An increase in research and development (R&D) activities in myotonic dystrophy is expected to drive market growth by enhancing the understanding of the disease, accelerating drug discovery, and creating innovative treatments. With more R&D, new therapeutic options such as gene therapies, antisense oligonucleotides, and targeted small molecules can be developed, addressing the unmet needs in myotonic dystrophy care. Increased R&D also attracts funding and partnerships, fostering a competitive environment that can lead to improved patient outcomes and market expansion.

LAS VEGAS, Nov. 27, 2024 /PRNewswire/ — DelveInsight’s ‘Myotonic Dystrophy Pipeline Insight 2024‘ report provides comprehensive global coverage of pipeline myotonic dystrophy therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the myotonic dystrophy pipeline domain.

Key Takeaways from the Myotonic Dystrophy Pipeline Report

• DelveInsight’s myotonic dystrophy pipeline report depicts a robust space with 20+ active players working to develop 22+ pipeline therapies for myotonic dystrophy treatment. 
• Key myotonic dystrophy companies such as Avidity Biosciences, Lupin, AMO Pharma, Harmony Biosciences, Arthex Biotech, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Juvena Therapeutics, EditForce, Inc., Kinea Bio, and others are evaluating new myotonic dystrophy drugs to improve the treatment landscape.
• Promising myotonic dystrophy pipeline therapies such as AOC 1001, Mexiletine, Tideglusib, Pitolisant, ATX-01, DYNE-101, VX-670, ARO-DM1, PGN EDODM1, JUV 161, EF-210, KNA129, and others are under different phases of myotonic dystrophy clinical trials.
• On November 7, 2024, ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression, announced that the US Food and Drug Administration has granted Rare Pediatric Designation (RPD) to ATX-01 for the treatment of myotonic dystrophy type 1.
• In November 2024, Dyne Therapeutics announced that the US Food and Drug Administration has cleared the Investigational New Drug application for DYNE-101, which is being evaluated in the ongoing, global Phase I/II ACHIEVE trial in adults with myotonic dystrophy type 1 (DM1). The ACHIEVE trial currently includes 56 participants and is fully enrolled through the 6.8 mg/kg Q8W cohort (approximate ASO dose).
• In August 2024, PepGen announced that both Health Canada and the United Kingdom Medicines and Healthcare products Regulatory Agency have cleared the Company’s clinical trial application (CTA) submissions for the FREEDOM2 trial, and PepGen expects to initiate patient dosing in the second half of 2024. FREEDOM2 is a Phase 2 randomized, double-blind, placebo-controlled, MAD clinical trial evaluating PGN-EDODM1 in approximately 24 adult patients with DM1 in Canada, the United Kingdom, and in the United States, subject to regulatory clearance for the treatment of myotonic dystrophy.
• In May 2024, Avidity Biosciences announced that the US Food and Drug Administration granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company’s lead clinical development program, for the treatment of myotonic dystrophy type 1.
• In April 2024, PepGen presented a poster on the PGN-EDODM1 program at the 14th International Myotonic Dystrophy Consortium (IDMC-14) Meeting titled “FREEDOM-DM1: Phase 1 Study Design to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1.”
• In March 2024, Entrada achieved a milestone under its global collaboration with Vertex related to the clinical advancement of Vertex’s Phase I/II clinical trial of VX-670, which triggered a USD 75 million payment. The Company expects to receive this payment in the second quarter of 2024. The Company is eligible to receive up to USD 485 million, inclusive of milestones achieved to date, for the successful achievement of certain research, development, regulatory and commercial milestones, and tiered royalties on future net sales for any products that may result from this collaboration agreement.
• In February 2024, PepGen Inc. announced that the US Food and Drug Administration had granted Fast Track designation to PGN-EDODM1, an investigational candidate for the treatment of myotonic dystrophy type 1.
• In January 2024, Juvena Therapeutics receives the FDA Orphan Drug Designation for JUV-161 for the treatment of Myotonic Dystrophy Type 1.

Request a sample and discover the recent advances in myotonic dystrophy treatment drugs @ Myotonic Dystrophy Pipeline Report

The myotonic dystrophy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage myotonic dystrophy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the myotonic dystrophy clinical trial landscape. 

Myotonic Dystrophy Overview

Myotonic dystrophy is a genetic disorder characterized by progressive muscle wasting and weakness. It is caused by mutations in specific genes that lead to an abnormal expansion of repetitive DNA sequences. The two main types of myotonic dystrophy are Myotonic Dystrophy Type 1 (DM1) and Type 2 (DM2). DM1 is caused by a CTG repeat expansion in the DMPK gene, while DM2 is linked to a CCTG repeat expansion in the CNBP gene. 

Symptoms of myotonic dystrophy can vary but often include muscle stiffness (myotonia), weakness, and atrophy, primarily affecting the distal muscles and those of the face. Additional symptoms may involve cataracts, cardiac arrhythmias, endocrine issues, and cognitive difficulties. Diagnosis typically involves a combination of clinical evaluation, family history assessment, and genetic testing to identify the specific mutations. Electromyography can also be used to detect myotonia.

Currently, there is no cure for myotonic dystrophy, and treatment focuses on managing symptoms. This may include physical therapy to maintain muscle function, medications to alleviate myotonia, and regular monitoring for cardiac issues. Genetic counseling is recommended for affected individuals and their families to understand the inheritance patterns and potential risks.

Find out more about myotonic dystrophy treatment drugs @ Drugs for Myotonic Dystrophy Treatment

A snapshot of the Myotonic Dystrophy Pipeline Drugs mentioned in the report:

Learn more about the emerging myotonic dystrophy pipeline therapies @ Myotonic Dystrophy Clinical Trials

Myotonic Dystrophy Therapeutics Assessment

The myotonic dystrophy pipeline report proffers an integral view of the myotonic dystrophy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

Scope of the Myotonic Dystrophy Pipeline Report 

• Coverage: Global 
• Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
• Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
• Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical
• Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy
• Therapeutics Assessment By Mechanism of Action: Myotonin protein kinase expression inhibitors, RNA interference, Sodium channel antagonists, Glycogen synthase kinase 3 beta inhibitors, Histamine H3 receptor antagonists, Histamine H3 receptor inverse agonists, MicroRNA inhibitor, RNA inhibitors, RNA splicing modulators
• Key Myotonic Dystrophy Companies: Avidity Biosciences, Lupin, AMO Pharma, Harmony Biosciences, Arthex Biotech, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Juvena Therapeutics, EditForce, Inc., Kinea Bio, and others.
• Key Myotonic Dystrophy Pipeline Therapies: AOC 1001, Mexiletine, Tideglusib, Pitolisant, ATX-01, DYNE-101, VX-670, ARO-DM1, PGN EDODM1, JUV 161, EF-210, KNA129, and others.

Dive deep into rich insights for new drugs for myotonic dystrophy treatment, visit @ Myotonic Dystrophy Drugs

Table of Contents

For further information on the myotonic dystrophy pipeline therapeutics, reach out @ Myotonic Dystrophy Treatment Drugs

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