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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 the treatment of Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I), a rare genetic muscle-wasting disorder. It is not yet approved for any indication and remains in clinical development.
BBP-418 targets FKRP, the fukutin-related protein. In LGMD2I, mutations in the FKRP gene impair the glycosylation of alpha-dystroglycan. BBP-418 is designed to act on this pathway to address the underlying defect.
BBP-418 is being developed by BridgeBio Pharma, Inc., which trades on the Nasdaq under the ticker BBIO. The company is advancing the program for LGMD2I.
BBP-418 is in the NDA/BLA stage of development. The program has completed Phase 2 and Phase 3 trials and is under regulatory review. It has not been approved by the FDA.
BBP-418 has been studied in three clinical trials: NCT04800874 (Phase 2), NCT05775848 (Phase 3), and NCT07678775 (Phase 3 open-label extension). All are active but not recruiting. The trials enrolled a total of 202 participants across the United States, Australia, and several European countries.
Yes, BBP-418 is also known as ribitol. The two names refer to the same investigational small molecule being developed by BridgeBio Pharma for LGMD2I.