Another setback for Ipsen's Albireo portfolio as Bylvay flunks phase 3 trial

 Three years after gaining three liver disease assets in its $952 million buyout of Albireo, Ipsen is struggling to develop the trio. 

In May, the company grounded two investigational candidates. And now Ipsen has reported the failure of Bylvay in a phase 3 study that was designed to expand its patient population.

The placebo-controlled Bold trial did not meet its primary endpoint, as Bylvay failed to extend the lives of biliary atresia (BA) patients who have undergone Kasai hepatoportoenterostomy (HPE) surgery. The procedure is performed on infants who have blocked bile ducts. It restores bile flow by connecting part of the small intestine directly to the liver.

Ipsen did not elaborate further on Bylvay's performance in the prospective indication, though it stressed that present "topline data is in line with the well-established safety profile of odevixibat in approved indications."

HPE can delay disease progression but does not always cure biliary atresia, which causes inflammation, fibrosis, cirrhosis and eventually liver failure. The only other option for these patients is a liver transplant, as there are no approved medicines for BA.

The condition is the No. 1 cause of liver transplants in children under the age 2, according to lead investigator Saul Karpen M.D., Ph.D., the chief scientific officer of the Stravitz-Sanyal Institute for Liver Disease and Metabolic Health at Virginia Commonwealth University.

“As the first global Phase 3 trial in biliary atresia, Bold has generated the most comprehensive dataset ever assembled in this disease,” Karpen added in a release. “Although the study did not meet its primary endpoint, the commitment of participating children and families has produced valuable insights that will deepen our understanding of biliary atresia and provide a crucial basis for future research and patient care.”

Bylvay, a once-daily oral, is a ileal bile acid transport (IBAT) inhibitor which diverts bile acid to the feces, reducing its reabsorption in the intestine. It was approved in 2021 as a treatment for progressive familial intrahepatic cholestasis (PFIC) and two years later to relieve cholestatic pruritis (itching) associated with another liver disorder, Alagille syndrome. 

Last year, Ipsen posted (PDF) sales of Bylvay at 180 million euros ($203 million), which were up 36% over 2024. In its current indications, Ipsen competes with Mirum Pharmaceuticals’ IBAT Livmarli, which raked in sales of $360 million in 2025, up 69% year over year. 

In May, Mirum posted a phase 2 win in primary sclerosing cholangitis (PSC) for an investigational IBAT inhibitor. A week later, in the same indication, Ipsen shut down an open-label phase 2 trial of IBAT candidate ritivixibat because of recruitment issues and said it was discontinuing development of the Albireo asset. Around the same time, the company also removed another former Albireo liver disease candidate, A2342, from its list of pipeline drugs.

Elsewhere, Ipsen has had a busy summer of business development. Four weeks ago, the company inked a $1.7 billion buyout of Kartos Therapeutics to gain a late-stage blood cancer candidate. A few days later, Ipsen scooped up Memo Therapeutics and its phase 2 BK polyomavirus candidate potravitug in a $700 million deal.

https://www.fiercepharma.com/pharma/another-setback-ipsens-albireo-portfolio-bylvay-flunks-phase-3-trial