Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BMN165/day · 1 trial · 1 indication
Blood phe concentration in subjects previously exposed to BMN165 who were administered BMN165 20 or 40 mg/day compared with those who were administered a matching placebo.
| Arm | Type | Description |
|---|---|---|
| BMN165 20mg/day | ACTIVE_COMPARATOR | BMN165 20mg/day self-administered daily |
| 20 mg/day Placebo | PLACEBO_COMPARATOR | 20 mg/day Placebo self-administered daily |
| BMN165 40mg/day | ACTIVE_COMPARATOR | BMN165 40mg/day self-administered daily |
| 40 mg/day Placebo | PLACEBO_COMPARATOR | 40 mg/day Placebo self-administered daily |
| Name | Type | Description |
|---|---|---|
| BMN165 20mg/day | DRUG | BMN165 20mg/day self-administered daily |
| BMN165 40mg/day | DRUG | BMN165 40mg/day self-administered daily |
| Placebo | DRUG | Non-drug placebo, self-administered daily |
INCLUSION CRITERIA Individuals eligible to participate in this study must meet all of the following criteria: * Have completed a prior BMN 165 study (PAL-003, 165-205, or 165-301) prior to screening * Have had a stable BMN 165 dose regimen for at least 14 days prior to screening * Are at least 18 ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| BioMarin Pharmaceutical Inc. | BMRN | 5 | PHASE3 | Pegvaliase |
| PTC Therapeutics, Inc. | PTCT | 2 | PHASE3 | PTC923 |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR444836 |
| Agios Pharmaceuticals, Inc. | AGIO | 1 | PHASE1 | AG-181 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
| Q32 Bio Inc | QTTB | 1 | - | HMI-102 |
BMN 165 is used for the treatment of phenylketonuria (PKU), a metabolic disorder. It is being developed by BioMarin Pharmaceutical Inc. as a monoclonal antibody therapy for patients with this condition.
BMN 165 is a monoclonal antibody, but its specific molecular target has not been disclosed in the available information. The drug is being studied for its effects on phenylketonuria, a metabolic disorder.
BMN 165 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 to evaluate the drug for phenylketonuria.
BMN 165 is in Phase 3 clinical development. It has completed two Phase 3 trials and one Phase 2 trial, all of which are finished. The drug is investigational and has not been approved by regulatory authorities.
BMN 165 has completed three clinical trials: NCT01560286, a Phase 2 study with 24 patients; NCT01819727, a Phase 3 study with 261 patients; and NCT01889862, a Phase 3 study with 215 patients. All trials were conducted in the United States.
BMN 165 is also known as rAvPAL-PEG, as indicated by the Phase 2 trial title. The drug is being developed under both names, with BMN 165 being the primary designation used in later-stage trials.