Dystrophin expressing chimeric cells (Dystrogen Therapeutics)

DUBLIN, Oct. 24, 2023 /PRNewswire/ -- The "Duchenne Muscular Dystrophy Drugs - Global Strategic Business Report" report has been added to ResearchAndMarkets.com's offering. The global market for Duchenne Muscular Dystrophy Drugs, valued at US$2 billion in 2022, is projected to grow to US$27.4 billion by 2030, with a remarkable compound annual growth rate (CAGR) of 39.1% during the analysis period of 2022-2030. Among the segments analyzed in the report, Exon Skipping is expected to lead the growth with a CAGR of 44.9%, reaching US$18.1 billion by the end of the forecast period. The Steroid Therapy segment is estimated to grow at a CAGR of 27.7% over the next 8 years. Key Market Insights: The U.S. Duchenne Muscular Dystrophy Drugs market is estimated at US$843.1 million in 2022. China, the world's second-largest economy, is forecasted to reach a market size of US$2.4 billion by 2030, with a CAGR of 46%. Japan and Canada are also noteworthy markets, expected to grow at 32.6% and 31.7% respectively from 2022 to 2030. Within Europe, Germany is forecasted to grow at approximately 34.8% CAGR. Select Competitors: Key players in the Duchenne Muscular Dystrophy Drugs market include: Acceleron Pharma, Inc. Akashi Therapeutics, Inc. BioMarin Pharmaceutical, Inc. Bristol-Myers Squibb Company Eli Lilly and Company Janssen Pharmaceuticals, Inc. Lexicon Pharmaceuticals, Inc. Nippon Shokubai Co., Ltd. Nobelpharma Co., Ltd. Pfizer, Inc. PTC Therapeutics, Inc. Sarepta Therapeutics Inc. Taiho Pharmaceutical Co., Ltd. Economic Outlook: The global economic outlook is showing signs of improvement, with growth recovery expected in the near term. While challenges such as slower-than-expected decline in global headline inflation, food and fuel inflation in developing countries, and high retail inflation persist, countries and governments are taking measures to address these issues. The rise of new technologies, including generative AI, applied AI, machine learning, next-generation software development, Web3, cloud and edge computing, quantum technologies, electrification, renewables, and climate technologies, is expected to open up new opportunities in the global investment landscape. These technologies have the potential to drive incremental growth and value to global GDP. The short-term economic landscape is expected to be a mix of challenges and opportunities, requiring resilience and adaptability from businesses and their leaders. For more detailed insights into the Duchenne Muscular Dystrophy Drugs market and global economic trends, please refer to the full report. What`s New? Special discussions on the global economic climate and market sentiment Coverage on global competitiveness and key competitor percentage market shares Market presence analysis across multiple geographies - Strong/Active/Niche/Trivial Online interactive peer-to-peer collaborative bespoke updates Access to digital archives and research platform Complimentary updates for one year Access to curated YouTube video transcripts of market sentiments shared by CEOs, domain experts and market influencers via interviews, podcasts, press statements and event keynotes Key Topics Covered: I. METHODOLOGY II. EXECUTIVE SUMMARY 1. MARKET OVERVIEW A Prelude to Duchenne Muscular Dystrophy Global Economic Update From Pandemic to War & Inflation: "Gloomy Outlook for 2023 Despite Signs of Easing Inflationary Conditions Here's How Inflationary Pressures Affect the Economy Here's What's Causing Inflationary Pressures in the Global Market Although Past Peak in 2023, Efforts to Solve the Inflation Puzzle Should Continue Global Economy Faces a Tough Climb Towards Recovery Made Rockier by the War, Anti-Inflation Fiscal Policies & Slower Than Desirable Easing of Inflationary Pressures: World Economic Growth Projections (Real GDP, Annual % Change) for the Years 2020 Through 2024 Impact of COVID-19 on Duchenne Muscular Dystrophy Drugs Market Competition Product Development as Hallmark of Global Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Drugs - Global Key Competitors Percentage Market Share in 2023 (E) Competitive Market Presence - Strong/Active/Niche/Trivial for Players Worldwide in 2023 (E) Global Market Overview and Prospects Global Duchenne Muscular Dystrophy Drugs Market Set for a Rapid Growth, Driven by Rising Prevalence A Review of the Approved DMD Drugs North America and Europe to Remain Lucrative Markets Molecular Therapy, Hospital/Clinic & Hospital Pharmacy: Hale & Hearty Segments of Duchenne Muscular Dystrophy Market Dynamic Factors Keeping Duchenne Muscular Dystrophy Market Growing Market Drivers Rising Incidence of DMD to Catalyze Growth DMD Prevalence (in 100,000 Population) in Select Countries: 2020 High Uptake of Novel Therapies & Drugs Market Restraints & Challenges Lack of Standardization for Clinical Efficacy Stringent Regulatory Regime Market Opportunities Product Developments & Rising Awareness Surge in Clinical Trials to Launch Novel Options Recent Market Activity 2. FOCUS ON SELECT PLAYERS 3. MARKET TRENDS & DRIVERS Rising R&D Investment to Boost DMD Market Select Therapy Candidates Under Development for Treating DMD Growing Government Support Propels Market Expansion Biologics - The Most Dynamic Segment of the Market Blossoming of Gene Therapy in Duchenne Muscular Dystrophy Treatment Domain Gene Therapy for DMD Treatment Elevidys, The First Ground-breaking Gene Therapy for Treatment of Duchenne Muscular Dystrophy (DMD) Children's National Hospital Starts Offering Newly Approved Gene Therapy for DMD Promising Gene Therapies Potential Side-Effects & Risks of Gene Therapies Vamorolon Holds Potential to Lessen Health Risks Linked with Extended Use of Steriods DT-DEC01 Offers Promise to Stabilize or Improve Motor Function AOC 1044 Receives Orphan Drug Status for DMD44 Italfarmaco Group Announces NDA Acceptance for Givinostat Targeting Duchenne Muscular Dystrophy Uptrend in Healthcare Expenditure Creates Substantial Opportunities World Healthcare Expenditure (In US$ Trillion) for the Years 2019, 2021 & 2023 4. GLOBAL MARKET PERSPECTIVE III. MARKET ANALYSIS IV. COMPETITION For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

New data from Axcella, PepGen, Dystrogen and Galecto are showing promise in challenging diseases, including NASH, Duchenne muscular dystrophy and myelofibrosis. Axcella's NASH Candidate Impresses In Phase IIb Axcella Therapeutics posted interim data Thursday from its Phase IIb EMMPACT study of AXA1125 in nonalcoholic steatohepatitis (NASH) confirming the candidate's ability to target multiple pathways simultaneously during disease treatment. Data from weeks 12 and 24 showed all participants experienced significantly greater changes from baseline in MRI-PDFF compared to the placebo group. The positive trend continued into the 24th week, though researchers noted they needed a larger group of subjects for it to be considered statistically significant. For this data set, around 30% of the target 270 participants were available, all with biopsy-confirmed stage II and stage III NASH. At week 12, the trial had 82 subjects, and 58 by week 24. Around 50% of the subjects have type II diabetes mellitus. They were randomized 1:1:1 to receive either 22.6 g or 33.9 g of AXA1125 twice a day or a placebo. Axcella expects to share topline, 48-week biopsy results in the first half of 2024. This news comes on the heels of a positive update from another company seeking to develop the next treatment for NASH. Oramed Pharmaceuticals recently shared promising mid-stage trial results from its oral insulin candidate ORMD-0801, showing a clinically meaningful reduction in liver fat from baseline at 12 weeks. The company is expected to share full details about this trial soon. PepGen Preps for Phase IIa DMD Study PepGen announced its Phase I trial of Duchenne muscular dystrophy treatment PGN-EDO51 in healthy adult males met the primary safety endpoint, enabling a move to Phase II. PepGen's lead clinical candidate, PGN-EDO51 uses its proprietary enhanced delivery oligonucleotide technology to deliver a therapeutic component designed to target the root cause of DMD. It is given intravenously and works by skipping exon 51 of the dystrophin transcript to restore the open reading frame and enable the production of a truncated dystrophin protein. PGN-EDO51 was tested in various doses on 32 healthy adult males, who were assessed for safety and tolerability, as well as oligonucleotide tissue concentration and exon skipping. The participants were given either 1, 5, 10 or 15 mg/kg of PGN-EDO51 or a placebo over the course of 28 days. PepGen is now preparing to launch a Phase IIa multiple ascending dose study in the first half of 2023. Full results from the Phase I study will be presented at an upcoming medical meeting. Dsytrogen's DMD Hopeful Shows Promise at 6 Months In other DMD news, Dystrogen Therapeutics shared positive initial results from the ongoing clinical trial of investigational engineered cell therapy DT-DEC01. Six months' worth of data demonstrated promising safety, functional and biomarker outcomes from the three subjects under the first low-dose cohort. The participants, aged six, seven and 15, showed improved duration and amplitude of motor unit potentials on EMG versus the pretreatment baseline. They were also observed to have improved grip strength and better 6MWD, NSAA, PODCI and PUL scores, among others. DC-DEC01 is an engineered chimeric cell that engrafts in cardiac and skeletal muscle to deliver full-length dystrophin gene and other healthy muscle cell components. The drug does not require immunosuppression. There was no evidence of immune reaction on blood assays during the six-month period in any of the patients. OncoNano's Fluorescence Imaging Agent Demonstrates Significant Detection OncoNano Medicine's ongoing Phase II clinical trial of pegsitacianine for the detection of residual disease after cytoreductive surgery delivered positive interim results from 27 participants. Data showed 55% of the participants demonstrated a significant detection of pathology-confirmed residual disease while being well-tolerated and without any serious adverse events. Pegsitacianine is a micellar fluorescence agent that can detect residual malignancies post-CRS and act as an adjunct to tactile and visual cues. The patients were given 1 mg/kg of the drug and underwent surgery with intra-operative imaging done between 24 and 72 hours post-dose. These findings could offer surgeons real-time optics to enhance their ability to detect cancerous tissues that might be left behind if only standard-of-care processes are implemented after CRS. The initial results were shared at an oral presentation at the World Molecular Imaging Congress in Florida. The study is still open to enrollment. Galecto's Myelofibrosis Drug Demonstrates Robust Collagen Fibrosis Reduction Galecto announced its ongoing Phase IIa MYLOX-1 study of GB2064 for the treatment of myelofibrosis delivered reduction in collagen fibrosis of the bone marrow equal to or greater than 1-grade in four out of five patients who received the drug for at least six months. All four participants demonstrated stable hemoglobin, thrombocyte and white blood cell count numbers and a stable spleen volume. None required transfusion. Two participants have already advanced into the extension phase since GB2064 maintained an acceptable tolerability profile. The trial started with 16 patients, of which eight have either continued to receive or completed the treatment. The other eight have discontinued due to either disease progression or adverse events. The most common AEs observed were gastrointestinal-related and manageable with standard therapy.