Delpacibart etedesiran Clinical Immunoconjugate, Unconventional ADC, AOC

Antibody Information

Entry ID	306
INN	Delpacibart etedesiran
Status	Clinical
Drug code(s)	AOC 1001
Brand name	None
mAb sequence source	mAb humanized
General Molecular Category	Immunoconjugate, Unconventional ADC, AOC
Format, general category	Full length Ab conjugate
Format details	None
Isotype (Fc)	IgG1
Light chain isotype	kappa
Linker	None
Ave. DAR	None
Conjugated/fused moiety	siRNA
Discovery method/technology	None

Therapeutic information

Target(s)	TfR
Indications of clinical studies	Myotonic dystrophy
Primary therapeutic area	Muscular disorders

Development stage information

Phase lengths*

*The graph represents early-stage clinical development phase lengths. For molecules approved or under evaluation for marketing authorization in the US is provided a complete overview of all clinical development phase lengths. Phase lengths are calculated from the start of the first in human (FIH) study (Start of clinical phase). “Start of Phase 2” bar represents Phase 1 length (Start of clinical phase to start of Phase 2); “Start of Phase 3” bar represents Phase 1+2 length (Start of clinical phase to start of Phase 3); “Date BLA/NDA submitted” bar represents Phase 1+2+3 length (Start of clinical phase to Date BLA/NDA submitted); and “Date of first US approval” bar represents Phase 1 to first US approval length (Start of clinical phase to Date of first US approval).

Most advanced stage of development (global)	Phase 3
Status	Active
Start of clinical phase (IND filing or first Phase 1)	July 15, 2021
Start of Phase 2	August 04, 2022
Start of Phase 3	May 15, 2025
Date BLA/NDA submitted to FDA
Year of first approval (global)	None
Date of first US approval
INN, US product name	None
US or EU approved indications	None

Company information

Company	Avidity Biosciences Inc.
Licensee/Partner	None
Comments about company or candidate	Jan 2025: Planned marketing application submissions in 2026, including in the U.S. and European Union May 8, 2024: Avidity Biosciences, Inc. announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1. https://aviditybiosciences.investorroom.com/2024-05-08-Avidity-Biosciences-Receives-FDA-Breakthrough-Therapy-Designation-for-Delpacibart-Etedesiran-AOC-1001-for-Treatment-of-Myotonic-Dystrophy-Type-1 NCT06411288 Phase 3 started in May 2024. March 2024: Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024. (https://aviditybiosciences.investorroom.com/2024-03-04-Avidity-Biosciences-Announces-Positive-AOC-1001-Long-term-Data-Showing-Reversal-of-Disease-Progression-in-People-Living-with-Myotonic-Dystrophy-Type-1-Across-Multiple-Endpoints-Same-Key-Endpoints-Agreed-for-Phase-3-HARBOR-TM-Trial) NCT05479981 Phase 2 in Muscular Dystrophy Type 1 Received clearance from the U.S. Food and Drug Administration to proceed with the Phase 1/2 MARINA trial under Avidity's initial new drug application in July 2021 Enrolled the first patient in the MARINA trial in October 2021, marking the first time a person has been dosed with an AOC AOC 1001 was granted Orphan Designation by the FDA and the European Medicines Agency (EMA) AOC 1001 was granted Fast Track Designation by the FDA in October 2021. August 2, 2021: Avidity Biosciences, Inc., a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) cleared the company to proceed with the Phase 1/2 MARINA™ clinical trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1).
Full address of company	San Diego, CA 92121 North America United States of America https://www.aviditybiosciences.com/contact-us/

Description/comment

AOC 1001, Avidity's lead program utilizing its Antibody Oligonucleotide Conjugates (AOC) platform, is designed to address the root cause of DM1 by reducing levels of DMPK, the disease-related mRNA. AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA) that targets DMPK mRNA.

Additional information

Anticipated events	Possible BLA / MAA in 2026
Factor(s) contributing to discontinuation	None