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DelveInsight’s, “Myotonic Dystrophy Pipeline Insight 2023” report provides comprehensive insights about 25+ companies and 28+ pipeline drugs in the Myotonic Dystrophy pipeline landscape. It covers the Myotonic Dystrophy pipeline drug profiles, including Myotonic Dystrophy clinical trials 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 pipeline products in this space.

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.

• The leading Myotonic Dystrophy Companies include Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, and others

• Promising Myotonic Dystrophy Pipeline Therapies 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.

• The Myotonic Dystrophy companies and academics are working to assess challenges and seek opportunities that could influence Myotonic dystrophy (DM) R&D. The Myotonic Dystrophy pipeline therapies under development are focused on novel approaches to treat/improve Myotonic dystrophy.

Request a sample and discover the recent breakthroughs happening in the Myotonic Dystrophy Pipeline landscape @ Myotonic Dystrophy Pipeline Outlook Report

Myotonic Dystrophy Overview

Myotonic Dystrophy (DM) is considered a subgroup of myopathy and the most common type of muscular dystrophy that begins in adulthood. 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.

Recent Developmental Activities in the Myotonic Dystrophy Treatment Landscape

• In September 2021, Expansion Therapeutics, Inc., announced the close of an $80 million Series B financing. Cormorant Asset Management led the financing with participation from new investors Westlake Village BioPartners, Surveyor Capital (a Citadel company) and Logos Capital as well as Series A investors RA Capital Management, 5AM Ventures, Kleiner Perkins, Sanofi Ventures and Novartis Venture Fund. Proceeds from the financing were used to advance the company’s small molecule RNA platform (SMiRNA™) to identify clinical candidates in myotonic dystrophy type 1 (DM1), amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and various tauopathies.

• In January 2021, Dewpoint Therapeutics, announced a new research collaboration with Pfizer for the development of potential therapeutics for the treatment of myotonic dystrophy type 1, DM1, a rare genetic disorder and one of two types of myotonic dystrophy. Under the agreement, Dewpoint received an upfront payment and is eligible to receive research, development, and sales milestones payments, totaling up to $239 million should all milestones be achieved.

• In August 2022, Arthex Biotech’s lead investigational product ATX-01 has been granted Orphan Drug Designation (ODD) by the US. 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.

For further information, refer to the detailed Myotonic Dystrophy Drugs Launch, Myotonic Dystrophy Developmental Activities, and Myotonic Dystrophy News, click here for Myotonic Dystrophy Ongoing Clinical Trial Analysis

Myotonic Dystrophy Emerging Drugs Profile

• Mexiletine: Lupin

Mexiletine (NaMuscla), is an investigational drug being developed by Lupin. NaMuscla, is the first and only antimyotonic agent licensed to treat symptomatic myotonia in adults with non-dystrophic myotonic disorders across Europe. In randomized controlled trials, NaMuscla (167 to 500 mg/day) has been shown to significantly reduce myotonia compared to placebo, reducing skeletal muscle hyperexcitability through its use-dependent, voltage-gated, sodium channel blocking actions which are independent of the cause of channel function. This resulted in an improvement in patient quality-of-life and other functional outcomes, with gastro-intestinal discomfort reported as the most common adverse event, demonstrating NaMuscla to be safe and well tolerated. Currently, the drug is being investigated in Phase III stage of Clinical trial evaluation for the treatment of Myotonic Dystrophy Type 1 and Type 2.

• AMO-02: 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. AMO-02 is an inhibitor that has been shown to normalize levels of GSK3ß in transgenic models and in ex vivo tissue samples in patients with DM1 and to reduce levels of the mRNA that is pathogenic for DM1.Currently the drug is being investigated in Phase II/III stage of Clinical trial evaluation for the treatment of Congenital Myotonic Dystrophy.

Myotonic Dystrophy Pipeline Therapeutics Assessment

There are approx. 25+ key Myotonic dystrophy companies which are developing the therapies for Myotonic dystrophy. The companies which have their Myotonic dystrophy drug candidates in the most advanced stage, i.e. Phase III include, Lupin.

Find out more about the Myotonic Dystrophy Pipeline Segmentation, Therapeutics Assessment, and Myotonic Dystrophy Emerging Drugs @ Myotonic Dystrophy Treatment Landscape

Scope of the Myotonic Dystrophy Pipeline Report

• Coverage- Global

• 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.

• 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.

• Myotonic Dystrophy Pipeline Segmentation: Product Type, Molecule Type, Mechanism of Action, Route of Action

Dive deep into rich insights for drugs for Myotonic Dystrophy Pipeline Companies and Therapies, click here @ Myotonic Dystrophy Unmet Needs and Analyst Views

Table of Content

1. Introduction
2. Executive Summary
3. Myotonic dystrophy Overview
4. Myotonic dystrophy Pipeline Therapeutics
5. Myotonic dystrophy Therapeutic Assessment
6. Myotonic dystrophy (DM) – DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Mexiletine: Lupin
9. Drug profiles in the detailed report…..
10. Early Stage Products (Phase II)
11. Pitolisant: Harmony Biosciences
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I)
14. DYNE-101: DYNE Therapeutics
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. ENTR-701: Entrda Therapeutics
18. Drug profiles in the detailed report…..
19. Inactive Myotonic dystrophy Products
20. Myotonic dystrophy Key Companies
21. Myotonic dystrophy Key Products
22. Myotonic dystrophy Unmet Needs
23. Myotonic dystrophy Market Drivers
24. Myotonic dystrophy Market Barriers
25. Myotonic dystrophy Future Perspectives and Conclusion
26. Myotonic dystrophy Analyst Views
27. Myotonic dystrophy Companies
28. Appendix

Got Queries? Find out the related information on Myotonic Dystrophy Mergers and acquisitions, Myotonic Dystrophy Licensing Activities @ Myotonic Dystrophy Recent Trends, and Future Perspectives

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