Achillion Pharmaceuticals reports the initiation of a phase 1 study with ACH-4471, the Company’s first orally-administered, highly potent and specific small molecule that has been shown to inhibit complement factor D. The phase 1 study is being conducted in healthy volunteers and is evaluating single ascending doses of ACH-4471 to assess safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD). The Company expects to report interim clinical data from this study during the second quarter of 2016.

“We believe our molecular-level understanding of factor D, based upon proprietary high-resolution x-ray structures, has enabled us to design ACH-4471 with the potential to be the first orally-bioavailable, highly differentiated treatment for PNH and other ultra-rare diseases. We look forward to advancing ACH-4471, with the goal of demonstrating its safety, tolerability, proof-of-mechanism and working to establish its potential benefit for patients,” commented Milind Deshpande, Ph.D., President and Chief Executive Officer.