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Plixorafenib

Phase 1

BRAF V600E Mutation | Small molecule | Other |Incyte Corporation|Last Updated: Apr 16, 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
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06610682A Trial to Evaluate CSF ctDNA and Safety of Plixorafenib Alone or With Retifanlimab in Patients With BRAF-altered GliomaEARLY_PHASE1 RECRUITING 24Apr 7, 2025Jun 30, 2028Apr 16, 20261 United States
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Study Endpoints
Primary Endpoints
Detection rate of BRAF-V600E ctDNA in CSF and/or plasma
surgery, baseline (C1D1) and pre-cycle 2 (week 4)

Proportion of patients with detectable ctDNA in CSF and/or plasma detected at surgery, baseline (C1D1), and pre-Cycle 2 (week 4).

Secondary Endpoints
Compare BRAF-V600 ctDNA levels in CSF and plasma after treatment among patients with different radiographic responses
up to 16 weeks
Radiographic response rate in each arm
up to 16 weeks
Percentage of patients with serious Treatment-Emergent Adverse Events (TEAEs) treated with plixorafenib alone or in combination with retifanlimab.
up to 24 months
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Plixorafenib alone (Arm A)EXPERIMENTALPatients will start the study drug (plixorafenib 900mg daily 30 minutes after a full meal or meal supplement) 7-28 days post-operatively, when clinically stable. Patients will take the drug daily by mouth continuously for 28-day cycles until progressive disease or up to 24 cycles. MRI will be performed post-operatively (between surgery and start of study drug) for evaluation of measurable disease, at the beginning of Cycle 2, then at the beginning of every odd cycle. Blood and CSF samples will be obtained on day of surgery, C1D1 (baseline), pre-C2 (week 4) and with every odd cycle up to and including C7 and EOT
Plixorafenib in combination with retifanlimab (Arm B)EXPERIMENTALParticipants will receive one dose of retifanlimab prior to surgery. Following surgery, the participants will start plixorafenib 7-28 days post-operatively, when clinically stable. Retifanlimab (administered by IV every 28 days) will be restarted after one cycle of plixorafenib or after the 2nd cycle of plixorafenib per physician discretion. Patients will take the drugs in 28-day cycles until progressive disease or up to 24 cycles. MRI, blood, CSF and other study assessments will be performed as for Arm A.
Interventions
NameTypeDescription
PlixorafenibDRUGPatients will start the study drug (plixorafenib 900mg daily 30 minutes after a full meal or meal supplement) 7-28 days post-operatively, when clinically stable. Patients will take the drug daily by mouth continuously for 28-day cycles until progressive disease or up to 24 cycles. MRI will be performed post-operatively (between surgery and start of study drug) for evaluation of measurable disease, at the beginning of Cycle 2, then at the beginning of every odd cycle. Blood and CSF samples will be obtained on day of surgery, C1D1 (baseline), pre-C2 (week 4) and with every odd cycle up to and including C7 and EOT.
RetifanlimabDRUGInvestigators will evaluate the sensitivity of ctDNA from plasma and CSF at baseline and over time in response to treatment with plixorafenib alone or in combination with retifanlimab in patients with BRAF-V600E mutant glioma refractory to prior therapies.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Arm A Only: * Patient must have received prior BRAF and/or MEK inhibitor therapy. 1. Prior RAF dimer disruptor or pan-RAF inhibitor not allowed 2. Prior immunotherapy allowed * The following intervals from previous treatments should have elapsed prior to enrollment: a. BRAFi/MEKi ...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06610682primaryCompletionDate: changed
LOWMay 24, 2026NCT06610682studyFirstPostDate: changed