Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as AEB1102 (Co-ArgI-PEG), AEB1102, Co-ArgI-PEG
Pegzilarginase · 5 trials · 4 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.
Includes significant changes in hematology, chemistry and coagulation laboratory studies as well as in physical exam and vital signs
the dose level at which no more than 1/6 patients experiences dose-limiting toxicity
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. |
| AEB1102 | EXPERIMENTAL | Each patient may receive AEB1102 administered IV for up to approximately 4 years. |
| AEB1102 Dose Escalation Cohort 1 | EXPERIMENTAL | 3 patients dosed at 0.01 mg/kg until MTD determined |
| AEB1102 Dose Escalation Cohort 2 | EXPERIMENTAL | 4 patients dosed at 0.02 mg/kg until MTD determined |
| AEB1102 Dose Escalation Cohort 3 | EXPERIMENTAL | 4 patients dosed at 0.04 mg/kg until MTD determined |
| AEB1102 Dose Escalation Cohort 4 | EXPERIMENTAL | 4 patients dosed at 0.08 mg/kg until MTD determined |
| AEB1102 Dose Escalation Cohort 5 | EXPERIMENTAL | 3 patients dosed at 0.12 mg/kg until MTD determined |
| AEB1102 Dose Escalation Cohort 6 | EXPERIMENTAL | 4 patients dosed at 0.18 mg/kg until MTD determined |
| AEB1102 Dose Escalation Cohort 7 | EXPERIMENTAL | 5 patients dosed at 0.27 mg/kg until MTD determined |
| AEB1102 Dose Escalation Cohort 8 | EXPERIMENTAL | 7 patients dosed at 0.40 mg/kg until MTD determined |
| AEB1102 Dose Escalation Cohort 9 | EXPERIMENTAL | 7 patients dosed at 0.33 mg/kg until MTD determined MTD determined at 0.33 mg/kg |
| AEB1102 Expansion | EXPERIMENTAL | Uveal: 11 patients dosed at 0.33 mg/kg Cutaneous Melanoma: 11 dosed at 0.33 mg/kg SCLC: 13 patients dosed at 0.33 mg/kg |
| 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 |
| AEB1102 | DRUG | modified human arginase I |
| Co-ArgI-PEG | DRUG | Administered IV |
| 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 therapy being developed for Arginase 1 Deficiency, a rare inherited disorder that causes elevated arginine levels, and it has also been studied in small-cell lung cancer. It is not yet approved and remains in clinical development.
Pegzilarginase has completed a Phase 3 trial in Arginase 1 Deficiency and a Phase 1 trial in small-cell lung cancer. No active trials are currently listed, so the program is not presently enrolling. It is investigational and has not been approved by the FDA.
Pegzilarginase is being developed by Spyre Therapeutics, Inc., which trades on the Nasdaq under the ticker SYRE. The company is responsible for the clinical program evaluating the drug in Arginase 1 Deficiency and small-cell lung cancer.
Pegzilarginase has been evaluated in two completed trials: NCT03921541, a Phase 3 placebo-controlled study in Arginase 1 Deficiency with 32 patients across the United States, Austria, Canada, France, Germany, Italy, and the United Kingdom, and NCT03371979, a Phase 1 study of Pegzilarginase plus pembrolizumab in extensive disease small-cell lung cancer with 68 patients.
No, Pegzilarginase is not FDA approved. It is an investigational drug that has completed Phase 3 and Phase 1 clinical trials but has not received marketing authorization. It remains in clinical development for Arginase 1 Deficiency and small-cell lung cancer.