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AEB1102

Phase 2

Arginase I Deficiency | Small molecule | Other |Spyre Therapeutics, Inc.|Last Updated: Jul 27, 2023

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment30
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03378531A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I DeficiencyPHASE2 COMPLETED 14Dec 7, 2017Dec 15, 2022Jul 27, 20237 United States, Canada +2
NCT02488044A Phase 1/2 Study of AEB1102 in Patients With Arginase I DeficiencyPHASE1 COMPLETED 16Jun 1, 2016Feb 1, 2019Jun 12, 20199 United States, Canada +2
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Study Endpoints
Primary Endpoints
Incidence of treatment-related adverse events
up to 4 years
Number of subjects with adverse events
weekly throughout the study, up to 14 weeks

Includes significant changes in hematology, chemistry and coagulation laboratory studies as well as in physical exam and vital signs

Secondary Endpoints
Cmax Cmin
up to 4 years
Number of subjects with a decrease from baseline in plasma arginine level
Baseline to 2, 4, 6, 8 weeks
Pharmacokinetic profile including Cmax, AUC, Tmax, T1/2 for each subject
At 15 min, 1, 2, 4, 8, 12, 24, 48, 72, and 120 hours following dose escalation
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AEB1102EXPERIMENTALEach patient may receive AEB1102 administered IV for up to approximately 4 years.
Interventions
NameTypeDescription
AEB1102DRUGmodified human arginase I
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Eligibility Criteria
Age Range2 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites7

Inclusion Criteria: 1. Complete treatment in Study CAEB1102-101A without experiencing any clinically significant adverse event or other unmanageable drug toxicity that would preclude continued dosing 2. Confirmation by the Investigator and the Sponsor determine that it is acceptable for the patient...

Countries:United StatesCanadaPortugalUnited Kingdom
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