RARITAN, N.J., October 3, 2023 – 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
“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.
|Free Paper Session Abstract #CA231214||Phase 1 Study Of JNJ-81201887 Gene Therapy In Geographic Atrophy (GA) Due To Age-related Macular Degeneration (AMD)|
|Speakers’ Corner Session Abstract #CA231118||Pooled Safety Analysis Of A Single Intravitreal Injection Of JNJ-1887 (Gene Therapy, AAVCAGsCD59) In Patients With Age-Related Macular Degeneration (AMD)|
|Speakers’ Corner Session Abstract #CA231637||Descriptive Analyses Of A Real-World Cohort Of Patients With Geographic Atrophy Secondary To Age-Related Macular Degeneration|
|Audio Narrated Free Paper Abstract #CA231346||Concept Elicitation Interviews To Obtain Patients’ Perspectives On The Symptoms And Impacts Of X-Linked Retinitis Pigmentosa|
|E-Poster Abstract #CA23429||Comorbidities Among Inherited Retinal Dystrophy Patients In Sweden: A National Patient Register Study|
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 www.retina.janssen.com.
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 www.janssen.com. 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 www.sec.gov, www.jnj.com 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.
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.
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.
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.
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.
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.
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
Boughman JA, Conneally PM, Nance WE. Population genetic studies of retinitis pigmentosa. Am J Hum Genet. 1980;32(2):223–235.
Fishman GA. Retinitis pigmentosa. Genetic percentages. Arch Ophthalmol. 1978;96(5):822–826.
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.
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