Retinal Telangiectasis

Twelve week data from the Ph1b/2a AMARONE trial reveals Restoret to be well-tolerated in patients with diabetic macular edema and neovascular age-related macular degeneration. Proof of concept established with strong visual and anatomic outcomes Treatment-naïve DME patients treated with Restoret monotherapy experienced a mean reduction in excess retinal thickness of 80% and a mean vision gain of 11.2 letters at 12 weeks NEW YORK & LONDON--(BUSINESS WIRE)-- Eyebiotech Limited (“EyeBio” or “the Company”) announced today the presentation of Week 12 data from its first-in-human Phase 1b/2a AMARONE trial of Restoret in patients with treatment-naïve diabetic macular edema (DME) and treatment-naïve neovascular age-related macular degeneration (NVAMD). The analysis was presented by Charles C. Wykoff, MD, PhD, Director of Research at Retina Consultants of Texas and Chairman of the Research and Clinical Trials Committee, Retina Consultants of America on February 8, 2024, at the Macula Society Meeting in Palm Springs, California. The presentation of Week 12 data from AMARONE demonstrated that Restoret was well-tolerated, with no drug-related adverse events, drug-related serious adverse events, or intraocular inflammation reported. Patients with DME (n=26) received Restoret as monotherapy, manifesting a mean improvement in best-corrected visual acuity of +11.2 letters and a mean reduction in retinal thickness of -143 microns, as measured by optical coherence tomography (OCT). Similar outcomes were observed in patients with NVAMD (n=5), who received Restoret in combination with aflibercept. The data demonstrated that multiple monthly doses of Restoret, as both monotherapy and in combination with aflibercept, were well-tolerated. “These data from AMARONE are unique and impressive,” stated Charles C. Wykoff, MD, PhD. “While anti-VEGF monotherapies have proven valuable, many patients still do not achieve optimal outcomes and there remains substantial unmet need for therapeutics with alternative mechanisms of action that can improve outcomes for patients with exudative retinal diseases including diabetic macular edema and wet macular degeneration. While early data thus far, it is exciting to see these meaningful anatomic and visual improvements using this differentiated and fascinating Wnt agonist, especially in the absence of VEGF inhibition.” “We are thrilled to be sharing first-in-human data from the Restoret development program,” said David R. Guyer, MD, Chief Executive Officer of EyeBio. “The AMARONE trial represents the first ever clinical use of a Wnt pathway agonist to address retinal disease, and we are encouraged by the preliminary safety and efficacy data we’ve seen thus far.” “Retinal diseases impact millions of people worldwide, and so we need to continue to innovate to help drive better outcomes for these patients,” said Tony Adamis, MD, Chief Scientific Officer of EyeBio. “There is a great deal of preclinical evidence that agonizing the Wnt pathway in the retina results in reduction in vascular leakage, and so it is very gratifying to generate the first clinical evidence that validates this pathway in the eye.” About the AMARONE Clinical Trial The Phase 1b/2a AMARONE (Anti-permeability Mechanism and Age-Related Ocular Neovascularization Evaluation) clinical trial is a multi-center two-part trial consisting of an open-label multiple ascending dose (MAD) safety study, and a single-masked comparative safety and preliminary efficacy study of intravitreal (IVT) Restoret (EYE103) in participants with diabetic macular edema (DME) and neovascular age-related macular degeneration (NVAMD). Four increasing doses of Restoret were tested sequentially in cohorts of 3 patients each during the MAD portion, and the three highest doses were subsequently tested in a larger cohort of patients during the proof of concept portion of AMARONE. Patients with DME received intravitreal Restoret monotherapy every month for three months, while patients with NVAMD received Restoret in combination with aflibercept 2mg every month for three months. All patients were followed for 12 weeks from their first dose. Quality control of the trial database and data cleaning were successfully performed by IDDI/EyeBio. Of note, one participant had their end of study visit erroneously substituted for their safety extension injection visit, which was corrected. Another participant’s final visual acuity measurement was felt to be erroneous by the Principal Investigator (PI) on 11/20/23 (35 letters vs. 53 letters at month 2). Because the patient had noted subjective improvement of their vision and the PI had observed clinical improvement on OCT and exam, the PI felt the recorded vision loss was not medically plausible. He brought the subject back for recheck 4 days later (after Eylea) on 11/24/23, at which time the patient’s visual acuity was restored to 59 letters. The PI corrected the final visit input to that measured on 11/24/23, which he felt was accurate and documented with a note to file. About Restoret, a Wnt agonist tetravalent tri-specific agonist antibody Restoret is an investigational tri-specific Wnt agonist antibody designed to address urgent unmet medical need in patients with back-of-the-eye diseases. Restoret aims to eliminate leakage in vascular diseases by activating the Wnt pathway to both restore and maintain the blood-retinal barrier. The result of a breakthrough in protein engineering and manufacturing, Restoret may enable the clinical translation of the extensively studied Wnt pathway for the first time in the eye. Published research has shown that Wnt signaling in the retina plays a central role in the maintenance of vascular integrity, and defects in Wnt signaling cause retinal vascular leakage. Restoret has demonstrated activity in preclinical ophthalmic models, including validation in genetic models. About EyeBio (Eyebiotech Limited) Eyebiotech Limited (EyeBio) is a clinical-stage, privately held ophthalmology biotechnology company dedicated to developing and delivering a new generation of therapies to protect, restore, and improve vision in patients with sight-threatening eye diseases. Founded in August 2021 by David Guyer, M.D., and Tony Adamis, M.D., EyeBio has a leadership team with a world class track record in ophthalmology drug development. With operations in the United States and the United Kingdom, EyeBio is building and advancing a pipeline of ocular therapies that combine scientifically robust targets with innovative translational approaches. EyeBio was seeded and created by SV Health Investors and raised a Series A in February 2022 led by SV Health Investors, alongside Samsara BioCapital and Jeito Capital, with additional financial backing from MRL Ventures Fund, the therapeutics-focused corporate venture fund of Merck & Co., Inc., Rahway, N.J., USA. EyeBio doubled the size of its original Series A raise in November 2023 with new investors Bain Capital Life Sciences, Omega Funds and Vertex Ventures HC. For more information, please see .

First presentation of long-term results demonstrated sustained visual and anatomic improvements among EYLEA HD patients through two yearsTARRYTOWN, N.Y., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the first presentation of positive two-year (96 weeks) results from the pivotal PULSAR trial investigating EYLEA® HD (aflibercept) Injection 8 mg with 12- and 16-week dosing regimens, compared to EYLEA® (aflibercept) Injection, in patients with wet age-related macular degeneration (wAMD). The results were presented with the 60-week results at the 23rd EURETINA Congress. “EYLEA HD was approved in the U.S. based in part on strong one-year efficacy and safety outcomes from PULSAR in wet age-related macular degeneration,” said Charles C. Wykoff, M.D., Ph.D., Director of Research at Retina Consultants of Texas and a trial investigator. “These two-year outcomes from PULSAR show that EYLEA HD can deliver sustained and consistent visual and anatomic benefits with extended dosing intervals in the longer-term – helping to reduce the treatment burden for patients with exudative retinal diseases and reinforcing the potential of EYLEA HD as a new standard of care.” PULSAR (N=1,009) is a double-masked, active-controlled pivotal trial evaluating non-inferiority of EYLEA HD 12-week (n=335) and 16-week (n=338) dosing regimens compared to an 8-week dosing regimen for EYLEA (n=336). All patients received three initial monthly doses. Patients receiving EYLEA HD could have their dosing intervals shortened to an every 8-week interval if protocol-defined criteria for disease progression were observed. Patients were only able to extend their dosing intervals in the second year by 4-week increments up to 24-weeks, if pre-specified criteria were met. The PULSAR trial met its primary endpoint, with EYLEA HD patients achieving clinically equivalent vision gains to EYLEA at 48 weeks. As previously announced, EYLEA HD demonstrated durable vision gains at extended dosing intervals at the end of two years, with the mean number of injections administered being 9.7 for the 12-week EYLEA HD group, 8.2 for the 16-week EYLEA HD group, and 12.8 for the EYLEA group. As presented at EURETINA, efficacy results for EYLEA HD patients who completed the two-year follow-up were as follows: Among all patients, 88% were on a ≥12-week dosing interval at the end of two years, with 71% meeting the extension criteria for ≥16-week dosing intervals.Of patients assigned to ≥16-week dosing regimen at baseline, 78% were eligible for ≥16-week dosing at the end of two years, with 53% eligible for ≥20-dosing week intervals. In PULSAR, the safety of EYLEA HD continued to be similar to EYLEA through two years and remained consistent with the known safety profile of EYLEA from previous clinical trials for wAMD. There were no cases of occlusive retinal vasculitis or endophthalmitis in the EYLEA HD groups. The rate of intraocular inflammation was 1.3% for the aflibercept 8 mg group and 2.1% for the EYLEA group. Anti-platelet trialists' collaboration-defined arterial thromboembolic treatment-emergent adverse events occurred in 1.8% of patients treated with aflibercept 8 mg and 3.3% of patients treated with EYLEA. The full EURETINA presentation is available on the Regeneron website. The two-year data from the pivotal PHOTON trial for aflibercept 8 mg in diabetic macular edema (DME) and diabetic retinopathy (DR) were previously presented at the American Society of Retina Specialists annual meeting in July 2023. EYLEA HD 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. About the EYLEA HD Clinical Trial ProgramPULSAR in wAMD and PHOTON in DME/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. About wAMD and Diabetic Eye DiseasewAMD is a retinal disease that may affect people as they age. It occurs when abnormal blood vessels grow and leak fluid under the macula, the part of the eye responsible for sharp central vision and seeing fine detail. This fluid can damage and scar the macula, which can cause vision loss. An estimated 1.4 million Americans have wAMD. DR is an eye disease characterized by microvascular damage to the blood vessels in the retina often caused by poor blood sugar control in people with diabetes. The disease generally starts as nonproliferative diabetic retinopathy (NPDR) and often has no warning signs or symptoms. NPDR may progress to proliferative diabetic retinopathy (PDR), a stage of the disease in which abnormal blood vessels grow onto the surface of the retina and into the vitreous cavity, potentially causing severe vision loss. DME can occur at any stage of DR as the blood vessels in the retina become increasingly fragile and leak fluid, potentially causing visual impairment. In the U.S., approximately 1.5 million adults are diagnosed with DME, while approximately 6 million people have DR without DME. About Ophthalmology at RegeneronAt 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, 11 years of real-world experience, and more than 64 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 INDICATIONSEYLEA HD (aflibercept) 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) 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 and retinal detachment (separation of retina from back of the eye) can occur. Inflammation in the eye has been reported with the use of EYLEA HD and EYLEA.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.Contact your doctor right away if you think you or your baby might be experiencing any side effects, including eye pain or redness, light sensitivity, or blurring of vision, after an injection.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 for 35 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous FDA-approved treatments and product candidates in development, almost all of which were homegrown in our laboratories. Regeneron’s medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, hematologic conditions, infectious diseases and rare diseases. Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, such as VelocImmune®, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center®, which is conducting one of the largest genetics sequencing efforts in the world. For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn. Forward-Looking Statements and Use of Digital Media This 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 for the treatment of patients with wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy; uncertainty of the utilization, market acceptance, and commercial success of Regeneron’s Products (such as EYLEA HD) and Regeneron’s Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the 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) 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® (aflibercept) Injection 2 mg and REGEN-COV® (casirivimab and imdevimab)), other litigation and other proceedings and government investigations relating to the Company and/or its operations, 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, 2022 and its Form 10-Q for the quarterly period ended June 30, 2023. 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

CUMBERLAND, R.I.--(BUSINESS WIRE)-- Neurotech Pharmaceuticals, Inc., a private clinical stage biotech company, announced today that Alex Gorsky has been elected to Neurotech’s board as Lead Director. Gorsky is the former Chairman and CEO of Johnson & Johnson. This press release features multimedia. View the full release here: Alex Gorsky (Photo: Business Wire) Under Gorsky’s leadership, Johnson & Johnson grew to become the world’s largest diversified healthcare company and one of the foremost innovators in research and development for emerging health technologies. His influence has shaped both the healthcare landscape and the greater business community through his work as a member of the Business Roundtable, the Business Council, and many industry groups. He currently sits on the boards of Apple, IBM, JPMorgan Chase, and the Travis Manion Foundation, as well as the Wharton School of the University of Pennsylvania Board of Advisors. “I am delighted to welcome Alex to Neurotech’s Board of Directors,” said Jim Mazzo, Neurotech’s Executive Chairman. “As a visionary in patient focused healthcare, his expertise and enthusiasm for innovative technology will greatly enrich Neurotech’s mission to revolutionize treatments for various ocular diseases using our encapsulated cell therapy.” Mr. Gorsky said, “I am honored to join Neurotech’s board and look forward to using my experience to help the company navigate in a rapidly evolving environment. I am inspired by the Lowy family’s long-term commitment. This unique scientific approach and novel technology for the treatment of chronic eye diseases has the potential to provide access to important health options for patients around the world.” About Macular telangiectasia type 2 Macular telangiectasia type 2 (MacTel), or idiopathic juxtafoveal macular telangiectasia type 2, is a rare neurodegenerative disease with characteristic alterations of the retinal vasculature and localized retinal degeneration.1 MacTel typically affects both eyes and causes a gradual deterioration in central vision. About NT-501 Implant Designed to be implanted into the vitreous cavity of the eye, the investigational NT-501 implant is a tiny hollow cylindrical membrane which encapsulates human epithelial cells genetically engineered to produce ciliary neurotropic factor (CNTF) continuously, a protein now clinically validated in Phase 3 clinical trials to slow the progression of MacTel. About Encapsulated Cell Therapy Encapsulated Cell Therapy (ECT) is an investigational first-in-class, platform technology that promotes continuous production of therapeutic proteins to the eye with the potential to treat a broad array of ocular diseases. It utilizes a proprietary, well-characterized retinal pigment epithelial cell line that is genetically engineered to produce therapeutically active biologics. The cells are encapsulated in a semi-permeable membrane that allows for selective passage of therapeutic proteins. The ECT platform is inserted during a single outpatient surgical procedure through a small scleral incision, and can also be removed through the same incision, if desired. ECT has the potential to address the current limitations of intraocular drug delivery by allowing for and ensuring patient compliance, while reducing the treatment burden with one surgical procedure that can deliver the drug for at least 2 years. About Neurotech Pharmaceuticals, Inc. Neurotech Pharmaceuticals, Inc. is a private clinical stage biotech company focused on developing transformative therapies for chronic eye diseases. The core platform technology, Encapsulated Cell Therapy (ECT), enables continuous production of therapeutic proteins to the eye. Neurotech is currently studying in the clinic ECT candidates to treat Macular telangiectasia type 2 and glaucoma. To learn more, visit . 1. Charbel Issa P, Gillies MC, Chew EY, Heeren TFC, Bird AC, Peto T, Holz FG, Scholl HPN (2013) Macular Telangiectasia Type 2. Prog. Retin. Eye Res. 34: 49-77.