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BioCryst Discontinues Development of BCX9930 and Shifts Focus

December 15, 2022

BioCryst Pharmaceuticals said that, based on new competitive data recently presented at the American Society of Hematology annual meeting, the company no longer believes that BCX9930 would be commercially competitive, and is discontinuing its development.

Photo: said Jon Stonehouse, president and CEO at BioCryst

The company said it will now fully focus on its complement inhibitor development efforts on BCX10013, a potential once-daily, oral Factor D (alternative pathway) inhibitor currently in clinical development, and pursue additional oral compounds for multiple targets across other complement pathways.

“With the new competitor efficacy data presented at ASH, and the limitations preventing us from optimizing the dosing of BCX9930 for increased efficacy, it is unlikely that BCX9930 could meet the new standard of care,” said Jon Stonehouse, president and CEO at BioCryst. “We have made this decision prior to fully investing in the pivotal development program and commercialization activities, and will focus on our potential best-in-class asset, BCX10013, and our other programs.”

Patients benefitting from BCX9930 in the clinical program may remain on therapy. As the program advances, the company plans to offer these patients an opportunity for access to BCX10013.

BioCryst expects to report preliminary data from healthy volunteers receiving single ascending doses and multiple ascending doses of BCX10013 in the first quarter of 2023. The preclinical and early clinical profile from approximately 90 healthy volunteers suggests BCX10013 could have the properties of a once-daily, oral therapy. Key goals of the ongoing clinical program include confirming this once-daily profile with healthy volunteer and patient data and establishing optimal dosing for pivotal studies.

In addition to BCX10013, which targets Factor D in the alternative pathway of complement, BioCryst is pursuing oral medicines directed at other targets across the classical, lectin, and terminal pathways of the complement system. The goal of the company’s overall complement program is to advance several oral compounds across multiple pathways in the complement system to treat many complement-mediated diseases.

The decision to discontinue the BCX9930 program will have a positive near-term financial impact for the company. The pause in the program earlier this year allowed the company to reduce 2022 operating expense guidance by approximately $100 million, primarily from reduced research and development expenses. The company now expects that 2023 R&D expenses will be similar to 2022 R&D expenses as the company focuses its investment on BCX10013 and its other complement programs. The company plans to provide full year 2023 operating expense guidance in the first quarter of 2023.

Author: Rare Daily Staff

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