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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 the treatment of Duchenne Muscular Dystrophy, a genetic muscle-wasting disorder. It is designed for patients whose disease is amenable to exon 51 skipping, a biomarker-selected population. BMN 351 has not been approved and remains in clinical development.
BMN 351 targets exon 51 of the dystrophin gene. By addressing exon 51, the therapy is intended to restore the reading frame of the dystrophin transcript so that a shortened but functional dystrophin protein can be produced. This mechanism applies to Duchenne Muscular Dystrophy patients with mutations correctable by exon 51 skipping.
BMN 351 is being developed by BioMarin Pharmaceutical Inc., which trades on the Nasdaq under the ticker BMRN. BioMarin is the sponsor of the clinical program evaluating the drug in Duchenne Muscular Dystrophy.
BMN 351 is in Phase 2 development. It is an investigational therapy and has not been approved by the FDA. The program includes an ongoing Phase 1/2 study and a Phase 2 open-label extension study, both of which remain active.
BMN 351 is being evaluated in two active trials. NCT06280209 is a Phase 1/2 study assessing safety, tolerability, pharmacokinetics, and pharmacodynamics in Duchenne Muscular Dystrophy. NCT07573631 is a Phase 2 open-label extension study evaluating long-term safety and efficacy. Both enroll males aged 4 years and older across Italy, the Netherlands, Spain, Turkey, and the United Kingdom.
The BMN 351 clinical program has a combined planned enrollment of 36 participants across its two active studies. NCT06280209 plans to enroll 18 participants, and the Phase 2 open-label extension study NCT07573631 also plans to enroll 18 participants. Both trials enroll male patients aged 4 years and older.