Zydus Therapeutics, Inc., a U.S.-based innovation arm of Zydus Lifesciences Ltd., today announced positive topline data from its pivotal EPICS-III Phase 2(b)/3 trial evaluating Saroglitazar Magnesium, a novel dual PPAR alpha/gamma agonist, in patients with Primary Biliary Cholangitis (PBC) who had an inadequate response or intolerance to ursodeoxycholic acid (UDCA).
Science Significance
Saroglitazar is the first PPAR alpha/gamma agonist to deliver positive Phase 3 results in PBC, a rare autoimmune liver disease with limited therapeutic options. In the EPICS-III trial, Saroglitazar achieved a 48.5% treatment difference in biochemical response compared to placebo (p<0.001), targeting key predictors of disease progression such as alkaline phosphatase (ALP) and bilirubin. These findings reinforce Saroglitazar’s potential to address bile acid toxicity and liver inflammation simultaneously — a dual mechanism with broad implications for rare liver disorders.
Regulatory Significance
Saroglitazar is the first PPAR alpha/gamma agonist to deliver positive Phase 3 results in PBC, a rare autoimmune liver disease with limited therapeutic options. In the EPICS-III trial, Saroglitazar achieved a 48.5% treatment difference in biochemical response compared to placebo (p<0.001), targeting key predictors of disease progression such as alkaline phosphatase (ALP) and bilirubin. These findings reinforce Saroglitazar’s potential to address bile acid toxicity and liver inflammation simultaneously — a dual mechanism with broad implications for rare liver disorders.
Business Significance
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This milestone strengthens Zydus’ innovation pipeline in specialty and rare liver diseases, extending its global biopharma footprint beyond generics into specialty therapeutics. By leveraging the group’s global R&D and manufacturing infrastructure, Zydus Therapeutics is poised to expand its presence in the U.S. specialty market. The successful progression of Saroglitazar validates Zydus’ strategy of innovation-led growth and positions it for potential entry into the rare disease therapeutics segment.
Patients’ Significance
For patients with PBC who remain symptomatic or progress despite standard UDCA therapy, Saroglitazar could represent a new class of treatment. With demonstrated biochemical improvements and balanced tolerability, this investigational therapy has the potential to reduce disease progression, improve quality of life, and delay transplant need.
Policy Significance
The advancement of Saroglitazar in PBC highlights the importance of FDA’s Orphan Drug and Fast Track pathways in expediting therapies for rare diseases. It reflects ongoing global policy emphasis on incentivizing innovation in orphan indications, where therapeutic choices are scarce but clinical burden is high.
Transaction Highlights
The EPICS-III trial (NCT05133336) was a multicenter, randomized, double-blind, placebo-controlled, seamless Phase 2b/3 study that enrolled 149 patients in a 2:1 ratio. The trial met its primary composite endpoint, achieving a 48.5% treatment difference in biochemical response at 52 weeks, and also met its key secondary endpoint with ALP normalization. Safety findings were favorable, with adverse events balanced between the Saroglitazar and placebo groups. Based on these results, Zydus intends to submit a New Drug Application to the U.S. FDA in early 2026. Full results will be presented at an upcoming scientific congress.
Source: Zydus Therapeutics, Inc. Press Release
