Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Pegzilarginase · 2 trials · 3 indications
The primary analysis will test the change in the level of plasma arginine between baseline and completion of week 24 assessments. It will compare the change from baseline in plasma arginine between participants treated with pegzilarginase and those treated with placebo.
1\. Number of participants experiencing treatment-related adverse events as assessed by CTCAE v4.0.
1\. Objective Response Rate (ORR:) per RECIST 1.1 • The Objective Response Rate (ORR) is defined as the percentage of subjects whose best objective response (BOR) is either complete response (CR) or partial response (PR). The ORR will be derived from the BOR according to response evaluation criteria in solid tumors (RECIST) v1.1 as assessed by the Investigator.
| Arm | Type | Description |
|---|---|---|
| Pegzilarginase | EXPERIMENTAL | Weekly IV infusions of pegzilarginase plus individualized disease management for 24 weeks |
| Placebo | PLACEBO_COMPARATOR | Weekly IV infusions of placebo plus individualized disease management for 24 weeks |
| Pegzilarginase Long Term Extension | EXPERIMENTAL | After completion of 24 weeks DB treatment, weekly IV infusions of pegzilarginase plus individualized disease management for an additional 150 weeks, with the option to receive treatment by SC after 8 weeks of the LTE study. |
| Pegzilarginase plus Pembrolizumab | EXPERIMENTAL | Phase 1 \& 2 |
| Name | Type | Description |
|---|---|---|
| Pegzilarginase | DRUG | Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated |
| Placebo | DRUG | Individualized disease management which includes severe protein restriction, essential amino acid supplementation and the ammonia scavengers when indicated |
| Pembrolizumab | DRUG | Administered IV |
Inclusion Criteria: Subjects are eligible to be included in the study only if all the following criteria apply: 1. The subject and/or parent/guardian provides written informed consent/assent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF)...
Pegzilarginase is an investigational drug being studied for Arginase I Deficiency and Small-cell Lung Cancer. In Arginase I Deficiency, also called hyperargininemia, it is being evaluated in a Phase 3 trial. For Small-cell Lung Cancer, it was studied in a Phase 1 trial in combination with pembrolizumab.
Pegzilarginase is being developed by Spyre Therapeutics, Inc., which trades under the ticker SYRE. The company is conducting clinical trials for the drug in Arginase I Deficiency and Small-cell Lung Cancer.
Pegzilarginase is in Phase 3 clinical development for Arginase I Deficiency, based on a completed Phase 3 trial. It was also studied in a completed Phase 1 trial for Small-cell Lung Cancer. The drug is investigational and not yet approved.
Pegzilarginase has two completed trials. NCT03921541 is a Phase 3 study in Arginase I Deficiency with 32 participants. NCT03371979 is a Phase 1 study of Pegzilarginase with pembrolizumab in Extensive Disease Small Cell Lung Cancer, enrolling 68 participants.
Pegzilarginase is not FDA approved. It is an investigational drug still in clinical development. Its most advanced trial is a completed Phase 3 study for Arginase I Deficiency, but approval status has not been established.