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LB-100

Phase 1

Myelodysplastic Syndromes | Small molecule | Hematology |Lixte Biotechnology Holdings, Inc.|Last Updated: Apr 8, 2019

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 Enrollment47
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03886662A Study of LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)PHASE1 RECRUITING 47Apr 1, 2019Jul 1, 2021Apr 8, 20191 United States
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Study Endpoints
Primary Endpoints
For Phase Ib - Number of patients with adverse events related to the study treatment as a measure of safety and tolerability of LB-100 study drug
From the first dose of the study drug to 30-days following last dose of the study drug

Number of patients with treatment-related adverse events as assessed by Common Terminology Criteria for Adverse Events v5.0 (CTCAE v5.0)

For Phase 2 - Best overall response rate of patients to the study treatment as a measure of efficacy of LB-100 study drug
At screening and then at the end of Cycle 3 and Cycle 6. (Each cycle is 21 days)

Best overall response rate of the patients to the study treatment as assessed by International Working Group (IWG) 2006 criteria

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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LB-100 for Intravenous administrationEXPERIMENTALPhase Ib: Escalating doses of LB-100 administered. Phase 2: Safe dose of LB-100 from phase Ib administered.
Interventions
NameTypeDescription
LB-100DRUGPhase Ib: Two escalating doses of LB-100 in two separate cohorts will be administered intravenously on days 1, 3 and 5 of a 21-day cycle over 120 minutes. Phase 2: Safe dose of LB-100 as determined from phase Ib will be administered intravenously on days 1, 3 and 5 of a 21-day cycle over 120 minutes.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Patient has signed the Informed Consent Form (ICF) and is able to comply with protocol requirements. 2. Patient has adequate organ function as defined by the following laboratory values: * Creatinine clearance (CrCl) ≥ 60ml/min * Total serum bilirubin \< 1.5 x Upper Li...

Countries:United States
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Competitive Landscape -Myelodysplastic Syndromes 76 trials
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT03886662primaryCompletionDate: changed
LOWMay 24, 2026NCT03886662studyFirstPostDate: changed