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Also known as BBP-418 (ribitol)
BBP-418 · 3 trials · 2 indications
| Arm | Type | Description |
|---|---|---|
| BBP-418 | EXPERIMENTAL | Drug: BBP-418. Single arm. |
| Placebo to Match BBP-418 | PLACEBO_COMPARATOR | The placebo will be identical to the BBP-418 Granules for Oral Solution in appearance, packaging, labeling, and storage conditions. |
| Cohort 1 | EXPERIMENTAL | Subjects will receive 6 grams of BBP-418 once daily x 90 days, then 12 grams twice daily (BID, a least 8 hours apart) of BBP-418 daily until study completion. |
| Cohort 2 | EXPERIMENTAL | Subjects will receive 6 grams of BBP-418 twice daily (BID, at least 8 hours apart) x 90 days, then 12 grams BID of BBP-418 daily until study completion. |
| Cohort 3 | EXPERIMENTAL | Subjects will receive 12 grams of BBP-418 twice daily (BID, at least 8 hours apart) x 90 days, then 12 grams BID of BBP-418 daily until study completion. |
| Name | Type | Description |
|---|---|---|
| BBP-418 (ribitol) | DRUG | Drug: BBP-418. Single arm. Participants who completed Study MLB-01-005 and meet eligibility criteria will receive BBP-418 (ribitol) taken orally twice daily at 9 or 12g (based on body weight measured) for up to 36 months. |
| Placebo | OTHER | A placebo matched for similar taste and appearance was compounded at the clinical pharmacy with sucralose as a 0.35 mg/mL oral solution in purified water (USP) in a glass container. Sucralose is similar in taste to the compounded drug product. |
| BBP-418 | DRUG | BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) for which no approved therapy currently exists. It targets the molecular defect at the source by supplying excess substrate to the mutant enzyme thus boosting glycosylation of muscle α-dystroglycan. The BBP-418 drug product will be packaged in sachets and provided in a carton for in-clinic and at home use. |
Inclusion Criteria: * Completed Study MLB-01-005 on study drug through the final clinic visit (Month 36 or another qualifying end-of-study visit as determined by the Sponsor). * The participant (or parent/guardian) who signs the ICF understands the study procedures and agrees to participate in the ...
BBP-418 is an investigational small molecule being developed for Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I), a genetic muscle disorder. It is also known as ribitol. The drug is currently in clinical development and has not been approved by the FDA.
The specific molecular target of BBP-418 is not disclosed in the available information. It is a small molecule being studied for its potential to treat LGMD2I, a form of muscular dystrophy. The mechanism of action has not been detailed in public clinical trial summaries.
BBP-418 is being developed by BridgeBio Pharma, Inc., a biopharmaceutical company traded on NASDAQ under the ticker BBIO. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with LGMD2I.
BBP-418 is in Phase 2 and Phase 3 clinical trials. A Phase 2 study (NCT04800874) is active but not recruiting, and two Phase 3 studies (NCT05775848 and NCT07678775) are also active but not recruiting. The drug is investigational and not yet FDA approved.
BBP-418 is being studied in three active trials: NCT04800874, a Phase 2 study in LGMD2I patients; NCT05775848, a Phase 3 efficacy and safety study; and NCT07678775, a Phase 3 open-label extension study. These trials are enrolling patients aged 12 and older across multiple countries.
Yes, BBP-418 is also known as ribitol. Clinical trial records refer to the drug as BBP-418 (ribitol), and the Phase 3 study NCT05775848 explicitly evaluates BBP-418 (Ribitol) in patients with LGMD2I.