Myotonic Dystrophy Clinical Trials and Studies 2025: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies by DelveInsight

DelveInsight's, "Myotonic Dystrophy Pipeline Insight" report provides comprehensive insights about 20+ companies and 27+ pipeline drugs in Myotonic Dystrophy pipeline landscape. It covers the Myotonic Dystrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Myotonic Dystrophy therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive Myotonic Dystrophy pipeline products in this space.

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Key Takeaways from the Myotonic Dystrophy Pipeline Report

* In February 2025:- Avidity Biosciences Inc.:- A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1. The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks.
* In January 2025:- ARTHEx Biotech S.L.:- The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo.
* DelveInsight's Myotonic Dystrophy pipeline report depicts a robust space with 20+ active players working to develop 27+ pipeline therapies for Myotonic Dystrophy treatment.
* The leading Myotonic Dystrophy Companies such as AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Aparito , and others.
* Dystrophy Therapies such as AOC 1001 (del-desiran), Tideglusib, ATX-01, IONIS-DMPKRx, VX-670, DYNE-101 , and others.

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Myotonic Dystrophy Emerging Drugs Profile

* Tideglusib: AMO Pharma

AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has potential for use in additional CNS, neuromuscular and other orphan indications. AM0-02 is a clinical stage investigational medicine for the treatment of the severe form of congenital myotonic dystrophy known as DM1 or Steinert disease. In cellular and animal models of DM1 as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3Ss) has been shown to increase.

* Pitolisant: Harmony Biosciences

Pitolisant (WAKIX) is a selective histamine 3 (H3) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H3 receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States. Currently the drug is being investigated in Phase II stage of Clinical trial evaluation for the treatment of Myotonic Dystrophy.

* ARO-DM1: Arrowhead Pharmaceuticals, Inc.

ARO-DM1 is an RNA interference (RNAi) conjugate designed to specifically silence DMPK mRNA in skeletal muscle. Published literature suggests that the silencing of aberrantly transcribed DMPK mRNA using ARO-DM1 may halt CUGexp-related spliceopathies in patients with DM1 leading to improved muscle strength and function. It belongs to a class of medicines called RNA therapeutics. The drug candidate is administered intravenously in the body. ARO-DM1 is currently in Phase I stage of development for the treatment of myotonic dystrophy.

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Myotonic Dystrophy Companies

AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Aparito, and others.

Myotonic Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

* Oral
* Intravenous
* Subcutaneous
* Parenteral
* Topical

Myotonic Dystrophy Products have been categorized under various Molecule types such as

* Recombinant fusion proteins
* Small molecule
* Monoclonal antibody
* Peptide
* Polymer
* Gene therapy

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Scope of the Myotonic Dystrophy Pipeline Report

* Coverage- Global
* Myotonic Dystrophy Companies- AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Aparito , and others.
* Myotonic Dystrophy Therapies- AOC 1001 (del-desiran), Tideglusib, ATX-01, IONIS-DMPKRx, VX-670, DYNE-101 , and others.
* Myotonic Dystrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Myotonic Dystrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Content

* Introduction
* Executive Summary
* Myotonic Dystrophy: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Myotonic Dystrophy- DelveInsight's Analytical Perspective
* Late Stage Products (Phase II/III)
* Tideglusib: AMO Pharma
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* Pitolisant: Harmony Biosciences
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* Comparative Analysis
* ARO-DM1: Arrowhead Pharmaceuticals, Inc.
* Drug profiles in the detailed report.....
* Preclinical and Discovery Stage Products
* Drug name: Company name
* Drug profiles in the detailed report.....
* Inactive Products
* Myotonic Dystrophy Key Companies
* Myotonic Dystrophy Key Products
* Myotonic Dystrophy- Unmet Needs
* Myotonic Dystrophy- Market Drivers and Barriers
* Myotonic Dystrophy- Future Perspectives and Conclusion
* Myotonic Dystrophy Analyst Views
* Myotonic Dystrophy Key Companies
* Appendix

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