Biomarin Pharmaceutical Inc. ((BMRN)) announced an update on their ongoing clinical study.
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Study Overview: BioMarin Pharmaceutical Inc. is conducting a clinical study titled ‘A Randomized, Double-Blind, Placebo-Controlled, Single Oral Dose Study Evaluating the Safety and Pharmacokinetics of BMN 349 in Homozygous for the Z Mutation of Alpha 1 Antitrypsin Gene (PiZZ) and Heterozygous for the Z Mutation (PiMZ/MASH).’ The study aims to assess the safety and pharmacokinetics of BMN 349, a potential treatment for individuals with specific genetic mutations affecting lung function.
Intervention/Treatment: The study tests BMN 349, a 250mg oral tablet, against a placebo. The drug is designed to improve safety and tolerability in participants with PiZZ or PiMZ/MASH mutations.
Study Design: This Phase 1 interventional study uses a randomized, parallel assignment model with triple masking (participant, care provider, investigator) to ensure unbiased results. Its primary purpose is treatment-focused, aiming to evaluate the drug’s safety profile.
Study Timeline: The study began on February 21, 2025, with the primary completion date yet to be announced. The latest update was submitted on July 21, 2025, indicating ongoing recruitment and data collection.
Market Implications: The progress of this study could significantly influence BioMarin’s stock performance, as positive safety results may boost investor confidence. The study’s outcome could also impact the competitive landscape in the treatment of genetic lung conditions, potentially positioning BioMarin as a leader in this niche market.
The study is currently ongoing, with further details available on the ClinicalTrials portal.