After years of research work backed by a multimillion-dollar federal contract, BioCryst ($BCRX) reported today that it is halting enrollment on its late-stage study for the flu drug peramivir after an interim analysis led trial monitors to conclude that it would be futile to continue and the trial should be terminated. After this latest setback, the company's chief medical officer says that it is "unlikely" that the biotech will continue development work needed for U.S. approval.
The ax dropped after investigators turned to the interim analysis to provide some clear idea of the difference in responses seen in the drug arm and the control group as they assessed how many patients would be needed to reach statistical significance. "The difference between peramivir and control groups for the primary endpoint was small and the recalculated sample size was greater than the predefined futility boundary of 320 subjects," BioCryst noted in a release, leaving out any data on efficacy.
BioCryst shares were once again battered by the fresh round of bad news, dropping 37% in pre-market trading. The trouble with peramivir--which was approved in Japan and Korea after gaining widespread attention during the swine flu epidemic--will also likely sound a sour note at federal Biomedical Advanced Research and Development Authority, which provided a $234.8 million contract for its development.
That's more bad news for a company that has run into some serious trouble as it attempts to remake itself into a hepatitis C player. BioCryst recently struck a deal to merge with Presidio Pharmaceuticals in a $101 million, all-stock deal tied to $60 million in fresh financing. Days later BioCryst was forced to announce that it had to reel back in its IND for a hep C program after the FDA concluded that the nuc's preclinical tox profile raised some serious safety concerns.
"The goal of this analysis was to reassess the sample size required for the trial, and to make adjustments to the study if necessary. Based on the DMC recommendation, we have suspended enrollment of patients in the trial," said Dr. William P. Sheridan, the chief medical officer of BioCryst Pharmaceuticals. "We are proceeding with a full analysis of unblinded data from the trial, and a final decision will be made following completion of the analysis and further discussions with our development partners; however, it is unlikely that peramivir development for US registration will continue."
- here's the press release