A late-breaking abstract presented at the AASLD Liver Meeting showed a “durable and sustained biochemical response” to seladelpar in patients with primary biliary cholangitis (PBC).
The ongoing, open-label, long-term, phase 3 ASSURE study assesses seladelpar, a novel selective peroxisome proliferator-activated receptor agonist, in patients with PBC who enrolled from the placebo-controlled, phase 3 RESPONSE trial or those who participated in other legacy clinical trials. This presentation included the pooled interim analysis of efficacy and safety outcomes.
Patients did not respond or were intolerant to first-line ursodeoxycholic acid. As of data cutoff on January 31, 2024, 337 patients (94% female; n=318) received seladelpar 10 mg daily, with 34 patients on treatment for 30 months and 90 patients on treatment for 24 or more months.
At baseline:
- Mean patient age was 58.1 years;
- Mean alkaline phosphatase (ALP) was 287.5 U/L;
- Mean total bilirubin (TB) was 0.85 mg/dL; and
- 16% (n=55) had cirrhosis.
At 12, 24, and 30 months, 73% (n=204/280), 73% (n=90/124), and 81% (n=30/37) of evaluable patients had a composite biochemical response to treatment (key efficacy endpoint). At those same time points, ALP normalized in 38% (n=106/280), 38% (n=47/124), and 41% (n=15/37) of patients.
At 6 months, many patients reported improvement in pruritus symptoms, with a mean change from baseline of −3.3 on the pruritus numeric rating scale among 99 evaluable patients. No serious treatment-related adverse events were reported.
“Seladelpar continues to appear safe and well tolerated, with no new safety signals or change in frequency of [adverse events] with up to 3 years of exposure,” the researchers concluded.
Reference
Lawitz E, Trivedi P, Kowdley K, et al. Long-term efficacy and safety of open-label seladelpar treatment in patients with primary biliary cholangitis: pooled interim results for up to 3 years from the ASSURE study. Late-Breaking Abstract 5044. Presented at the 2024 American Association for the Study of Liver Diseases’ 75th Liver Meeting; November 15–19, 2024; San Diego.
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