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Fimepinostat

Phase 1

Diffuse Intrinsic Pontine Glioma | Small molecule | Oncology |Curis, Inc.|Last Updated: Jul 11, 2025

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 Enrollment30
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03893487Fimepinostat in Treating Brain Tumors in Children and Young AdultsEARLY_PHASE1 ACTIVE NOT_RECRUITING 30Aug 7, 2019Aug 31, 2027Jul 11, 202519 United States, Switzerland
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Study Endpoints
Primary Endpoints
Penetration of fimepinostat across the blood brain barrier (BBB)
During surgery or biopsy

Will be analyzed using descriptive statistics from results of fimepinostat concentrations measured within the tumor tissue collected at the time of a standard of care surgery or biopsy. Within each strata, Simon's two-stage design will be used.

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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Treatment (fimepinostat, tumor resection)EXPERIMENTALPatients receive fimepinostat by mouth once daily, on Days -2 to 0. Within 2 hours of receiving fimepinostat on Day 0, patients undergo tumor resection or biopsy as part of their standard of care. MAINTENANCE PHASE: Patients receive fimepinostat by mouth, once daily for days 1-5 each week. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity for up to 12 months from the time treatment begins. Should patients continue to derive clinical benefit, and not experience excess toxicity or progression, patients can continue to receive drug for up to 24 months or longer pending discussion with study chairs and study sponsor.
Interventions
NameTypeDescription
FimepinostatDRUGFimepinostat capsules
Therapeutic Conventional SurgeryPROCEDUREUndergo surgery
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Eligibility Criteria
Age Range3 Years — 39 Years
SexALL
Healthy VolunteersNo
Study Sites19

Inclusion Criteria: * Patients must have one of the following histologically confirmed diagnoses (histologic confirmation from initial diagnosis acceptable, as appropriate): * Stratum A: Newly diagnosed diffuse intrinsic pontine glioma (WHO grade II-IV) - this stratum does not require tissue con...

Countries:United StatesSwitzerland
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT03893487primaryCompletionDate: changed
LOWMay 24, 2026NCT03893487studyFirstPostDate: changed