other material
confidence high
sentiment positive
materiality 0.85
Travere reports positive Phase 1/2 COMPOSE Cohort 6 data for pegtibatinase in HCU
Travere Therapeutics, Inc.
- Pegtibatinase 2.5 mg/kg twice weekly reduced total homocysteine (tHcy) by mean 67.1% vs placebo increase of 0.6%.
- All 4 treated patients achieved mean tHcy below 100 µM threshold; one patient with low baseline achieved normalization.
- No treatment-related serious adverse events, anaphylaxis, or discontinuations in cohort 6.
- Methionine reduced and cystathionine elevated, consistent with desired CBS enzyme activity.
- Company plans to initiate Phase 3 program by end of 2023 after regulatory engagement.