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 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. The company is conducting clinical trials for this investigational drug in the United States and United Kingdom.
BMN 349 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory agencies and is still undergoing clinical trials to evaluate its safety and efficacy in humans.
BMN 349 is being studied in a Phase 1 clinical trial identified as NCT06738017, titled 'Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants.' This active trial is enrolling 6 participants with Alpha 1-Antitrypsin Deficiency and is being conducted in the United States and United Kingdom.
Yes, BMN 349 is a small molecule drug. It is being developed as an oral or injectable therapeutic for Alpha 1-Antitrypsin Deficiency, a rare disease, and is currently in Phase 1 clinical testing.