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mRNA-3705

Phase 1

Methylmalonic Acidemia | Small molecule | Other |Moderna, Inc.|Last Updated: Dec 23, 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 Enrollment56
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05295433An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705PHASE1 RECRUITING 56 — — Mar 8, 2022Apr 2, 2034Dec 23, 202512 United States, Canada +4
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Study Endpoints
Primary Endpoints
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Drug-Related TEAEs, Unrelated TEAEs, Adverse Events of Special Interests (AESIs), Serious Adverse Events (SAEs) and TEAEs Leading to Discontinuation
Baseline up to follow-up period (up to 6 years + 6 months)
Secondary Endpoints
Percent Change in Plasma Methylmalonic Acid and 2-Methylcitric Acid (2-MC) Levels (Primary Biomarkers) From Baseline up to 18 Months
Baseline, Year 6 (Treatment Period) + Month 6 (Follow up)
Pre- and Postdose Human Methylmalonyl-Coenzyme A Mutase (hMUT) mRNA and SM-86 Levels
Baseline up to 6 years
Change From Baseline in Pretreatment and Post-treatment Annualized Metabolic Decompensation Events (MDEs) Rate
Baseline, Year 6 (Treatment Period) + Month 6 (Follow up)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
mRNA-3705EXPERIMENTALParticipants will receive mRNA-3705 at the same dose levels at the same dosing interval (every 2 weeks \[Q2W\], or every 3 weeks \[Q3W\]) last received in the clinical study of mRNA-3705 in which they initially participated, unless the Sponsor recommends modification.
Interventions
NameTypeDescription
mRNA-3705DRUGA sterile liquid for injection
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Eligibility Criteria
Age Range1 Year — N/A
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: * Completed the assigned dose regimen treatment time period in other clinical studies of mRNA-3705 or is eligible for early transition to this study because they missed more than 3 consecutive doses of study drug due to coronavirus disease 2019 (COVID-19) vaccination during Stud...

Countries:United StatesCanadaFranceNetherlandsSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05295433primaryCompletionDate: changed
LOWMay 24, 2026NCT05295433studyFirstPostDate: changed