Recent Updates
Recently added Catalysts

Del-desiran

Phase 3

Myotonic Dystrophy Type 1 | Small molecule | Other |Atrium Therapeutics, Inc.|Last Updated: Apr 23, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
UNCONTROLLEDDMC
Total Trials1
Total Enrollment230
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07008469Global Open-Label Extension Study of Del-desiran for the Treatment of DM1PHASE3 ENROLLING BY_INVITATION 230Jul 25, 2025Oct 1, 2030Apr 23, 202626 United States, Canada +5
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
TEAEs
From enrollment to the end of treatment at 4 years

Incidence of treatment emergent adverse events

Unlock Study Endpoints
Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Del-desiranEXPERIMENTALDel-desiran (AOC 1001) will be administered seven times per year for up to 4 years.
Interventions
NameTypeDescription
Del-desiran (AOC 1001)DRUGDel-desiran will be administered by intravenous (IV) infusion.
Unlock Study Design Details
Eligibility Criteria
Age Range16 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites26

FROM A PARENT STUDY Key Inclusion Criteria: * Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator. Key Exclusion Criteria: * Bre...

Countries:United StatesCanadaFranceGermanyItalyJapanNetherlands
Unlock Eligibility Criteria
Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07008469primaryCompletionDate: changed
LOWMay 24, 2026NCT07008469studyFirstPostDate: changed