Press release
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Opus Genetics to Host Virtual KOL Event on OPGx-LCA5 as a Treatment for LCA5- Associated Inherited Retinal Disease on December 11, 2024 6-month efficacy and safety data from ongoing Phase 1/2 trial of OPGx-LCA5 to be presented FARMINGTON HILLS, Mich., Dec. 3, 2024 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and other ophthalmic disorders, today announced that it will host a virtual key opinion leader (KOL) event on Wednesday, December 11, 2024 at 4:00 PM ET. To register, click here. The event will feature Jean Bennett, MD, PhD, and Tomas Aleman, MD, from the University of Pennsylvania, along with Christine Kay, MD, and Arshad Khanani, MD, MA, FASRS, who will discuss detailed, patient level 6-month efficacy and safety data on OPGx-LCA5, an adeno- associated virus (AAV)-based gene therapy, in Phase 1/2 development for LCA5-associated inherited retinal disease (IRD). The event will focus on the OPGx-LCA5 clinical data and will dive into the key efficacy assessments in detail. The presenters will also highlight the current unmet need with respect to LCA5-associated inherited retinal disease, next steps in the development program, and the potential of Opus Genetics’ therapeutic approach. A live question and answer session will follow the formal presentations. Jean Bennett, MD, PhD is the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine and continues to serve as director of the Center for Advanced Retinal and Ocular Therapeutics (CAROT) at the University of Pennsylvania. In addition to Dr. Bennett’s positions at the University of Pennsylvania, she has been an Investigator at the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia (CHOP) for more than a decade. She also co-founded life science companies Spark Therapeutics (acquired by Roche), GenSight Biologics and Limelight Bio. Dr. Bennett received her PhD in Zoology and Cell Biology from the University of California, Berkeley, and obtained her MD from Harvard University. She also completed postdoctoral fellowships in Radiobiology and Environmental Health at the University of California, San Francisco, Human Genetics at the Yale School of Medicine and Development Genetics at the Johns Hopkins University School of Medicine. She received her BS in Biology from Yale University. Tomas Aleman, MD is the Irene Heinz-Given and John LaPorte Research Professor at the Perelman School of Medicine, University of Pennsylvania. He is an Attending Physician in the Department of Ophthalmology at the Hospital of the University of Pennsylvania. He is part of the
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Retinal Degeneration Center at Scheie Eye Institute and the Center for Advanced Retinal & Ocular Therapeutics (CAROT), also at the University of Pennsylvania. Dr. Aleman has extensive expertise in the study of hereditary retinal degenerations. Most of the studies in which he has been investigator have aimed to lead patients with incurable retinal degenerations into clinical trials as well as in the detailed characterization of patients with these conditions. Dr. Aleman received an M.D. with High Honors from Sup de Cien Med de la Habana and completed a fellowship at the Scheie Eye Institute. Christine N. Kay, MD is a board-certified ophthalmologist and vitreoretinal surgeon and has been conducting clinical research since 2012. Her focus and passion are inherited retinal diseases, including but not limited to Stargardt disease, Retinitis Pigmentosa, and Achromatopsia. Dr. Kay is the Director of Research at Vitreoretinal Associates in Gainesville, Florida. She and her research team aim to improve the lives of people diagnosed with these and other diseases, as well as prevent blindness. Currently, the clinical trial team is conducting over 20 different studies targeting a multitude of retinal diseases. She was previously an Assistant Professor at the University of Florida and is currently Affiliate faculty at the University of South Florida. Dr. Kay is a member of the Macula Society, Retina Society, and American Society of Retina Specialists. Arshad M. Khanani, MD, MA, FASRS is the Managing Partner, Director of Clinical Research, and Director of Fellowship at Sierra Eye Associates, as well as a Clinical Professor at the University of Nevada, Reno School of Medicine. Dr. Khanani founded the clinical research department at Sierra Eye Associates, which has since become one of the nation's leading centers for clinical research. He has served as the principal investigator in over 120 clinical trials and has been a top enroller for several Phase 1-3 trials. He is also at the forefront of several collaborative studies examining real-world outcomes for newly approved treatments. Additionally, Dr. Khanani has been the first to perform surgical procedures in various clinical trials focused on sustained drug delivery and gene therapy. His extensive body of work includes numerous publications in highly regarded journals such as The Lancet, Ophthalmology, and JAMA Ophthalmology. Dr. Khanani is a lead principal investigator for several ongoing clinical trials and contributes to national and international clinical trial steering committees and scientific advisory boards. These efforts are aimed at developing new treatment options for patients with retinal diseases. A sought-after speaker, he is frequently invited to present at major national and international meetings. In 2021, Dr. Khanani founded the Clinical Trials at the Summit meeting to foster discussion on clinical trial design and data. Dr. Khanani is a distinguished member of both the Macula Society and the Retina Society, and he has earned various honors throughout his career. In 2019, he was named Healthcare Heroes Physician of the Year by Nevada Business Magazine for his dedication to ophthalmology. In 2023, he was chosen to deliver the prestigious Ernst Bodenheimer Memorial Lecture at the Wilmer Eye Institute, Johns Hopkins University. Dr. Khanani was also the recipient of the American Society of Retina Specialists (ASRS) Presidents’ Young Investigator Award in 2021 and the ASRS Presidential Award in 2024.
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About OPGx-LCA5 OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. New 6-month efficacy and safety data on OPGx-LCA5 for treatment of LCA5-associated inherited retinal disease will be presented at a virtual KOL event on December 11, 2024. About Opus Genetics Opus Genetics is a clinical-stage ophthalmic biotechnology company developing gene therapies to treat patients with inherited retinal diseases (IRDs) and other treatments for ophthalmic disorders. The pipeline includes adeno-associated virus (AAV)-based gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. The company’s most advanced gene therapy program is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein and is currently being evaluated in a Phase 1/2 open-label, dose-escalation trial. BEST1 gene therapy is designed to address mutations in the BEST1 gene, which is associated with retinal degeneration; a Phase 1/2 study is expected to be initiated in 2025. The pipeline also includes Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist to reduce pupil size, and APX3330, a novel small-molecule inhibitor of Ref-1 to slow the progression of non-proliferative diabetic retinopathy. Phentolamine Ophthalmic Solution 0.75% is currently being evaluated in Phase 3 trials for presbyopia and dim (mesopic) light vision disturbances. For more information, please visit www.opusgtx.com. Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning expectations regarding data from and future enrollment for our clinical trials and the effectiveness of our pipeline of indications and gene therapies. These forward- looking statements relate to us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 and in our other filings with the U.S. Securities and Exchange Commission (the “SEC”). Readers are
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cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise. These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, without limitation: • Our ability to successfully integrate the business of former Opus Genetics Inc. and manage our expanded combined product pipeline; • Our ability to develop and obtain regulatory approval for newly acquired gene therapies to treat inherited retinal diseases; • Our ability to obtain and maintain orphan drug designation or rare pediatric disease designation for our current and future product candidates; • The success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts; • Regulatory requirements or developments; • Changes to or unanticipated events in connection with clinical trial designs and regulatory pathways; • Delays or difficulties in the enrollment of patients in clinical trials; • Substantial competition; • Rapid technological change; • Our development of sales and marketing infrastructure; • Future revenue losses and profitability; • Changes in capital resource requirements; • Risks related to our inability to obtain sufficient additional capital to continue to advance our product candidates and our preclinical programs; • Domestic and worldwide legislative, regulatory, political and economic developments; • Our dependency on key personnel; • Changes in market opportunities and acceptance; • Reliance on third parties to conduct our clinical trials and supply and manufacture drug supplies;
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• Future, potential product liability and securities litigation; • System failures, unplanned events, or cyber incidents; • The substantial number of shares subject to potential issuance associated with our equity line of credit arrangement; • Risks that our licensing or partnership arrangements may not facilitate the commercialization or market acceptance of our product candidates; • Future fluctuations in the market price of our common stock; • The success and timing of commercialization of any of our product candidates; • Obtaining and maintaining our intellectual property rights; and • The success of mergers and acquisitions. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive. Readers are urged to carefully review and consider the various disclosures made by us in this report and in our other reports filed with the SEC that advise interested parties of the risks and factors that may affect our business. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Contacts Corporate Investor Relations Nirav Jhaveri CFO ir@opusgtx.com Corey Davis, Ph.D. LifeSci Advisors cdavis@lifesciadvisors.com