First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH

Summary

This is a 3-part Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single (Part 1) and multiple (Part 2) ascending doses of BCX9930 in healthy subjects and in subjects with paroxysmal nocturnal hemoglobinuria (PNH; Part 3). Pharmacokinetics is an analysis of how the body handles the study drug BCX9930 and pharmacodynamics is an analysis of the activity that the study drug BCX9930 may have in the body.

Timeline

Start

2020-03-03

Primary completion

2020-11-11

Completion

2021-01-25

Outcome

Results reported · no primary-endpoint met/missed verdict (typical of a dose-finding / safety study)

release “In an ongoing dose ranging trial of BCX9930 in patients with PNH, BCX9930 was safe and well tolerated” biocryst.com ↗

Drugs

Indications