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GMI-1070

Phase 2

Sickle Cell Disease | Small molecule | Hematology |GlycoMimetics, Inc.|Last Updated: May 13, 2020

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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment96
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01119833Study of GMI-1070 for the Treatment of Sickle Cell Pain CrisisPHASE2 COMPLETED 81May 1, 2010Dec 1, 2013May 13, 202022 United States, Canada
NCT00911495Study of Intravenous GMI-1070 in Adults With Sickle Cell DiseasePHASE1 COMPLETED 15May 1, 2009Sep 1, 2010May 11, 20203 United States
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Study Endpoints
Primary Endpoints
Reduction in time to resolution of vaso-occlusive crisis
Up to 7 days or resolution

Including pain score, feeling ready to leave the hospital, and actual time of leaving the hospital

Safety as Measured by the Number of Participants With Adverse Events
28 days
Secondary Endpoints
Safety during the study
Up to 28 days post last dose
Pharmacokinetics
Baseline thru 36 hrs post last dose
Markers of inflammation and cell stickiness in the blood
Up thru 28 days post last dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
GMI-1070EXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
GMI-1070DRUGIntravenous GMI-1070 given twice a day during hospital stay for sickle cell pain crisis
PlaceboDRUGIntravenous Placebo given twice a day during hospital stay for sickle cell pain crisis
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Eligibility Criteria
Age Range12 Years — 60 Years
SexALL
Healthy VolunteersNo
Study Sites22

Inclusion Criteria: 1. 12 to 60 years of age 2. Confirmed diagnosis of sickle cell disease (HbSS or HbS-β0thalassemia) 3. Diagnosis of VOC at the time of enrollment 4. Hospitalized or in process of admission at the time of enrollment 5. Able to receive the first dose of study drug within 24 hours o...

Countries:United StatesCanada
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