Wet Macular Degeneration

– DURAVYU 2.7mg demonstrated an early and sustained improvement in BCVA with a gain of +8.9 letters compared to baseline - – DURAVYU 2.7mg demonstrated an early and sustained anatomical improvement mirroring BCVA results with a 68 micron reduction in CST - – Favorable safety profile continues with no DURAVYU-related ocular or systemic SAEs to date – – Full topline data anticipated in Q1 2025 – Oct. 28, 2024 -- EyePoint Pharmaceuticals, Inc. (NASDAQ: EYPT), a company committed to developing and commercializing innovative therapeutics to improve the lives of patients with serious retinal diseases, today announced positive interim 16-week data for the ongoing Phase 2 VERONA clinical trial evaluating DURAVYU, an investigational sustained delivery therapy delivering patent-protected vorolanib, a selective tyrosine kinase inhibitor formulated in proprietary bioerodible Durasert E™, for patients with diabetic macular edema (DME). DURAVYU 2.7mg demonstrated an early, sustained, and clinically meaningful improvement in best-corrected visual acuity (BCVA) and anatomical control versus the aflibercept control arm. A favorable safety and tolerability profile continued for both DURAVYU arms. The 2.7mg dose is also being evaluated in the Phase 3 pivotal trials for wet AMD. The Company expects to report the full topline results in the first quarter of 2025, once all patients complete the trial. “We are encouraged and excited by these highly positive interim results demonstrating clinically meaningful functional and concomitant structural improvement with a continued favorable safety profile of DURAVYU,” said Jay S. Duker, M.D., President and Chief Executive Officer of EyePoint. “DME is a prevalent disease with a significant need for more durable treatments. The interim data from the VERONA trial demonstrates that after a single DURAVYU 2.7mg treatment there was a meaningful, early and maintained improvement in BCVA paired with strong anatomical improvement in retinal thickness, demonstrating the potential for DURAVYU in DME as a sustained delivery therapy. One of the most notable aspects of these data is that both DURAVYU doses showed an immediate benefit over aflibercept control in both BCVA and CST. We believe these compelling interim results support DURAVYU’s potential to advance to non-inferiority pivotal trials in DME. With pivotal wet AMD clinical trials underway and promising DME interim data, DURAVYU has the potential to be the first sustained release therapy to market in two significant indications.” All patients (n=27) have completed the week 16 visit. DURAVYU 2.7mg demonstrated an early and sustained improvement in both BCVA and CST (central subfield thickness) as measured by optical coherence tomography (OCT). BCVA improved +8.9 letters versus +3.2 letters for aflibercept control compared to baseline. CST improved 68.1 microns versus 30.5 microns for aflibercept control compared to baseline. Visual and anatomical gains were observed at Week 4 demonstrating the immediate bioavailability of DURAVYU. Positive trend in BCVA and anatomy continued for patients that have reached the Week 24 visit. Continued positive safety and tolerability profile with no DURAVYU-related ocular or systemic serious adverse events. Additionally, there were no cases of: Endophthalmitis Retinal vasculitis (occlusive or non-occlusive) Insert migration to the anterior chamber Intraocular inflammation (IOI) 82% of eyes in the DURAVYU 2.7mg arm were supplement-free versus 50% in the aflibercept control arm at 16 weeks. “DME is a sight-threatening complication of diabetes that can lead to severe visual loss and eventual blindness,” said Charles Wykoff, M.D., Ph.D., Director of Research, Retina Consultants of Texas and Co-Chair of EyePoint’s Scientific Advisory Board. “There remains a significant need for differentiated and longer-acting treatments, as the current standard of care involves frequent intravitreal injections that can be a burden and have been associated with under-treatment. The interim data from the Phase 2 VERONA trial suggests promising activity in patients with active DME versus aflibercept alone and a favorable safety profile. These results support the potential for DURAVYU to bring substantial value to patients through stable, durable disease control.” “Reducing the treatment burden in patients with DME is a critical unmet need,” said Adam Gerstenblith, M.D., a principal investigator in the VERONA clinical trial and vitreoretinal surgeon at Mid Atlantic Retina Specialists. “As a clinician dedicated to advancing retinal care, I am encouraged by the interim clinical data demonstrating the potential for DURAVYU 2.7mg to extend treatment intervals while improving vision without sacrificing anatomy. The VERONA trial is an important step in the pursuit of treatment options for patients that are safe and durable, and I am pleased to be participating in research that has the potential to shift the treatment paradigm in DME and ultimately improve patient outcomes.” VERONA is a randomized, controlled, single-masked, open label Phase 2 trial of DURAVYU in DME patients previously treated with a standard-of-care anti-VEGF therapy. The trial enrolled 27 patients assigned to one of two intravitreal doses of DURAVYU (1.3mg or 2.7mg) or aflibercept control. The primary efficacy endpoint of the VERONA trial is time to first supplemental aflibercept injection up to 24 weeks based on established supplement criteria. Key secondary endpoints include safety, mean change in best corrected visual acuity (BCVA), mean change in central subfield thickness (CST) as measured by optical coherence tomography (OCT) and change in diabetic retinopathy severity scale (DRSS) over time. More information about the trial is available at clinicaltrials.gov (identifier: NCT06099184). Diabetic macular edema (DME) is the leading cause of vision loss in people with type 1 and type 2 diabetes. DME results when damaged blood vessels leak fluid into the macula, the central portion of the retina responsible for the sharp vision needed for routine tasks such as driving or reading. This resulting retinal swelling can cause blurred vision and may lead to severe vision loss or even blindness. DME is a common form of sight-threatening retinopathy in people with diabetes, with approximately 28 million people afflicted worldwide. As the prevalence of diabetes continues to grow, an increased number of people will be affected by diabetic eye diseases such as DME. The current standard of care for patients experiencing DME include intravitreal injections of short-acting anti-VEGF biologics, corticosteroids, or laser photocoagulation which can become a burden on patients, caregivers, and physicians due to the longevity of the disease. DURAVYUTM, f/k/a EYP-1901, is being developed as a potential paradigm-altering treatment for patients suffering from VEGF-mediated retinal diseases. DURAVYU delivers vorolanib, a potent, selective and patent-protected tyrosine kinase inhibitor (TKI) as a solid bioerodible insert using EyePoint’s proprietary sustained-release Durasert E™ technology. Vorolanib brings a new mechanistic approach to the treatment of VEGF-mediated retinal diseases as a pan-VEGF receptor inhibitor, inhibiting all VEGF receptors. Further, in an in-vivo model of retinal detachment, vorolanib demonstrated neuroprotection and may have antifibrotic benefits as it also blocks PDGF. DURAVYU is shipped and stored at ambient temperature and is administered with a standard intravitreal injection in the physician's office. DURAVYU is immediately bioavailable with zero-order kinetics release for up to nine months. Positive data from both the Phase 1 DAVIO and Phase 2 DAVIO 2 clinical trials of DURAVYU in wet AMD demonstrated clinically meaningful efficacy data with stable visual acuity and CST and a favorable safety profile. Further, data from DAVIO 2 demonstrated an impressive treatment burden reduction of approximately 88% at eight months, six months after treatment with DURAVYU, with over 80% of patients supplement-free or receiving only one supplemental anti-VEGF injection through up to eight months, six months after treatment with DURAVYU. The data from the DAVIO 2 clinical trial supported the advancement of the wet AMD program and the initiation of the Phase 3 LUGANO trial, with the LUCIA pivotal trial to follow by year end 2024. DURAVYU is also currently being studied in the Phase 2 VERONA trial for diabetic macular edema (DME). Full topline data is expected in the first quarter of 2025. EyePoint (Nasdaq: EYPT) is a clinical-stage biopharmaceutical company committed to developing and commercializing innovative therapeutics to help improve the lives of patients with serious retinal diseases. The Company's pipeline leverages its proprietary bioerodible Durasert E™ technology for sustained intraocular drug delivery. The Company’s lead product candidate, DURAVYU™ (f/k/a EYP-1901), is an investigational sustained delivery treatment for VEGF-mediated retinal diseases combining vorolanib, a selective and patent-protected tyrosine kinase inhibitor with bioerodible Durasert E™. DURAVYU is presently in Phase 3 global, pivotal clinical trials as a sustained delivery treatment for wet age-related macular degeneration (wet AMD), the leading cause of vision loss among people 50 years of age and older in the United States, and in a Phase 2 clinical trial in diabetic macular edema (DME). EyePoint expects full topline data from the Phase 2 clinical trial in DME in Q1 2025 and topline data from both Phase 3 pivotal trials in wet AMD in 2026. Pipeline programs include EYP-2301, a TIE-2 agonist, razuprotafib, formulated in Durasert E™ to potentially improve outcomes in serious retinal diseases. The proven Durasert® drug delivery technology has been safely administered to thousands of patient eyes across four U.S. FDA approved products. EyePoint Pharmaceuticals is headquartered in Watertown, Massachusetts. Vorolanib is licensed to EyePoint exclusively by Equinox Sciences, a Betta Pharmaceuticals affiliate, for the localized treatment of all ophthalmic diseases outside of China, Macao, Hong Kong and Taiwan. DURAVYU™ has been conditionally accepted by the FDA as the proprietary name for EYP-1901. DURAVYU is an investigational product; it has not been approved by the FDA. FDA approval and the timeline for potential approval is uncertain. The content above comes from the network. if any infringement, please contact us to modify.

97% reduction in treatment burden at nine months after treatment with ABBV-RGX-314 Data consistent with that from multiple previous studies demonstrating favorable safety and efficacy profile Well tolerated with zero cases of intraocular inflammation in a setting of no prophylactic steroids Data highlight the potential of ABBV-RGX-314 to treat both eyes in wet AMD ROCKVILLE, Md., Oct. 21, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced positive data from the Phase II fellow eye sub-study evaluating the subretinal delivery of ABBV-RGX-314 in patients with bilateral wet age-related macular degeneration (wet AMD). The new data were presented at the American Academy of Ophthalmology (AAO) meeting by Arshad Khanani, M.D., M.A., FASRS, Director of Clinical Research at Sierra Eye Associates, Reno, NV. "The results presented at AAO from the Phase II sub-study, the first to evaluate a gene therapy in fellow eyes for wet AMD, demonstrate that ABBV-RGX-314 could be a treatment for patients with bilateral disease and add to the already-robust data demonstrating ABBV-RGX-314's potential to impact the treatment paradigm for patients with wet AMD," said Curran Simpson, President and Chief Executive Officer of REGENXBIO. "With more treated patients and the longest-term data of any gene therapy program for wet AMD, REGENXBIO, with our partner AbbVie, are well-positioned to bring the first gene therapy to market with the hope of preserving long-term vision for millions of patients globally with wet AMD." "The majority of our patients with wet AMD eventually have bilateral disease and face a substantial treatment burden with frequent lifelong injections in both eyes. This leads to suboptimal real-world vision outcomes with current standard of care," said Dr. Khanani. "The fellow eye dosing data with ABBV-RGX-314 is a milestone for the field of gene therapy for common retinal diseases, as this is the first time we have performed bilateral treatment for wet AMD patients. These results, combined with the durable treatment effect up to four years shown in long-term follow up, highlight the potential of ABBV-RGX-314 as a one-time effective treatment option for patients with wet AMD." Data and Safety Summary The fellow eye sub-study was designed to evaluate the safety and efficacy of a single dose (1.3x1011 GC/eye) of ABBV-RGX-314 using subretinal delivery in the fellow eye of previously treated patients. The second eye was treated with ABBV-RGX-314 approximately one year or more after administration of ABBV-RGX-314 in the first eye. This dose is being evaluated in the ongoing pivotal trials of ABBV-RGX-314 and is similar to one of the doses evaluated in the Phase I/IIa trial, which demonstrated durable treatment effect up to four years in a long-term follow up study. The fellow eye sub-study data at nine months includes nine patients who received ABBV-RGX-314 using subretinal delivery in the Phase I/IIa or bridging studies and elected to receive treatment in their second eye. Prior to ABBV-RGX-314 administration, these patients had a high treatment burden in the fellow eye and had received an average of nine anti-VEGF injections in the year prior to entering the study, including anti-VEGF injections intended to be longer-lasting treatments. At nine months post-administration of ABBV-RGX-314, key findings from the Phase II fellow eye sub-study include: 97% reduction in annualized anti-VEGF treatment burden 100% of patients required either zero or one supplemental injection 78% of patients were completely injection-free Additionally, patients demonstrated sustained best-corrected visual acuity (BCVA) and central retinal thickness (CRT) at nine months. ABBV-RGX-314 produced similar levels of aqueous protein in both treated eyes. As of September 11, 2024, ABBV-RGX-314 was well tolerated in the treated fellow eye with no drug related serious adverse events. No cases of intraocular inflammation, chorioretinitis, vasculitis, occlusion or hypotony were observed. No prophylactic steroids were used in this trial, other than those typically used in vitrectomy surgery. Common adverse events included mild retinal pigmentary changes occurring in periphery and post-operative conjunctival hemorrhage, which all resolved within days to weeks. Data presented is available on the "Publications" section of the REGENXBIO website at . About ABBV-RGX-314 ABBV-RGX-314, being developed in collaboration with AbbVie, is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. ABBV-RGX-314 consists of the NAV® AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). ABBV-RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina. REGENXBIO is advancing research in two separate routes of administration of ABBV-RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye. About Wet AMD Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long frequent, repeated intraocular injections to maintain efficacy. Due to the burden of treatment, it is difficult for patients to adhere to frequent injections, which can lead to a decline in vision over time. ABOUT REGENXBIO Inc. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit . FORWARD-LOOKING STATEMENTS This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2023, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at . All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Zolgensma® is a registered trademark of Novartis Gene Therapies. SCS Microinjector® is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO. CONTACT: Dana Cormack Corporate Communications [email protected] George E. MacDougall Investor Relations [email protected] SOURCE REGENXBIO Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clearside Biomedical reported positive results from a Phase 2b trial of its tyrosine kinase inhibitor in patients with wet age-related macular degeneration, bolstering its prospects of bringing a different approach to an eye disorder that has been dominated by anti-VEGFs. In the Phase 2b, Clearside’s CLS-AX injection met the primary endpoint of stable best corrected visual acuity from baseline to 36 weeks in people with wet AMD, according to a Wednesday release . The company’s shares $CLSD were up by as much as 30% premarket Wednesday. CLS-AX also met the study’s secondary endpoints, including reducing injection frequency by around 84% over six months compared with the average number of anti-VEGF injections patients had in the 24 weeks prior to screening. None of the CLS-AX patients needed additional dosing of anti-VEGF drugs at up to 12 weeks. At up to 24 weeks, 67% of the patients didn’t need additional dosing, which is when CLS-AX was redosed. The eye disorder occurs when abnormal blood vessels grow in the macula in the retina, leading to blurred vision or a blind spot. CLS-AX is a “proprietary suspension” of the TKI axitinib for injection into the area between the sclera and choroid in the back of the eye. Originally developed by Pfizer, axitinib won FDA approval in 2019 to treat renal cell carcinoma with the brand name Inlyta. Clearside said planning for a Phase 3 study is already underway. The company believes the pan-VEGF blockade triggered by TKIs could unlock better efficacy versus existing drugs, which tend to target VEGF-A only. VEGF approaches like Regeneron’s Eylea and Novartis’ Lucentis are often administered once every four weeks to begin with and less often after, but CLS-AX is being tested with a less frequent dosing regimen right from the start. The 60-participant Phase 2b used Eylea as an active control. CLS-AX has the potential “to significantly decrease anti-VEGF treatment burden for patients with wet AMD,” Chardan analysts said in August. The same month, Stifel analysts described the Phase 2b trial design as “thoughtful” because it allows for retreatment with anti-VEGFs, which should “generate more clinically relevant data to guide real-world practice.” Safety wise, CLS-AX was well-tolerated in the Phase 2b, with no serious adverse events observed and similar discontinuation rates between the treatment and control arms.