BioCryst Pharmaceuticals, Inc. announced that the company will provide updates on berotralstat, an oral kallikrein inhibitor for hereditary angioedema (HAE), and BCX9930, an oral Factor D inhibitor for complement-mediated diseases, this week at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco. New drug application (NDA) submitted to U.S Food and Drug Administration in December 2019. APeX-J trial in Japan met its primary endpoint (p=0.003) for prevention of HAE attacks, and berotralstat was safe and generally well-tolerated; JNDA submission to Japanese Pharmaceuticals and Medical Devices Agency (PMDA) on-track for First Quarter 2020; Marketing authorization application to European Medicines Agency (EMA) on-track for first quarter of 2020. As previously announced, results from an ongoing three part Phase 1 trial of BCX9930 showed rapid, sustained and >95% suppression of the alternative pathway (AP) of the complement system at 100 mg every 12 hours, as measured by the AP Wieslab® assay. In two initial multiple ascending dose (MAD) assessment cohorts, healthy volunteers received 50 mg or 100 mg of oral BCX9930 or placebo (each MAD cohort randomized 10:2) administered every 12 hours for seven days. Healthy volunteers in the MAD cohorts were prophylactically dosed with the broad-spectrum antibiotic, amoxicillin/clavulanate. BCX9930 was safe and generally well tolerated at all doses studied in single ascending dose and MAD cohorts. There were no serious adverse events. A clinically benign rash was observed in some healthy volunteers in the MAD (two in the 50 mg cohort, seven in the 100 mg cohort), which was self-limited and resolved in 4-8 days after onset. The company has now completed an additional MAD cohort with 50 mg of oral BCX9930 or placebo administered every 12 hours for 14 days, with vaccination instead of an antibiotic. Key observations from the additional MAD cohort include: Benign rash (similar to prior MAD cohorts) that was self-limited and resolved in 4 to 8 days post-onset seen in seven healthy volunteers; Successfully dosed-through benign rash, with rash resolving on-drug, in both patients who continued dosing, per protocol; Biopsies of rashes from multiple subjects confirm benign assessment; The company is on-track to report proof of concept data in paroxysmal nocturnal hemoglobinuria (PNH) patients in first half 2020.