Iqirvo® (elafibranor) data shows efficacy and safety for up to 3 years in patients with PBC with improvements in fatigue and pruritus

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PARIS, FRANCE, 15 November 2024 Ipsen (Euronext: IPN; ADR: IPSEY) announced today late-breaking data for Iqirvo® (elafibranor 80 mg tablets) from an interim analysis of the ongoing open-label extension of the Phase III ELATIVE® study at the American Association for the Study of Liver Disease (AASLD) congress. The late-breaking presentations (Abstract #5041 and Abstract #5042) report on biomarkers of cholestasis, stabilization of surrogate markers of liver fibrosis and moderate-to-severe pruritus data for up to three years in Iqirvo-treated patients. Additionally, exploratory endpoints in fatigue and sleep were evaluated using patient-reported outcomes tools.

“Over three years, Iqirvo data suggest sustained efficacy and support the safety profile of the medicine. Importantly, when patients tell me they are less impacted by itch and fatigue—that matters to me as a physician,” said Dr. Kris Kowdley, Director at The Liver Institute Northwest, Washington and a primary investigator on the ELATIVE study. “Treatment with Iqirvo had an impact on symptoms of pruritus and surrogate markers of fibrosis, which are important findings for people living with PBC.”

“Fatigue is a symptom often reported by people living with PBC and is also very challenging to manage,” said Dr. Mark Swain, Department of Medicine, Cumming School of Medicine, University of Calgary, Canada. “Patients treated with Iqirvo reported improvement in fatigue and sleep, across several patient-reported outcome measures.”

The open-label extension (OLE) included 138 patients who completed the double-blind period of the Phase III ELATIVE® study1. This interim analysis was performed after at least one year of treatment with Iqirvo in the OLE (up to three years total). In patients receiving three years of continuous treatment with Iqirvo across the double-blind period and OLE (n=13), 85 percent had a biochemical response (n=11/13; ALP <1.67 x ULN, with ≥ 15% reduction from baseline and total bilirubin ≤ ULN) and 39 percent achieved ALP normalization (n=5/13) at week 156. Surrogate markers of liver fibrosis, liver stiffness measurements (n=23) and enhanced liver fibrosis (ELF™) (n=19) scores, suggest stabilization when measured from baseline to week 130. In patients continuously receiving Iqirvo for up to 156 weeks, pruritus improvements were sustained for patients with moderate or severe pruritus at baseline (n=5).

No new safety findings were observed. The most common treatment-emergent adverse events (>10 percent) occurring more frequently in patients treated with Iqirvo than placebo in the double-blind period of the trial (abdominal pain, diarrhea, nausea and vomiting) were also reported in the OLE.

The impact of Iqirvo on fatigue and sleep were investigated as an exploratory endpoint in the OLE.2 Changes in fatigue or sleepiness (including normal sleep) were reviewed from baseline to week 104 looking at the minimal clinically important differences and categorical changes (n=48). Fatigue and sleep improvements for patients treated with Iqirvo were observed at week 104 across three patient-reported outcome (PRO) tools. In patients with moderate-to-severe fatigue or excessive sleepiness at baseline, clinically meaningful improvements were observed after 104 weeks of treatment with Iqirvo in 56 percent (n=18) of patients according to the PRO Measurement Information System (PROMIS) Fatigue Short Form 7a, 50 percent (n=24) of patients according to the fatigue domain of the PBC-40, and 69 percent (n=16) of patients according to the Epworth Sleepiness Scale (ESS). These are interim data and have not been submitted to regulatory agencies. A confirmatory study of Iqirvo is ongoing (NCT06016842).

“People living with PBC tell us just how devastating this disease can be for patients and their families,” said Sandra Silvestri, EVP and Chief Medical Officer, Ipsen. “Data like these continue to provide prescribers with a clear rationale for Iqirvo. As the first-in-class PPAR approved for the treatment of PBC, Iqirvo is on track to be the treatment of choice for patients living with PBC. Ipsen is committed to being a leader the rare liver community can count on.”

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