Achillion Announces First Dosing in Phase 1 Multiple Ascending Dose Study of ACH-5228 Next-Generation Oral Factor D Inhibitor in Healthy Volunteers

- ACH-5228, with improved potency and half-life, increases alternative pathway inhibition –

- Achillion planning Investigational New Drug (IND) submission in fourth quarter of 2019 -

NEW HAVEN, CT, USA I January 31, 2019 I Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients and families affected by complement-mediated diseases, today announced dosing of the first healthy volunteer in a Phase 1 multiple ascending dose (MAD) study of ACH-5228, one of the company’s next-generation oral, small-molecule factor D inhibitors.

In the completed single ascending dose study, ACH-5228 demonstrated enhanced potency as well as improved pharmacokinetic properties compared to the first-generation factor D inhibitor, ACH-4471. These attributes are projected to provide greater inhibition of the alternative pathway (AP) and reduced dosing frequency for patients with immune-related diseases associated with the AP of the complement system.

“This important clinical milestone for our next-generation oral factor D inhibition program marks an exciting time at Achillion as we pursue safe, effective and innovative treatments for patients with complement-mediated diseases,” said Steven Zelenkofske D.O., Executive Vice President, Chief Medical Officer at Achillion. “With ACH-5228’s profile, we aim to evolve our oral factor D portfolio to enable alternative pathway inhibition throughout a variety of rare diseases. I look forward to working with our clinical development team to potentially broaden treatment options for patients who may benefit by increased therapeutic availability.”

About the ACH-5228 Phase 1 MAD Study
This Phase 1 trial is a randomized, placebo-controlled, multiple-ascending dose study of oral ACH-5228 administered to healthy volunteers outside the United States. Approximately 38 individuals are expected to be enrolled in four separate cohorts. The primary endpoint for the trial is an evaluation of safety and tolerability. Secondary endpoints include assessments of pharmacokinetics (PK), pharmacodynamics (PD), and evaluation of alternative pathway inhibition biomarkers to establish a PK/PD relationship for ACH-5228. Achillion anticipates completing the study in the third quarter of 2019 and submitting an IND to the U.S. Food and Drug Administration supporting continued development for ACH-5228 in the fourth quarter of 2019.

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

About Achillion Pharmaceuticals
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN) is a clinical-stage biopharmaceutical company focused on advancing its oral factor D inhibitors into late-stage development and commercialization. Achillion is initially focusing its drug development activities on alternative pathway-mediated diseases where there are no approved therapies or where existing therapies are inadequate for patients. Potential indications being evaluated for its compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune complex mediated membranoproliferative glomerulonephritis (IC-MPGN). Each of the product candidates in the Company’s oral factor D portfolio was discovered in its laboratories and is wholly owned. To advance its investigational product candidates into Phase 3 clinical trials and commercialization, the Company plans to work closely with key stakeholders including healthcare professionals, patients, regulators and payors. More information is available at

SOURCE: Achillion Pharmaceuticals

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