Clinical Trials

Date: 2017-04-06

Type of information: Treatment of the first patient

phase: 2

Announcement: treatment of the first patient

Company: Achillion Pharmaceuticals (USA - CT)

Product: ACH-4471

Action mechanism: factor D inhibitor. ACH-4471 is a potent and highly specific factor D inhibitor. Primary and secondary pharmacology studies, as well as non-clinical toxicity studies up to 28 days, have been completed. ACH-4471 was dosed orally in non-human studies and demonstrated the ability to block the activation of the alternative pathway. ACH-4471 was been shown to substantially decrease hemolysis and C3b deposition on red blood cells from patients with PNH, and achieved dose-dependent inhibition of cell killing in Atypical hemolytic uremic syndrome (aHUS) in the modified HAM assay. Achillion initiated a first-in-human trial with ACH-4471 in February 2016.

Disease: paroxysmal nocturnal hemoglobinuria (PNH)

Therapeutic area: Rare diseases - Immunological diseases

Country: New Zealand

Trial details: The purpose of this study is to determine the safety and effectiveness of ACH-0144471 in currently untreated patients with paroxysmal nocturnal hemoglobinuria. (NCT03053102)

Latest news:

• • On April 6, 2017, Achillion Pharmaceuticals announced initiation of patient dosing in a Phase 2 open-label study of ACH-4471, Achillion's first orally-administered, small molecule factor D inhibitor, for patients with paroxysmal nocturnal hemoglobinuria (PNH). This proof of concept study will assess the efficacy, safety, and pharmacokinetics of ACH-4471 in untreated patients with PNH.
• The primary objective of the study is to assess the change-from-baseline in serum lactate dehydrogenase (LDH) levels, a sensitive biomarker for intravascular hemolysis, during 28 days of dosing. The protocol allows for intra-patient dose-escalation with patients initially receiving 100 mg three times daily of ACH-4471 with the ability to increase dosage during the treatment period. Secondary endpoints being assessed include changes in hemoglobin and red blood cell levels, complement pathway biomarkers, such as Bb and factor D, levels, pharmacokinetics, and safety. Interim results from this study are anticipated during the second quarter of 2017.

 

Is general: Yes