BioCryst halts enrollment in all studies for its lead pipeline candidate — shares crash
BioCryst is taking a beating Friday morning after reporting it would pause enrollment in three studies for its lead pipeline program.
In a terse statement put out right before the market opened, the Durham, NC-based biotech said it discovered elevated serum creatinine levels in some patients, and will halt recruitment while it investigates. Elevated creatine levels can be a sign of kidney dysfunction.
The patients were taking the experimental drug known as BCX9930, an oral Factor D inhibitor being evaluated in three clinical studies.
After Friday’s opening bell, BioCryst $BCRX shares fell more than 30%, to $11.92.
Two of the biotech’s clinical studies were examining the experimental drug in paroxysmal nocturnal hemoglobinuria (PNH), one in an open-label setting compared to an active comparator and one controlled against placebo. Both were being evaluated as a monotherapy. BioCryst’s plan was to study BCX9930 in a range of complement-mediated diseases.
According to the studies’ clinicaltrials.gov webpages, BioCryst had expected to enroll about 140 patients between the two trials, and it’s not immediately clear how many had taken the drug as of Friday’s update. A company spokesperson declined to comment, saying BioCryst is not providing details on the studies.
The pause marks a setback for the biotech’s pipeline a little over a year after it won its first FDA approval for Orladeyo, a daily pill meant to prevent painful episodes in patients with the rare disease hereditary angioedema (HAE). BioCryst moved quickly to capitalize on the approval, signing a $250 million deal with Royalty Pharma three days after the greenlight.
Late last year, the biotech also moved to raise some new cash, getting another $350 million from Royalty and new investors. Some of the money was planned for advancing BCX9930 to its NDA and building out additional clinical trials, BioCryst said at the time.
This article has been updated to include BioCryst’s response to an Endpoints News request for comment.