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FDA places partial clinical hold on BioCryst’s lead candidate, BCX9930

Published on 09/05/22 at 09:17am

The FDA has imposed a partial clinical hold on BioCryst’s Factor D inhibitor, BCX9930. The clinical hold arrives around a month following the Druham, NC-based biotech company’s halt of three studies for the inhibitor.

Early last month, BioCryst announced that it was voluntarily pausing enrollment in clinical trials evaluating its lead pipeline candidate, BXC9930, after elevations in serum creatinine were observed in some patients.

Factor D is a protein which is involved in the alternative complement pathway of the complement system. It is a serine protease, an enzyme that cleaves peptide bonds in proteins.

The early April decision to pause trials testing BCX9930 in patients with paroxysmal nocturnal hemoglobinuria (PNH) was spurred by safety signals. The company shared that the trials were paused to investigate elevated serum creatinine levels, a signal of impaired kidney function.

PNH is a rare disease marked by the destruction of red blood cells, blood clots, and poor bone marrow function. It is a life-threatening disease with a significant variation of symptoms patient to patient. While some individuals may display mild symptoms that remain stable for many years, others have serious symptoms that can progress to cause life-threatening complications.

“As we complete our investigation, we will continue to be comprehensive and deliberate, with a primary focus on patient safety,” said Chief Medical officer William Sheridan. “After consultation with regulators, we will determine the next step for the BCX9930 program.”

Under the conditions of the hold, the company is unable to enroll new patients. However, patients who have already been treated with BCX9930 can continue, if no other treatment option is available to them.

Ana Ovey

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