Polypoidal choroidal vasculopathy

The ELEVATUM study showed clinically meaningful improvement in vision and reduction in retinal fluid in people with diabetic macular edema (DME) treated with Vabysmo who identify as African American, Black, Hispanic and Latino1 Efficacy and safety from this phase IV study were consistent with data from the Vabysmo phase III DME studies1These racial and ethnic groups are disproportionately affected by diabetes and at higher risk of developing DME, a leading cause of vision loss2-3 Basel, 18 October 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today positive topline one-year results from the open-label, single-arm phase IV ELEVATUM study evaluating Vabysmo® (faricimab) for the treatment of diabetic macular edema (DME) in people from racial and ethnic groups that are often underrepresented in clinical trials.4 Initial data from 124 participants in the United States (US) showed that after one year of treatment with Vabysmo, administered every eight weeks, participants could read an additional 12.3 letters on average – equivalent to about two and a half lines on an eye chart. Results among major racial and ethnic groups represented in this study were similar. Hispanic and Latino participants started the study with the most severe disease and had an average vision gain of 14.1 letters from baseline at one year, equivalent to nearly three lines on an eye chart. African American and Black participants gained an average of 11.3 letters from baseline at one year. Vabysmo was well tolerated, with no new safety events identified.1 These data were presented in a late-breaking oral presentation at the American Academy of Ophthalmology (AAO) 2024 Annual Meeting in Chicago, Illinois, on 18 October.1 The study is the first retina trial for historically underrepresented populations. “Vabysmo has been shown to be an effective first-line treatment for diabetic macular edema, and for the first time, we have data specifically demonstrating its ability to improve vision in Black, African American, Hispanic and Latino patients who are disproportionately impacted by this condition,” said investigator Jeremiah Brown, M.D., of Retina Consultants of Texas, who presented the data at AAO. “As a clinician who serves patients from these communities that are so often underrepresented in clinical trials, I believed it was important to take part in this groundbreaking study, and hope the findings will inform and improve the care we provide to our patients in the clinic daily.” Results were consistent with the phase III YOSEMITE and RHINE DME studies.5 A secondary endpoint showed robust retinal drying with Vabysmo across these racial and ethnic groups, who, on average, achieved a decrease of 206.3 microns in central subfield thickness (CST) from baseline.1 Reducing CST indicates retinal drying, which is an important clinical measure, as swelling from excess fluid in the back of the eye is associated with distorted and blurred vision.6 “We established ELEVATUM to specifically evaluate Vabysmo in underrepresented populations,” said Nilesh Mehta, Roche’s Global Therapeutic Area Head for Ophthalmology. “Including diverse populations and perspectives is part of our broader Roche Diversity, Equity & Inclusion (DE&I) strategy and is essential if we want to improve scientific understanding of diabetic macular edema and ultimately improve standard of care for all people living with this condition.” Among the 124 patients, 45% self-identified as Hispanic or Latino, and 48% as Black or African American.1 The study was designed to facilitate enrolment and promote retention of underrepresented patients. For example, ELEVATUM was conducted at sites that treat a high proportion of these populations in urban, rural and community-based locations. In addition, eligibility criteria allowed participants with a haemoglobin A1c (HbA1c) level up to 12%.4 An HbA1c test measures a person’s average blood sugar levels over the past three months and is used to diagnose diabetes.7 Typically, the threshold for DME trials is an HbA1c level of 10%. However, HbA1c levels can be higher in Black, African American, Hispanic and Latino populations compared with Caucasians, meaning a lower HbA1c threshold can inadvertently lead to the exclusion of patients from various ethnic and racial groups.8-9 To date, Vabysmo is approved in more than 100 countries for DME and neovascular or ‘wet’ age-related macular degeneration, and in over 30 countries for macular edema following retinal vein occlusion (RVO). More than five million doses of Vabysmo have been distributed globally since its initial US approval in 2022.10-15 About ELEVATUM4 ELEVATUM (NCT05224102) is a phase IV, multicentre, open-label, single-arm study designed to evaluate Vabysmo® (faricimab) as a treatment for diabetic macular edema (DME) in patients that have been historically underrepresented in clinical trials, including people who self-identify as Black, African American, Hispanic or Latino. Trial participants have not been treated with an anti‑vascular endothelial growth factor before the study. They receive treatment every four weeks with Vabysmo up to week 20, followed by treatment every eight weeks up to week 52. The primary endpoint is change from baseline in best corrected visual acuity at week 56. Secondary endpoints include safety and change in central subfield thickness from baseline over time. About Diabetic Macular Edema Affecting around 29 million people globally, diabetic macular edema (DME) is a vision-threatening retinal condition associated with blindness and decreased quality of life when left untreated.16-17 DME occurs when damaged blood vessels leak into and cause swelling in the macula – the central area of the retina responsible for the sharp vision needed for reading and driving.18-19 The number of people with DME is expected to grow as the prevalence of diabetes increases.20 About the Vabysmo® (faricimab) clinical development programmeRoche has a robust phase III clinical development programme for Vabysmo. The programme includes AVONELLE-X, an extension study of TENAYA and LUCERNE, evaluating the long-term safety and tolerability of Vabysmo in neovascular or ‘wet’ age-related macular degeneration (nAMD), and RHONE-X, an extension study of YOSEMITE and RHINE, evaluating the long-term safety and tolerability of Vabysmo in diabetic macular edema (DME).21-22 The POYANG study is evaluating Vabysmo in adult treatment-naive patients with choroidal neovascularisation secondary to pathologic myopia.23 Roche has initiated several phase IV studies, including the ELEVATUM study of Vabysmo in underrepresented patient populations with DME and the SALWEEN study of Vabysmo in a subpopulation of nAMD highly prevalent in Asia.4, 24 Roche has also initiated the VOYAGER study, a global real-world data collection platform, and supports several other independent studies to further understand retinal conditions with a high unmet need.25 About Vabysmo® (faricimab)Vabysmo is the first bispecific antibody approved for the eye.11, 26 It targets and inhibits two signalling pathways linked to a number of vision-threatening retinal conditions by neutralising angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A). Ang-2 and VEGF-A contribute to vision loss by destabilising blood vessels, causing new leaky blood vessels to form and increasing inflammation. By blocking pathways involving Ang-2 and VEGF-A, Vabysmo is designed to stabilise blood vessels.5, 26 Vabysmo is approved in more than 100 countries around the world, including the United States (US), Japan, the United Kingdom and the European Union (EU) for people living with neovascular or ‘wet’ age-related macular degeneration and diabetic macular edema, and in more than 30 countries, including the US, EU and Japan, for people living with macular edema following retinal vein occlusion. Review by other health authorities is ongoing.10-15 About Roche in OphthalmologyRoche is focused on saving people’s eyesight from the leading causes of vision loss through pioneering therapies. Through our innovation in the scientific discovery of new potential drug targets, personalised healthcare, molecular engineering, biomarkers and continuous drug delivery, we strive to design the right therapies for the right patients. We have the broadest retina pipeline in ophthalmology, which is led by science and informed by insights from people with eye diseases. Our pipeline includes innovative treatments across different modalities, such as antibodies, and gene and cell therapies targeting multiple vision-threatening conditions, including retinal vascular and diabetic eye diseases, geographic atrophy, and autoimmune conditions, such as thyroid eye disease and uveitic macular edema. Applying our extensive experience, we have already brought breakthrough ophthalmic treatments to people living with vision loss. Susvimo® (previously called Port Delivery System with ranibizumab) 100 mg/mL for intravitreal use via ocular implant is the first United States (US) Food and Drug Administration-approved refillable eye implant for neovascular or ‘wet’ age-related macular degeneration (nAMD) that continuously delivers a customised formulation of ranibizumab over a period of months.27, 28 Vabysmo® (faricimab) is the first bispecific antibody approved for the eye, which targets and inhibits two signalling pathways linked to a number of vision-threatening retinal conditions by neutralising angiopoietin-2 and vascular endothelial growth factor-A.5, 11, 12, 26 Vabysmo is approved around the world for people living with nAMD, diabetic macular edema and macular edema following retinal vein occlusion.10-15 Lucentis® (ranibizumab injection)* was the first treatment approved to improve vision in people with certain retinal conditions.29 About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com. *Lucentis® (ranibizumab injection) was developed by Genentech, a member of the Roche Group. Genentech retains commercial rights in the United States and Novartis has exclusive commercial rights for the rest of the world. All trademarks used or mentioned in this release are protected by law.References[1] Emanuelli A. ELEVATUM, the First Industry Sponsored Trial Focused on Underrepresented Patients in Retina in the US. Presented at: American Academy of Ophthalmology (AAO) Annual Meeting; 2024, October 18; Chicago, IL, USA.[2] Coney J, Scott A. Racial disparities in the screening and treatment of diabetic retinopathy. Journal of the National Medical Association. 2022;114 (2):171-181.[3] Lee R, et al. Epidemiology of diabetic retinopathy, DME and related vision loss. Eye and Vision. 2015; 2, 17.[4] Clinical Trials.gov. A Study to Investigate Faricimab Treatment Response in Treatment-Naive, Underrepresented Patients With Diabetic Macular Edema (ELEVATUM) [Internet; cited October 2024]. Available from: https://www.clinicaltrials.gov/study/NCT05224102.[5] Wykoff C, et al. Efficacy, durability, and safety of intravitreal faricimab with extended dosing up to every 16 weeks in patients with DME (YOSEMITE and RHINE): Two randomised, double-masked, phase III trials. The Lancet. 2022; 399:741-755.[6] US National Institutes of Health - National Eye Institute. Macular edema. 2023. [Internet; cited October 2024]. Available from: https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/macular-edema.[7] Eyth E, Naik R. Hemoglobin A1C. 2023. [Internet; cited October 2024]. Available from: https://www.ncbi.nlm.nih.gov/books/NBK549816/.[8] Cavagnolli G, et al. Effect of ethnicity on HbA1c levels in individuals without diabetes: Systematic review and meta-analysis. PloS one. 2017;12 (2).[9] Borkar DS, et al. Understanding the impact of HbA1c thresholds on DME clinical trial eligibility by race. Presented at: AAO 2023; November 3-6, 2023; San Francisco, CA, USA. Poster PO492.[10] European Medicines Agency. Summary of product characteristics, Vabysmo. 2022. [Internet; cited October 2024]. Available from: https://www.ema.europa.eu/en/documents/product-information/vabysmo-epar-product-information_en.pdf.[11] US FDA. Highlights of prescribing information, Vabysmo. 2023 [Internet; cited October 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761235s003lbl.pdf.[12] Medicines and Healthcare products Regulatory Agency approves faricimab through international work-sharing initiative. [Internet; cited October 2024]. Available from: https://www.gov.uk/government/news/mhra-approves-faricimab-through-international-work-sharing-initiative.[13] Chugai obtains regulatory approval for Vabysmo, the only bispecific antibody in the ophthalmology field, for additional indication of macular edema associated with RVO. [Internet; cited October 2024]. Available from: https://www.chugai-pharm.co.jp/english/news/detail/20240326160000_1054.html.[14] Chugai obtains regulatory approval for Vabysmo, the first bispecific antibody in ophthalmology, for nAMD and DME. [Internet; cited October 2024]. Available from: https://www.chugai-pharm.co.jp/english/news/detail/20220328160002_909.html.[15] Roche data on file.[16] Im JHB, et al. Prevalence of DME based on optical coherence tomography in people with diabetes: a systematic review and meta-analysis. Survey of Ophthalmology. 2022 Jul-Aug;67(4):1244-1251.[17] The Lancet. Diabetes: A defining disease of the 21st century. Lancet. 2023 24;401 (10394):2087.[18] All About Vision. Macula lutea. [Internet; cited October 2024]. Available from: https://www.allaboutvision.com/resources/macula.[19] National Eye Institute. Diabetic Retinopathy. [Internet; cited October 2024]. Available from: https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/diabetic-retinopathy.[20] Liu E, et al. DME: Clinical risk factors and emerging genetic influences. Clinical and Experimental Optometry. 2017;100:569–76.[21] Clinical Trials.gov. A study to evaluate the long-term safety and tolerability of Vabysmo in participants with nAMD (AVONELLE-X). [Internet; cited October 2024]. Available from: https://clinicaltrials.gov/ct2/show/NCT04777201. [22] Clinical Trials.gov. Clinical Trials.gov. A study to evaluate the long-term safety and tolerability of Vabysmo in participants with diabetic macular edema (DME) (RHONE-X). [Internet; cited October 2024]. Available from: https://clinicaltrials.gov/ct2/show/NCT04432831.[23] Clinical Trials.gov. A study to evaluate the efficacy and safety of faricimab in patients with choroidal neovascularization secondary to pathologic myopia (POYANG). [Internet; cited October 2024]. Available from: https://www.clinicaltrials.gov/study/NCT06176352.[24] APVRS. Design and rationale of the SALWEEN trial: A phase IIIb/IV study of faricimab, a dual angiopoietin-2 and vascular endothelial growth factor-a inhibitor, in patients with polypoidal choroidal vasculopathy. [Internet; cited October 2024]. Available from: https://2022.apvrs.org/abstract/?code=200351.[25] Clinical Trials.gov. A real-world study to gain clinical insights into Roche ophthalmology products (VOYAGER). [Internet; cited October 2024]. Available from: https://clinicaltrials.gov/ct2/show/NCT05476926.[26] Heier JS, et al. Efficacy, durability, and safety of intravitreal faricimab up to every 16 weeks for nAMD (TENAYA and LUCERNE): two randomised, double-masked, phase III, non-inferiority trials. The Lancet. 2022; 399:729-40.[27] US FDA. Highlights of prescribing information, Susvimo. 2021. [Internet; cited October 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761197s000lbl.pdf.[28] Holekamp N, et al. Archway randomised phase III trial of the Port Delivery System with ranibizumab for neovascular age-related macular degeneration (nAMD). Ophthalmology. 2021.[29] US FDA. Highlights of prescribing information, Lucentis. 2014. [Internet; cited October 2024]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2012/125156s0069s0076lbl.pdf. Roche Global Media RelationsPhone: +41 61 688 8888 / e-mail: media.relations@roche.com Hans Trees, PhDPhone: +41 79 407 72 58 Sileia UrechPhone: +41 79 935 81 48 Nathalie AltermattPhone: +41 79 771 05 25 Lorena CorfasPhone: +41 79 568 24 95 Simon GoldsboroughPhone: +44 797 32 72 915 Karsten KleinePhone: +41 79 461 86 83 Nina MählitzPhone: +41 79 327 54 74 Kirti PandeyPhone: +49 172 6367262 Yvette PetillonPhone: +41 79 961 92 50 Dr. Rebekka SchnellPhone: +41 79 205 27 03 Attachment 18102024_MR_Vabysmo ELEVATUM study _en

HAYWARD, Calif., Oct. 15, 2024 /PRNewswire/ -- Avirmax Biopharma Inc. (ABI) team will attend AAO 2024, the American Academy of Ophthalmology's annual meeting in Chicago, October 17-21, 2024. Avirmax will exhibit at booth #5357 to showcase the latest progress of the ABI gene therapy ocular programs. Continue Reading With a mission of unlocking the great potential of AAV based genetic medicine for treating retinal diseases, Avirmax Biopharma has innovated proprietary AAV2.N54 capsids optimized for superior gene delivery to the macula retina on basis of AAV2, with safe intravitreal administration aims to provide a long-lasting therapeutic effect and potentially reduces the need for frequent intravitreal injections. Manufacturing at Avirmax's in-house cGMP facility using proprietary VSaf™ virus free Sf9 cell lines enable product safety and cost of gene therapy vector production to be 5-10% of the traditional approaches, unlocking a great potential for the next generation genetic medicines be made accessible to millions of retinal patients worldwide. Avirmax Biopharma Is Unlocking Potential of Genetic Medicine, Preserving Vision for Millions of Patients Post this "Avirmax Biopharma's innovative pipeline products focus on many ophthalmic unmet needs," said Chief Medical Officer, Wendy Murahashi, MD. Avirmax Biopharma's lead candidate ABI-110 for wet AMD including PCV – currently enrolling patients for Phase I/IIa trials in the US () – showed significantly improved tropism to the macular retina over wildtype serotypes and AAV2.7m8 in mice, rabbits, pigs, and non-human primates. The ABI-201 programs for geographic atrophy (GA), dry AMD, proliferative diabetic retinopathy (PDR), and the ABI-902 pipeline targeting multiple types of glaucoma are currently under nonclinical development. About Avirmax Inc. Avirmax Biopharma, Inc., based in the San Francisco Bay Area, is a clinical stage biopharmaceutical company developing next-generation gene therapies for wet AMD, PCV, dry AMD, glaucoma and diabetic retinopathy. Utilizing proprietary AAV vector technologies, Avirmax Biopharma aims to deliver safe, effective, and accessible gene therapies to improve patient outcomes and preserve vision. Visit us at avirmax.com for more information. Contact June Song, Associate Director for Operations Email: [email protected] Tel: +1-510-641-0201 Address 25503 Whitesell Street Hayward 94545 CA USA SOURCE Avirmax WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Three new post-hoc analyses highlight EYLEA HD rapid and sustained fluid control and consistent safety profile over two years in patients with wet age-related macular degeneration (wAMD) New indirect comparison evaluates disease control of EYLEA HD and faricimab across different pivotal Phase 3 trials in wAMD TARRYTOWN, N.Y., Sept. 11, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced new analyses of EYLEA HD® (aflibercept) Injection 8 mg and EYLEA® (aflibercept) Injection 2 mg will be presented at the Annual Meeting of the European Society of Retina Specialists (EURETINA) in Barcelona from September 19 to 22, 2024. "The presentations at EURETINA reinforce the efficacy and safety profile of EYLEA HD and the ability to extend dosing intervals leading to significant and positive impacts on patients with wet age-related macular degeneration and diabetic macular edema,” said Boaz Hirshberg, MD, Senior Vice President, Clinical Development, Internal Medicine at Regeneron. “EYLEA HD continues on its way to becoming the new standard of care for these retinal diseases based on its differentiated clinical profile, and its strong familiarity and satisfaction among retinal specialists.” Among the presentation highlights are several new post-hoc analyses of the pivotal PULSAR trial for EYLEA HD in wet age-related macular degeneration (wAMD). These include first-time oral presentations of: An analysis applying disease activity criteria from the faricimab Phase 3 wAMD trials to EYLEA HD to evaluate the impact on the decision for extended dosing intervals.An analysis evaluating the impact of EYLEA HD on sustained fluid control throughout two years of treatment as measured by central retinal thickness and best-corrected visual acuity.The safety of EYLEA HD from an analysis evaluating intraocular pressure outcomes through week 96. Additional data to be shared at the meeting include analyses of the PHOTON trial in diabetic macular edema and a pooled safety analysis across the CANDELA, PHOTON and PULSAR trials. EYLEA HD (known as Eylea™ 8 mg in the European Union and Japan) is being jointly developed by Regeneron and Bayer AG. In the U.S., Regeneron maintains exclusive rights to EYLEA and EYLEA HD. Bayer has licensed the exclusive marketing rights outside of the U.S., where the companies share equally the profits from sales of EYLEA and EYLEA HD following any regulatory approvals. The following abstracts for EYLEA HD and EYLEA will be presented at EURETINA: Abstract titleLead author Presentation date, time (CEST), locationEYLEA HDA 96-week PULSAR subgroup analysis: similar visual and anatomic improvements with aflibercept 8 mg every 12 weeks or longer and 2 mg every 8 weeks, as defined by baseline BCVA, CRT, CNV type, and raceJean-Francois Korobelnik, MDOral Presentation in Free Paper SessionDate: September 19Time: 12:06–12:12 CESTSession 3: AMDIntraocular pressure outcomes with intravitreal aflibercept 8 mg and 2 mg in patients with neovascular age-related macular degeneration through Week 96 of the PULSAR trialPaolo Lanzetta, MDOral Presentation at Speakers’ CornerDate: September 19Time: 13:50–13:55 CESTSession 2: AMDA PULSAR phase 3 trial post-hoc analysis: Evaluating the timing and magnitude of control of disease activity with aflibercept 8 mg and faricimab, applying similar disease activity criteria across different pivotal Phase 3 trials for nAMDMichael Stewart, MDOral Presentation in Free Paper Session Date: September 19Time: 16:48–16:54 CEST Session 5: AMDA 96-week PULSAR Phase 3 trial post-hoc analysis: Rapid and sustained fluid control with aflibercept 8mg every 12 weeks or longer, as defined by fluid-free status at Weeks 16, 48, and 96 stratified by baseline CRT and BCVAPraveen J Patel, MDOral Presentation in Free Paper Session Date: September 19Time: 15:24–15:30 CESTSession 4: AMDImpact of baseline central retinal thickness on vision among patients with diabetic macular edema: post hoc analysis of the Phase 2/3 PHOTON trialDavid Lally, MDOral Presentation in Free Paper Session Date: September 20Time: 9:06–9:12 CESTSession 6: Diabetes & Vascular DiseasesPooled safety analysis of the CANDELA, PHOTON, and PULSAR trials up to 96 weeks demonstrates comparable safety profiles with aflibercept 8 mg and 2 mgAndreas Stahl, MDAudio-Narrated Free Paper Abstract Available on terminals throughout congressWeek 48 outcomes in aflibercept 8 mg and 2 mg treated patients by prior DME treatment status: a subgroup analysis of the Phase 2/3 PHOTON trialMarion Munk, MD, PhDAudio-Narrated Free Paper Abstract Available on terminals throughout congressIntravitreal aflibercept 8 mg for diabetic macular edema: week 96 efficacy outcomes by baseline characteristics in the Phase 2/3 PHOTON trialDeepali Varma, MDAudio-Narrated Free Paper AbstractAvailable on terminals throughout congressOutcomes of patients with DME and baseline BCVA of 20/50 or worse, and 20/40 or better who were treated with aflibercept 8 mg and 2 mg: a post-hoc analysis of the Phase 2/3 PHOTON trialJustus G. Garweg, MDAudio-Narrated Free Paper Abstract Available on terminals throughout congressIntraocular pressure outcomes with aflibercept 8 mg and 2 mg in patients with diabetic macular edema through week 48 of the Phase 2/3 PHOTON trialDilraj S. Grewal, MDAudio-Narrated Free Paper AbstractAvailable on terminals throughout the congressKey baseline disease characteristics in nAMD are not linked to treatment interval extension of aflibercept 8 mg: a post-hoc 96-week PULSAR analysisJavier Zarranz-Ventura, MDAudio-Narrated Free Paper Abstract Available on terminals throughout congressComparable efficacy and safety with aflibercept 8 mg at extended dosing intervals beyond q16 versus 2 mg q8 in Asian patients with nAMD in PULSAR through Week 96Timothy Lai, MDFree Paper SessionDate: September 20Time: 11:48–11:54 CESTSession 2: AMDAflibercept 8 mg monotherapy results in regression of polypoidal lesions that is maintained over 96 weeks in patients with PCV in the PULSAR Phase 3 trialRufino Silva, MDFree Paper SessionDate: September 20Time: 8:30–8:36 CESTSession 1: AMD EYLEA Efficacy and safety outcomes from the FIREFLEYE next study of children 3 years of age with retinopathy of prematurity treated with intravitreal aflibercept versus laser in the randomized FIREFLEYE studyAndreas Stahl, MDOral PresentationDate: September 20Time: 12:15-12:20 CESTSession: Results of Clinical Trials and Late Breaking SessionThe XTEND study: 3-year results from a global observational study investigating proactive dosing regimens with intravitreal aflibercept 2 mg in neovascular age-related macular degeneration in routine clinical practiceClare Bailey, MDAudio-Narrated Free Paper AbstractAvailable on terminals throughout congressFinal, 3-year results from the 8 highest recruiting countries included in the global, observational XTEND study of real-world proactive regimens with intravitreal aflibercept 2 mg in patients with neovascular age-related macular degenerationJean-Francois Korobelnik, MDAudio-Narrated Free Paper Abstract Available on terminals throughout congress About the EYLEA HD Clinical Trial Program PULSAR in wAMD and PHOTON in DME/diabetic retinopathy (DR) are double-masked, active-controlled pivotal trials that are being conducted in multiple centers globally. In both trials, patients were randomized into 3 treatment groups to receive either: EYLEA HD every 12 weeks, EYLEA HD every 16 weeks, or EYLEA every 8 weeks. The lead sponsors of the trials were Bayer for PULSAR and Regeneron for PHOTON. Patients treated with EYLEA HD in both trials had 3 initial monthly doses, and patients treated with EYLEA received 3 initial doses in PULSAR and 5 in PHOTON. In the first year, patients in the EYLEA HD groups could have their dosing intervals shortened down to an every 8-week interval if protocol-defined criteria for disease progression were observed. Intervals could not be extended until the second year of the study. Patients in all EYLEA groups maintained a fixed 8-week dosing regimen throughout their participation in the trials. CANDELA was a Regeneron-sponsored Phase 2 trial investigating the safety and efficacy of EYLEA HD extended dosing regimens compared to EYLEA in wAMD patients. About Ophthalmology at Regeneron At Regeneron, we relentlessly pursue groundbreaking innovations in eye care science to help maintain the eye health of the millions of Americans impacted by vision-threatening conditions. Over a decade ago, our breakthrough scientific research resulted in the development of EYLEA, a vascular endothelial growth factor (VEGF) inhibitor designed to block the growth of new blood vessels and decrease the ability of fluid to pass through blood vessels in the eye. EYLEA has since brought fundamental change to the retinal disease treatment landscape and is supported by a robust body of research that includes eight pivotal Phase 3 trials, 12 years of real-world experience, and more than 70 million EYLEA injections globally. Regeneron continues to advance our anti-angiogenesis expertise with new solutions with the aim of offering optimal flexibility for a broad group of patients and eye care professionals. This includes EYLEA HD, which has been developed with the aim of extending the time between injections, while maintaining the vision gains, anatomic benefits and safety previously observed with EYLEA. IMPORTANT SAFETY INFORMATION AND INDICATIONS INDICATIONS EYLEA HD® (aflibercept) Injection 8 mg is a prescription medicine approved for the treatment of patients with Wet Age-Related Macular Degeneration (AMD), Diabetic Macular Edema (DME), and Diabetic Retinopathy (DR). EYLEA® (aflibercept) Injection 2 mg is a prescription medicine approved for the treatment of patients with Wet Age-Related Macular Degeneration (AMD), Macular Edema following Retinal Vein Occlusion (RVO), Diabetic Macular Edema (DME), Diabetic Retinopathy (DR), and Retinopathy of Prematurity (ROP) (0.4 mg). IMPORTANT SAFETY INFORMATION EYLEA HD and EYLEA are administered by injection into the eye. You should not use EYLEA HD or EYLEA if you have an infection in or around the eye, eye pain or redness, or known allergies to any of the ingredients in EYLEA HD or EYLEA, including aflibercept.Injections into the eye with EYLEA HD or EYLEA can result in an infection in the eye, retinal detachment (separation of retina from back of the eye) and, more rarely, serious inflammation of blood vessels in the retina that may include blockage. Call your doctor right away if you or your baby (if being treated with EYLEA for Retinopathy of Prematurity) experience eye pain or redness, light sensitivity, or a change in vision after an injection.In some patients, injections with EYLEA HD or EYLEA may cause a temporary increase in eye pressure within 1 hour of the injection. Sustained increases in eye pressure have been reported with repeated injections, and your doctor may monitor this after each injection.In infants with Retinopathy of Prematurity (ROP), treatment with EYLEA will need extended periods of ROP monitoring.There is a potential but rare risk of serious and sometimes fatal side effects, related to blood clots, leading to heart attack or stroke in patients receiving EYLEA HD or EYLEA.The most common side effects reported in patients receiving EYLEA HD were cataract, increased redness in the eye, increased pressure in the eye, eye discomfort, pain, or irritation, blurred vision, vitreous (gel-like substance) floaters, vitreous detachment, injury to the outer layer of the eye, and bleeding in the back of the eye.The most common side effects reported in patients receiving EYLEA were increased redness in the eye, eye pain, cataract, vitreous detachment, vitreous floaters, moving spots in the field of vision, and increased pressure in the eye.The most common side effects reported in pre-term infants with ROP receiving EYLEA were separation of the retina from the back of the eye, increased redness in the eye, and increased pressure in the eye. Side effects that occurred in adults are considered applicable to pre-term infants with ROP, though not all were seen in clinical studies.You may experience temporary visual changes after an EYLEA HD or EYLEA injection and associated eye exams; do not drive or use machinery until your vision recovers sufficiently.For additional safety information, please talk to your doctor and see the full Prescribing Information for EYLEA HD and EYLEA. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. Please click here for full Prescribing Information for EYLEA HD and EYLEA. About RegeneronRegeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases. Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite®, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases. For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X. Forward-Looking Statements and Use of Digital MediaThis press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, the nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Products”) and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Product Candidates”) and research and clinical programs now underway or planned, including without limitation EYLEA HD® (aflibercept) Injection 8 mg and EYLEA® (aflibercept) Injection 2 mg; uncertainty of the utilization, market acceptance, and commercial success of Regeneron’s Products (such as EYLEA HD and EYLEA) and Regeneron’s Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the studies (as well as additional analyses of such studies) discussed or referenced in this press release, on any of the foregoing; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s Product Candidates and new indications for Regeneron’s Products; the ability of Regeneron’s collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and Regeneron’s Product Candidates; the ability of Regeneron to manage supply chains for multiple products and product candidates; safety issues resulting from the administration of Regeneron’s Products and Regeneron’s Product Candidates in patients, including serious complications or side effects in connection with the use of Regeneron’s Products and Regeneron’s Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize Regeneron’s Products and Regeneron’s Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron’s Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement of Regeneron’s Products (such as EYLEA HD and EYLEA) from third-party payers, including private payer healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payers and new policies and procedures adopted by such payers; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron’s Products and Regeneron’s Product Candidates; the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license, collaboration, or supply agreement, including Regeneron’s agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable) to be cancelled or terminated; the impact of public health outbreaks, epidemics, or pandemics (such as the COVID-19 pandemic) on Regeneron's business; and risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA), other litigation and other proceedings and government investigations relating to the Company and/or its operations (including the pending civil proceedings initiated or joined by the U.S. Department of Justice and the U.S. Attorney's Office for the District of Massachusetts), the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron’s business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron’s filings with the U.S. Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2023 and its Form 10-Q for the quarterly period ended June 30, 2024. Any forward-looking statements are made based on management’s current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron. Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise. Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron's media and investor relations website (https://investor.regeneron.com) and its LinkedIn page (https://www.linkedin.com/company/regeneron-pharmaceuticals). Contacts: Media RelationsMary HeatherTel: +1 914-847-8650mary.heather@regeneron.comInvestor RelationsMark HudsonTel: +1 914-847-3482mark.hudson@regeneron.com