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safusidenib

Phase 3

Glioma | Small molecule | Oncology |Nuvation Bio Inc.|Last Updated: Mar 5, 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment365
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05303519SIGMA (Safusidenib in IDH1 Mutant Glioma Maintenance)PHASE3 RECRUITING 365Jun 5, 2023Dec 1, 2030Mar 5, 202645 United States, Australia +1
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Study Endpoints
Primary Endpoints
Part 1: Incidence of adverse events (AEs) and serious adverse events (SAEs)
From participants sign ICF to 30 days after last dose,average 2 years

calculate Percentage and numbers of participants with treatment emergent adverse events (TEAEs) and serious adverse events (SAEs) assessed by CTCAE 5.0

Part 2: Progression-free survival (PFS) assessed by Blinded Independent Central Review (BICR) per Response Assessment in Neuro-Oncology (RANO) 2.0
From randomization until the date of first documented disease progression, average 2 years

PFS is defined as the time from randomization to the date of the first documented disease progression assessed by BICR per RANO 2.0 or death (by any cause in the absence of disease progression).

Part 3 Objective Response Rate (ORR) (Complete Response (CR), Partial Response (PR) and Minor Response (MR)) assessed by Blinded Independent Central Review (BICR) per Response Assessment in Neuro-Oncology (RANO) 2.0
From the first dose of study drug until the date of first documented disease progression, average 18 months
Secondary Endpoints
Part 1: Cmax of safusidenib
on Cycle 1 Day 1 and Day 8 (every cycle is 28 days)
Part 1: Tmax of safusidenib
on Cycle 1 Day 1 and Day 8 (every cycle is 28 days)
Part 1: AUC8h of safusidenib
on Cycle 1 Day 1 and Day 8 (every cycle is 28 days)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
safusidenib 125mg bid (part 1)EXPERIMENTALsafusidenib 125mg bid administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with safusidenib until disease progression or development of other unacceptable toxicity.
safusidenib 250mg bid (part 1)EXPERIMENTALsafusidenib 250mg bid administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with safusidenib until disease progression or development of other unacceptable toxicity.
safusidenib 500mg qd (part 1)EXPERIMENTALsafusidenib 500mg qd administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with safusidenib until disease progression or development of other unacceptable toxicity.
safusidenib 375mg bid (part 1)EXPERIMENTALsafusidenib 375mg bid administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with safusidenib until disease progression or development of other unacceptable toxicity.
safusidenib 500mg bid (part 1)EXPERIMENTALsafusidenib 500mg bid administered continuously as a single agent dosed orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with safusidenib until disease progression or development of other unacceptable toxicity.
safusidenib 250mg bid (Part 2)EXPERIMENTALsafusidenib administered continuously as dosed single agent orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment until disease progression or another reason for discontinuation occurs.
placebo (Part 2)PLACEBO_COMPARATORPlacebo administered continuously as dosed single agent orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with placebo until disease progression or another reason for discontinuation occurs.
safusidenib 250mg bid (Part 3)EXPERIMENTALsafusidenib administered continuously as dosed single agent orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment until disease progression or another reason for discontinuation occurs.
Interventions
NameTypeDescription
safusidenibDRUGsafusidenib administered continuously as dosed single agent orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with agent safusidenib until disease progression or development of other unacceptable toxicity.
PlaceboDRUGPlacebo administered continuously as dosed single agent orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with placebo until disease progression or another reason for discontinuation occurs.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites45

Key Inclusion Criteria for Part 1: 1. Patient must be ≥ 18 years of age at the time of signing the informed consent form (ICF). 2. Patient must have histologically confirmed recurrent or progressive WHO Grade 2 glioma or Grade 3 glioma with IDH1 R132H or R132C mutation confirmed by immunohistochemi...

Countries:United StatesAustraliaChina
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05303519primaryCompletionDate: changed
LOWMay 24, 2026NCT05303519studyFirstPostDate: changed