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 [https://www.delveinsight.com/report-store/myotonic-dystrophy-pipeline-insight?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr]' 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.
https://mma.prnewswire.com/media/1082265/3528414/DelveInsight_Logo.jpg [https://mma.prnewswire.com/media/1082265/3528414/DelveInsight_Logo.jpg]
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 [https://www.delveinsight.com/sample-request/myotonic-dystrophy-pipeline-insight?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr]
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 [https://www.delveinsight.com/sample-request/myotonic-dystrophy-pipeline-insight?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr]
A snapshot of the Myotonic Dystrophy Pipeline Drugs mentioned in the report:
Drugs Company Phase MoA RoA AOC 1001 Avidity Biosciences Phase III Myotonin protein kinase expression inhibitors; RNA interference Intravenous Tideglusib AMO Pharma Phase III Glycogen synthase kinase 3 beta inhibitors Oral Pitolisant Harmony Biosciences Phase II Histamine H3 receptor antagonists; Histamine H3 receptor inverse agonists Oral PGN EDODM1 Pepgen Corporation Phase II RNA splicing modulators Intravenous ATX-01 Arthex Biotech Phase I/ II MicroRNA inhibitor Intravenous DYNE-101 Dyne Therapeutics Phase I/ II Myotonin protein kinase expression inhibitor Intravenous VX-670 Vertex Pharmaceuticals Phase I/ II RNA inhibitors Intravenous ARO-DM1 Arrowhead Pharmaceuticals, Inc. Phase I Myotonin protein kinase expression inhibitors Intravenous
Learn more about the emerging myotonic dystrophy pipeline therapies @ Myotonic Dystrophy Clinical Trials [https://www.delveinsight.com/sample-request/myotonic-dystrophy-pipeline-insight?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr]
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 [https://www.delveinsight.com/sample-request/myotonic-dystrophy-pipeline-insight?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr]
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 [https://www.delveinsight.com/sample-request/myotonic-dystrophy-pipeline-insight?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr]
Related Reports
Myotonic Dystrophy Market [https://www.delveinsight.com/report-store/myotonic-dystrophy-market?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr]
Myotonic Dystrophy Market Insights, Epidemiology, and Market Forecast - 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key myotonic dystrophy companies, including 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, among others.
Myotonic Dystrophy Epidemiology Forecast [https://www.delveinsight.com/report-store/myotonic-dystrophy-epidemiology-forecast?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr]
Myotonic Dystrophy Epidemiology Forecast - 2032 report delivers an in-depth understanding of the disease, historical and forecasted myotonic dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
Duchenne Muscular Dystrophy Market [https://www.delveinsight.com/report-store/duchenne-muscular-dystrophy-market?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr]
Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast - 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Duchenne muscular dystrophy companies, including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics, among others.
Duchenne Muscular Dystrophy Pipeline [https://www.delveinsight.com/report-store/duchenne-muscular-dystrophy-pipeline-insight?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr]
Duchenne Muscular Dystrophy Pipeline Insight - 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others.
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.
Contact Us
Shruti Thakur
info@delveinsight.com [mailto:info@delveinsight.com]
+14699457679
www.delveinsight.com [https://www.delveinsight.com/]
Logo: https://mma.prnewswire.com/media/1082265/3528414/DelveInsight_Logo.jpg [https://mma.prnewswire.com/media/1082265/3528414/DelveInsight_Logo.jpg]
View original content:https://www.prnewswire.co.uk/news-releases/myotonic-dystrophy-clinical-trial-pipeline-boom-as-over-20-companies-leading-the-charge-in-research-and-development--delveinsight-302317210.html [https://www.prnewswire.co.uk/news-releases/myotonic-dystrophy-clinical-trial-pipeline-boom-as-over-20-companies-leading-the-charge-in-research-and-development--delveinsight-302317210.html]
Photo: https://mma.prnewswire.com/media/1082265/3528414/DelveInsight_Logo.jpg DelveInsight Business Research, LLP
