye with high unmet need, with the purpose to protect the sight of individuals impacted by retinal diseases. Alkeus’ breakthrough-designated lead candidate, gildeuretinol acetate (ALK-001), is a new chemical entity currently being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). For further information, please contact: Media Relations media@alkeuspharma.com Website:

Determined the high dose of OCU410ST to be the maximum tolerated doseNo serious adverse events have been reportedApproved proceeding to Phase 2 using high and medium doses of OCU410ST MALVERN, Pa., Oct. 22, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU410ST GARDian clinical trial recently convened and approved enrollment for the second phase of the Phase 1/2 clinical trial. OCU410ST (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for Stargardt disease. “The DSMB has recommended moving forward with Phase 2 enrollment, as safety data indicates that OCU410ST appears to be safe and well-tolerated to date,” said Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas & Retina Consultants of America, and a lead study investigator. “The safety and tolerability profile of OCU410ST remains encouraging as the clinical trial has progressed and continues to bring hope to patients with Stargardt disease, which still has no FDA-approved treatments.” The first phase of the Phase 1/2 clinical trial was an open-label, dose-ranging study that enrolled nine patients to receive either a low (3.75 x 1010 vg/mL), medium (7.5 x 1010 vg/mL), or high (2.25 x 1011 vg/mL) dose of OCU410ST administered via subretinal injection. No serious adverse events (SAEs) have been reported, and the DSMB determined the high dose to be the maximum tolerated dose (MTD). Stargardt disease is the most common form of inherited macular dystrophy. Symptoms of bilateral central vision loss typically begin in childhood and gradually worsen over time. “We are enthusiastic about the potential of OCU410ST to be the first one-time novel modifier gene therapy for Stargardt disease,” said Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen. “We are encouraged by the prospect of addressing a substantial unmet medical need for the estimated 100,000 Stargardt patients in the U.S. and Europe.” The GARDian clinical trial is currently being performed at 6 leading retinal surgery centers across the U.S. About Stargardt DiseaseStargardt 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 OCU410STOCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks. About Ocugen, Inc.Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn. Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release. Contact:Tiffany HamiltonHead of CommunicationsTiffany.Hamilton@ocugen.com