CENTAUR is a randomized, placebo-controlled phase 2 trial of orally administered, fixed-dose sodium phenylbutyrate-taurursodiol (PB-TURSO), which significantly slowed functional decline in ALS participants. After the trial, 92% of participants elected to continue taking PB-TURSO, also known as AMX0035, in an open-label extension study.
Vital status was obtained for 135 of 137 original CENTAUR participants. Median overall survival was 25.0 months for participants originally randomized to PB-TURSO and 18.5 months for those originally randomized to placebo, leading to 6.5-month longer median survival for PB-TURSO versus placebo. Combined with results from CENTAUR, these findings suggest that PB-TURSO has both functional and survival benefits in ALS.