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Vorasidenib

Phase 1

Astrocytoma | Small molecule | Oncology |Merck & Company, Inc.|Last Updated: Mar 12, 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
RandomizedNO_TREATMENT_CONTROLLEDBiomarker
Total Trials1
Total Enrollment60
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05484622Study of Vorasidenib and Pembrolizumab Combination in Recurrent or Progressive IDH-1 Mutant GliomaPHASE1 ACTIVE NOT_RECRUITING 60 — — Jan 20, 2023Aug 30, 2027Mar 12, 202617 United States
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Study Endpoints
Primary Endpoints
Safety Lead-in Phase: Percentage of Participants With Dose-limiting Toxicities (DLTs)
First 21 days of dosing (Cycle 1) in safety lead-in phase
Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
approximately up to 19 months
Percentage of Tumor-infiltrating Lymphocyte (TIL) Cells in Surgically Resected Tumors Following Treatment With Vorasidenib + Pembrolizumab Compared to Untreated Control Tumors
approximately 2 months

TIL is defined as the percentage of tumor-infiltrating lymphocyte cells on a logarithmic scale.

Secondary Endpoints
Overall Survival (OS)
Up to approximately 55 months
AUC: Area Under the Plasma Concentration-Time Curve of Vorasidenib
approximately 16 months
Cmax: Maximum Observed Plasma Concentration of Vorasidenib
approximately 16 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Safety Lead-In Phase: Vorasidenib + PembrolizumabEXPERIMENTALParticipants will receive vorasidenib orally, once daily (QD) in combination with pembrolizumab 200 mg intravenous (IV) infusion, once every 3 weeks (Q3W) in each 21-day cycle until disease progression, unacceptable toxicity or other discontinuation criteria are met.
Randomized Perioperative Phase: Vorasidenib + PembrolizumabEXPERIMENTALParticipants will receive vorasidenib recommended combination dose (RCD) determined in the Safety Lead-in phase, orally, QD from Day 1 to 28 in combination with pembrolizumab 200 mg IV infusion, Q3W on Days 1 and 22 of a 28-day cycle prior to surgery.
Randomized Perioperative Phase: Vorasidenib OnlyEXPERIMENTALParticipants will receive vorasidenib orally, QD from Day 1 to 28 of a 28-day cycle prior to surgery.
Randomized Perioperative Phase: Untreated Control GroupNO_INTERVENTIONParticipants will not receive any treatment prior to surgery.
Interventions
NameTypeDescription
VorasidenibDRUGAdministered orally as tablets.
PembrolizumabDRUGAdministered as IV infusion.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites17

Inclusion Criteria: 1. Have Karnofsky Performance Status (KPS) of ≥ 70%. 2. Have expected survival of ≥ 3 months. 3. Have histologically confirmed Grade 2 or Grade 3 glioma (per the 2016 or 2021 World Health Organization \[WHO\] Classification of Tumors of the central nervous system) 4. Have: 1...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT05484622studyFirstPostDate: changed