Myotonic Dystrophy (DM) - Pipeline Insight, 2022

Myotonic Dystrophy (DM) - Pipeline Insight, 2022

DelveInsight’s, “Myotonic dystrophy (DM) - Pipeline Insight, 2022,” report provides comprehensive insights about 25+ companies and 28+ pipeline drugs in Myotonic dystrophy (DM) 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 (DM) Understanding Myotonic dystrophy (DM): 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. "Myotonic dystrophy (DM)- Pipeline Insight, 2022" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Myotonic dystrophy (DM) pipeline landscape is provided which includes the disease overview and Myotonic dystrophy (DM) treatment guidelines. The assessment part of the report embraces, in depth Myotonic dystrophy (DM) 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, 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 in Myotonic dystrophy (DM) R&D. The therapies under development are focused on novel approaches to treat/improve in Myotonic dystrophy (DM). • 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. This segment of the Myotonic dystrophy (DM) 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 (DM) Emerging Drugs • 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. Further product details are provided in the report…….. Myotonic dystrophy (DM): Therapeutic Assessment This segment of the report provides insights about the different Myotonic dystrophy (DM) drugs segregated based on following parameters that define the scope of the report, such as: • Major Players in Myotonic dystrophy (DM) There are approx. 25+ key companies which are developing the therapies for Myotonic dystrophy (DM). The companies which have their Myotonic dystrophy (DM) drug candidates in the most advanced stage, i.e. Phase III include, Lupin. • Phases DelveInsight’s 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) along with the details of • Pre-clinical and Discovery stage candidates • Discontinued & Inactive candidates • Route of Administration Myotonic dystrophy (DM) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as • Intravenous • Oral • Subcutaneous Molecule Type Products have been categorized under various Molecule types such as • Small Molecule • Antidepressants • Product Type Drugs have been categorized under various product types like Mono, Combination and Mono/Combination. Myotonic dystrophy (DM): Pipeline Development Activities The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Myotonic dystrophy (DM) 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 (DM) drugs. Myotonic dystrophy (DM) Report Insights • Myotonic dystrophy (DM) Pipeline Analysis • Therapeutic Assessment • Unmet Needs • Impact of Drugs Myotonic dystrophy (DM) 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 (DM) drugs? • How many Myotonic dystrophy (DM) 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 (DM)? • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Myotonic dystrophy (DM) 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 (DM) and their status? • What are the key designations that have been granted to the emerging drugs? Key Players • 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 Key Products • 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

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