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R906289 Monosodium

Phase 1

Low Risk Myelodysplastic Syndromes | Small molecule | Hematology |Rigel Pharmaceuticals, Inc.|Last Updated: Oct 21, 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
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment86
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05308264Study of R289 in Patients With Lower-risk Myelodysplastic Syndromes (LR MDS)PHASE1 RECRUITING 86Sep 12, 2022Dec 1, 2026Oct 21, 202515 United States
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Study Endpoints
Primary Endpoints
Safety and Tolerability
2 Year

* Incidence of adverse events (AEs) * Incidence of discontinuation or interruptions of R289 due to AEs * Incidence of dose limiting toxicities (DLTs)

Secondary Endpoints
Outcome Measure: Preliminary Efficacy
24 Weeks for Primary Efficacy; 8 Weeks for Characterized PK
Characterize pharmacokinetics (PK)
8 Weeks
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ExperimentalEXPERIMENTALESCALATION PHASE: Dose Level 1: 250mg PO qd Dose Level 2: 500mg PO qd Dose Level 3: 750 mg PO qd Dose Level 4: 250 mg PO bid Dose Level 5: 500 mg PO AM/250 mg PO PM Dose Level 6: 500 mg PO bid EXPANSION PHASE (randomized 1:1): Dose Level 1: 250 mg PO qd Dose Level 2: 250 mg PO bid
Interventions
NameTypeDescription
R906289 Monosodium (R289 Na)DRUGDrug: R906289 Monosodium (R289 Na) R906289 Monosodium (250mg PO qd, 250mg PO bid, 500 mg PO qd, 500 mg PO bid, 750 mg PO qd, split dose - 500 mg PO AM/250 mg PO PM)
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites15

Inclusion Criteria: * Patient must be ≥ 18 years of age at the time of signing the informed consent. * Must have definitive diagnosis of MDS with very low, low, or intermediate-1 risk (International Prognostic Scoring System (IPSS)-R ≤ 3.5) and ≤5% bone marrow myeloblasts. * Must be relapsed, refra...

Countries:United States
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Competitive Landscape -Myelodysplastic Syndromes 76 trials
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05308264primaryCompletionDate: changed
LOWMay 24, 2026NCT05308264studyFirstPostDate: changed