Another blow to NASH, disappointing trial results spell end for darkhorse player Albireo
Another NASH program is headed to the scrap heap.
Albireo $ALBO, a Boston biotech that took an unconventional route to treating the fatty liver disease, announced yesterday that they were ending NASH development after they received disappointing results from a Phase II study. Just last week CEO Ron Cooper, on their Q2 call, talked up their offbeat approach and the potential for their lead molecule to be used in combination with agents now in development at other countries.
On Tuesday, he emphasized that Albireo’s investment in the program was minimal.
“We wanted to investigate the potential of elobixibat in NASH and allocated minimal resources to an exploratory Phase 2 study,” Cooper said in a statement. “Based on the results of this study, we have made the decision not to pursue further development of elobixibat in NASH.”
Compared to the stock-melting hits companies such as GenFit have taken after their trial failures, Albireo emerges from their NASH exploration relatively unscathed. Their lead programs are in other liver conditions, bile atresia and PFIC, and the company took only a 4.85% hit on the news, dropping from $27.01 to $25.70.
Still, the failed program is another blow to a field that has seen little but failure since JP Morgan 2019, when multiple companies seemed poised to break into a market estimated to have millions of patients and room for multiple mega-blockbusters. Since then Gilead, GenFit and a series of smaller biotechs have seen major trial failures. And, after the FDA surprisingly rejected an Intercept drug that showed mixed results in Phase III, developers have been left in limbo about what the bar for efficacy will be.
Oddly enough, Albireo actually hit the primary endpoint on their study. Cooper insisted that he saw the disease, largely driven by obesity and similar health factors, as fundamentally a cardiovascular ailment.
So unlike Intercept, Genfit and the rest of the biotechs that focused on fibrosis and NASH resolution, Albireo set up a trial designed to prove their lead drug, elobixibat, could improve patients’ cholesterol and show “proof-of-concept” on liver-specific endpoints, such as liver fat and function. If so, it could be used in tandem with other future NASH drugs, as researchers increasingly turn to combination therapies for the disease.
Ultimately, though, elobixibat could do only the former. In the 47-person study, patients on the treatment arm saw their cholesterol fall almost twice as much as patients on the placebo arm (-20.5 mg/dL vs -11.1 mg/dL), but there was little to no change in liver endpoints. Liver fat fell by 2.6% for the treatment arm, as measured by MRI, and there was “no meaningful change” in levels of alanine aminotransferase, a liver enzyme commonly used to measure function.