A new review article titled “Compstatin: a C3-targeted complement inhibitor reaching its prime for bedside intervention.” by Mastellos et al., has been accepted for publication in the European Journal of Clinical Investigation. The review analyzes why complement component C3 represents an attractive target for therapeutic modulation of the complement cascade and describes in detail the optimization effort which has led to the structure-function refinement of compstatin and its novel derivatives such as AMY-101, with improved inhibitory potency, pharmacokinetic profile and efficacy in a number of clinically relevant primate models of disease. The review discusses translational challenges in complement drug discovery and peptide drug development and analyzes concerns related to systemic C3 interception; despite existing challenges, AMY-101 emerges as a highly promising complement inhibitor therapeutic.
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