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Low dose of MRM-3379

Phase 2

Fragile X Syndrome | Small molecule | Rare Disease |Mirum Pharmaceuticals, Inc.|Last Updated: Aug 24, 2026

Target and mechanism

Molecular targetPDE4D
Target classEnzyme
ModalitySmall molecule

Also known as [14C]-MRM-3379, MRM-3379

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment60

FDA Designations

No designations recorded

Clinical trial landscape

Low dose of MRM-3379 · 1 trial · 1 indication

Phase 2 1
NCT07209462Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)Fragile X Syndrome
RECRUITING60 Analytics
PHASE2RECRUITING
Study of MRM-3379 in Male Participants With Fragile X Syndrome (BLOOM)
Fragile X SyndromeUnlock trial analytics

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 to 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)

LOWAug 24, 2026NCT07209462lastUpdatePostDate: changed
LOWAug 24, 2026NCT07209462lastUpdatePostDate: changed
LOWAug 7, 2026NCT07209462lastUpdatePostDate: changed
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LOWJul 28, 2026NCT07209462lastUpdatePostDate: changed
LOWJul 28, 2026NCT07209462lastUpdatePostDate: changed
LOWJul 17, 2026NCT07209462lastUpdatePostDate: changed
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LOWJun 16, 2026NCT07209462lastUpdatePostDate: changed
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LOWJun 10, 2026NCT07209462lastUpdatePostDate: changed
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Frequently asked questions about Low dose of MRM-3379

What is MRM-3379 used for in Fragile X Syndrome?

MRM-3379 is an investigational small molecule being studied for Fragile X Syndrome, a rare genetic condition. It is currently in Phase 2 clinical development, with an active trial evaluating its effects in male participants aged 13 years and older with Fragile X Syndrome.

What does MRM-3379 target?

MRM-3379 targets PDE4D, an enzyme involved in cellular signaling pathways. By inhibiting PDE4D, the drug aims to modulate these pathways, which may be relevant to the treatment of Fragile X Syndrome.

Who is developing MRM-3379?

MRM-3379 is being developed by Mirum Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol MIRM. The company is conducting clinical trials to evaluate the drug's safety and efficacy.

What phase is MRM-3379 in?

MRM-3379 is in Phase 2 clinical development for Fragile X Syndrome. It is also being studied in a Phase 1 trial in healthy volunteers to evaluate its absorption, distribution, metabolism, and excretion. The drug is investigational and not yet approved.

What clinical trials is MRM-3379 in?

MRM-3379 is being studied in two clinical trials. NCT07209462 is a Phase 2 study in male participants with Fragile X Syndrome, and NCT07712718 is a Phase 1 study of [14C]-MRM-3379 in healthy male volunteers to assess its pharmacokinetics.

Is MRM-3379 the same as [14C]-MRM-3379?

MRM-3379 and [14C]-MRM-3379 are related forms of the same drug. [14C]-MRM-3379 is a radiolabeled version used in a Phase 1 study to track the drug's absorption, distribution, metabolism, and excretion in healthy volunteers.