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Recently added Catalysts

PEG asparaginase

Phase 1

Lymphoblastic Leukemia | Small molecule | Oncology |Amgen Inc.|Last Updated: Jun 3, 2026

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 Trials1
Total Enrollment90
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05848687TINI 2: Total Therapy for Infants With Acute Lymphoblastic Leukemia IIPHASE1 RECRUITING 90Nov 3, 2023Dec 1, 2033Jun 3, 202624 United States, Canada
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Study Endpoints
Primary Endpoints
Minimal Residual Disease
5 years and 2 months

Proportion of patients who are minimal residual disease positive at the end of Induction Intensification

Secondary Endpoints
Ziftomenib Minimum safe and Biologically-Effective Dose in Combination with Chemotherapy
5 years and 6 months
Event Free Survival
8 years
Overall Survival
8 years
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
TreatmentEXPERIMENTALParticipants who meet eligibility criteria will receive remission induction, induction intensification, consolidation I, reinduction block I, reinduction block II, consolidation II, and Maintenance. Interventions: Dexamethasone, Mitoxantrone, PEG-asparaginase, Bortezomib, Vorinostat, Mercaptopurine, Methotrexate and Vincristine, Blinatumomab, Ziftomenib
Interventions
NameTypeDescription
DexamethasoneDRUGGiven orally (PO) or naso-gastrically (NG) or intravenously (IV).
MitoxantroneDRUGGiven IV
PEG asparaginaseDRUGGiven IV
BortezomibDRUGGiven IV
VorinostatDRUGTaken PO or NG
MercaptopurineDRUGGiven PO or NG.
MethotrexateDRUGGiven IV, IM or PO
BlinatumomabDRUGWill be administered at 15 mcg/m2/day for 28 days following induction and reinduction
ZiftomenibDRUG3+3 dose escalation will be done. Dose level 1 will start at 75% of the adult recommended phase two dosing which has been established in phase I studies. Based on tolerability, we will either de-escalate to 50% RP2D (dose level -1) or escalate to 100% RP2D
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Eligibility Criteria
Age RangeN/A — 1 Year
SexALL
Healthy VolunteersNo
Study Sites24

Inclusion Criteria: * Patient is ≤ 365 days of age at the time of diagnosis. * Patient has newly diagnosed CD19 positive acute lymphoblastic leukemia (ALL) or acute undifferentiated leukemia. Subjects with bilineage or biphenotypic acute leukemia are eligible provided they express CD19. Patients wi...

Countries:United StatesCanada
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Recent Changes (Last 90 Days)
LOWJun 4, 2026NCT05848687lastUpdatePostDate: changed
LOWJun 4, 2026NCT05848687lastUpdatePostDate: changed
LOWJun 4, 2026NCT05848687lastUpdatePostDate: changed
LOWJun 4, 2026NCT05848687lastUpdatePostDate: changed
LOWJun 4, 2026NCT05848687lastUpdatePostDate: changed
LOWMay 26, 2026NCT05848687primaryCompletionDate: changed
LOWMay 24, 2026NCT05848687studyFirstPostDate: changed