RARE Daily

BioCryst Pauses Enrollment in BCX9930 Clinical Trials

April 8, 2022

In a two-sentence statement, BioCryst Pharmaceuticals said it had paused enrollment in three clinical trials of its lead candidate BCX9930 for the treatment of complement-mediated diseases while it investigates why some patients developed elevated serum creatinine levels.

Shares of the company fell 30 percent on the news.

During the investigation, the company will not enroll new patients in the REDEEM-1, REDEEM-2, or RENEW clinical trials, BioCryst wrote, but patients currently enrolled in the trials will continue taking the study drug at this time.

BCX9930, 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.

REDEEM-1 is a randomized, open-label, active comparator-controlled study of the efficacy and safety of oral BCX9930 monotherapy in PNH patients with an inadequate response to a C5 inhibitor.

REDEEM-2 is a randomized, placebo-controlled study to evaluate the efficacy and safety of oral BCX9930 as monotherapy versus placebo in PNH patients not currently receiving complement inhibitor therapy.

BioCryst is also preparing to initiate a proof-of-concept trial of BCX9930 in renal complement-mediated diseases such as C3 glomerulopathy, IgA nephropathy, and primary membranous nephropathy.

In early December 2020, just days after receiving U.S. regulatory approval to market Orladeyo for hereditary angioedema, BioCryst Pharmaceuticals entered into a deal with Royalty Pharma and Athyrium Capital Management for $325 million in funding to support its launch. One year later, the company entered into another deal with Royalty Pharma, this time also with OMERS Capital Markets, for $350 million in new funding to support the continued development of BCX9930.

Author: Rare Daily Staff

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