Annexon Outlines 2024 Priorities with Late-Stage Clinical Milestones Across Upstream Complement Portfolio for Autoimmune, Ophthalmic and Neurodegenerative Diseases

ANX005 Phase 3 Pivotal Data in Guillain-Barré Syndrome (GBS) On Track for First Half of 2024;
Potential to be First Approved Treatment for GBS Patients in the U.S.

ANX007 Global Pivotal Program in Geographic Atrophy (GA) to Initiate Mid-2024;
First Pivotal Trial Using Vision Preservation as Primary Outcome Measure in GA

Clinical Proof-of-Concept Study of ANX1502 First-in-Kind Oral Small Molecule Inhibitor of the Classical Pathway to Initiate in First Half of 2024

Recent Successful $125 Million Financing Extends Operating Runway into Second Quarter 2026

BRISBANE, Calif., Jan. 07, 2024 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement-based medicines for people living with devastating inflammatory-related diseases, today outlined its strategic priorities for 2024 with late-stage clinical milestones, including ANX005 for Guillain-Barré syndrome (GBS), ANX007 for geographic atrophy (GA) and its first-in-kind oral small molecule complement inhibitor, ANX1502, for a range of autoimmune indications.

“Our distinct classical complement portfolio has been built over 10 years of research focused on stopping C1q-driven inflammation activated on diseased tissue in complement-mediated diseases of the body, brain and eye. The robust and consistent functional outcome data generated by our flagship and next wave programs has reinforced our founding thesis of stopping the classical complement disease process where it starts,” said Douglas Love, president and CEO of Annexon.

“This is a pivotal time for Annexon, with registration programs for our two lead candidates, numerous late- and mid-stage clinical catalysts expected across our portfolio, and a strong balance sheet to fuel our priority programs to meaningful inflection points. We are proud of what we’ve accomplished over our decade-long history and are excited by the potential to achieve our goal of bringing transformative therapies to millions of patients with debilitating autoimmune, ophthalmic and neurodegenerative diseases.”

2024 Strategic Priorities
Annexon is sharply focused on mid- to late-stage development of three value-driving programs:

ANX005 for GBS: Report Pivotal Data from Phase 3 Trial in GBS in First Half of 2024

• GBS is a serious autoimmune condition of the nervous system that can lead to sudden paralysis and even death in otherwise healthy individuals. GBS impacts more than 12,000 people annually in the U.S. and Europe, and there are currently no approved therapies for GBS in the U.S.
• Annexon is conducting a randomized, double-blind, placebo-controlled, multi-center Phase 3 clinical trial (N=241) designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of ANX005 in patients with GBS. The study completed enrollment in 2023 and the company is on-track to report Phase 3 data in the first half of 2024.
• ANX005 was granted orphan drug designation by the EMA for the treatment of GBS based on a meta-analysis of past studies with ANX005 and intravenous immunoglobulin (IVIg). In a completed proof-of-concept, placebo-controlled Phase 1b trial, ANX005 showed rapid and consistent improvement in muscle strength that translated into observable gains in health status, including a reduction in the need of mechanical ventilation, as well as improvement in neuronal damage and clinical function in patients with GBS.