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Complement inhibition as a therapeutic strategy in retinal disorders
Enoch Kassa1, Thomas A Ciulla2, Rehan M Hussain3
1a Department of Ophthalmology , Indiana University School of Medicine , Indianapolis , IN , USA.
Expert Opinion on Biological Therapy
|January 29, 2019
Summary
Complement inhibition shows promise for treating dry age-related macular degeneration (AMD) and Stargardt disease, though halting geographic atrophy (GA) progression requires further investigation.
Area of Science:
- Ophthalmology
- Immunology
- Genetics
Background:
- Dry age-related macular degeneration (AMD) and Stargardt Macular Dystrophy (STGD1) cause progressive vision loss due to macular atrophy.
- Complement-associated inflammation is implicated as a key factor in the pathogenesis of both diseases.
Purpose of the Study:
- To review the current landscape of complement inhibitors for treating dry AMD and STGD1.
- To assess the efficacy and safety of various complement inhibition strategies in clinical trials.
Main Methods:
- Review of clinical trial data for complement inhibitors targeting factors D, C3, C5, and properdin.
- Analysis of outcomes from Phase 1, 2, and 3 trials investigating these therapies for geographic atrophy (GA).
Main Results:
- Several complement inhibitors, including lampalizumab (factor D) and eculizumab (C5), failed to halt GA progression in Phase 3 or 2 studies.
- APL-2 (C3 inhibitor) demonstrated potential in reducing GA growth in Phase 2, advancing to Phase 3.
- Avacincaptad pegol (C5 inhibitor) and gene therapy approaches are under investigation in early-phase trials.
Conclusions:
- Complement inhibition has not yet definitively halted GA progression in Phase 3 trials for dry AMD and STGD1.
- Further research and clinical trials are warranted to explore the therapeutic potential of complement inhibitors, including novel C3 inhibitors and gene therapy.
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