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Low dose of MRM-3379

Phase 2

Fragile X Syndrome | Small molecule | Other |Mirum Pharmaceuticals, Inc.|Last Updated: Jun 5, 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
RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment60
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07209462Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)PHASE2 RECRUITING 60Nov 22, 2025Oct 1, 2027Jun 5, 202615 United States
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Study Endpoints
Primary Endpoints
Incidence and severity of treatment-emergent adverse events and Withdrawal due to adverse events and withdrawal due to adverse events
Baseline to Week 12
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Arm 1: Low Dose of MRM-3379EXPERIMENTALParticipants 16-45 years of age randomized to this arm will receive the low dose of MRM-3379
Arm 2: Middle dose of MRM-3379EXPERIMENTALParticipants 16-45 years of age randomized to this arm will receive the middle dose of MRM-3379
Arm 3: High dose of MRM-3379EXPERIMENTALParticipants 16-45 years of age randomized to this arm will receive the high dose of MRM-3379
Arm 4 :PlaceboPLACEBO_COMPARATORParticipants 16-45 years of age randomized to this arm will receive Placebo
Arm 5: Low dose of MRM-3379 Open-LabelEXPERIMENTALParticipants 13 to \< 16 years of age will receive the low dose of MRM-3370 Open-Label
Interventions
NameTypeDescription
Low dose of MRM-3379DRUGOral capsule
Middle Dose of MRM-3379DRUGOral capsule
High dose of MRM-3379DRUGOral capsule
PlaceboDRUGCapsules matched to study drug without the active pharmaceutical ingredient
Low dose of MRM-3379 Open-LabelDRUGOral capsule
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Eligibility Criteria
Age Range13 Years — 45 Years
SexMALE
Healthy VolunteersNo
Study Sites15

Inclusion Criteria: * Willing and able to provide signed informed consent/assent. Where local regulations permit inclusion of participants deemed unable to provide informed consent, a legally authorized representative must provide informed consent on the participant's behalf, and the participant mu...

Countries:United States
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Recent Changes (Last 90 Days)
LOWJun 5, 2026NCT07209462lastUpdatePostDate: changed
LOWJun 5, 2026NCT07209462lastUpdatePostDate: changed
LOWJun 5, 2026NCT07209462lastUpdatePostDate: changed
LOWJun 5, 2026NCT07209462lastUpdatePostDate: changed
LOWJun 4, 2026NCT07209462lastUpdatePostDate: changed
LOWJun 4, 2026NCT07209462lastUpdatePostDate: changed
LOWJun 4, 2026NCT07209462lastUpdatePostDate: changed
LOWJun 4, 2026NCT07209462lastUpdatePostDate: changed
LOWMay 26, 2026NCT07209462primaryCompletionDate: changed
LOWMay 24, 2026NCT07209462studyFirstPostDate: changed