BioCryst Pharmaceuticals (NASDAQ:BCRX) perks up 4% premarket on light volume in response to encouraging Phase 1 results on oral Factor D inhibitor BCX9930 in 72 healthy volunteers.
The candidate was safe and generally well-tolerated while showing rapid and sustained suppression of the alternative pathway of the complement system at a dose of 100 mg every 12 hours.
There was a clinical benign rash observed in some participants which was self-limited and resolved four-to-eight days after onset. There were no discontinuations.
Development will now advance to a proof-of-concept study in patients with paroxysmal nocturnal hemoglobinuria (PNH) who failed to respond adequately to Alexion Pharmaceuticals’ (NASDAQ:ALXN) Soliris (eculizumab) or Ultomiris (ravulizumab-cwvz) and treatment-naïve PNH patients. Topline data should be available in H1 2020.
Management will host a conference call this morning at 8:30 am ET to discuss the results.
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