An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed the MEX-DM-302 Study (ATLAS Study)

This is an open-label extension study intended to evaluate the long-term safety and efficacy of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2) who have completed the parent study MEX-DM-302.

Start Date27 Feb 2025

Sponsor / Collaborator

Lupin Ltd

[+1]

NCT06862596

/ RecruitingPhase 2/3IIT

A Multicenter, Randomized, Placebo-controlled, Double-blind Clinical Trial: the Efficacy and Safety of Mexiletine Hydrochloride for Amelioration of Motor Dysfunction in Spinal and Bulbar Muscular Atrophy

The purpose of this clinical trial is to evaluate the efficacy and safety of mexiletine hydrochloride in patients with spinal and bulbar muscular atrophy.
The main questions it aims to answer are:
Does mexiletine hydrochloride improve the ALSFRS-R score in spinal and bulbar muscular atrophy patients?
Participants will:
Take mexiletine hydrochloride or a placebo every day for 3 months Visit the hospital once every 4 weeks for evaluations.

Start Date26 Feb 2025

Sponsor / Collaborator

Nagoya University

CTR20243596

/ CompletedNot Applicable

南京泽恒医药技术开发有限公司研制的盐酸美西律片与KYOWA CritiCare Co., Ltd公司持证的盐酸美西律片（商品名：Mexiletine Hydrochloride ®，规格：50 mg）在中国健康受试者中进行的随机、开放、单次给药、两周期、两序列、交叉、空腹及餐后状态下的生物等效性研究

[Translation] A randomized, open-label, single-dose, two-period, two-sequence, crossover, fasting and fed bioequivalence study of mexiletine hydrochloride tablets developed by Nanjing Zeheng Pharmaceutical Technology Development Co., Ltd. and mexiletine hydrochloride tablets (trade name: Mexiletine Hydrochloride ®, specification: 50 mg) licensed by KYOWA CritiCare Co., Ltd. in healthy Chinese subjects

以南京泽恒医药技术开发有限公司研制的盐酸美西律片（规格：50 mg）为受试制剂，以持证商为KYOWA CritiCare Co., Ltd公司的盐酸美西律片（商品名：Mexiletine Hydrochloride®，规格：50 mg）为参比制剂，考察两制剂在空腹及餐后状态下单次给药的药代动力学参数及相对生物利用度，评价两制剂是否具有生物等效性。同时评价两种制剂在健康人体中的安全性和耐受性。

[Translation]

Mexiletine Hydrochloride Tablets (Specification: 50 mg) developed by Nanjing Zeheng Pharmaceutical Technology Development Co., Ltd. were used as the test preparation, and Mexiletine Hydrochloride Tablets (trade name: Mexiletine Hydrochloride®, Specification: 50 mg) from KYOWA CritiCare Co., Ltd. were used as the reference preparation. The pharmacokinetic parameters and relative bioavailability of the two preparations after single administration in the fasting and postprandial states were investigated to evaluate whether the two preparations were bioequivalent. At the same time, the safety and tolerability of the two preparations in healthy humans were evaluated.

Start Date24 Oct 2024

Sponsor / Collaborator

Nanjing Zeheng Pharmaceutical Technology Development Co., Ltd.

100 Clinical Results associated with Mexiletine Hydrochloride

100 Translational Medicine associated with Mexiletine Hydrochloride

100 Patents (Medical) associated with Mexiletine Hydrochloride

3,008

Literatures (Medical) associated with Mexiletine Hydrochloride

01 Mar 2025·Journal of clinical neuromuscular disease

What is in the Myopathy Literature?

Review

Author: Isfort, Michael ; Lacomis, David

Abstract:

This update begins with the incidence and features of statin-associated muscle symptoms, which may often be misattributed. Articles on potential muscle mitochondria dysfunction from statins follow, along with recommendations for possibly avoiding statins in some patients with genetic myopathies. Next, autoimmune myopathies, including immune-mediated necrotizing myopathy, myositis with antimitochondrial antibodies, and overlap myositis with lupus, as well as the role of myxovirus protein A identification in muscle specimens, are addressed. The next section includes reports on the significance of elevated serum aldolase with normal creatine kinase and recommended approaches to evaluate a patient with rhabdomyolysis. A cluster of reports on muscle imaging, particularly using ultrasound and magnetic resonance imaging, are covered. They include studies of inherited and inflammatory myopathies and neck extensor myopathy on topics such as imaging features, patterns of involvement, diagnostic utility, and correlation with histopathology. Last, there are discussions on mexiletine versus lamotrigine for nondystrophic myotonias and the treatment of fatty acid oxidation disorders in adults.

01 Mar 2025·KOREAN JOURNAL OF PHYSIOLOGY & PHARMACOLOGY

Human induced pluripotent stem cell-cardiomyocytes for cardiotoxicity assessment: a comparative study of arrhythmiainducing drugs with multi-electrode array analysis

Article

Author: Park, Soon-Jung ; Park, Na Kyeong ; Park, Yun-Gwi ; Moon, Sung-Hwan ; Choi, Seong Woo ; Choi, Hyung Kyu ; Choi, Ji-Hee

Reliable preclinical models for assessing drug-induced cardiotoxicity are essential to reduce the high rate of drug withdrawals during development. Human induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) have emerged as a promising platform for such assessments due to their expression of cardiacspecific ion channels and electrophysiological properties. In this study, we investigated the effects of eight arrhythmogenic drugs-E4031, nifedipine, mexiletine, JNJ303, flecainide, moxifloxacin, quinidine, and ranolazine-on hiPSC-CMs derived from both healthy individuals and a long QT syndrome (LQTS) patient using multielectrode array systems. The results demonstrated dose-dependent changes in field potential duration and arrhythmogenic risk, with LQTS-derived hiPSC-CMs showing increased sensitivity to hERG channel blockers such as E4031. Furthermore, the study highlights the potential of hiPSC-CMs to model disease-specific cardiac responses, providing insights into genetic predispositions and personalized drug responses. Despite challenges related to the immaturity of hiPSC-CMs, their ability to recapitulate human cardiac electrophysiology makes them a valuable tool for preclinical cardiotoxicity assessments. This study underscores the utility of integrating patientderived hiPSC-CMs with advanced analytical platforms, such as multi-electrode array systems, to evaluate drug-induced electrophysiological changes. These findings reinforce the role of hiPSC-CMs in drug development, facilitating safer and more efficient screening methods while supporting precision medicine applications.

16 Feb 2025·ChemSusChem

Stable and Versatile Pd Precursors for the Preparation of Robust Pd Catalysts Under Continuous‐Flow

Article

Author: Yokoyama, Takashi ; Abe, Yuka ; Sakurada, Naoya ; Yamada, Tsuyoshi ; Kobayashi, Kwihwan ; Niwa, Kosuke ; Ikawa, Takashi ; Sajiki, Hironao

Abstract:

To achieve global sustainability, the chemical and engineering communities require the development of versatile precursors that can be used to synthesize robust catalysts. To meet this demand, we developed a new Pd precursor for highly efficiently incorporating fine Pd‐metal into supports. An atmospherically stable Pd precursor (Pd‐80) was prepared by thermally promoting the aerobic oxidation of tetrakis(triphenylphosphine)palladium. The physical properties of Pd‐80 were investigated by NMR spectroscopy, SEM, XPS, solvent‐relaxation NMR spectroscopy, and dynamic light scattering (DLS) experiments. We also prepared a cordierite‐supported Pd catalyst (Pd/cordierite) by stirring Pd‐80 and cordierite powder in chloroform at room temperature. Pd/cordierite selectively catalyzed the hydrogenation of various reducible functional groups, including alkynes, azides, nitro groups, olefins, CO2Bn, N‐Cbz, O‐Bn, aromatic ketones, and styrene oxide, in continuous‐flow hydrogenation reactions. The Pd/cordierite‐catalyzed continuous‐flow hydrogenation of nitrobenzene derivatives afforded the corresponding anilines, with catalytic activities maintained for over 250 h of continuous operation with a turnover number (TON) of 61,090.

News (Medical) associated with Mexiletine Hydrochloride

17 Feb 2025

·www.biospace.com

Opinion: Companies Take Multiple Tacks Against Myotonic Dystrophy Type 1

horillaz/ Getty Images With its recent data drop for an oligonucleotide candidate, Dyne Therapeutics signals it may become a frontrunner in this disease space alongside Avidity Biosciences, Lupin and AMO Pharma. Last month, Dyne Therapeutics announced positive results in a Phase I/II trial of its candidate DYNE-101. The milestone places Dyne among the companies closest to developing a disease-modifying treatment for myotonic dystrophy type 1. Myotonic dystrophy type 1 (DM1) is a form of muscular dystrophy caused by DMPK mutations on chromosome 19. It affects muscles and many organs across multiple systems that depend on muscular activity. Also known as Steinert disease, it is characterized by the inability to relax muscles at will. DM1 affects 140,000 people in the United States alone, and although there are drugs that address its symptoms, there are no currently approved disease-modifying treatments for the condition. There are, however, more than two dozen investigational drugs in development for DM1. Among the companies looking to fill the unmet need is Dyne, whose DYNE-101 is an antisense oligonucleotide fused with a fragment antibody that binds to the transferrin receptor 1 in muscles. In the Phase I/II ACHIEVE trial , key findings included DMPK RNA level reductions, CASI-22 improvements, early and sustained improvement in myotonia as measured by grip myotonia assessment, and multiple other strength and timed assessments. The trial utilized a multiple ascending dose cohort to identify 6.8 mg/kg every 8 weeks as the optimal dose for future studies. Dyne stated in its press release that it plans to initiate a global registrational expansion cohort of the trial to potentially support a submission for FDA Accelerated Approval in the first half of 2026. Meanwhile, Avidity Biosciences is developing AOC 1001, an antibody oligonucleotide conjugate that targets the DMPK gene to treat DM1. Also known as del-desiran, this investigational treatment is currently being studied in the Phase III HARBOR trial and Avidity has announced plans to submit marketing applications in 2026. In March 2024, Avidity announced positive long-term del-desiran data from the Phase II MARINA-OLE trial showing reversal of disease progression in people living with DM1 across multiple endpoints compared to a matched natural history study population over one year. Those endpoints included grip myotonia assessment, muscle strength and activities of daily living. According to Avidity, additional data from the ongoing Phase I/II MARINA trial for AOC 1001, in which patients were split into placebo-controlled single-dose and multiple ascending dose arms, will be released in the first quarter of this year. While clinical-stage investigational oligonucleotide-based therapy candidates are catching up, repurposed drugs such as metformin, tideglusib and mexiletine are also undergoing Phase III trials to treat DM1. Metformin , a biguanide currently approved for type 2 diabetes mellitus, is currently slated for Phase III trials in DM1. In previous Phase II trials, metformin increased the 6-minute walking distance for DM1 patients versus placebo. While it has positive benefits ranging from anticancer effects to cardiovascular protection to neuroprotection, metformin is also associated with adverse effects such as gastrointestinal discomfort and lactic acidosis in high doses. Another approach is AMO Pharma ’s tideglusib (also known as AMO-02), a glycogen synthase kinase 3 beta inhibitor currently in Phase III clinical trials. Despite not reaching its primary endpoint in the Phase II/III REACH-CDM trial due to a strong placebo effect, tideglusib had achieved clinically and statistically significant benefits in various functional and objective assessments as of May 2024. As a result, after meeting with the FDA, AMO Pharma is forging ahead with a Phase III trial of tideglusib. Elsewhere, Lupin is working on repurposing mexiletine, a class I antiarrhythmic drug that is currently in Phase III trials for DM1. In contrast to previous trials that used mexiletine capsules for immediate-release effects, Lupin intends to use mexiletine granules for prolonged-release oral suspension formulations for its Phase III HERCULES and ATLAS trials. Like tideglusib, mexiletine reached Phase III despite less-than-stellar mid-stage results. In its case, Phase II trial data demonstrated no significant change in 6-minute walk distance at 6 months for patients on mexiletine capsules compared to placebo. Also worthy of mention is ARTHex. While clinical data is scant for the company’s ATX-01, it was enrolling patients into its Phase I/IIa ArthemiR trial as of October 2024. The drug is based on microRNA technology. As progress currently stands, antibody-oligonucleotide conjugates appear to be more effective at targeting the genetic source of DM1 and have a more convincing mechanism of action than small molecules and repurposed drugs. However, even though genetic therapies are advancing quickly, the DM1 space is risky. Consider that Ionis Pharmaceuticals stopped working on its antisense compound IONIS-DMPK-2.5Rx in January 2017 after failing to achieve adequate concentration levels in muscles in Phase I/II trials. If all goes well in the late-stage gene therapy space for DM1, expect new treatments to hit the market within the next three to four years at the earliest.

Phase 3OligonucleotidePhase 2Clinical ResultDrug Approval

27 Nov 2024

·www.prnewswire.com

Myotonic Dystrophy Clinical Trial Pipeline Boom as Over 20 Companies Leading the Charge in Research and Development | DelveInsight

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 ' 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. 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 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 A snapshot of the Myotonic Dystrophy Pipeline Drugs mentioned in the report: Learn more about the emerging myotonic dystrophy pipeline therapies @ Myotonic Dystrophy Clinical Trials 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 Table of Contents For further information on the myotonic dystrophy pipeline therapeutics, reach out @ Myotonic Dystrophy Treatment Drugs Related Reports Myotonic Dystrophy Market 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 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 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 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 [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Fast TrackPhase 2Orphan Drug

06 Nov 2023

·www.prnewswire.com

Emerging Market Trends in Muscular Dystrophy: A DelveInsight Analysis of Becker Muscular Dystrophy, Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, and Limb-girdle Muscular Dystrophy

Muscular dystrophies are a group of genetic muscle disorders characterized by progressive muscle weakness. There are more than 40 different variations, and the most common ones in children are Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). LAS VEGAS, Nov. 6, 2023 /PRNewswire/ -- Muscular dystrophies encompass a range of muscle disorders resulting from genetic mutations. Over time, these conditions lead to muscle weakness, which hinders everyday activities. Muscular dystrophy takes various forms, each targeting specific muscle groups and manifesting at different ages with varying degrees of severity. It can either be hereditary or occur as a spontaneous genetic mutation. Additionally, there may be multiple genetic subtypes within each specific type of muscular dystrophy, and individuals with the same subtype may exhibit varying symptoms. DelveInsight has recently released a series of epidemiology-based market reports focusing on muscular dystrophy including Becker Muscular Dystrophy, Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, and Limb-girdle Muscular Dystrophy. These reports include a comprehensive understanding of current treatment practices, historical and forecasted patient pool, emerging drugs, market share of individual therapies, and historical and forecasted market size from 2019 to 2032 segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan]. Additionally, the reports feature exhaustive analysis of prominent companies working with their emerging candidates in different stages of clinical development. Let's deep dive into the assessment of these muscular dystrophy reports market individually. Becker Muscular Dystrophy Market Becker muscular dystrophy (BMD) is a genetic disorder that primarily impacts muscle structure. It leads to the gradual deterioration of limb, cardiac, and skeletal muscles. Interestingly, involuntary muscles remain unaffected. BMD is considered a milder form of Duchenne muscular dystrophy (DMD). Typically, BMD manifests in one's teenage years or early adulthood, and its progression is slower and less predictable compared to DMD. As per DelveInsight analysis, it can be observed that prevalence of BMD is high in the 7MM, and similarly, high rate of treatment is seen in patients. The approach to BMD treatment centers on symptom management since there is no definitive cure available. Instead, there are treatments designed to alleviate symptoms and enhance muscle functionality. It's essential for individuals living with BMD to maintain their physical conditioning and muscle usage. While there are no FDA-approved drugs specifically for BMD, researchers are actively investigating various therapeutic interventions. As per DelveInsight analysis, the dynamics of the becker muscular dystrophy market are anticipated to change in the coming years owing to the positive outcomes of the emerging pipeline candidates during the developmental stage by key players that are under phase III, II and I clinical development have the potential to create a significant positive shift in BMD market size. Becker Muscular Dystrophy Pipeline Therapies and Companies EDG-5506: Edgewise Therapeutics, Inc./Medpace, Inc. Vamorolone: ReveraGen BioPharma, Inc./Santhera Pharmaceuticals For a comprehensive view of the Becker muscular dystrophy market, check out the Becker Muscular Dystrophy Market Assessment Myotonic Dystrophy Market Myotonic dystrophy, an umbrella term for two rare genetic disorders with autosomal dominant inheritance patterns, impacts various bodily systems, featuring a clinical triad of progressive muscle weakness, myotonia, and early-onset cataracts. These disorders are classified into two primary types: DM type 1 (DM1) and DM type 2 (DM2). DelveInsight estimated that the total diagnosed prevalent patient population of myotonic dystrophy in the 7MM countries was close to 80K cases in 2021. As per the estimates, the US had the highest patient population of myotonic dystrophy in 2021. Although numerous pre-clinical advancements have been made, there is currently no targeted disease-modifying treatment available. The mainstay of management primarily involves monitoring for complications and adhering to standard care practices, such as utilizing assistive devices, administering hormone therapy, and providing pain relief. Several clinical studies have systematically assessed the efficacy of therapeutic agents in managing the condition. However, the scarcity of scientific evidence, coupled with the complex and diverse manifestations of the disease, poses significant challenges for healthcare providers when it comes to identifying and selecting appropriate medications. To tap the uncovered myotonic dystrophy market, few players have targeted the unmet need fortreatment. As per DelveInsight analysis, the myotonic dystrophy market is expected to grow from USD 80 million in 2021 at a significant CAGR by 2032. The current pipeline consists of both mid and late-stage candidates. The few promising therapies inthe mid and late-stage development are expected to experience significant reconstitution during the forecastperiod. Hence, more research and studies are needed to develop potential therapies that cater to the unmet need for curative and disease-modifying therapy in myotonic dystrophy patients. Myotonic Dystrophy Pipeline Therapies and Companies AMO-02 (tideglusib): AMO Pharma Limited Mexiletine: Lupin Ltd. Pitolisant: Harmony Biosciences, LLC AOC 1001: Avidity Biosciences, Inc. DYNE-101: Dyne Therapeutics To gain a deeper understanding of the myotonic dystrophy market, be sure to explore the Myotonic Dystrophya Market Outlook Facioscapulohumeral Muscular Dystrophy (FSHD) Market Facioscapulohumeral muscular dystrophy (FSHD), a genetically inherited disorder, progressively weakens muscles and significantly reduces the functional capacity of those affected. Recent years have witnessed substantial progress in our understanding of how epigenetic factors play a role in this condition. FSHD can be categorized into two subtypes based on the mechanisms of reduced methylation and failed epigenetic repression. Type 1 accounts for approximately 95% of FSHD cases and is characterized by the loss of D4Z4 repeats. The remaining 5% fall into Type 2, where methylation and epigenetic changes occur independently of deletions. Regardless of the subtype, FSHD leads to the same outcome. As per DelveInsight analysis, in the year 2021, the total prevalent cases of FSHD were 78K in the 7MM which are expected to grow during the study period, i.e., 2019–2032. As of now, there are no officially approved therapies for managing FSHD. However, individuals dealing with FSHD can find relief by utilizing orthotic devices designed to support their feet. These devices are particularly effective in addressing common FSHD symptoms like foot drop and shoulder weakness. Depending on the degree of foot drop, a custom-made ankle-foot orthosis (AFO) can provide significant benefits. In cases where knee extensor weakness may worsen gait with an AFO, alternatives like floor reaction ankle-foot orthosis (FRAFO) or advanced knee-ankle-foot orthosis (KAFO) may be considered. Regular exercise, especially hydrotherapy, plays a crucial role in preserving mobility and pain management. Additionally, weight management, potentially through dietary adjustments, is essential to reduce strain on already weakened muscles. It's important to note that there is currently no cure for FSHD, but there are treatments and devices available to alleviate many of its symptoms. Non-steroidal anti-inflammatory drugs (NSAIDs) are commonly prescribed to enhance comfort and mobility. According to DelveInsight, the FSHD market in 7MM is expected to witness a major change in the study period (2019–2032). As per estimates, in 2021, the total FSHD market size was USD 25.55 million which is expected to rise by 2032. This increase in market size is mainly due to extensive research and development activities of pharmaceutical companies, along with the expected launch of approved therapies. Facioscapulohumeral Muscular Dystrophy Pipeline Therapies and Companies Losmapimod: Fulcrum Therapeutics/GSK GYM329 (RO7204239/RG 6237): Roche Explore in-depth for a comprehensive understanding of the FSHD Clinical Trials Limb-girdle Muscular Dystrophy Market Limb-girdle muscular dystrophies (LGMD), also known as pelvofemoral muscular dystrophy or proximal muscular dystrophy, encompass a group of infrequent genetic disorders characterized by the progressive wasting and weakening of voluntary muscles located in the hip and shoulder regions. This muscle weakness and atrophy gradually advance and can eventually impact other muscle groups in the body. As per data from the National Organization for Rare Disorders, LGMD affects both males and females in roughly equal proportions. The exact prevalence of LGMD remains uncertain, but estimates suggest a range between one in 14,500 to one in 123,000 individuals. It's important to note that the age at which symptoms manifest can vary significantly, even within the same family. Additionally, the relative frequencies of the different LGMD types differ across populations, though it is worth mentioning that LGMD2G, 2H, and 2J are exceedingly rare on a global scale. Currently, there is no known cure for LGMD. However, there are supportive therapies designed to manage and alleviate its symptoms. The approach to LGMD management is individualized, focusing on addressing specific symptoms that each patient experiences. Treatment options may involve physical and occupational therapy to enhance muscle strength and prevent contractures, the utilization of assistive devices like canes, braces, walkers, or wheelchairs to aid mobility, corrective surgery for skeletal issues like scoliosis, and regular monitoring of cardiac and respiratory health for potential complications associated with certain LGMD subtypes. According to DelveInsight's' estimates, the limb girdle muscular dystrophy market in 7MM is expected to show a positive growth, during the forecast period (2023–2032), mainly attributed to anticipated launch of emerging therapy and the increasing cases of LGMD. Limb-girdle Muscular Dystrophy Pipeline Therapies and Companies SRP-6004: Sarepta Therapeutics, Inc. LION-101: Asklepios Biopharmaceutical, Inc. BBP-418 (ribitol): ML Bio Solutions, Inc. To access a complete analysis of the limb-girdle muscular dystrophy market, visit Limb-girdle Muscular Dystrophy Market Assessment Trending Genetic Disorders Reports Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2032 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 DMD companies, including Pfizer, Santhera Pharmaceuticals, ReveraGen BioPharma, Taiho Pharmaceutical, FibroGen, Sarepta Therapeutics, Capricor Therapeutics, Daiichi Sankyo, Italfarmaco, Antisense Therapeutics, Solid Biosciences, among others. Alpha-1 Antitrypsin Deficiency Market Alpha-1 Antitrypsin Deficiency Market Insights, Epidemiology, and Market Forecast – 2032 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 A1ATD companies, including Arrow Head Pharmaceuticals, Mereo Biopharma, Kamada Ltd, Grifols, CSL Behring, Shire, LFB Biotechnologies, among others. Wilson Disease Market Wilson Disease Market Insights, Epidemiology, and Market Forecast – 2032 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 Wilson disease companies, including Orphalan, AstraZeneca, Vivet Therapeutics, Pfizer, Ultragenyx Pharmaceutical, among others. Cystic Fibrosis Market Cystic Fibrosis Market Insights, Epidemiology, and Market Forecast – 2032 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 cystic fibrosis companies, including Verona Pharmaceuticals, Eloxx Pharmaceuticals, Inc., Santhera Pharmaceuticals, Laurent Pharmaceuticals Inc., Vertex Pharmaceuticals, among others. About DelveInsight DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +1(919)321-6187 Logo: SOURCE DelveInsight Business Research, LLP

Phase 3

100 Deals associated with Mexiletine Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D00639	Mexiletine Hydrochloride	C01BB02	DB00379

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Non dystrophic myotonia	European Union	Lupin Ltd	18 Dec 2018
Non dystrophic myotonia	Iceland	Lupin Ltd	18 Dec 2018
Non dystrophic myotonia	Liechtenstein	Lupin Ltd	18 Dec 2018
Non dystrophic myotonia	Norway	Lupin Ltd	18 Dec 2018
Lung Abscess	Japan	Chemix, Inc.	08 Jul 2005
Peritonitis	Japan	Chemix, Inc.	08 Jul 2005
Pneumonia	Japan	Chemix, Inc.	08 Jul 2005
Arrhythmias, Cardiac	Japan	Taiyo Pharma Co., Ltd.	31 Jan 1985
Diabetic Neuropathies	Japan	Taiyo Pharma Co., Ltd.	31 Jan 1985

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Myotonic Dystrophy	Phase 3	France	Lupin Ltd	03 Sep 2021
Myotonic Dystrophy	Phase 3	-	Lupin Ltd	03 Sep 2021
HIV Infections	Phase 2	United States	Boehringer Ingelheim GmbH	31 Aug 2001
HIV Infections	Phase 2	Tanzania	Boehringer Ingelheim GmbH	31 Aug 2001
Neuropathy, Painful	Phase 2	United States	Boehringer Ingelheim GmbH	31 Aug 2001
Neuropathy, Painful	Phase 2	Tanzania	Boehringer Ingelheim GmbH	31 Aug 2001

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ESC2024	Not Applicable	Timothy Syndrome	-	Mexiletine	blgulsjzwf(pjcjguwyzp) = lbtjiyzhrn fqmaozhjhf (facduiyoic )	-	01 Sep 2024
AAN2024	Not Applicable	Migraine Disorders	-	Mexiletine 450 mg	zvofatqjlg(mwkctuxeiq) = Dizziness during lidocaine administration (p-value=0.0008) and lower tolerated lidocaine infusion rates (p-value=0.001) were predictive of shorter number of days on mexiletine treatment. A lower starting dose should be considered on these patients bccknlauug (fzczejcbwv ) View more	-	09 Apr 2024
				Mexiletine 600 mg
MDS2023	Not Applicable	Myotonia	183	Mexiletine	kkaecubosy(vfopubwokt) = mexiletine significantly causes some GIT symptoms compared to placebo (RR 3.7, 95% CI [1.79 to 7.64]) qrmjjnpuum (xtwqdxxoiv )	Positive	27 Aug 2023
EAN2023	Not Applicable	-	-	Mexiletine	ynryqbhxrh(cwteszyrow) = ukhsjhkmjo pmrjxscjnd (fxgydtmthl )	-	28 Jun 2023
ESC2020	Not Applicable	Long Qt Syndrome 2	20	mexiletine	aizpiyohef(bdbsunffwm) = htscyqdamq pvhatqfebt (qhzsfviyds )	-	28 Aug 2020
2531 (AAN2020)	Phase 2	Amyotrophic Lateral Sclerosis	20	Placebo	uaiyvdktjg(huzhpwbskr) = tiazpmvchj nptnhohumv (rowwrgkvfs, 2.2) View more	Positive	14 Apr 2020
				Mexiletine 300 mg	dojxtxjapq(gcetewwzrp) = lmdlnwuiqs crdpueiffx (tkynxtrbhs, 0.315 - 1.102)
NCT02781454 (Mexiletine-2, CTgov)	Phase 2	Amyotrophic Lateral Sclerosis	20	Mexiletine (Mexiletine, 300 Milligrams)	bypxeiscat(ginxeoobbm) = hdyuwfpgti apbitgywgv (tkdmvbcsal, 2.323) View more	-	05 Dec 2019
				Mexiletine (Mexiletine, 600 Milligrams)	bypxeiscat(ginxeoobbm) = jbzkisbrrx apbitgywgv (tkdmvbcsal, 2.207) View more
WCN2019	Not Applicable	Non dystrophic myotonia	-	Safinamide	xivsverivi(rjzqkzkftv) = kjktzpinpj wdtfhvvpil (wqgqeogwpr ) View more	-	15 Oct 2019
				Mexiletine	xivsverivi(rjzqkzkftv) = wsjejgtkix wdtfhvvpil (wqgqeogwpr ) View more
NCT02260388 (PAIN-CONTRoLS, CTgov)	Phase 4	Pain \| Sensory polyneuropathy	402	Nortriptyline (Nortriptyline)	lotieeslaf(aldhmgbays) = hbbibvloit ucqseulurq (sfvdgbospn, 8.9) View more	-	06 Jul 2018
				Duloxetine (Duloxetine)	lotieeslaf(aldhmgbays) = nqsnskmifh ucqseulurq (sfvdgbospn, 9.9) View more
AAN2018	Not Applicable	Non dystrophic myotonia	30	Mexiletine	fpdcdmogoo(qcixljdvjx) = vuchkhnsyo ebjvxsmwke (gewbqbrhfr ) View more	Positive	10 Apr 2018

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