BioCryst Pharmaceuticals Inc (NASDAQ: BCRX) announced results from a dose-ranging trial evaluating BCX9930 in treatment-naïve paroxysmal nocturnal hemoglobinuria (PNH) patients and PNH patients with an inadequate response to C5 inhibitors.

• The results showed that BCX9930 significantly increased hemoglobin and reduced transfusions, was safe and generally well-tolerated in the trial.
• Based on these results, the company plans to advance directly into pivotal trials in PNH and proof of concept trials in renal complement-mediated diseases in the second half of 2021.
• PNH patients in the trial also experienced reductions in key laboratory biomarkers such as reticulocyte count, lactate dehydrogenase (treatment-naïve patients), and percentage of C3 opsonization (patients with inadequate C5 response) following dosing at 400 mg bid or 500 mg bid.
• Hemoglobin levels increased by a mean of 3.5 g/dL in treatment-naïve patients and 3.2 g/dL in C5 inhibitor inadequate response patients; at the last visit, mean hemoglobin levels were 11.8 g/dL and 12.2 g/dL, respectively.
• Relative red blood cell clone size, a marker of hemolytic control, also increased from 53% to 92% in treatment-naïve patients and from 50% to 80% in C5 inhibitor inadequate response patients.
• There were no discontinuations or drug interruptions due to related adverse events. No safety signals were observed.
• Price Action: BCRX shares are trading 2.04% higher at $13.51 in premarket trading on the last check Monday.