Meet the new Regenerate, same as the old Regenerate

Intercept hopes that a new analysis of its Regenerate study of Ocaliva in Nash will pave the way to FDA approval. However, it is hard to see what has changed for the project, which received a complete response letter in Nash in 2020. This time, a three-doctor panel evaluated liver biopsy slides, versus just one reader when Regenerate first read out in February 2019. Overall, the new analysis echoed the original one: Regenerate met just one of its two co-primary endpoints, at only the higher of two doses studied. This can be deemed a success, as the study merely needed to hit on one outcome. However, there are still lingering questions about Ocaliva’s toxicity. Pruritus was again an issue, especially with the higher 25mg dose. Intercept also described an increase in adverse gallbladder and liver events with the high dose, without giving actual numbers, and a temporary increase in LDL-cholesterol with Ocaliva overall. These events look “monitorable and manageable”, Intercept’s chief medical officer, Michelle Berrey, said several times during a conference call today. Now to find out if the FDA agrees. Investors seem pessimistic: the company’s already beaten-down stock slumped 8% this morning.