New York, USA, Feb. 15, 2023 (GLOBE NEWSWIRE) -- Myotonic Dystrophy Pipeline Appears Robust With 25+ Key Pharma Companies Actively Working in the Therapeutics Segment | DelveInsight
Myotonic Dystrophy (DM) is considered a subgroup of myopathy and the most common type of muscular dystrophy that begins in adulthood.The current therapeutic developments at RNA or DNA levels should be applicable in principle to the CNS. Therapeutic molecular tools to eliminate toxic RNAs, release the sequestrated mediators, or to remove expanded repeats in the DMPK gene should also be efficient in brain cells. The most recent strategies to counteract MDs concern gene therapy and the repurposing of drugs. Maximizing health and functional independence is the goal of supportive management. This is geared towards monitoring and treating all the medical issues associated with DM.The rapid technological advancements in cellular and molecular biology in genomics research have contributed greatly to the growing gene therapy market in recent years.
DelveInsight’s 'Myotonic Dystrophy Pipeline Insight – 2023' report provides comprehensive global coverage of available, marketed, and 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 25+ active players working to develop 28+ pipeline therapies for myotonic dystrophy treatment.
- Key myotonic dystrophy companies such as Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana, Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, and others are evaluating new drugs for myotonic dystrophy to improve the treatment landscape.
- Promising myotonic dystrophy pipeline therapies in various stages of development include Mexiletine, Tideglusib, Pitolisant, AOC 1001, DYNE-101, NXEN04, Research programme: neuromuscular gene therapies, ENTR-701, ATX-01, NT-0200, NT 0231 F, ENZ-001, ENZ003, AT 466, Dyne-102, PGN EDODM1, Research Programme: Type1 Muscular Dystrophy, Research programme: myotonic dystrophy therapeutics, and others.
- In August 2022, the US granted Arthex Biotech’s lead investigational product ATX-01 Orphan Drug Designation (ODD). Food and Drug Administration (FDA). ATX-01 is under development for the treatment of Myotonic Dystrophy Type 1 (DM1), a serious, debilitating, and life-threatening disease with no known cure or currently available drug treatment.
- In July 2022, Dyne Therapeutics, Inc. announced that the New Zealand Medicines and Medical Devices Safety Authority cleared its clinical trial application to initiate its Phase I/II multiple ascending doses (MAD) clinical trials of DYNE-101 in patients with myotonic dystrophy type 1 (DM1).
- In May 2022, AMO Pharma Limited announced that current investors had made additional investments in the Company in response to progress in AMO Pharma's potentially pivotal REACH-CDM study, a double-blind, placebo-controlled, randomized clinical trial to assess the efficacy and safety of AMO-02 (tideglusib) for the treatment of congenital myotonic dystrophy (CDM1).
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
Steinert, Batten, and Gibb identified myotonic dystrophy (DM) as a rare progressive disorder that is characterized by weakness, especially in muscles. Alterations in the central nervous system also characterize it. There are two major types of myotonic dystrophy—Steinert disease or myotonic dystrophy type 1 (DM1) and a milder form, DM2 or proximal myotonic myopathy. The myotonic dystrophy symptoms vary from person to person and even among the subtypes of myotonic dystrophy despite the overlap. These constellations of myotonic dystrophy symptoms make myotonic dystrophy a multifaceted and complex disorder.
A thorough clinical evaluation, a detailed patient and family history, and identifying characteristic physical findings may lead to a diagnosis of myotonic dystrophy. Several laboratory tests, including blood tests, electromyography (EMG), magnetic resonance imaging (MRI), muscle biopsy, and genetic testing, can help to clarify the clinical myotonic dystrophy diagnosis. Though various diagnostic options exist, the definitive test for myotonic dystrophy is a genetic test.
Currently, no approved treatment provides a permanent cure for myotonic dystrophy, but researchers are looking into ways to help people with these disorders. The current myotonic dystrophy treatment focuses on each individual’s specific symptoms.
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:
| Drugs | Company | Phase | MoA | RoA |
| Mexiletine | Lupin | Phase III | Sodium channel antagonists | Oral |
| AMO-02 | AMO Pharma | Phase II/III | Glycogen synthase kinase 3 beta inhibitors | Oral |
| Pitolisant | Harmony Biosciences | Phase II | Histamine H3 receptor antagonists; Histamine H3 receptor inverse agonists | Oral |
| AOC 1001 | Avidity Biosciences | Phase II | Myotonin protein kinase modulators; RNA interference | Intravenous |
| DYNE-101 | Dyne Therapeutics | Phase I/II | Myotonin protein kinase expression inhibitors; RNA interference | Intravenous |
| ENTR-701 | Entrada Therapeutics | Preclinical | RNA inhibitors | Parenteral |
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 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: Intravenous, Oral, Subcutaneous
- Therapeutics Assessment By Molecule Type: Small Molecule, Antidepressants
- Therapeutics Assessment By Mechanism of Action: Sodium channel antagonists, Glycogen synthase kinase 3 beta inhibitors, Histamine H3 receptor antagonists, Histamine H3 receptor inverse agonists, Myotonin protein kinase expression inhibitors, RNA interference, Myotonin protein kinase modulators, RNA inhibitors
- Key Myotonic Dystrophy Companies: Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana, Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, and others.
- Key Myotonic Dystrophy Pipeline Therapies: Mexiletine, Tideglusib, Pitolisant, AOC 1001, DYNE-101, NXEN04, Research programme: neuromuscular gene therapies, ENTR-701, ATX-01, NT-0200, NT 0231 F, ENZ-001, ENZ003, AT 466, Dyne-102, PGN EDODM1, Research Programme: Type1 Muscular Dystrophy, Research programme: myotonic dystrophy therapeutics, and others.
Dive deep into rich insights for new drugs for myotonic dystrophy treatment; visit @ Myotonic Dystrophy Medications
Table of Contents
| 1. | Myotonic Dystrophy Pipeline Report Introduction |
| 2. | Myotonic Dystrophy Pipeline Report Executive Summary |
| 3. | Myotonic Dystrophy Pipeline: Overview |
| 4. | Analytical Perspective In-depth Commercial Assessment |
| 5. | Myotonic Dystrophy Clinical Trial Therapeutics |
| 6. | Myotonic Dystrophy Pipeline: Late Stage Products (Pre-registration) |
| 7. | Myotonic Dystrophy Pipeline: Late Stage Products (Phase III) |
| 8. | Myotonic Dystrophy Pipeline: Mid Stage Products (Phase II) |
| 9. | Myotonic Dystrophy Pipeline: Early Stage Products (Phase I) |
| 10. | Myotonic Dystrophy Pipeline Therapeutics Assessment |
| 11. | Inactive Products in the Myotonic Dystrophy Pipeline |
| 12. | Company-University Collaborations (Licensing/Partnering) Analysis |
| 13. | Key Companies |
| 14. | Key Products in the Myotonic Dystrophy Pipeline |
| 15. | Unmet Needs |
| 16. | Market Drivers and Barriers |
| 17. | Future Perspectives and Conclusion |
| 18. | Analyst Views |
| 19. | Appendix |
For further information on the myotonic dystrophy pipeline therapeutics, reach out @ Myotonic Dystrophy Treatment Drugs
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