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Del-desiran

Phase 3

Myotonic Dystrophy Type 1 | Small molecule | Rare Disease |Atrium Therapeutics, Inc.|Last Updated: Aug 19, 2026

Target and mechanism

Target class-Siran (Sirna)
ModalitySmall molecule

Also known as Del-desiran (AOC 1001)

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment199

FDA Designations

No designations recorded

Clinical trial landscape

Del-desiran · 1 trial · 8 indications

Phase 3 1
NCT07008469Global Open-Label Extension Study of Del-desiran for the Treatment of DM1Myotonic Dystrophy Type 1
ENROLLING BY_INVITATION199 Analytics
PHASE3ENROLLING BY_INVITATION
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
Myotonic Dystrophy Type 1Unlock trial analytics

Study Endpoints

Primary Endpoints

TEAEs
From enrollment to the end of treatment at 4 years

Incidence of treatment emergent adverse events

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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.
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Eligibility Criteria

Age Range16 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites35

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 StatesCanadaDenmarkFranceGermanyItalyJapanNetherlandsSpainUnited Kingdom
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Recent Changes (Last 90 Days)

LOWAug 19, 2026NCT07008469Enrollment: 230 → 199
LOWAug 19, 2026NCT07008469Enrollment: 230 → 199
LOWJul 24, 2026NCT07008469startDate: changed
LOWJul 24, 2026NCT07008469startDate: changed

Frequently asked questions about Del-desiran

What is Del-desiran used for?

Del-desiran is an investigational small molecule being developed for the treatment of Myotonic Dystrophy Type 1 (DM1), a rare genetic disorder. It is currently in Phase 3 clinical development and is being studied in an open-label extension trial for patients with DM1.

Who makes Del-desiran?

Del-desiran is being developed by Atrium Therapeutics, Inc., a biopharmaceutical company. The company is conducting a Phase 3 clinical trial of Del-desiran for the treatment of Myotonic Dystrophy Type 1.

What phase is Del-desiran in?

Del-desiran is currently in Phase 3 clinical development. It is being evaluated in an open-label extension study for the treatment of Myotonic Dystrophy Type 1. The drug is investigational and has not been approved by regulatory authorities.

What clinical trials is Del-desiran in?

Del-desiran is being studied in a Phase 3 open-label extension trial with the identifier NCT07008469. This trial is enrolling patients with Myotonic Dystrophy Type 1 by invitation and aims to enroll approximately 199 participants across multiple countries.

Is Del-desiran the same as AOC 1001?

Yes, Del-desiran is also known as AOC 1001. It is an investigational small molecule being developed by Atrium Therapeutics, Inc. for the treatment of Myotonic Dystrophy Type 1.

How does Del-desiran work?

Del-desiran is a small molecule with a target class of -siran, which indicates it is a small interfering RNA (siRNA) therapeutic. It is designed to target the underlying cause of Myotonic Dystrophy Type 1, though the specific molecular target is not disclosed.