Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BMN 165 · 2 trials · 1 indication
Hypersensitivity AEs will be identified in two ways: * Broad Algorithmic anaphylactic reaction Standardized MedDRA Queries (SMQ) * Modified Hypersensitivity SMQ to include above additional preferred terms
All patients will have their plasma Phenylalanine (Phe) assessed. Please note that "Pharmacokinetics Sub-study" was not performed, and thus no results are available.
| Arm | Type | Description |
|---|---|---|
| BMN 165, 20mg/day | ACTIVE_COMPARATOR | Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 mg/day or 40 mg/day. The randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600 to 900 μmol/L and \> 900 μmol/L). |
| BMN 165, 40mg/day | ACTIVE_COMPARATOR | Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 mg/day or 40 mg/day. The randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600 to 900 μmol/L and \> 900 μmol/L). |
| Group 1 | EXPERIMENTAL | 4-8 week induction of rAvPAL-PEG at 2.5 mg, followed by titration to maintenance dose |
| Name | Type | Description |
|---|---|---|
| BMN 165 | DRUG | After informed consent, eligible subjects will be randomized (1:1) to titrate up to one of two dose regimens: 20 mg/day or 40 mg/day. All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the Induction Period, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly BMN 165 dose to a daily dose regimen of 20 mg/day or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 mg/day or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until a minimum of approximately 26 weeks or a maximum of 36 weeks in the study. |
| BMN 165 (rAvPAL-PEG) | BIOLOGICAL | Subcutaneous injection of rAvPAL-PEG administered from 1 time up to 5 times per week between 2.5mg up to a maximum of 375mg for 24 weeks. |
INCLUSION CRITERIA Individuals eligible to participate in this study must meet all of the following criteria: * A current diagnosis of PKU with the following: * Current blood Phe concentration \>600 µmol/L at screening and * Average blood Phe concentration of \>600 µmol/L over the past 6 mont...
| 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.