Pegcetacoplan Shows Benefit for Macular Function in Geographic Atrophy

Dr. Jaclyn L. Kovach presented new analyses at ARVO 2024, highlighting ANX007's beneficial effects on macular function in patients with geographic atrophy. The Phase 2 ARCHER study demonstrated broad-based protection against vision loss, with plans for pivotal Phase 3 trials in 2024.

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Pegcetacoplan Shows Benefit for Macular Function in Geographic Atrophy

Pegcetacoplan Shows Benefit for Macular Function in Geographic Atrophy

Dr. Jaclyn L. Kovach presented new neuroprotective analyses at the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting in Seattle, highlighting the beneficial effects of continuous pegcetacoplan treatment on macular function in patients with geographic atrophy (GA). The data, from the Phase 2 ARCHER study of ANX007, a first-in-class non-pegylated antigen-binding fragment (Fab), demonstrated broad-based protection against vision loss.

Why this matters: This breakthrough in treating geographic atrophy could lead to improved vision outcomes for millions of people worldwide, and its implications could extend to other diseases involving the classical complement pathway. As the global population ages, innovative treatments like ANX007 will play a critical role in addressing the growing burden of age-related diseases.

Key findings from the study included dose-dependent and time-dependent protection in Best-Corrected Visual Acuity (BCVA) 15-letter loss, statistically significant protection of Low Light Visual Acuity (LLVA), reduction of photoreceptor loss measured by the Ellipsoid Zone (EZ) layer via Optical Coherence Tomography (OCT), and meaningful slowing of lesion growth near the fovea. New preclinical data also showed that C1q blockade protected photoreceptor synapses, cells, and retinal function in a model of photoreceptor damage.

ANX007, developed by Annexon Inc., is designed to block C1q and activation of the classical complement cascade locally in the eye with an intravitreal formulation. The company plans to initiate the global pivotal Phase 3 ARCHER II trial vs sham control in mid-2024 and the pivotal Phase 3 head-to-head ARROW trial vs SYFOVRE pegcetacoplan injection in the second half of 2024.

"2024 is an exciting and potentially transformative year for Annexon with multiple value-creating catalysts culminating from a decade of research in developing treatments designed to stop harmful classical complement pathway inflammation where it starts," said Douglas Love, President and CEO of Annexon. The company, which is advancing a late-stage clinical platform of novel therapies for complement-mediated neuroinflammatory diseases, reports having approximately $264.9 million in cash and cash equivalents as of March 31, 2024, with an anticipated runway into mid-2026.

The promising Phase 2 results for ANX007 in GA, along with the planned initiation of pivotal Phase 3 trials, position Annexon as a key player in the development of innovative treatments for complement-driven diseases. The data presented by Dr. Kovach at ARVO 2024 provide strong evidence for the neuroprotective effects of pegcetacoplan in preserving visual function and retinal structures in patients with geographic atrophy.

Key Takeaways

  • ANX007 shows promise in treating geographic atrophy, a leading cause of vision loss.
  • Treatment slows vision loss, protects photoreceptors, and reduces lesion growth.
  • Phase 3 trials to begin in 2024, with potential to transform treatment of age-related diseases.
  • ANX007 blocks C1q and classical complement cascade, reducing inflammation in the eye.
  • Annexon has $264.9M in cash, with runway into mid-2026, to advance its clinical platform.