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Safusidenib

Phase 3

Grade 2 Glioma (Astrocytoma or Oligodendroglioma) With an IDH1 Mutation | Small molecule | Oncology |Nuvation Bio Inc.|Last Updated: Aug 28, 2026

Target and mechanism

Molecular targetIDH1
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment140

FDA Designations

FAST_TRACK

Clinical trial landscape

Safusidenib · 3 trials · 16 indications

Phase 3 2Phase 2 1
NCT07712757Safety and Efficacy Study of Safusidenib in Participants With Grade 2 IDH1-Mutant GliomaGrade 2 Glioma (Astrocytoma or Oligodendroglioma) With an IDH1 Mutation
NOT YET_RECRUITING140 Analytics
NCT05303519SIGMA (Safusidenib in IDH1 Mutant Glioma Maintenance)Glioma
RECRUITING365 Analytics
PHASE3NOT YET_RECRUITING
Safety and Efficacy Study of Safusidenib in Participants With Grade 2 IDH1-Mutant Glioma
Grade 2 Glioma (Astrocytoma or Oligodendroglioma) With an IDH1 MutationUnlock trial analytics
PHASE3RECRUITING
SIGMA (Safusidenib in IDH1 Mutant Glioma Maintenance)
GliomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Progression Free Survival (PFS) assessed by Blinded Independent Central Review (BICR) per modified Response Assessment in Neuro-Oncology (RANO) 2.0
From the date of randomization until the date of first documented disease progression, approximately 30 months

PFS, defined as time from date of randomization to the date of the first occurrence of radiographic disease progression per modified RANO 2.0 assessed by BICR or death from any cause, whichever occurs earlier

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
Objective Response Rate (ORR) per modified Response Assessment in Neuro-Oncology (RANO) 2.0 assessed by Blinded Independent Central Review (BICR)
From the date of first dose of study drug until the date of first documented disease progression, approximately 18 months

ORR defined as the proportion of participants with the confirmed best overall response of Complete Response (CR), Partial Response (PR) or Minor Response (MR) per modified RANO 2.0, assessed by BICR

Secondary Endpoints

Objective Response Rate (ORR) assessed by BICR per modified RANO 2.0
From the date of randomization until the date of first documented disease progression, approximately 30 months
Time to Next Intervention (TTNI)
From the date of randomization until the date of the start of another anticancer treatment or date of death, approximately 30 months
PFS assessed by the Investigator per modified RANO 2.0
From the date of randomization until the date of first documented disease progression, approximately 30 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
SafusidenibEXPERIMENTALSafusidenib 250 mg BID
PlaceboPLACEBO_COMPARATORPlacebo BID
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 twice daily as a single agent dosed orally on Days 1 through 28 of a 28-day cycle. Participants may continue treatment until disease progression or another reason for discontinuation occurs.
PlaceboDRUGPlacebo administered twice daily as a single agent dosed orally on Days 1 through 28 of a 28-day cycle. Participants may continue treatment with placebo until disease progression or another reason for discontinuation occurs.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo

Key Inclusion Criteria: * Expected survival of ≥12 months. * At least 1 prior surgery for glioma (biopsy, sub-total resection, or gross total resection), with the most recent surgery having occurred at least 90 days (or at least 28 days if biopsy only and postoperative changes have resolved per BIC...

Countries:United StatesAustraliaChina
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Recent Changes (Last 90 Days)

LOWAug 28, 2026NCT05303519lastUpdatePostDate: changed
LOWAug 28, 2026NCT07703436lastUpdatePostDate: changed
LOWAug 28, 2026NCT05303519lastUpdatePostDate: changed
LOWAug 28, 2026NCT07703436lastUpdatePostDate: changed
LOWAug 27, 2026NCT07703436lastUpdatePostDate: changed
LOWAug 27, 2026NCT07703436lastUpdatePostDate: changed
LOWAug 24, 2026NCT07703436lastUpdatePostDate: changed
LOWAug 24, 2026NCT07703436lastUpdatePostDate: changed
LOWAug 11, 2026NCT07703436lastUpdatePostDate: changed
LOWAug 11, 2026NCT07703436lastUpdatePostDate: changed
LOWJul 17, 2026NCT07712757NEW_TRIAL: changed
LOWJul 17, 2026NCT07712757NEW_TRIAL: changed
LOWJul 14, 2026NCT07703436NEW_TRIAL: changed
LOWJul 14, 2026NCT07703436NEW_TRIAL: changed
LOWJul 2, 2026NCT05303519lastUpdatePostDate: changed
LOWJul 2, 2026NCT05303519lastUpdatePostDate: changed
LOWJul 2, 2026NCT05303519lastUpdatePostDate: changed
LOWJul 2, 2026NCT05303519lastUpdatePostDate: changed
LOWJun 11, 2026NCT05303519lastUpdatePostDate: changed
LOWJun 11, 2026NCT05303519lastUpdatePostDate: changed

Frequently asked questions about Safusidenib

What is Safusidenib used for?

Safusidenib is an investigational small molecule being studied for the treatment of IDH1-mutant gliomas, including Grade 2 glioma (astrocytoma or oligodendroglioma) with an IDH1 mutation. It is being evaluated in clinical trials for patients with these brain tumors.

What does Safusidenib target?

Safusidenib is a kinase inhibitor, as indicated by its '-nib' suffix, and is being developed to target IDH1-mutant gliomas. It is designed to interfere with kinase activity in cancer cells that harbor IDH1 mutations.

Who makes Safusidenib?

Safusidenib is being developed by Nuvation Bio Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol NUVB. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with IDH1-mutant glioma.

What phase is Safusidenib in?

Safusidenib is currently in Phase 3 clinical development. It has received Fast Track designation from the FDA. The drug is investigational and has not been approved by regulatory authorities for commercial use.

What clinical trials is Safusidenib in?

Safusidenib is being studied in multiple trials, including the Phase 3 SIGMA trial (NCT05303519) in IDH1-mutant glioma, a Phase 2 study (NCT07703436) in patients who discontinued vorasidenib due to progressive disease, and a Phase 3 trial (NCT07712757) in Grade 2 IDH1-mutant glioma.

Is Safusidenib the same as vorasidenib?

No, Safusidenib is not the same as vorasidenib. Safusidenib is a separate investigational drug being developed by Nuvation Bio. One of its clinical trials specifically enrolls patients with IDH1-mutant glioma who discontinued vorasidenib treatment due to progressive disease.