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BioCryst Resumes Enrollment in BCX9930 Clinical Program
—U.S. Food and Drug Administration lifts partial clinical hold— RESEARCH TRIANGLE PARK, N.C., Aug. 04, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc.
About this update from Biocryst Pharmaceuticals, Inc.
[{"type":"text","content":"—U.S. Food and Drug Administration lifts partial clinical hold—\nRESEARCH TRIANGLE PARK, N.C., Aug. 04, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has lifted its partial clinical hold on the BCX9930 program. The company will resume enrollment in global clinical trials under revised protocols at a reduced dose of 400 mg twice daily of BCX9930. This includes the REDEEM-1 and REDEEM-2 pivotal trials in patients with paroxysmal nocturnal hemoglobinuria (PNH) and the RENEW proof-of-concept trial in patients with C3 glomerulopathy (C3G), immunoglobulin A nephropathy (IgAN) and primary membranous nephropathy (PMN). Clinical evidence and recent laboratory studies have informed the company’s hypothesis that crystals form in the kidneys of some patients. The company believes that lowering the dose to 400 mg and ensuring adequate hydration will dilute the concentration of drug in the urine below the threshold where crystals can form. On April 8, 2022, BioCryst announced that the company had voluntarily paused enrollment in BCX9930 clinical trials while it investigated observed elevations in serum creatinine (SCr) seen in some patients at the 500 mg twice daily dose. The FDA subsequently placed the program on a partial clinical hold. Patients already enrolled in the trials and demonstrating clinical benefit were able to continue on therapy. “Now that the FDA has lifted the partial clinical hold, we can take the next step toward our goal of finding a safe and effective dose for BCX9930. We expect this can be accomplished in a reasonable time frame after resuming enrollment, in a relatively small number of patients given the rate and timing of the SCr rises in patients prior to the enrollment pause. If we are successful, we will invest more significantly in BCX9930 to tap the full potential of reaching many patients suffering from a number of alternative pathway diseases, and, if we are not successful, we will stop investment in BCX9930 and move on to other molecules in our pipeline,” said Jon Stonehouse, president and chief executive officer of BioCryst. PNH patients naive to C5 inhibitor therapy who received 400 mg twice daily in the Phase 1 clinical program (n=7) experienced a mean change from baseline increase in hemoglobin of 4.3 g/dL and required...