BioMarin’s BMN 349 Study: A New Hope for Alpha 1-Antitrypsin Deficiency

Biomarin Pharmaceutical Inc. ((BMRN)) announced an update on their ongoing clinical study.

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 evaluate the safety and pharmacokinetics of BMN 349, a potential treatment for Alpha 1-Antitrypsin Deficiency, an inherited condition that can lead to lung and liver disease.

The intervention being tested is BMN 349, a 250mg oral tablet, designed to assess its safety and tolerability in participants with specific genetic mutations. Participants will receive either BMN 349 or a placebo in a single dose and will be monitored for any adverse events and abnormalities in laboratory and lung function tests.

This Phase 1 study employs a randomized, parallel assignment model with triple masking, meaning that the participant, care provider, and investigator are unaware of the treatment allocation. The primary purpose of the study is treatment-focused, aiming to ensure the safety of the drug.

The study began on November 27, 2024, and the latest update was submitted on September 2, 2025. These dates are crucial as they indicate the study’s progress and the timeline for potential results, which can impact market expectations.

The ongoing study could influence BioMarin’s stock performance positively if the results demonstrate BMN 349’s safety and efficacy, potentially boosting investor confidence. This study also positions BioMarin in a competitive stance within the pharmaceutical industry, particularly in the niche market of genetic disorder treatments.

The study is currently recruiting, with further details available on the ClinicalTrials portal.