Data from Pharmawand - Curated by EPG Health - Date added 09 November 2019

Intercept Pharmaceuticals announced additional supportive data from the first reported analyses of the Phase III REGENERATE study examining the effects of Ocaliva (obeticholic acid) on noninvasive assessments of liver fibrosis and patient-reported outcomes (PROs). As previously reported, in the primary efficacy analysis, once-daily Ocaliva 25 mg met the primary endpoint of fibrosis improvement (at least 1 stage) with no worsening of NASH in 23.1% of patients compared to 11.9% of placebo patients at the planned 18-month interim analysis (p=0.0002 vs. placebo). In a new analysis of the interim data presented at The Liver Meeting, Ocaliva-treated patients in the primary ITT group showed time- and dose-dependent improvements compared to placebo across commercially available noninvasive tests, including blood tests of fibrosis (Fibrosis-4 [FIB-4] index, AST to platelet ratio index [APRI], and FibroSURE) as early as three months after treatment initiation. In addition, vibration-controlled transient elastography [VCTE], an imaging assessment of liver stiffness and a surrogate of fibrosis, decreased from baseline in both Ocaliva groups but increased with placebo at 18 months.

In a responder analysis, improvements in noninvasive tests mirrored shifts in fibrosis stage, with the greatest improvements observed in patients achieving >1 fibrosis stage reduction. In contrast to patients treated with placebo, Ocaliva-treated patients with no change in fibrosis stage also had marked improvement in noninvasive tests. The authors concluded that the improvements observed in histologic non-responders suggest Ocaliva’s therapeutic benefit may not be adequately captured by categorical histologic fibrosis staging at 18 months.

Two new analyses being presented at The Liver Meeting focused on PROs in REGENERATE’s expanded ITT population, which consisted of the primary ITT population (n=931), plus an exploratory cohort of 287 NASH patients with stage 1 liver fibrosis and additional risk factors who were at increased risk of progression to cirrhosis (n=1,218). PROs related to pruritus were assessed using the chronic liver disease questionnaire for NASH (CLDQ-NASH), Work Productivity and Activity Impairment (WPAI) and EQ-5D tools. At the start of study treatment, 21% of patients reported significant pruritus (CLDQ Itch score<=4) at baseline, which negatively impacted all PRO domains. Following randomization, PROs were measured every six months through the 18-month interim analysis. Despite a dose-dependent increase in pruritus in the OCA 10 mg and 25 mg arms, pruritus with OCA 25 mg did not have a detectable negative impact on PROs. The new data will be presented at The Liver Meeting 2019, the Annual Meeting of the American Association for the Study of Liver Diseases.


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