Myotonic Dystrophy Pipeline Assessment 2024 | In-depth Insights Into the Emerging Drugs, Latest FDA Approvals, Clinical Trials, Ter, Key Companies

Myotonic Dystrophy Pipeline Assessment 2024 | In-depth Insights Into the Emerging Drugs, Latest FDA Approvals, Clinical Trials, Ter, Key Companies
Myotonic Dystrophy Pipeline Insight
The leading companies working in the Myotonic Dystrophy Market include 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.

As per DelveInsight’s assessment, globally, about 25+ key pharma and biotech companies are working on 28+ pipeline drugs in the Myotonic Dystrophy therapeutics landscape based on different Routes of Administration (RoA), Mechanism of Action (MoA), and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.

Myotonic Dystrophy Pipeline Insight, 2024” report by DelveInsight outlines a comprehensive assessment of the present clinical/non-clinical development activities and growth prospects across the Myotonic Dystrophy Market. 

The Myotonic Dystrophy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers, acquisition, funding, designations, and other product-related details.

Myotonic Dystrophy Pipeline Analysis

The report provides insights into: 

  • The report provides detailed insights into the emerging therapies for the treatment of Myotonic Dystrophy and the aggregate therapies developed by major pharma companies.
  • It accesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of clinical development.
  • It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
  • The report evaluates the drugs that are 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. 
  • It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Myotonic Dystrophy market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.  

Major Clinical, Commercial, and Regulatory Activities in the Myotonic Dystrophy Therapeutics Market

  • 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 milestone payments totaling up to $239 million, should all milestones be achieved.
  • In August 2024, Arthex Biotech’s lead investigational product, ATX-01, was granted Orphan Drug Designation (ODD) by the US. Food and Drug Administration (FDA). ATX-01 is under development for treating Myotonic Dystrophy Type 1 (DM1), a serious, debilitating, and life-threatening disease with no known cure or currently available drug treatment.

DelveInsight’s Myotonic dystrophy pipeline report covers around 28+ products under different phases of clinical development like – 

  • Late-stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I)
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates

Route of Administration

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

  • Intravenous
  • Oral
  • Subcutaneous

Molecule Type

The Report Covers the Pipeline Products Under various Molecule types, such as

  • Small Molecule
  • Antidepressants

Learn How the Ongoing Clinical & Commercial Activities will Affect the Myotonic Dystrophy Therapeutic Segment @

Myotonic Dystrophy Therapeutics Landscape

There are approx. 25+ key companies which are developing therapies for Myotonic dystrophy. Currently, Lupin is leading the therapeutics market with its Myotonic dystrophy drug candidates in the most advanced stage of clinical development among all the key players.

The Leading Companies in Myotonic Dystrophy Therapeutics Market Include

  • Lupin
  • AMO Pharma
  • Harmony Biosciences
  • Avidity Biosciences
  • Dyne Therapeutics
  • Nexien BioPharma
  • Locana, Inc.
  • Entrada Therapeutics
  • Arthex Biotech
  • NeuBase Therapeutics
  • Enzerna
  • Astellas Gene Therapies
  • Dyne Therapeutics
  • Pepgen Corporation
  • Sangamo Therapeutics
  • Syros Pharmaceuticals
  • And Many Others

Myotonic Dystrophy Drugs Covered in the Report Include:

  • Mexiletine: Lupin
  • AMO-02: AMO Pharma
  • And Many More

Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Key Companies @

Table of Content

1. Report Introduction

2. Executive Summary

3. Myotonic Dystrophy Current Treatment Patterns

4. Myotonic Dystrophy – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Myotonic Dystrophy Late Stage Products (Phase-III)

7. Myotonic Dystrophy Mid-Stage Products (Phase-II)

8. Myotonic Dystrophy Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Myotonic Dystrophy Discontinued Products

13. Myotonic Dystrophy Product Profiles

14. Key Companies in the Myotonic Dystrophy Market

15. Key Products in the Myotonic Dystrophy Therapeutics Segment

16. Dormant and Discontinued Products

17. Myotonic Dystrophy Unmet Needs

18. Myotonic Dystrophy Future Perspectives

19. Myotonic Dystrophy Analyst Review  

20. Appendix

21. Report Methodology

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Kritika Rehani
Email: Send Email
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States