ANX007 / Annexon Biosci - LARVOL DELTA

Annexon Reports First Quarter 2025 Financial Results, Portfolio Progress and Key Anticipated Milestones (GlobeNewswire) - "FDA Meeting for Tanruprubart (formerly ANX005), the First Potential Targeted Therapy for GBS, Scheduled for Second Quarter 2025 Ahead of Planned BLA Submission; Open-Label Tanruprubart FORWARD Study Designed to Broaden Patient and Healthcare Community Experience in North America and Europe, Initiating in Second Quarter 2025; Accelerated Enrollment in Phase 3 ARCHER II Trial on Pace for Completion in Third Quarter 2025 for ANX007, the First Potential Treatment for Dry AMD with GA; Pivotal Topline Data Expected in the Second Half of 2026."

FDA event • New trial • P3 data: top line • Age-related Macular Degeneration • Immunology

Annexon to Present on the Neuroprotective Effects of ANX007 at the 2025 ARVO Annual Meeting and the Retina World Congress (GlobeNewswire) - "Phase 2 ARCHER Data Support Therapeutic Potential for ANX007 to Preserve Vision in Patients who have Dry Age-related Macular Degeneration (AMD) with Geographic Atrophy (GA). Phase 3 ARCHER II Trial Enrolling Globally....Annexon...today announced presentations on the neuroprotective effect of ANX007, including consistent benefits of C1q inhibition against inflammation and neuronal damage across diseases. The presentations will take place at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting being held May 4-8 in Salt Lake City, Utah and at the 2025 Retina World Congress being held May 8-11 in Fort Lauderdale, Florida....ARCHER II is a global, randomized, double-masked, sham-controlled Phase 3 trial expected to enroll approximately 630 patients with geographic atrophy (GA)....Topline data are expected in the second half of 2026."

Clinical data • P2 data • P3 data • Age-related Macular Degeneration • Huntington's Disease • Ophthalmology

Microglia-induced neuronal injury attenuation with C1q Inhibition: Outcomes in Geographic Atrophy (GA) and Huntington's Disease (HD) (ARVO 2025) - P2, P2a | "Methods In ARCHER (NCT04656561), 270 patients were randomized 1:1:1 to intravitreal (IVT) administration of ANX007 5 mg monthly (EM), 5 mg every other month (EOM), or matched sham for 12 months...Describe the big picture and the implications of your findings, not the study itself and the associated details. This study utilizes two prospectice studies to demonstrate that inhibition of C1q preserves visual acuity and reduces ellipsoid zone loss in geographic atrophy and improves clinical function measures in Huntington's Disease (HD), suggesting that anti-C1q therapy may provide neuroprotection against inflammation and neuronal damage and loss induced by downstream complement components."

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology

Annexon Announces Presentations Highlighting ANX007 Functional and Structural Differentiation in Geographic Atrophy at the Macula Society 48th Annual Meeting (GlobeNewswire) - "Annexon, Inc...today announced presentations on ANX007 in geographic atrophy (GA) at the Macula Society 48th Annual Meeting being held February 12-15 in Charlotte Harbor, Florida."

P2 data • Age-related Macular Degeneration

Annexon Provides 2025 Outlook with Strong Momentum Accelerating into Breakthrough Year (GlobeNewswire) - "ANX005 for GBS: Next Milestone: BLA Submission targeted for first half of 2025....ANX007 in GA: Phase 3 ARCHER II trial enrollment to be completed in second half of 2025; data expected in second half of 2026....ANX1502 for Autoimmune Conditions: Next Milestone: Clinical proof of concept data in CAD and update on future target indications in first quarter of 2025."

Clinical data • Enrollment status • FDA filing • P3 data • Immunology • Ophthalmology • Rare Diseases

Preservation of Vision by ANX007: Clinical Results and Anatomical Changes From the Phase 2 ARCHER Trial (AAO 2024) - "Ellipsoid zone area loss and BCVA 15-letter loss were reduced in ANX007-treated versus sham-treated eyes. The results suggest a drug-related photoreceptor protective effect."

Clinical • P2 data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology

ARCHER II: A Study Investigating the Efficacy and Safety of Intravitreal (IVT) Injections of ANX007 in Participants With Geographic Atrophy (GA) (clinicaltrials.gov) - P3 | N=630 | Recruiting | Sponsor: Annexon, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

ARCHER: A Study Investigating the Efficacy and Safety of Intravitreal Injections of ANX007 in Patients With Geographic Atrophy (clinicaltrials.gov) - P2 | N=270 | Completed | Sponsor: Annexon, Inc. | Active, not recruiting ➔ Completed | Trial completion date: Dec 2023 ➔ Sep 2023

Trial completion • Trial completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

ARCHER II: A Study Investigating the Efficacy and Safety of Intravitreal (IVT) Injections of ANX007 in Participants With Geographic Atrophy (GA) (clinicaltrials.gov) - P3 | N=630 | Not yet recruiting | Sponsor: Annexon, Inc.

New P3 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Annexon Reports First Quarter 2024 Financial Results and Key Anticipated Milestones (GlobeNewswire) - "ANX005 in GBS: Topline data from the pivotal, randomized, placebo-controlled Phase 3 trial expected in the second quarter of 2024. Initial data from RWE comparability protocol with IGOS expected in first half 2025 to support a planned BLA submission. ANX007 in GA: Global pivotal Phase 3 ARCHER II trial vs. sham control expected to initiate in mid-2024. Pivotal Phase 3 head-to-head ARROW trial vs. SYFOVRE (pegcetacoplan injection) planned to initiate in the second half of 2024. Expect to host an R&D Day on GA and therapeutic potential of ANX007 in mid-2024. ANX1502 in CAD: POC trial evaluating the pharmacodynamics and efficacy of an oral tablet formulation in CAD anticipated to provide initial data in the second half of 2024....R&D expenses were $21.0 million for the quarter ended March 31, 2024, reflecting the advancement of the Company’s priority programs, including GBS, GA and ANX1502, compared to $32.3 million for the quarter ended March 31, 2023."

Clinical data • Commercial • New P3 trial • P3 data: top line • Immunology • Ophthalmology • Rare Diseases

Protection Against Vision Loss by ANX007: Results from the Phase 2 ARCHER Clinical Trial (ARVO 2024) - "ARCHER is the first clinical trial demonstrating visual function protection through 12 months of treatment. Lesion type did not drive the ANX007 protective effect against vision loss. ANX007 is effective in patients with both foveal and non-foveal involvement.These ARCHER findings support a broad-based neuroprotective mechanism of ANX007 in preserving photoreceptor synapses, cells and retinal function."

Clinical • P2 data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology

Treatment of Geographic Atrophy Secondary to AMD With Intravitreal ANX007, a Selective Classical Complement Inhibitor: Results of the ARCHER Study (AAO 2023) - No abstract available

Late-breaking abstract • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology

Treatment of Geographic Atrophy Secondary to Age-Related Macular Degeneration With Intravitreal ANX007: Results of the ARCHER Study (ASRS 2023) - No abstract available

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Safety and Target Engagement of Complement C1q Inhibitor ANX007 in Neurodegenerative Eye Disease: Results from Phase I Studies in Glaucoma. (PubMed, Ophthalmol Sci) - "Further investigation in neurodegenerative ocular diseases is warranted. Proprietary or commercial disclosure may be found after the references."

Journal • P1 data • CNS Disorders • Glaucoma • Ophthalmology • C1Q

Pharmacokinetic and Target Engagement Measures of ANX007, an Anti-C1q Antibody Fragment, Following Intravitreal Administration in Nonhuman Primates. (PubMed, Invest Ophthalmol Vis Sci) - "Based on its mechanism of action inhibiting C1q and its downstream activity, ANX007 is predicted to mitigate tissue damage driven by classical complement activation in the retina. These data support further clinical evaluation of ANX007."

Journal • PK/PD data • Age-related Macular Degeneration • CNS Disorders • Dry Age-related Macular Degeneration • Glaucoma • Ophthalmology

ARCHER: A Study Investigating the Efficacy and Safety of Intravitreal Injections of ANX007 in Patients With Geographic Atrophy (clinicaltrials.gov) - P2 | N=270 | Active, not recruiting | Sponsor: Annexon, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Inhibition of C1q Protects Photoreceptor Synapses in a Light Damage Model and is a Potential Treatment for Geographic Atrophy (GA) (Macula 2022) - "We provide the first evidence of C1q deposition on photoreceptor synapses in a light exposure model of retinal damage in mice and in human GA tissue. Preliminary results suggest that inhibiting C1q protects against photoreceptor neuron damage. A randomized, multi-center, double-masked Phase 2 trial to evaluate the efficacy and safety of intravitreal ANX007 in reducing the area of GA, as evaluated by fundus autofluorescence, is underway."

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Immunology • Inflammation • Retinal Disorders

Evaluating Intravitreal ANX007, a Novel C1q Inhibitor, in the Treatment of GA Secondary to AMD: The ARCHER Study (AAO 2021) - "Conclusion Data supports evaluation of ANX007 for treatment of GA. A global, randomized Phase 2 trial to evaluate ANX007 (monthly or bimonthly) in reducing area of GA is under way."

Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Glaucoma • Ophthalmology

[VIRTUAL] Pharmacokinetics and target engagement of intravitreal administration of ANX007, an anti-C1q antibody fragment, in nonhuman primates (ARVO 2021) - "Following IVT administration, ANX007 distributes to relevant sites of neurodegenerative ophthalmic disease within the retina, with clear evidence of C1q target engagement. Engagement of C1q in the aqueous humor reflects inhibition in the vitreous and retinal tissue, and is hypothesized to be sufficient to mitigate classical complement activation in neurodegenerative ophthalmic disease. This data supports further clinical evaluation of ANX007 for the treatment of such diseases."

PK/PD data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Glaucoma • Ophthalmology

Annexon Advances Classical Complement Platform with Initiation of Global Phase 2 ARCHER Trial in Patients with Geographic Atrophy (GlobeNewswire) - P2, N=240; NCT04656561 (ARCHER); Sponsor: Annexon, Inc.; "Annexon, Inc...announced that patient dosing has begun in its Phase 2 ARCHER study of its anti-C1q therapy, ANX007, to treat Geographic Atrophy (GA). GA, also known as atrophic age-related macular degeneration (AMD) or dry AMD, can lead to blindness caused by damaged and dying retinal cells. Currently, there are no approved treatment options to prevent onset or progression of GA. 'Geographic atrophy can have a profound impact on patients who lose their ability to drive, read and carry out normal daily functions,' said Peter K. Kaiser, M.D., Professor of Ophthalmology, Cole Eye Institute, Cleveland Clinic. 'Targeting aberrant complement activity associated with the GA process has demonstrated great promise. Evaluating treatments that may reduce the rate of GA lesions growth and preserve visual function is important for these patients that have no treatment options today."

Media quote

A Study Investigating the Efficacy and Safety of Intravitreal Injections of ANX007 in Patients With Geographic Atrophy (clinicaltrials.gov) - P2; N=240; Recruiting; Sponsor: Annexon, Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

A Study Investigating the Efficacy and Safety of Intravitreal Injections of ANX007 in Patients With Geographic Atrophy (clinicaltrials.gov) - P2; N=240; Not yet recruiting; Sponsor: Annexon, Inc.

Clinical • New P2 trial • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders