Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as OT-58, TVT-058, OT-058
Pegtibatinase · 3 trials · 1 indication
Incidence of treatment-emergent AEs
Incidence of hypermethioninemia
Incidence of hypomethioninemia
Proportion of participants requiring dietary protein rescue for participants with hypomethioninemia
Change between baseline and average of 6 to 12 week (6, 8, 10, and 12 week) plasma tHcy levels in participants receiving pegtibatinase vs. placebo. Baseline tHcy level defined as average of Week -3, Week -1, and Day 1 pre-dose plasma tHcy
Incidence of AEs (by type, severity and relationship to study drug)
Presence and levels of anti-pegtibatinase antibodies in plasma as measured by antibody titers
Presence and levels of anti-PEG antibodies in plasma as measured by antibody titers
The number and percentage of participants who develop hypermethioninemia during treatment, based on plasma methionine concentrations exceeding the protocol-defined threshold. Participants meeting the protocol-defined threshold may undergo dietary management or study treatment modifications, as appropriate.
The number and percentage of participants who develop hypomethioninemia during treatment, based on plasma methionine concentrations below the protocol-defined threshold. Participants meeting the protocol-defined threshold may receive dietary protein supplementation or study treatment modifications, as appropriate.
The proportion of participants who require initiation of dietary protein supplementation during study treatment to manage protocol-defined low plasma methionine concentrations.
| Arm | Type | Description |
|---|---|---|
| pegtibatinase | EXPERIMENTAL | - |
| placebo | PLACEBO_COMPARATOR | - |
| Pegtibatinase (Cohort 1-6) | ACTIVE_COMPARATOR | Double-Blind Treatment Cohorts (≥12 to ≤65 years) |
| Placebo (Cohort 1-6) | PLACEBO_COMPARATOR | Double-Blind Treatment Cohorts (≥12 to ≤65 years) |
| Pegtibatinase (Cohort 7) | EXPERIMENTAL | Pediatric Open-label Treatment Cohort (≥5 to \<12 years) |
| Name | Type | Description |
|---|---|---|
| Pegtibatinase | DRUG | Pegtibatinase BIW |
| Placebo | OTHER | volume-matched saline SC BIW |
Inclusion Criteria: * Participants active in the COMPOSE study or who completed the 24-week blinded period on study intervention (pegtibatinase or placebo) in the Phase 3 HARMONY study Exclusion Criteria: * Participant permanently discontinued from study intervention treatment in a previous pegti...
Pegtibatinase is an investigational therapy being developed for classical homocystinuria (HCU), a rare inherited metabolic disorder. It is designed for patients receiving standard of care treatment. The clinical program includes participants aged 5 to 65 years with classical HCU.
Pegtibatinase targets cystathionine beta-synthase (CBS), the enzyme deficient in classical homocystinuria. By targeting CBS, the drug aims to address the underlying metabolic defect in this rare disease.
Pegtibatinase is being developed by Travere Therapeutics, Inc., which trades on the Nasdaq under the ticker TVTX. Travere is a biopharmaceutical company focused on rare diseases.
Pegtibatinase is in Phase 3 clinical development for classical homocystinuria. The program includes three active trials: two Phase 3 studies and one Phase 1 study. It is investigational and has not been approved by the FDA.
Pegtibatinase is being evaluated in three active clinical trials. The Phase 3 ENSEMBLE long-term extension study is NCT06431893, the Phase 3 study in patients receiving standard of care is NCT06247085, and the Phase 1 COMPOSE study is NCT03406611. All are currently recruiting or enrolling by invitation.