BioCryst Pharmaceutical Inc. (NASDAQ:BCRX) has released new data from the treatment-naïve paroxysmal nocturnal hemoglobinuria (PNH)patients. This is for the patients who receive doses via 400 mg of oral Factor D inhibitor, BCX9930, in the on-going dose ascending trial.
BCX9930 well tolerated with a favorable safety profile in the study
The company’s Oral BCX993 is driving quick and dose-reliant reductions in main biomarkers that include LDH. It also increased hemoglobin levels in PNH patients in the study. Most importantly, the increase in hemoglobin levels was maintained in the patients without transfusion. BCX9930 has been well-tolerated and was safe at all doses in the study without any drug-related adverse effects reported.
William Sheridan, the company’s chief medical officer, said that they are delighted with the safety profile and clinical benefits observed in oral BCX9930 as monotherapy for PNH patients. Sheridan added that these are excellent results, and the company is planning to commence advanced development studies in 2021 in several complement-mediated hematology as well as nephrology diseases.
Already BCX9930 has been granted Orphan Drug and Fast Track designations from the FDA for PNH. The company has confirmed that it plans to meet the regulators in Q4 2020 to deliberate on the advanced development program for BCX9930.
PNH patients showed improvement in hemoglobin levels
The seven patients treated in the study were severely ill and had received LDH pre-treatment form 4.8 to 11 x ULC, indicating active hemolysis. Two subjects had thrombotic, kidney, and lung complications from PNH, while four have compromised marrow function. Results indicate that the four treatment-naïve PNH subjects that received more than 6 weeks of therapy at 400mg bid showed considerable clinical improvements.
There was an increase in hemoglobin levels and the size of PNH red-blood-cell clone in the four patients which approached that of PNH granulocyte clone. This is an indication of near-complete complement-mediated hemolysis control. The common adverse events were mild-to-moderate headaches lasting between one and three days, with one patient reporting a mild rash after dosing at 100mg bid.