Dublin, March 01, 2024 (GLOBE NEWSWIRE) -- The "Myotonic Dystrophy - Pipeline Insight, 2024" drug pipelines has been added to ResearchAndMarkets.com's offering.
This "Myotonic Dystrophy- Pipeline Insight, 2024" report provides comprehensive insights about 24+ companies and 25+ 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.
The repor 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, 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 (GSK3?) has been shown to increase.
AMO-02 is an inhibitor that has been shown to normalize levels of GSK3? 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.
ENTR-701: Entrada Therapeutics
ENTR-701, a proprietary Endosomal Escape Vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer, is the second novel clinical candidate from Entrada's growing pipeline of EEV therapeutics. ENTR-701 is designed to address the underlying cause of myotonic dystrophy type 1 through allele-specific targeting and blocking of the excess repeat-containing transcripts in dystrophia myotonica protein kinase mRNA.
In doing so, ENTR-701 has the potential to restore the function of muscle blind-like proteins, correct the mis-splicing and aberrant expression of downstream transcripts and restore normal muscle function. Data from preclinical studies of ENTR-701 suggest correction of disease relevant biomarkers in various muscle groups. Currently, the product is in preclinical stage of development for the treatment of Myotonic dystrophy.
Myotonic Dystrophy: Therapeutic Assessment
Major Players in Myotonic Dystrophy
There are approx. 24+ 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 25+ products under different phases of clinical development like
- Late stage products (Phase 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
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 Players
- AMO Pharma
- Harmony Biosciences
- Entrada Therapeutics
- Arrowhead Pharmaceuticals, Inc.
- Arthex Biotech
- NeuBase Therapeutics
- Enzerna
Key Products
- Tideglusib
- Pitolisant
- ENTR-701
- ARO-DM1
- ATX-01
- NT-0200
- NT 0231 F
- ENZ-001
For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/6r15ev
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