NEW HAVEN, CT, USA I December 20, 2017 I Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN), a pharmaceutical company focused on advancing small molecule inhibitors of factor D in the complement alternative pathway, today announced that the Company has begun dosing subjects in a first-in-human phase 1 trial of ACH-5228, a next-generation, oral small-molecule factor D inhibitor.

“During 2017, we reported interim data from our on-going phase 2 clinical trials, in PNH and in C3G, which demonstrated that factor D inhibition represents a truly novel, first-in-class approach to potentially treating these devastating diseases. As we continue to execute on our global clinical development strategy with ACH-4471, our next-generation compounds, including ACH-5228, represent strategic optionality as we look to expand the breadth of diseases that could potentially be treated via AP inhibition,” commented Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion.

ACH-5228: Phase 1 Healthy Volunteer Development Program

This initial phase 1 trial is a randomized, placebo-controlled, single-ascending dose study of ACH-5228 administered to healthy volunteers. Approximately 28 subjects are expected to be enrolled. The primary endpoint for the trial is evaluation of safety and tolerability. Secondary endpoints include assessments of PK, PD, and evaluation of alternative pathway inhibition in ex vivo laboratory assessments of blood samples from subjects in order to establish a PK/PD relationship for ACH-5228. The Company expects to report interim clinical data from this study during the second half of 2018. (ANZCTR UTN: U1111-1203-1371)

About the Achillion Complement Factor D Platform

Achillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop small molecule drug candidates that are oral inhibitors of complement factor D. Factor D is an essential serine protease involved in the complement pathway, a part of the innate immune system. Achillion’s complement platform is focused on seeking to advance small molecule compounds that inhibit factor D and can potentially be used in the treatment of immune-related diseases in which complement alternative pathway plays a critical role. Potential indications being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN), and geographic atrophy (GA).

About Achillion Pharmaceuticals

Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) is a science-driven, patient-focused company seeking to leverage its strengths across the continuum from discovery to commercialization in its goal of providing better treatments for people with serious diseases. The company employs a highly-disciplined discovery and development approach that has allowed it to build a platform of potent and specific complement inhibitors. Achillion is rapidly advancing its efforts to become a fully-integrated pharmaceutical company with a goal of bringing life-saving medicines to patients with rare diseases. More information is available at

SOURCE: Achillion Pharmaceuticals