Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Pegvaliase · 2 trials · 1 indication
The blood Phe measurement following 72 weeks on study is defined as the average of the Week 69 and Week 73 blood Phe measurements. Change from baseline in blood Phe will be compared between participants in the active (pegvaliase) and the control (diet-only) treatment arms. Baseline blood Phe concentration at Part 1 was defined as the average of 3 pre-treatment measurements collected between Screening/Run-in (2) and Day 1 pre-dose (1).
Treatment-emergent adverse events (TEAE) is defined as any AE that newly appeared or worsened in severity after first dose of pegvaliase (pegvaliase arm) or on or after study day 1 (diet-only arm) until 30 days after last dose of the study. Serious adverse event (SAE).
The anti-pegvaliase total antibody (TAb) method captures drug-binding antibodies comprised of different isotypes and specificities. Antibody Positivity = number of subjects testing positive at any study visit / total number of subjects at study visit. The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
The presence of immunoglobulin G (IgG) Antibodies against phenylalanine ammonia lyase (PAL) is measured overtime. The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
The presence of immunoglobulin M (IgM) Antibodies against PAL (phenylalanine ammonia lyase) is measured overtime. PAL IgM was positive if the titer value was greater than or equal to the median of all baseline PAL IgM titers. The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
The presence of IgG Antibodies against polyethylene glycol (PEG) is measured overtime The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
The presence of IgM Antibodies against PEG (polyethylene glycol) is measured overtime The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
Neutralizing antibodies (NAbs) capable of inhibiting the enzymatic activity of pegvaliase were measured. The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
A treatment-emergent AE was defined as any adverse event (AE) newly appearing or worsened in severity following initiation of study drug until 4 weeks after last dose of pegvaliase
Change in blood phenylalanine (Phe) concentration from Parent Study baseline (naïve/pretreatment).
| Arm | Type | Description |
|---|---|---|
| Pegvaliase | EXPERIMENTAL | Treatment Arm |
| Diet Only | OTHER | Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72, initiating pegvaliase treatment beginning Week 73 and, from Weeks 73 through 215. |
| Name | Type | Description |
|---|---|---|
| Pegvaliase | DRUG | Pegvaliase 2.5mg/10mg/20mg/40mg/60mg self-administered from 1 time up to 7 times a week |
| Diet Only | OTHER | Diet Control |
Inclusion Criteria * Is 12 to 17 years old (US), inclusive, or 12 to 15 years (EU), inclusive, at the start of the Screening/Run-in Period (Day -28). * Diagnosis of PKU and failure to maintain recommended blood Phe levels on existing management (sapropterin dihydrochloride and Phe-restricted diet) ...
| 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 |
Pegvaliase is an investigational therapy being developed for the treatment of Phenylketonuria (PKU), a metabolic disorder. It is currently in Phase 3 clinical development and is not yet approved by the FDA. The drug is being studied in both adults and adolescents with PKU.
Pegvaliase is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. BioMarin is conducting Phase 3 clinical trials to evaluate the safety and efficacy of Pegvaliase in patients with Phenylketonuria (PKU).
Pegvaliase is currently in Phase 3 clinical development for the treatment of Phenylketonuria (PKU). It is an investigational drug and has not received FDA approval. Two Phase 3 trials have been conducted or are ongoing, with one completed and one active but not recruiting.
Pegvaliase has been studied in two Phase 3 clinical trials. The first, NCT03694353, evaluated self-administered injections of Pegvaliase in adults with PKU and has been completed. The second, NCT05270837, is an active trial evaluating Pegvaliase in adolescents aged 12-17 with PKU, conducted in the United States and Germany.
Pegvaliase is not FDA approved. It is an investigational drug currently in Phase 3 clinical trials for the treatment of Phenylketonuria (PKU). The drug is being studied in both adult and adolescent populations, but it has not yet received regulatory approval.