Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BMN 349 · 1 trial · 1 indication
Number of participant AEs, SAEs, DLTs, AESIs per physician's assessment, abnormal laboratory tests through whole blood samples, abnormal pulmonary function spirometry tests, and 12-lead ECG parameters changes from baseline following a single oral dose of BMN 349
| Arm | Type | Description |
|---|---|---|
| Group A (PiZZ) | OTHER | 5:1 (349:Placebo) |
| Group B (PiMZ) | OTHER | 5:1 (349:Placebo) |
| Name | Type | Description |
|---|---|---|
| BMN 349 | DRUG | 250mg oral tablet |
| Placebo | DRUG | 250mg oral tablet |
Inclusion Criteria: * Participants must have confirmation of PiZZ or PiMZ genotype * Females and males, of any race, 18 to 75 years of age * Nonsmokers, defined as not using tobacco or nicotine-containing products for at least 6 months prior to Screening Exclusion Criteria: * International normal...
BMN 349 is an investigational small molecule being developed for the treatment of Alpha 1-Antitrypsin Deficiency, a rare genetic condition. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
BMN 349 targets Z-AAT, the misfolded alpha-1 antitrypsin protein that accumulates in the liver of patients with Alpha 1-Antitrypsin Deficiency. By targeting Z-AAT, the drug aims to address the underlying cause of the disease.
BMN 349 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company listed on NASDAQ under the ticker symbol BMRN. The company is conducting clinical trials to evaluate the drug's safety and efficacy.
BMN 349 is in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is studying the drug in adult participants with Alpha 1-Antitrypsin Deficiency.
BMN 349 is being studied in a Phase 1 clinical trial with the identifier NCT06738017. This trial is titled 'Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants' and is currently active but not recruiting. The trial is enrolling 6 participants in the United States and United Kingdom.