Ipsen (Euronext: IPN; ADR: IPSEY; reference: 0MH6) has announced late-breaking Phase III data from its ELSPIRE study, showing that 85.3% of patients with primary biliary cholangitis (PBC) receiving IQIRVO® (elafibranor) achieved normalisation of alkaline phosphatase (ALP) at Week 52, compared with 23.3% on placebo. The results are being presented at The Liver Meeting® 2026, hosted by the American Association for the Study of Liver Diseases, and simultaneously published in HEPATOLOGY. The data are also being shared with regulatory authorities, a step that investors in the rare liver disease space may be watching closely.

Key Points

  • Ipsen SA (Euronext: IPN; ADR: IPSEY; reference: 0MH6)
  • ELSPIRE Phase III trial of IQIRVO® in PBC patients with ALP between 1–1.67× upper limit of normal met its primary endpoint at Week 52
  • 85.3% of IQIRVO-treated patients achieved ALP normalisation versus 23.3% on placebo (p<0.0001); data submitted to regulatory authorities
  • Investors should monitor regulatory authority responses and progress of the ongoing Phase IV ELFINITY real-world study

ELSPIRE Trial Design and 85.3% ALP Normalisation Result in a Previously Unstudied PBC Population

ELSPIRE was a randomised, double-blind, placebo-controlled Phase III study enrolling 92 adults across 10 countries whose ALP levels fell between 1 and 1.67 times the upper limit of normal — a population not previously included in registrational trials of IQIRVO. Patients were randomised 2:1 to receive IQIRVO 80 mg once daily (n=62) or placebo (n=30). The primary endpoint, ALP at or below the upper limit of normal at Week 52, was achieved by 85.3% of IQIRVO-treated patients versus 23.3% receiving placebo, a statistically significant difference (p<0.0001).

Secondary Endpoints Show Broad Biochemical Improvements Across ALP, Bilirubin and Liver Enzymes

Beyond the primary outcome, IQIRVO demonstrated an adjusted mean ALP reduction of 36.5% from baseline at Week 52, compared with 4.2% for placebo. Complete biochemical response — defined as normalisation of total bilirubin, ALP, AST, ALT, albumin and INR — was achieved by 64.3% of IQIRVO patients versus 16.7% on placebo (p<0.0001). Total bilirubin also improved, with a 15-percentage-point adjusted difference in change from baseline (p=0.0260), and GGT was reduced by an adjusted mean difference of 36.3 U/L versus placebo (p=0.0002). The company stated IQIRVO was well tolerated, with no new safety signals and low treatment discontinuation rates.

ELFINITY Phase IV Real-World Study Adds Early Evidence on Fatigue and Pruritus in Clinical Practice

Alongside the ELSPIRE data, Ipsen presented six-month interim results from its ongoing global Phase IV ELFINITY study, which is assessing IQIRVO in routine clinical practice with plans to recruit approximately 424 patients over up to five years of follow-up. At six months, 57.5% of evaluable patients met the study's biochemical response endpoint. Among patients with moderate-to-severe fatigue at baseline, 42.3% achieved a clinically meaningful improvement on the PBC-40 Fatigue scale, while patients with moderate-to-severe pruritus showed meaningful improvements across multiple itch severity measures.

Ipsen's Expanding Rare Cholestatic Liver Disease Portfolio and Regulatory Milestones for IQIRVO

IQIRVO, a dual PPARα/δ agonist developed by GENFIT and licensed exclusively by Ipsen in 2021, holds approvals in the US, EU, UK, Canada, Australia, Brazil and 13 other countries for PBC. The ELSPIRE data are being shared with regulatory authorities, though the company did not disclose specific timelines or outcomes anticipated from that process. Ipsen also highlighted its broader rare cholestatic liver disease portfolio, covering progressive familial intrahepatic cholestasis and Alagille syndrome, underscoring its strategic focus on conditions with limited existing treatment options.