Biomarin Pharmaceutical Inc. ((BMRN)) announced an update on their ongoing clinical study.
Elevate Your Investing Strategy:
- Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence.
BioMarin Pharmaceutical Inc. is conducting a Phase 1/2 study titled A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy. The study aims to evaluate the safety and tolerability of BMN 351 in patients with Duchenne Muscular Dystrophy (DMD) who have a genetic mutation suitable for exon 51 skipping. This research is significant as it targets a severe genetic disorder with limited treatment options.
The intervention being tested is BMN 351, an anti-sense oligonucleotide administered intravenously. It is designed to skip exon 51 in the dystrophin gene, potentially offering a new therapeutic approach for DMD patients.
The study is interventional with a non-randomized, sequential model and no masking, focusing on treatment. Participants are divided into cohorts receiving different doses of BMN 351, administered weekly over varying durations.
The study began on January 26, 2024, with primary completion expected in 2025. The latest update was submitted on June 16, 2025. These dates are crucial for tracking the study’s progress and anticipating results.
This update could positively influence BioMarin’s stock performance and investor sentiment, as successful results may enhance the company’s portfolio in the competitive DMD treatment market.
The study is ongoing, and further details are available on the ClinicalTrials portal.