CymaBay Therapeutics' Lead Drug Candidate Hits Main Goal In Pivotal Study For Liver Damage

CymaBay Therapeutics Inc CBAY released topline results from its Phase 3 pivotal RESPONSE study of seladelpar for adult patients with primary biliary cholangitis (PBC). 

PBC is a chronic disease in which the small bile ducts in the liver become inflamed and are eventually destroyed. When no bile ducts exist, bile builds up and causes liver damage. 

Also Read: Genfit's Miss Is CymaBay Therapeutics' Gain: Analyst Says Rival Data Leaves Room for Seladelpar To Emerge As Best-In-Class.

The trial achieved the primary and all key secondary endpoints and supports advancement to regulatory discussions and filing for regulatory approval with the FDA, the UK Medicines and Healthcare Products Regulatory Agency, and the European Medicines Agency.

A total of 61.7% of patients on seladelpar 10 mg (n=128) met the primary composite endpoint related to serum alkaline phosphatase and bilirubin at 12 months versus 20.0% on placebo (n=65; p<0.0001). 

The anti-cholestatic effect of seladelpar was supported by the normalization of alkaline phosphatase at 12 months (key secondary endpoint) in 25.0% of patients on seladelpar vs. zero on placebo (p<0.0001). 

The least-squares mean percent reduction in alkaline phosphatase at 12 months was 42.4% in the seladelpar group vs. 4.3% in the placebo group (p<0.0001).

Seladelpar, compared to placebo, also demonstrated a statistically significant reduction in pruritus or itch (key secondary endpoint) after six months of treatment. 

Overall, safety was comparable between placebo and seladelpar groups and was consistent with previous studies. 

Additional analyses of RESPONSE are ongoing.

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