Last month, BioCryst trimmed down its pipeline to two drugs in complement-mediated diseases: BCX9930 and BCX10013. Fast-forward 45 days and BCX9930 is headed to the garbage can, too.
The decision to abandon BCX9930 comes after BioCryst caught a glimpse of its competitors’ hand at the American Society of Hematology meeting last week. After taking a look at what else is out there for diseases including paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy, BioCryst determined that BCX9930 would not be competitive. Discontinuing development will have a positive near-term financial impact on the company and clear the way for focus to shift to BCX10013, also an oral factor D inhibitor.
BCX9930 was by far BioCryst’s most advanced candidate, with two mid-phase studies underway in PNH as well as a third phase 2 in kidney diseases. Patients in these studies who are seeing benefit will be allowed to continue on the therapy, and the company plans to offer access to BCX10013.
BCX9930 was previously subject to a partial clinical hold from the FDA, which was lifted earlier this year with a revised protocol to bring down dosing. The pause in clinical development allowed BioCryst to save $100 million in R&D operating expenses for the year, according to the Thursday press release.
“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 BioCryst CEO Jon Stonehouse in the press release.
The company made the decision before more money was invested in a pivotal development program and commercialization activities, Stonehouse said. Heading into 2023 with a thinner pipeline, BioCryst will have R&D expenses similar to 2022.
That leaves BCX10013, which is being tested in healthy volunteers. A readout is expected in the first quarter of 2023. BioCryst is working on confirming that the therapy can be used as a once-daily oral, so the early studies are looking to establish optimal dosing for later trials.
BioCryst said it is continuing to develop other oral complement system meds but did not provide further details.
Back in November, BioCryst also cited the competitive environment as reason to abandon an ultra-rare bone disease therapy, which helped save $100 million. The company at the time signaled a shift towards complement-mediated diseases specifically BCX10013 and BCX9930.