december, 2018

17decAll DayACHN Phase 2 ACH-4471

Event Details

ACH-4471 for C3 glomerulopathy (C3G) and Paroxysmal nocturnal hemoglobinuria (PNH). Phase 2 interim data due December 17, 2018.

ACH-4471, Complement Factor D Inhibitor for PNH and C3G

Achillion’s first generation oral factor D inhibitor, ACH-4471 is being evaluated for safety and efficacy with Phase 2 clinical programs in both paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy (C3G).

The PNH program consists of two trials: A Phase 2 clinical trial in untreated PNH patients where ACH-4471 is being assessed as a monotherapy; the second trial is Phase 2 clinical trial evaluating ACH-4471 in patients who are inadequately controlled or sub-optimally responding to eculizumab, which is a therapy for patients with PNH.

The C3G program consists of three currently recruiting Phase 2 clinical trials: a 14-day biomarker study, a six-month blinded, placebo-controlled study, and a 12-month open label study.

More information is available at

ACH-5228 and ACH-5548, Complement Factor D Inhibitors

ACH-5228 and ACH-5548 are next-generation oral factor D inhibitors currently in Phase 1 clinical trials. In preclinical studies, these compounds demonstrated enhanced potency as well as improved pharmacokinetic properties that may allow for a reduced dosing frequency.

Achillion plans to provide interim data on all open-label ACH-4471 clinical trials as well as Phase 1 safety and pharmacokinetics interim data from the single ascending dose trials of its next-generation factor D compounds, ACH-5228 and ACH-5548, on December 17, 2018.

Achillion Pharmaceuticals News


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