Vonaprument

Expedited Enrollment Surpassing 630 Targeted Participants Underscores the Strong Demand for a Therapy Focused on Vision Preservation Phase 3 ARCHER II Trial Design and Rationale to Be Presented at ASRS, a Leading Meeting for Retina Science and Innovation Topline Pivotal Phase 3 Data Expected in the Second Half of 2026 Potential for Vonaprument to Be the First Treatment Approved in Europe and the U.S. for Dry AMD with Geographic Atrophy Based on Protection of Visual Acuity and Structural Measures BRISBANE, Calif., July 24, 2025 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company advancing a late-stage clinical platform of novel therapies for people living with devastating classical complement-mediated neuroinflammatory diseases of the body, brain, and eye, today announced complete enrollment of the Phase 3 ARCHER II trial of vonaprument (formerly ANX007) for dry age-related macular degeneration (AMD) with geographic atrophy (GA), and the company remains on track to report topline data in the second half of 2026. Vonaprument is a first-in-kind, non-pegylated antigen-binding fragment (Fab) designed to block C1q locally in the eye with an intravitreal formulation. The neuroprotective effect and consistent benefits of C1q inhibition were previously demonstrated by vonaprument in a Phase 2 trial. Vonaprument is the only investigational therapy for dry AMD with GA to show significant vision preservation based upon best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA). ARCHER II is a global, randomized, double-masked, sham-controlled pivotal trial designed to assess both visual acuity and structural measures to satisfy the global registration path in the U.S. and Europe. Vonaprument has received Priority Medicine (PRIME) designation in Europe and Fast Track designation in the U.S. “Achieving and surpassing our enrollment goal ahead of schedule in ARCHER II highlights the strong interest for a treatment that can help patients preserve their vision,” said Douglas Love, president and chief executive officer of Annexon. “ARCHER II is designed to confirm the protection of visual acuity and visual structures demonstrated previously in our Phase 2 trial, and we look forward to reporting topline pivotal data in the second half of 2026. The power of our classical complement platform continues to achieve differentiated functional benefits across indications, and we remain committed to our goal of making and shaping markets with first-in-kind medicines that will help millions of patients live their best lives.” An oral presentation by David Eichenbaum, M.D., Director of Research at the Retina Vitreous Associates of Florida on the design and rationale of the pivotal Phase 3 ARCHER II trial will take place on Thursday, July 31, 2025, at the American Society of Retina Specialists (ASRS) annual meeting held in Long Beach, CA. Dr. Eichenbaum, a Principal Investigator of the ARCHER II trial, added, “The global reach of ARCHER II has increased awareness of vonaprument’s innovative mechanism and compelling Phase 2 results. With no approved therapies in Europe and limited options in the U.S., there is an urgent need for a treatment for GA that protects vision, the most clinically meaningful outcome for patients. I’m encouraged by the robust design of ARCHER II and look forward to helping advance this differentiated therapy worldwide.” ASRS Annual Meeting Oral Presentation Dry AMD Symposium 2 “ARCHER II, a Phase 3 Randomized Clinical Trial of ANX007 in Patients With Dry AMD and GA: Study Design and Rationale Informed by Outcomes From ARCHER” Presenter: David A. Eichenbaum, MD, FASRS, Retina Specialist, Retina Vitreous Associates of Florida Date/Time: Thursday, July 31 at 2:59 pm Pacific Time (PT) About Phase 3 ARCHER II Trial ARCHER II is a global, randomized, double-masked, sham-controlled Phase 3 trial that has enrolled more than 630 patients with advanced dry AMD/geographic atrophy (GA). Patients will be randomized 2:1 to receive a monthly dose of vonaprument or sham procedure. The primary endpoint is the prevention of ≥15-letter loss of best corrected visual acuity (BCVA), which represents three lines on the standard Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart. The primary analysis will occur at least 12 months from dosing initiation. Proportion of patients experiencing BCVA ≥15-letter loss is a well-established functional endpoint that has served as the basis for numerous ophthalmology drug approvals by the Food and Drug Administration (FDA) and European Medicines Agency (EMA). Secondary endpoints in ARCHER II include safety, low-luminance visual acuity (LLVA), and photoreceptor integrity (EZ). Topline data are expected in the second half of 2026. About Vonaprument (formerly ANX007) and Phase 2 ARCHER Trial Vonaprument is an antigen-binding fragment (Fab) antibody designed as a first-in-kind therapeutic to selectively inhibit C1q, the initiating molecule of the classical complement pathway and a key driver of neurodegeneration. In advanced dry age-related macular degeneration (AMD) or geographic atrophy (GA), C1q binds to photoreceptor synapses, causing aberrant activation of the classical pathway with synapse loss, inflammation and neuronal damage that results in vision loss. Intravitreal administration of vonaprument fully stopped C1q and classical pathway activation. In animal models, the murine analog of vonaprument protected against loss of photoreceptor synapses and cells to preserve function. Vonaprument has been granted Fast Track designation from the Food and Drug Administration and is the first therapeutic candidate for the treatment of GA to receive Priority Medicine (PRIME) designation in the EU, which provides early and proactive support to developers of promising medicines that may offer a major therapeutic advantage over existing treatments or benefit to patients without treatment options. In the randomized, multi-center, double-masked, sham-controlled Phase 2 ARCHER clinical trial, vonaprument demonstrated consistent protection against vision loss across multiple measures in a broad population of patients with dry AMD and GA. Vonaprument provided statistically significant, time and dose-dependent protection from vision loss as measured by ≥ 15 letter loss on reading an eye chart with best corrected visual acuity (BCVA≥15), the widely accepted and clinically-meaningful functional endpoint. Significant protection from vision loss was also shown in other prespecified measures of BCVA and visual function, including low luminance visual acuity (LLVA) and low luminance visual deficit (LLVD). Vonaprument’s treatment effect increased over the course of the on-treatment portion of the study, suggesting that vonaprument may provide a growing and durable treatment effect over time. While benefit gained against vision loss was maintained during the subsequent six-month off-treatment period, the rate of decline for BCVA ≥ 15-letter vision after treatment termination began to parallel that of sham, providing additional support for the observed on-treatment protection. Vonaprument was also shown to protect key retinal structures important for vision, including significant protection of photoreceptors as measured by optical coherence tomography (OCT) and supported by slowing of loss of retinal pigment epithelial cells (RPE) near the fovea, as measured by fundus autofluorescence (FAF). Vonaprument was generally well-tolerated through month 12, with no increase in choroidal neovascularization (CNV) rates between the treated and sham arms and no events of retinal vasculitis reported. About Dry AMD and Geographic Atrophy Dry age-related macular degeneration (AMD) is the most common form of AMD and geographic atrophy (GA) is an advanced form of dry AMD, an eye disease that is the leading cause of blindness in the elderly. Dry AMD and GA are chronic progressive neurodegenerative disorders of the retina involving the loss of photoreceptor synapses and cells in the outer retina. GA affects an estimated one million people in the United States and eight million people globally, severely limiting their independence and causing frustration, anxiety and emotional hardship. Effective treatments that preserve vision are still needed, as no currently approved therapies have been shown in clinical trials to significantly prevent vision loss. About Annexon Annexon Biosciences (Nasdaq: ANNX) is developing therapeutics that stop classical complement-driven neurodegeneration as first-in-kind treatments for millions of people living with serious neuroinflammatory diseases of the body, brain and eye. Our novel scientific approach focuses on C1q, the initiating molecule of classical complement’s potent inflammatory pathway that when misdirected can lead to tissue damage and loss. By targeting C1q, our immunotherapies are designed to stop this neuroinflammatory cascade in disease before it starts. Our pipeline spans three diverse therapeutic areas – autoimmune, neurodegenerative and ophthalmic diseases – and includes targeted investigational drug candidates designed to address the unmet needs of over 8 million people worldwide. Annexon’s mission is to deliver game-changing therapies to patients so that they can live their best lives. To learn more visit annexonbio.com. Forward Looking Statements This press release contains 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. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “design,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “positioned,” “potential,” “predict,” “seek,” “should,” “suggest,” “target,” “on track,” “will,” “would” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. All statements other than statements of historical facts contained in this press release are forward-looking statements. These forward-looking statements include, but are not limited to, the ability of vonaprument to block upstream C1q, the clinical and regulatory status of vonaprument; vonaprument’s distinct potential neuroprotective mechanism of action and potential to protect visual acuity and visual structures; the potential therapeutic benefit of vonaprument; timing and results from the Phase 3 ARCHER II trial; the potential for vonaprument to be the first treatment approved in Europe and the U.S. for dry AMD with GA; Annexon’s goal to making and shaping markets with first-in-kind medicines; and Annexon’s ability to rigorously advance mid- to late-stage clinical trials and continue development of the company’s portfolio. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: the ongoing off-treatment follow-up portion of the ARCHER trial and final results from the ARCHER trial; the company’s history of net operating losses; the company’s ability to obtain necessary capital to fund its clinical programs; the early stages of clinical development of the company’s product candidates; the effects of public health crises on the company’s clinical programs and business operations; the company’s ability to obtain regulatory approval of and successfully commercialize its product candidates; any undesirable side effects or other properties of the company’s product candidates; the company’s reliance on third-party suppliers and manufacturers; the outcomes of any future collaboration agreements; and the company’s ability to adequately maintain intellectual property rights for its product candidates. These and other risks are described in greater detail under the section titled “Risk Factors” contained in the company’s Annual Report on Form 10-K and Quarterly Reports on Form 10-Q and the company’s other filings with the SEC. Any forward-looking statements that the company makes in this press release are made pursuant to the Private Securities Litigation Reform Act of 1995, as amended, and speak only as of the date of this press release. Except as required by law, the company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise. Investor Contact: Joyce Allaire LifeSci Advisors, LLC jallaire@lifesciadvisors.com Media Contact: Beth Keshishian 917-912-7195 beth@bethkeshishian.com

GA treatments are advancing rapidly, with oral, gene, and cell therapies emerging as contenders to intravitreal injections in preserving vision. Innovation in GA is no longer a luxury-it’s a necessity. Next-gen therapies must deliver more than anatomical slowing; they must give patients functional hope and real-world relief.” — DataM Intelligence AUSTIN, TX, UNITED STATES, June 9, 2025 / EINPresswire.com / -- Geographic Atrophy (GA) , a late-stage and irreversible form of dry age-related macular degeneration (AMD), has long symbolized a dead end in the journey to preserve sight. Characterized by the progressive degeneration of the retinal pigment epithelium, photoreceptors, and surrounding support tissue, GA leads to central vision loss-a critical blow to quality of life for millions of older adults. As the global population ages, GA’s burden grows heavier, with an estimated 5 million people affected worldwide, including over 1 million in the U.S. alone. Yet, for years, treatment options remained virtually nonexistent. Book Your Free CI Consultation Call: https://www.datamintelligence.com/strategic-insights/ci/geographic-atrophy-ga That paradigm is now shifting. A Landscape Defined by Milestone Approvals Until recently, patients and physicians had no pharmacologic recourse. But in the past two years, the U.S. Food and Drug Administration has greenlit two targeted therapies that mark a significant breakthrough: Syfovre (pegcetacoplan) by Apellis Pharmaceuticals and Izervay (avacincaptad pegol) by Astellas Pharma. Both are intravitreal injections that target the complement system, a key contributor to retinal inflammation and cell death in GA. Syfovre inhibits complement component C3, offering broad suppression across the complement cascade. Izervay, in contrast, selectively inhibits C5, aiming for efficacy with a potentially more favorable safety profile. Their approval represents a long-awaited step forward, transforming a previously untreatable condition into a manageable disease-at least in theory. However, enthusiasm is tempered by real-world complexity. Monthly or bimonthly eye injections carry risks-most notably intraocular inflammation, a safety signal associated with Syfovre-and impose a significant treatment burden on elderly patients. Additionally, while these drugs can slow the progression of GA lesions, they do not restore lost vision or significantly improve functional outcomes like reading speed or contrast sensitivity. The Pipeline Awakens: A New Generation of Therapies As of mid-2025, the treatment landscape is in the midst of a competitive and technological renaissance. More than half a dozen late-stage candidates are racing toward the market, aiming to challenge or complement the status quo. Regeneron’s pozelimab, a C5 inhibitor in Phase III trials, is perhaps the most direct competitor. Leveraging Regeneron’s strong retina portfolio, it poses a high threat to existing market leaders. Meanwhile, Annexon’s ANX007, a C1q inhibitor, represents a mechanistically novel approach targeting upstream complement activity with added neuroprotective benefits. But the most disruptive contenders come from outside the intravitreal injection space. Belite Bio’s Tinlarebant, an oral RBP4 inhibitor, offers a daily pill that sidesteps the logistical and emotional challenges of eye injections. Similarly, Gildeuretinol by Alkeus is exploring the vitamin A pathway with oral systemic delivery aimed at reducing retinal stress. Adding further depth to the field are therapies exploring regenerative and long-term solutions. Roche’s OpRegen, a subretinal cell replacement therapy, seeks to regenerate lost retinal pigment epithelium cells. Johnson & Johnson’s gene therapy candidate JNJ-1887 aims for durable complement inhibition via a single intravitreal injection-a potential game-changer if successful. Market Momentum and the Rise of the Target Opportunity Profile (TOP) With multiple late-phase contenders poised for readouts between 2025 and 2027, developers are increasingly judged by their Target Opportunity Profile (TOP)-a multi-dimensional framework assessing not just efficacy and safety, but also delivery method, durability, innovation, and functional benefit. A high-value therapy in this evolving landscape must go beyond anatomical slowing. It must preserve or improve visual function, minimize side effects, and ideally reduce the frequency of administration. Oral drugs like Tinlarebant and Gildeuretinol score high on patient convenience, while gene and cell therapies offer the tantalizing prospect of single-dose, long-lasting solutions. From a payer and provider perspective, differentiation is key. Novel mechanisms of action such as ANX007’s C1q inhibition or AVD-104’s siglec-targeted nanoparticle technology introduce innovation that could address patient subtypes who are non-responsive to existing drugs. Moreover, dosing frequency and route of administration are taking center stage. The real-world success of any emerging GA therapy hinges on its ability to reduce clinic visits and improve compliance among a predominantly elderly population. Sustained-delivery implants, gene therapies, or oral regimens have clear advantages in this context. Competitive Positioning: A Strategic Chessboard While Syfovre and Izervay currently dominate the market, they face increasing competition from therapies designed for ease-of-use and differentiated clinical value. Regeneron’s pozelimab is a direct competitor with strong corporate backing. Belite’s Tinlarebant and Alkeus’s Gildeuretinol address a different market segment entirely-patients wary of intravitreal injections. Annexon’s ANX007 could appeal to early-stage GA patients thanks to its neuroprotective mechanism. Cell-based and gene therapies, though still in earlier stages, present long-term threats with the potential to redefine treatment expectations. However, scalability, delivery challenges, and cost-effectiveness will be critical hurdles to overcome. A Market Poised for Reimagination The GA market, currently valued in the low billions, is set for robust growth through the end of the decade. But more than financial metrics, it is the transformation in patient experience and clinical possibility that will define this new era. To achieve commercial success and physician buy-in, future GA therapies must offer a balance of safety, efficacy, innovation, and convenience. Emerging treatments will only be as strong as the real-world outcomes they can deliver-preserved independence, improved daily function, and hope for those slowly going blind. Download Free CI Sample Report: https://www.datamintelligence.com/strategic-insights/ci/geographic-atrophy-ga Final Word Geographic atrophy is no longer a therapeutic void. With a rapidly advancing pipeline, the market is shifting from a mindset of damage control to one of proactive preservation-and even potential restoration-of vision. As new entrants race toward approval, they must answer a singular question: Can they improve not just the lesion, but the life behind the lens? Read Related CI Reports: 1. Lupus Nephritis | Competitive Intelligence 2. Immune Thrombocytopenia (ITP) |Competitive Intelligence Sai Kiran DataM Intelligence 4market Research LLP +1 877-441-4866 email us here Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.