Please describe your practice as a clinician,how many patients with   PNH do you see on a yearly basis? Can you describe the standard of care, and take us through the most promising upcoming treatments, or interesting clinical trials in this space?

How much of an unmet need is there in PNH, and how important is targeting Factor D in the alternative pathway of complement, as an endpoint? How would you evaluate Factor D inhibitors’ importance in the area of PNH?

Could you please comment the results, where, in the SAD assessment to date, cohorts of healthy volunteers received a single dose of 1 mg, 3 mg, 10 mg, 40 mg, 80 mg or 110 mg of oral BCX10013 or placebo. In the MAD assessment to date, cohorts of healthy volunteers received 20 mg, 40 mg or 80 mg of oral BCX10013 or placebo administered once daily for seven days (20 mg cohort) or 14 days (40 mg and 80 mg cohorts).

Following single BCX10013 dose administration, the onset of AP inhibition occurred within one hour and increased in a dose-dependent manner. At 110 mg, the highest dose studied to date, AP activity was suppressed by a mean of 97.8 percent at 24 hours post-dose. Suppression of AP activity by BCX10013 was assessed using the AP Wieslab® assay, which measures functional activity of the complement system. In the MAD studies with once-daily dosing, exposure to BCX10013 was approximately dose proportional over the studied dose range and steady-state was achieved in 7 to 14 days with modest accumulation.

How likely would you be to prescribe BCX10013, judging by the clinical trial results to date?