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BMN 351 · 2 trials · 2 indications
The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.
The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.
| Arm | Type | Description |
|---|---|---|
| BMN 351 | EXPERIMENTAL | Participants from 351-201 enrolling in 351-202 will initially receive BMN 351 at the dose level at which they completed 351-201. Once all 351-201 participants have completed their Week 25 visit in 351-202, available safety and PD data will be analyzed and a single optimal dose level will be selected for 351-202. All participants from 351-201 will transition to that dose level at their next applicable visit. |
| Cohort 1A | EXPERIMENTAL | Cohort 1A will consist of both a single ascending dose (SAD) part and a multiple ascending dose (MAD). BMN 351 will be administered once every 2 weeks during the SAD portion of the study for up to 8 weeks and once weekly during the MAD portion for up to 89 weeks. |
| Cohort 1B | EXPERIMENTAL | BMN 351 low dose will be administered once weekly for up to 97 weeks |
| Cohort 2 | EXPERIMENTAL | BMN 351 medium dose will be administered once weekly for up to 73 weeks |
| Cohort 3 | EXPERIMENTAL | BMN 351 high dose will be administered once weekly for up to 48 weeks |
| Name | Type | Description |
|---|---|---|
| BMN 351 | DRUG | Anti-sense Oligonucleotide BMN 351 will be administered intravenously |
Inclusion Criteria: * Participants must have completed 351-201 without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study * Currently receiving treatment with oral corticosteroids, on a stable dose regimen during 351-201, and must remain on a consi...
BMN 351 is an investigational small molecule being developed for Duchenne Muscular Dystrophy (DMD), a genetic neuromuscular disorder. It is currently in Phase 1 clinical development, meaning it has not yet been approved by regulatory authorities and is still being studied for safety and efficacy in patients.
BMN 351 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. The company is conducting clinical trials to evaluate the drug's safety, tolerability, pharmacokinetics, and pharmacodynamics in participants with Duchenne Muscular Dystrophy.
BMN 351 is in Phase 1 clinical development. The primary study is a Phase 1/2 trial that is active but not recruiting participants. An open-label extension study, listed as Phase 2, is also enrolling by invitation to evaluate long-term safety and efficacy. The drug remains investigational and is not FDA approved.
BMN 351 is being studied in two clinical trials. The first is NCT06280209, a Phase 1/2 study assessing safety, tolerability, pharmacokinetics, and pharmacodynamics in participants with Duchenne Muscular Dystrophy. The second is NCT07573631, an open-label extension study evaluating long-term safety and efficacy. Both trials enroll males aged 4 years and older.
No alternative names for BMN 351 have been reported. It is identified solely by its developmental code name BMN 351 in clinical trial registrations and by the developer, BioMarin Pharmaceutical Inc. Patients and researchers should refer to it by this name when searching for trial information.
The Phase 1/2 trial NCT06280209 has a planned enrollment of 18 male participants with Duchenne Muscular Dystrophy. It is a controlled, open-label study that is not randomized or double-blinded. The extension study NCT07573631 also enrolls 18 participants and is open-label. Both trials are conducted in Italy, Netherlands, Spain, Turkey, and the United Kingdom.