BioCryst to Present Data at the 2020 ASH Annual Meeting

On November 12, 2020 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported that preclinical data on BCX9930, an oral Factor D inhibitor, will be presented at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held as a virtual event, December 5-8, 2020 (Press release, BioCryst Pharmaceuticals, NOV 12, 2020, View Source [SID1234570761]).

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The abstract is available on the ASH (Free ASH Whitepaper) website at www.hematology.org.

Preclinical Characterization of BCX9930, a Potent Oral Complement Factor D Inhibitor, Targeting Alternative Pathway-Mediated Diseases Including Paroxysmal Nocturnal Hemoglobinuria (PNH); Poster #1680, Sunday, December 6, 2020, 10:00 a.m. – 6:30 p.m. ET
About BCX9930
Discovered by BioCryst, BCX9930 is a novel, oral, potent and selective small molecule inhibitor of Factor D currently in Phase 1 clinical development for the treatment of complement-mediated diseases. The U.S. Food and Drug Administration (FDA) has granted both Fast Track status and Orphan Drug designation for BCX9930 in PNH. In an ongoing dose ranging trial of BCX9930 in patients with PNH, BCX9930 was safe and well tolerated, with no drug-related serious adverse events. As a Factor D inhibitor, BCX9930 is designed as an oral monotherapy that can address both intravascular and extravascular hemolysis in PNH patients. Treatment-naïve PNH patients who have received more than six weeks of therapy at a monotherapy dose of 400 mg bid showed rapid and dose-dependent reductions in key biomarkers, including LDH, and increases in hemoglobin levels that were maintained without transfusions.