CD59

Five abstracts to be presented, including new real-world research and data on Janssen's investigational gene therapy JNJ-1887 RARITAN, N.J., Oct. 3, 2023 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that five company-sponsored presentations will be featured during the European Society of Retina Specialists (EURETINA) 2023 annual meeting, taking place in Amsterdam, from October 5-8. The Company's presentations will include safety analysis data from two Phase 1 trials of the investigational gene therapy JNJ-81201887 (JNJ-1887): one trial in patients with geographic atrophy (GA), an advanced stage and severe form of age-related macular degeneration (AMD), and one trial in wet AMD (Abstracts #CA231214 and #CA231118).1,2 In addition, new research highlighting descriptive analyses of real-world GA trial populations, patients' perspectives on the symptoms and impact of X-linked retinitis pigmentosa (XLRP) and comorbidities among inherited retinal dystrophy patients will also be shared (Abstracts #CA231637, #CA231346 and #CA23429).3,4,5 Continue Reading Dare to Envision Courageous Science Normal Anatomy of the Retina Experience the full interactive Multichannel News Release here: "These data at EURETINA 2023 help bring us one step closer to a new era of innovation in retina," said James List, M.D., Ph.D., Global Therapeutic Area Head, whose team oversees a portfolio of programs including Retina at Janssen Research & Development, LLC. "We're committed to building on this momentum – and doing so with boldness and urgency – as we advance our mission to restore and preserve vision for those living with retinal diseases." GA is an advanced form of AMD.6 It affects more than five million individuals globally and is a leading cause of blindness in people over 65 years of age.5 It has a devastating impact on these patients and their quality of life, including their ability to read, drive and perform other day-to-day activities.5 XLRP is a rare inherited condition estimated to impact one in 40,000 people globally.7,8 People with XLRP have progressive vision loss, starting in childhood with night blindness.9 Over time, they lose their peripheral vision, and by approximately age 40, many are legally blind.8 Currently, there are no approved treatments for XLRP.8 A complete listing of the Company's abstracts being featured at the EURETINA Annual Meeting is provided below. Abstracts can also be found on the EURETINA website. Janssen will also have an interactive Retina Medical Affairs exhibit booth (location #12.115) at the EURETINA annual meeting. For more information about Janssen Retina's research and portfolio, please visit . About JNJ-1887 JNJ-81201887 (JNJ-1887), formerly referred to as AAVCAGsCD59, is an investigational one-time gene augmentation therapy for the treatment of people with geographic atrophy (GA), an advanced form of age-related macular degeneration (AMD). JNJ-1887 is designed to increase local expression of a soluble form of CD59 (sCD59) to possibly reduce MAC formation and protect retinal cells, thus potentially slowing and preventing disease progression. The Phase 2b PARASOL clinical trial (NCT05811351) is actively enrolling patients to study JNJ-1887 for the treatment of adults 60 or older with advanced dry AMD with GA. JNJ-1887 has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) and Advanced Therapy Medicinal Product (ATMP) designation by the European Medicines Agency (EMA). About botaretigene sparoparvovec (bota-vec) Botaretigene sparoparvovec (bota-vec) is being investigated in collaboration with MeiraGTx Holdings plc for the treatment of patients with X-linked retinitis pigmentosa (XLRP) caused by disease-causing variants in the eye-specific form of the RPGR gene. Through a one-time administration, bota-vec is designed to deliver functional copies of the RPGR gene that may counteract the loss of retinal cells with the goal of preserving and potentially restoring vision for those living with XLRP. The Phase 3 LUMEOS clinical trial (NCT04671433) has surpassed enrollment and is evaluating participants to study bota-vec for the treatment of patients living with XLRP with disease-causing variants in the RPGR gene. Bota-vec has been granted Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and PRIority MEdicines (PRIME), Advanced Therapy Medicinal Product (ATMP) and Orphan designations by the European Medicines Agency (EMA). About the Janssen and MeiraGTx Strategic Collaboration In January 2019, Janssen Research & Development, LLC entered into a worldwide collaboration and license agreement with MeiraGTx Holdings plc, a clinical-stage gene therapy company, to develop, manufacture and commercialize its clinical-stage inherited retinal disease portfolio. Botaretigene sparoparvovec (bota-vec) is being developed as part of a collaboration and license agreement. In addition to forming a research collaboration to explore new targets for other inherited retinal diseases, Janssen is working with MeiraGTx to build core capabilities in viral vector design, optimization and manufacturing. About the Janssen Pharmaceutical Companies of Johnson & Johnson At Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension. Learn more at . Follow us at @JNJInnovMed and @JanssenUS. Cautions Concerning Forward-Looking Statements This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding botaretigene sparoparvovec and JNJ-81201887. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended January 1, 2023, including in the sections captioned "Cautionary Note Regarding Forward-Looking Statements" and "Item 1A. Risk Factors," and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at , or on request from Johnson & Johnson. Neither Janssen Research & Development, LLC nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments. ________________________________ 1 Holz F et al. Phase 1 Study of JNJ-81201887 Gene Therapy in Geographic Atrophy (GA) Due to Age-related Macular Degeneration (AMD). Abstract #CA231214. European Society of Retina Specialists 2023 Annual Meeting. 2 Fischer, DM et al. Pooled Safety Analysis of a Single Intravitreal Injection of JNJ-1887 (Gene Therapy, AAVCAGsCD59) in Patients With Age-Related Macular Degeneration (AMD). Abstract #CA231118. European Society of Retina Specialists 2023 Annual Meeting. 3 Besirli, C et al. Concept elicitation interviews to obtain patients' perspectives on the symptoms and impacts of X-linked retinitis pigmentosa. Abstract #CA231346. European Society of Retina Specialists 2023 Annual Meeting. 4 Wennberg, A et al. Comorbidities Among Inherited Retinal Dystrophy Patients in Sweden: A National Patient Register Study. Abstract #CA23429. European Society of Retina Specialists 2023 Annual Meeting. 5 Gale, SL et al. Descriptive analyses of a real-world cohort of patients with geographic atrophy secondary to age-related macular degeneration. Abstract #CA231637. European Society of Retina Specialists 2023 Annual Meeting. 6 Cohen, MN et al. Phase 1 Study of JNJ-81201887 Gene Therapy in Geographic Atrophy (GA) Due to Age-related Macular Degeneration (AMD). Abstract #30071749. Presented at the 2022 American Academy of Ophthalmology Annual Meeting 7 Boughman JA, Conneally PM, Nance WE. Population genetic studies of retinitis pigmentosa. Am J Hum Genet. 1980;32(2):223–235. 8 Fishman GA. Retinitis pigmentosa. Genetic percentages. Arch Ophthalmol. 1978;96(5):822–826. 9 Wang DY, Chan WM, Tam PO, et al. Gene mutations in retinitis pigmentosa and their clinical implications. Clin Chim Acta. 2005;351(1-2):5-16. Me d i a Inquiries: Jennifer Silvent Mobile +1 973-479-9845 Rachael Jarnagin Mobile +1 415-705-9023 Investor Relations: Raychel Kruper Office +1 732-524-6164 [email protected] Click to Access Audio SOURCE Johnson & Johnson Innovative Medicine

SEATTLE--( BUSINESS WIRE )--Omeros Corporation (Nasdaq: OMER) today announced that the slides presented yesterday in the “late-breaker” session at the 2023 European Hematology Association (EHA) Congress in Frankfurt, Germany are now available on Omeros’ website . The presentation, which details data from a pre-specified interim analysis from the ongoing Phase 1b clinical trial of Omeros’ lead MASP-3 inhibitor OMS906 in complement-inhibitor-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH), was identified by EHA’s Scientific Program Committee as one of the top five late-breaking submissions and selected for oral presentation. The presentation, entitled OMS906, A mannan-binding lectin-associated serine protease-3 (MASP-3) Inhibitor, Normalizes Hemoglobin Levels in Treatment-naïve PNH Patients: Interim Data from a Proof-of-Concept Clinical Trial, was delivered to a large audience by Jens Panse, MD, Senior Physician and Deputy Director of the Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, and Managing Medical Director of the Center for Integrated Oncology, Aachen, Germany. Also available on Omeros’ website is a second presentation at EHA – Alternative Pathway MASP-3 Inhibitor OMS906: Results from a First-in-Man Phase 1 Study in Healthy Subjects and Study Design of Two Ongoing Clinical Trials in Patients with PNH – by Morag Griffin, MBChB, FRCPath, Consultant in Haematology of St. James University Teaching Hospital, Leeds, United Kingdom. This poster presentation describes findings from a single-ascending dose study evaluating OMS906 safety, pharmacokinetics and pharmacodynamics in healthy subjects. About PNH Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening acquired disorder that causes production of red blood cells lacking certain surface proteins (CD55 and CD59), making them vulnerable to destruction by the complement system. This leads to intravascular hemolysis (destruction of red blood cells within blood vessels) and extravascular hemolysis (when damaged red blood cells are removed by macrophages in the spleen or liver). Left untreated, PNH is associated with debilitating anemia, a high risk of thrombosis, fatigue, and a severely reduced survival rate. There remains a significant unmet need for PNH therapies, as a large proportion of patients treated with C5 inhibitors continue to experience hemolysis and approximately one third of them still require red blood cell transfusions. About OMS906 OMS906 is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the complement system’s alternative pathway. The complement system plays a central role in inflammation and becomes activated as a result of tissue damage or microbial infection. Responsible for the conversion of pro-complement factor D to complement factor D, MASP-3 is believed to be the premier target in the alternative pathway. MASP-3 has the lowest native circulating level and low relative clearance compared to the other alternative pathway proteins and, unlike C5 and C3 blockers, MASP-3 inhibition leaves intact the lytic arm of the classical pathway, important for fighting infection. Also, unlike other components of the alternative pathway, MASP-3 is believed not to be an acute phase reactant, which could provide a significant advantage to MASP-3 inhibitors, like OMS906, over other alternative pathway inhibitors. MASP-3 inhibitors are thought to have preventive or therapeutic effects across a broad range of diseases including PNH, hemolytic uremic syndrome (HUS), atypical HUS, traumatic brain injury, arthritis, wet age-related macular degeneration, ischemia-reperfusion injury, transplant-related complications and other immune-related disorders. Through its growing and exclusive intellectual property position, Omeros controls the use of MASP-3 inhibitors across a wide range of alternative pathway-related and other diseases and disorders. About Omeros Corporation Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders including complement-mediated diseases, cancers, and addictive and compulsive disorders. Omeros’ lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application pending before FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). Narsoplimab is also in multiple late-stage clinical development programs focused on other complement-mediated disorders, including IgA nephropathy, COVID-19, and atypical hemolytic uremic syndrome. Omeros’ long-acting MASP-2 inhibitor OMS1029 is currently in a Phase 1 clinical trial. OMS906, Omeros’ inhibitor of MASP-3, the key activator of the alternative pathway of complement, is advancing across multiple clinical programs for alternative pathway-related diseases, including paroxysmal nocturnal hemoglobinuria (PNH) and complement 3 (C3) glomerulopathy. For more information about Omeros and its programs, visit www.omeros.com .

– Late-breaking abstract detailing interim data from ongoing clinical trial of OMS906 in treatment-naïve PNH patients selected for June 11, 2023 oral presentation – SEATTLE--(BUSINESS WIRE)-- Omeros Corporation (Nasdaq: OMER) today announced that data from a pre-specified interim analysis of its ongoing Phase 1b clinical trial of OMS906, the company’s lead MASP-3 inhibitor, in complement-inhibitor-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening hemolytic blood disorder, will be shared at the 2023 European Hematology Association (EHA) Congress in Frankfurt, Germany. Identified as one of the top five late-breaking submissions by the Scientific Program Committee, the abstract was selected for oral presentation. The presentation, entitled OMS906, A mannan-binding lectin-associated serine protease-3 (MASP-3) Inhibitor, Normalizes Hemoglobin Levels in Treatment-naïve PNH Patients: Interim Data from a Proof-of-Concept Clinical Trial, will be delivered by Jens Panse, MD, Senior Physician and Deputy Director of the Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, and Managing Medical Director of the Center for Integrated Oncology, Aachen, Germany. Dr. Panse’s presentation will be delivered at the late-breaking oral session scheduled for 9:45-11:15 CEST on Sunday, June 11, 2023 and will be available by livestream to registered meeting attendees via the congress platform. The presentation abstract (#LB2714) was embargoed by EHA until 16:00 CEST today but now is freely accessible on EHA’s website. Following Dr. Panse’s presentation, Omeros will make available on its website the associated slides. Also at this EHA congress, Morag Griffin, MBChB, FRCPath, Consultant in Haematology of St. James University Teaching Hospital, Leeds, United Kingdom, will present a poster describing findings from a single-ascending dose study evaluating OMS906 safety, pharmacokinetics and pharmacodynamics in healthy subjects. Dr. Griffin’s presentation is scheduled for 18:00-19:00 CEST on Friday, June 9, 2023 and will be available for viewing by registered attendees on the online EHA platform throughout the duration of the congress (June 8-11, 2023). The presentation abstract (#P787) is available to everyone on EHA’s website. About PNH Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening acquired disorder that causes production of red blood cells lacking certain surface proteins (CD55 and CD59), making them vulnerable to destruction by the complement system. This leads to intravascular hemolysis (destruction of red blood cells within blood vessels) and extravascular hemolysis (when damaged red blood cells are removed by macrophages in the spleen or liver). Left untreated, PNH is associated with debilitating anemia, a high risk of thrombosis, fatigue, and a severely reduced survival rate. There remains a significant unmet need for PNH therapies, as a large proportion of patients treated with C5 inhibitors continue to experience hemolysis and approximately one third of them still require red blood cell transfusions. About OMS906 OMS906 is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the complement system’s alternative pathway. The complement system plays a central role in inflammation and becomes activated as a result of tissue damage or microbial infection. Responsible for the conversion of pro-complement factor D to complement factor D, MASP-3 is believed to be the premier target in the alternative pathway – it has the lowest native circulating level and low relative clearance compared to the other alternative pathway proteins and, unlike C5 and C3 blockers, MASP-3 inhibition leaves intact the lytic arm of the classical pathway, important for fighting infection. Also, unlike other components of the alternative pathway, MASP-3 is believed not to be an acute phase reactant, which could provide a significant advantage to MASP-3 inhibitors, like OMS906, over other alternative pathway inhibitors. MASP-3 inhibitors are thought to have preventive or therapeutic effects across a broad range of diseases including paroxysmal nocturnal hemoglobinuria (PNH), hemolytic uremic syndrome (HUS), atypical HUS, traumatic brain injury, arthritis, wet age-related macular degeneration, ischemia-reperfusion injury, transplant-related complications and other immune-related disorders. Through its growing and exclusive intellectual property position, Omeros controls the use of MASP-3 inhibitors across a wide range of alternative pathway-related and other diseases and disorders. About Omeros Corporation Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders including complement-mediated diseases, cancers, and addictive and compulsive disorders. Omeros’ lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application pending before FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). Narsoplimab is also in multiple late-stage clinical development programs focused on other complement-mediated disorders, including IgA nephropathy, COVID-19, and atypical hemolytic uremic syndrome. Omeros’ long-acting MASP-2 inhibitor OMS1029 is currently in a Phase 1 clinical trial. OMS906, Omeros’ inhibitor of MASP-3, the key activator of the alternative pathway of complement, is advancing across multiple clinical programs for alternative pathway-related diseases, including paroxysmal nocturnal hemoglobinuria (PNH) and complement 3 (C3) glomerulopathy. For more information about Omeros and its programs, visit .