BioCryst’s Resumes PNH Drug Trial as FDA Lifts Partial Hold

The U.S. Food and Drug Administration has lifted its partial clinical hold on BioCryst Pharmaceuticals’ BCX9930 program, allowing the company to resume enrollment in global clinical trials under revised protocols at a reduced dose of 400 mg twice daily of BCX9930.

Photo: Jon Stonehouse, president and CEO of BioCryst

The changes affect 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).

The company said clinical evidence and recent laboratory studies have informed its 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.

BCX9930 is a novel, oral, potent and selective small molecule Factor D inhibitor that the company says could offer a significant advance in therapy for patients with paroxysmal nocturnal hemoglobinuria (PNH) and other complement-mediated diseases.

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,” said Jon Stonehouse, president and CEO of BioCryst. “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.”

Author: Rare Daily Staff