Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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 small molecule being developed for the treatment of classical homocystinuria (HCU), a rare genetic disorder. It is currently in Phase 3 clinical development and is not yet approved by regulatory authorities.
Pegtibatinase is being developed by Travere Therapeutics, Inc., a biopharmaceutical company publicly traded under the ticker symbol TVTX. The company is conducting multiple clinical trials to evaluate the drug's safety and efficacy in patients with homocystinuria.
Pegtibatinase is currently in Phase 3 clinical development. It is being studied in a placebo-controlled Phase 3 trial (NCT06247085) and a long-term extension study (NCT06431893), both of which are actively recruiting or enrolling participants.
Pegtibatinase is being evaluated in three clinical trials: NCT03406611 (COMPOSE), a Phase 1 study in patients with classical HCU; NCT06247085, a Phase 3 placebo-controlled study; and NCT06431893 (ENSEMBLE), a long-term extension study. All trials are currently active.
Pegtibatinase is also known by the name COMPOSE in the context of one of its clinical trials, but no other alternative drug names have been reported. It is a distinct investigational agent being developed specifically for homocystinuria.