Achillion Pharmaceuticals, Inc. announced that it achieved threshold enrollment in the Phase 2 trials of ACH-4471 for patients with C3 glomerulopathy (C3G), a devastating disease affecting the kidney for which there is no approved therapy. ACH-4471 will be evaluated in the six-month blinded, placebo-controlled trial (11 patients) and the 12-month open-label trial (12 patients). About the ACH-4471 Phase 2 C3G Trials: The objective of the six and 12-month proof-of-concept trials is to evaluate the safety and efficacy of the oral, small molecule factor D inhibitor, ACH-4471, in patients diagnosed with C3G. In the six-month trial, patients are blinded and randomized 1:1 to receive either ACH-4471 or placebo. The 12-month trial is open label. The measures being evaluated include changes in clinical manifestations of C3G including proteinuria and estimated glomerular filtration rate (eGFR) at the end of the treatment period and changes in kidney biopsy from baseline. ACH-4471, Complement Factor D Inhibitor: The Company’s first-generation oral complement factor D inhibitor, ACH-4471, is currently being evaluated for efficacy with Phase 2 clinical programs in both paroxysmal nocturnal hemoglobinuria (PNH) and C3G and has demonstrated preliminary proof-of-concept in both indications. The PNH program consists of a Phase 2 clinical trial evaluating ACH-4471 in patients who are inadequately controlled or sub-optimally responding to eculizumab. Additionally, patients continue to dose in the PNH monotherapy extension trial.