The Therapeutic Pipeline for Geographic Atrophy - Summary - MDSpire

The Therapeutic Pipeline for Geographic Atrophy

  • By

  • Benedict Uzorka, BS

  • Jessica D. Randolph, MD

  • March 1, 2026

  • 11 min

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Objective:

To review the current clinical-stage therapies in development for geographic atrophy (GA) associated with age-related macular degeneration (AMD).

Key Findings:
  • Pegcetacoplan and Avacincaptad pegol have shown modest reductions in lesion growth in clinical trials, with specific percentages noted.
  • Vonaprument is currently being tested for its ability to prevent vision loss, with expected outcomes from the ongoing trials.
  • Elamipretide has shown some functional improvements despite not meeting primary endpoints in trials, indicating potential for further research.
  • Gene therapies like JNJ-81201887 and CTx001 are in various stages of clinical evaluation, with specific trial phases and goals outlined.
Interpretation:

The development of therapies for GA reflects a shift towards targeting specific disease mechanisms, particularly through complement inhibition and gene therapy, with the potential for more effective and durable treatments.

Limitations:
  • Long-term safety and efficacy data for new therapies are still pending, which may affect clinical adoption.
  • Some therapies have shown only modest efficacy in reducing lesion growth, raising questions about their overall impact on patient outcomes.
Conclusion:

The therapeutic landscape for GA is evolving with promising investigational therapies that may offer improved outcomes for patients suffering from this debilitating condition.

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