DelveInsight’s, “Myotonic Dystrophy Pipeline Insight 2026” report provides comprehensive insights about 20+ companies and 22+ 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 pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Myotonic Dystrophy Pipeline Report

• On June 08, 2026- Vertex Pharmaceuticals Incorporated announced a phase 1/2 study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
• On June 01, 2026- Sanofi initiated a Phase 1/Phase 2 study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and efficacy of SAR446268 in knocking down dystrophia myotonica protein kinase (DMPK) messenger ribonucleic acid (mRNA) levels and improving neuromuscular function in DM1 participants receiving a single intravenous (IV) administration of SAR446268. The study consists of a dose escalation part (Part A) during which single ascending doses of SAR446268 will be evaluated in 3 distinct cohorts and an optional fourth dose cohort. Once a safe and effective dose is identified, additional participants will be treated in Part B, the dose expansion phase of the study.
• On June 01, 2026- Dyne Therapeutics conducted a phase 3 study consists of three periods: a Screening period (up to 8 weeks), Placebo-Controlled Period (48 weeks) and a Long-Term Extension Period (24 weeks). An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety and tolerability data of this study at regular intervals.
• DelveInsight’s Myotonic Dystrophy pipeline report depicts a robust space with 20+ active players working to develop 22+ pipeline therapies for Myotonic Dystrophy treatment.
• The leading Myotonic Dystrophy Companies such as AMO Pharma, Lupin, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Aparito and others.
• Promising Myotonic Dystrophy Pipeline Therapies such as AOC 1001, Mexiletine, PGN-EDODM1, SomatoKine/IPLEX, AOC 1001 (del-desiran), Tideglusib, ATX-01 and others.

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Myotonic Dystrophy Overview

Myotonic dystrophy is a chronic, progressive, autosomal dominant disorder characterized by muscle weakness and myotonia, which refers to the delayed relaxation of muscles after contraction. There are two major forms recognized based on clinical and molecular presentation: Myotonic dystrophy type I (DM1), known as Steinert disease, and myotonic dystrophy type II (DM2), or proximal myotonic myopathy which is a milder variety of DMI. These are progressive, multisystem genetic disorders. Clinical presentation is diverse and can range from asymptomatic electrical myotonia to severe weakness and disability, including cardiac conduction defects, infertility, cataracts, and insulin resistance.

Myotonic Dystrophy Emerging Drugs Profile

• Del-desiran (AOC 1001): Novartis AG

Del-desiran, utilizing Avidity’s AOC platform technology, is designed to address the underlying genetic cause of DM1 by reducing levels of toxic DMPK mRNA. Del-desiran consists of a proprietary monoclonal antibody that binds to transferrin receptor 1 (TfR1) and is conjugated to a siRNA that targets DMPK mRNA. Del-desiran has received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). Del-desiran was also the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. This drug was developed by Avidity Biosciences which has been fully acquired by Novartis AG. Currently, the drug is being evaluated in the Phase III stage of its development 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 and 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. This drug belongs to a class of medicines called RNA therapeutics. Preclinical data shows ARO-DM1 achieved greater than 80% silencing of DMPK in skeletal muscle, which was maintained for longer than 85 days. Currently, the drug is being evaluated in the Phase II stage of its development for the treatment of Myotonic Dystrophy.

• ATX-01: Arthex Biotech

ATX-01 is an antimiR oligonucleotide designed to target microRNA 23b(miR-23b), which is involved in the pathogenesis of DM1. It has been demonstrated, in human DM1 myoblast cell lines and in two murine models, that ATX-01 has a unique, dual mechanism of action which reduces toxic DMPK mRNA and increases MBNL protein production. ATX-01 was discovered through ARTHEx’s in-house discovery engine, which was built to identify, design and optimize novel gene expression modulators and ensure their preferential delivery to target tissues affected by the disease. Currently, the drug is being evaluated in the Phase I/II stage of its development for the treatment of Myotonic Dystrophy.

• SAR446268: Sanofi

SAR446268 employs a vectorized RNA interference (RNAi) approach to silence DMPK expression through a single administration. By reducing DMPK transcripts, the gene therapy aims to eliminate the abnormal and toxic RNA foci responsible for splicing defects in muscle tissue, thereby restoring normal splicing and improving muscular function. This approach has the potential to address key symptoms of the disease, including progressive muscle weakness, difficulty relaxing muscles (myotonia), and effects on multiple body systems including heart, lungs, and endocrine functions. The US Food and Drug Administration (FDA) has granted fast track designation to SAR446268 in September 2025. Currently, the drug is being evaluated in the Phase I/II stage of its development for the treatment of Myotonic Dystrophy.

The Myotonic Dystrophy pipeline report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Myotonic Dystrophy with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Myotonic Dystrophy Treatment.
• Myotonic Dystrophy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Myotonic Dystrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Myotonic Dystrophy market.

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Myotonic Dystrophy Companies

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

The 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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

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

• Oligonucleotide
• Peptide
• Small molecule

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Scope of the Myotonic Dystrophy Pipeline Report

• Coverage- Global
• Myotonic Dystrophy Companies- AMO Pharma, Lupin, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Aparito and others.
• Myotonic Dystrophy Pipeline Therapies- AOC 1001, Mexiletine, PGN-EDODM1, SomatoKine/IPLEX, AOC 1001 (del-desiran), Tideglusib, ATX-01 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

Which companies are leading the race in Myotonic Dystrophy drug development? @ Myotonic Dystrophy Emerging Drugs and Major Companies

Table of Contents

1. Introduction
2. Executive Summary
3. Myotonic Dystrophy: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Myotonic Dystrophy– DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Mexiletine: Lupin
9. Mid-Stage Products (Phase II)
10. Pitolisant: Harmony Biosciences
11. Early Stage Products (Phase I)
12. Comparative Analysis
13. ARO-DM1: Arrowhead Pharmaceuticals, Inc.
14. Preclinical and Discovery Stage Products
15. Drug name: Company name
16. Inactive Products
17. Myotonic Dystrophy Key Companies
18. Myotonic Dystrophy Key Products
19. Myotonic Dystrophy- Unmet Needs
20. Myotonic Dystrophy- Market Drivers and Barriers
21. Myotonic Dystrophy- Future Perspectives and Conclusion
22. Myotonic Dystrophy Analyst Views
23. Myotonic Dystrophy Key Companies
24. Appendix

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