Shares of Albireo Pharma (NASDAQ:ALBO) are up by 41.4% as of 11:49 a.m. EDT on Tuesday, after soaring by as much as 79% earlier today. These gains are a result of the biopharmaceutical company reporting positive data from a phase 3 clinical trial for odevixibat, one of its lead pipeline candidates. The study investigated the safety and efficacy of odevixibat as a treatment for progressive familial intrahepatic cholestasis (PFIC).
PFIC is a rare genetic disease; patients with this condition have difficulty draining bile, a digestive fluid in the liver. The buildup of bile in the liver leads to symptoms such as pruritus (intense itching). There are several forms of PFIC, but Albireo Pharma's phase 3 study focused on PFIC1 and PFIC2. During the trial, odevixibat "reduced serum bile acid responses and improved pruritus assessments," thereby meeting its primary endpoint.
The medicine was also well tolerated and even showed a safety profile comparable to that of a placebo. Said Richard Thompson, principal investigator of the study: "These data demonstrate that odevixibat reduced serum bile acids and improved pruritus in patients with PFIC. Coupled with the favorable safety and tolerability profile odevixibat exhibited, these data underscore the potential to improve upon the current standard of care, which typically consists of off-label medications or invasive surgeries including transplant."
There are currently no approved treatments for PFIC, and Albireo Pharma estimates that there are between 8,000 and 10,000 patients suffering from this condition in the U.S. and Europe. And considering the results the healthcare company released today, it seems likely that odevixibat will become the first approved drug for PFIC. Given those factors, it isn't surprising that enthusiastic investors are bidding up shares of Albireo Pharma today.