Alonso, Ra Pharmaceuticals, Inc
A Macrocyclic Peptide for Complement-Mediated Diseases
Inappropriate activation of complement C5 leads to RBC destruction in the rare, acquired disease paroxysmal nocturnal hemoglobinuria (PNH), and has been implicated in other serious indications. RA101495 is a potent, synthetic, macrocyclic peptide that binds C5 and inhibits its activation through a novel mechanism. A Phase 1 study in healthy volunteers has been completed, supporting the advancement of this product into Phase 2 studies in patients with PNH and other indications.