Myotonic Dystrophy - Pipeline Insight, 2024

This “Myotonic Dystrophy - Pipeline Insight, 2024” report provides comprehensive insights about 20+ companies and 27+ pipeline drugs in Myotonic Dystrophy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Myotonic Dystrophy: Understanding

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.

Clinical presentation is diverse and can range from asymptomatic electrical myotonia to severe weakness and disability, including cardiac conduction defects, infertility, cataracts, and insulin resistance. A congenital form of DM type 1 is associated with an apparent developmental disability.

Myotonia is characterized by impaired relaxation of muscles after voluntary contraction due to repetitive depolarization of the muscle membrane. Myotonia, due to myotonic dystrophy, improves with repeated exercise and is worsened by exposure to cold. In 1992, the mutation responsible for DM1 was identified as a CTG expansion located in the 3′ untranslated region of the dystrophia myotonica-protein kinase gene (DMPK).

The pathophysiology of DMI is related to the number of CTG repeats, with more repeats correlating with more severe disease. Those with less than 35 repeats are considered normal, and those that manifest with clinical symptoms typically have greater than 50. Maternal transmissions will often result in greater CTG expansion. Additionally, the newly expanded alleles have a bias for further expansions that leads to the genetic phenomenon of anticipation when transmitted from one generation to the next.

MDs are genetic disorders caused by localised mutations of DNA. Such DNA mutations result in a lack of dystrophin protein in DMD and an alteration of the protein production in DMs. No curative therapies are available to treat the pathogenic causes, and the identification of new therapeutic approaches to target genetic mutations is urgently needed. The most recent strategies to counteract MDs concern gene therapy and 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. Physical and occupational therapy is recommended for strengthening weakened muscles, evaluation for orthotics, and durable medical equipment needs. Speech-language pathology (SLP) is required for dysphagia and swallowing studies or dysarthria as indicated. SLP is also utilized for intellectual disabilities and learning strategies.

"Myotonic Dystrophy- Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Myotonic Dystrophy pipeline landscape is provided which includes the disease overview and Myotonic Dystrophy treatment guidelines. The assessment part of the report embraces, in depth Myotonic Dystrophy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Myotonic Dystrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

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

Myotonic Dystrophy Emerging Drugs Chapters

This segment of the Myotonic Dystrophy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II/III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Myotonic Dystrophy Emerging Drugs

Tideglusib: 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.

Pitolisant: Harmony Biosciences

Pitolisant (WAKIX) is a selective histamine 3 (H3) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H3 receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States. Currently the drug is being investigated in Phase II stage of Clinical trial evaluation for the treatment of Myotonic Dystrophy.

ARO-DM1: Arrowhead Pharmaceuticals, Inc.

ARO-DM1 is an RNA interference (RNAi) conjugate designed to specifically silence DMPK mRNA in skeletal muscle. Published literature suggests that the silencing of aberrantly transcribed DMPK mRNA using ARO-DM1 may halt CUGexp-related spliceopathies in patients with DM1 leading to improved muscle strength and function. It belongs to a class of medicines called RNA therapeutics. The drug candidate is administered intravenously in the body. ARO-DM1 is currently in Phase I stage of development for the treatment of myotonic dystrophy.

Myotonic Dystrophy: Therapeutic Assessment

This segment of the report provides insights about the different Myotonic Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Myotonic Dystrophy

• There are approx. 20+ key 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 II/III include, AMO Pharma.

Phases

The report covers around 27+ products under different phases of clinical development like

• Late stage products (Phase II/III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

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

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Myotonic Dystrophy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Myotonic Dystrophy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Myotonic Dystrophy drugs.

Myotonic Dystrophy Report Insights

• Myotonic Dystrophy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Myotonic Dystrophy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Myotonic Dystrophy drugs?
• How many Myotonic Dystrophy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Myotonic Dystrophy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Myotonic Dystrophy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Myotonic Dystrophy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• AMO Pharma
• Harmony Biosciences
• Avidity Biosciences
• Dyne Therapeutics
• Vertex Pharmaceuticals
• Arrowhead Pharmaceuticals, Inc.
• Pepgen Corporation
• Aparito

Key Products

• Tideglusib
• Pitolisant
• AOC 1001
• DYNE-101
• VX-670
• ARO-DM1
• PGN EDODM1
• IONIS-877864

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