Retinitis Pigmentosa

Johnson & Johnson’s gene therapy for a genetic disease that leads to vision loss called X-linked retinitis pigmentosa (XLRP) failed a pivotal trial, according to an update to a J&J healthcare provider website. At one year, the gene therapy, called bota-vec, didn’t meet its primary endpoint in the Phase 3 LUMEOS study, though results were “directionally supportive,” according to the website , which appears to have been updated Friday. The primary endpoint measured the change in patients’ ability to navigate through a vision-guided mobility assessment maze. “We’re working to understand the totality of the data, inclusive of the clinical relevance of improvement shown on the majority of secondary endpoints, as we evaluate strategic options and next steps,” a J&J spokesperson told Endpoints News via email. The Phase 3 study fail comes as large biopharma companies are pulling back from AAV gene therapy work. Vertex became the latest company to stop research on AAV gene therapy, Endpoints reported Friday. And Pfizer, Takeda, Roche and Biogen have previously cut back on AAV gene therapy research or clinical programs. J&J acquired the gene therapy, known as bota-vec, from MeiraGTx for $65 million upfront in 2023 after previously making licensing deals for three eye disease gene therapies, including bota-vec, from the smaller biotech. The gene therapy delivers functional copies of the RPGR gene via viral packages called adeno-associated viruses, or AAVs. That gene is mutated in people with X-linked retinitis pigmentosa, a disease that primarily impacts boys and men. The Phase 3 study had enrolled 95 participants and split them into three groups: low-dose gene therapy, high-dose gene therapy, or deferred treatment — a group of patients who served as the comparator arm and were meant to receive the gene therapy after the primary endpoint measurement. A Biogen gene therapy for XLRP failed a mid-stage study in 2021. Beacon Therapeutics is studying a gene therapy for the rare eye disease, and last year raised $170 million. Editor’s note: This story was updated to include a statement from a J&J spokesperson.

SAN DIEGO, April 29, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc. (NASDAQ: BLTE), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the Company will give two poster presentations at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting being held May 4–8, 2025, in Salt Lake City, Utah. The presentations will highlight baseline patient characteristics from the ongoing DRAGON trial and pharmacokinetic and pharmacodynamic properties from the ongoing DRAGON II trial, both in Stargardt disease. Poster Presentations: Title: Baseline Characteristics of Adolescent Stargardt Disease Subjects Participating in a Phase 3 Study of Tinlarebant (DRAGON Trial)Presenting Author: Dr. Ruifang SuiPresentation Number: 1463Session Number: 215Session Title: Retinitis Pigmentosa and IRD ISession Date/Time: Monday, May 5, 8:30-10:15 a.m. MDT Title: Pharmacokinetic and Pharmacodynamic Properties of an Oral Investigational Treatment for Stargardt Disease in Adolescent Japanese PatientsPresenting Author: Dr. Kaoru FujinamiPresentation Number: 1431Session Number: 215Session Title: Retinitis Pigmentosa and IRD ISession Date/Time: Monday, May 5, 8:30-10:15 a.m. MDT About Tinlarebant (a/k/a LBS-008)Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in Stargardt Disease type 1 (STGD1) and also contribute to disease progression in geographic atrophy, or advanced dry age-related macular degeneration (AMD). Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and Sakigake Designation in Japan for the treatment of STGD1. Stargardt Disease (STGD1)STGD1 is the most common inherited macular dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by mutations in a retina-specific gene (ABCA4), which results in progressive accumulation of bisretinoids leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging systems have helped ophthalmologists identify and monitor disease progression. Currently, there are no FDA approved treatments for STGD1. Importantly, the appearance of bisretinoids, followed by retinal cell death, and progressive loss of vision is also observed in geographic atrophy (GA) patients. Therefore, Belite Bio is evaluating safety and efficacy of Tinlarebant in GA patients in a 2-year Phase 3 study (PHOENIX). About Belite BioBelite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite’s lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook or visit us at www.belitebio.com. Media and Investor Relations Contact:Jennifer Wu ir@belitebio.com Julie Fallonbelite@argotpartners.com

MALVERN, Pa., April 29, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa (Phase 3 LiMeliGhT clinical trial), OCU410ST for the treatment of Stargardt disease (Phase 2/3 pivotal confirmatory clinical trial), and OCU410 for the treatment of geographic atrophy (Phase 2 ArMaDa clinical trial), at The Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting at the Calvin L. Rampton Salt Palace Convention Center in Salt Lake City, Utah from May 4-8, 2025, and Retina World Congress at the Marriott Harbor Beach Resort in Ft. Lauderdale, Florida from May 8-11, 2025. “We look forward to sharing more about the potential of our modifier gene therapy platform and the meaningful results we are seeing in the clinic during these two important meetings for the retina community,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Ocugen remains on track to deliver on our commitment to file three Biologics License Applications (BLAs)/Marketing Authorization Applications (MAAs) in the next three years—potentially addressing significant unmet medical need for large patient populations through our gene-agnostic approach.” The ARVO Annual Meeting is a premiere gathering for eye and vision scientists from across the globe, students, and those in affiliated fields to share the latest research findings and collaborate on innovative solutions. Retina World Congress brings together leading retina specialists from every continent to achieve a global scientific and clinical exchange in retinal health. Ocugen’s presence in Utah kicks off with the Company Showcase at Eyecelerator, presented by Dr. Huma Qamar, Chief Medical Officer at Ocugen, and continues through presentations and thought leadership engagement at ARVO. Eyecelerator @ Park City 2025 Session: Retina—Gene Therapy and Novel Mechanisms of Action Showcase Location: Grand Hyatt Deer Valley, Strawberry Ballroom, Park City, UT Date: Friday, May 2, 2025 Time: 2:06 p.m. MDT Presenter: Dr. Huma Qamar ARVO Exhibitor Education Forum Two-Year Follow-Up of a Phase 1/2 Clinical Trial for the Safety and Efficacy of OCU400 Novel Modifier Gene Therapy for Retinitis Pigmentosa Location: Exhibitor Floor, Section 1037 Date: Monday, May 5, 2025 Time: 2 p.m. MDT Presenter: Benjamin Bakall, MD, Ph.D., Assistant Clinical Professor, University of Arizona, College of Medicine–Phoenix, and Director for Clinical Research, Director for The Inherited Retinal Disease and Visual Function Clinic, Associated Retina Consultants Safety and Efficacy of OCU410ST: A Phase 1/2 Trial of a Novel Modifier Gene Therapy for Stargardt Disease (GARDian) Location: Exhibitor Floor, Section 1037 Date: Tuesday, May 6, 2025 Time: 2 p.m. MDT Presenter: Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin An Evaluation of the Safety and Efficacy of Novel Modifier Gene Therapy OCU410 for the Treatment of Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration Location: Exhibitor Floor, Section 1037 Date: Wednesday, May 7, 2025 Time: 2 p.m. MDT Presenter: Neena Haider, Ph.D., Faculty Harvard Medical School and Founder, CEO, Shifa Precision Paper Session Preliminary Safety and Efficacy of OCU410 for Treatment of Geographic Atrophy: Phase 1/2 OCU410: The Age-related Macular Degeneration (ArMaDa) Study Update Presentation Number: 3675 Session Number and Title: 358/AMD Clinical research II Location: Ballroom J Date: Tuesday, May 6, 2025 Time: 4:15 p.m. MDT Presenter: Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin Poster Session A0513: Safety and Efficacy of OCU410ST for the Treatment of Stargardt Disease: Phase 1/2 Study Update Location: Hall A-E Date: Thursday, May 8, 2025 Time: 2 p.m. MDT Presenter: Ramiro Maldonado, MD, Duke Center for Ophthalmic Genetics, Duke Pediatric Retina, Adult vitreo-Retinal diseases Dr. Qamar will represent Ocugen at Retina World Congress to share the Company presentation and serve alongside notable retinal surgeons and industry peers during a panel discussion. Retina World Congress Retina Unplugged Inherited and Rare Retinal Diseases Session Moderators: Rishi P. Singh, MD, FASRS and Kourous A. Rezaei, MD Location: Grand Ballroom Date: Thursday, May 8, 2025 Time: 10:35 am – 11:12 a.m. EDT Ocugen is committed to bringing game-changing therapies to treat inherited retinal diseases as well as blindness diseases affecting millions to market and working even harder to provide access to patients globally. About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn. Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release. Contact: Tiffany Hamilton AVP, Head of Communications Tiffany.Hamilton@ocugen.com