Delpacibart Etedesiran

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

Enrollment in global Phase 3 HARBOR™ study for del-desiran in DM1 is on track Avidity initiated biomarker cohort for del-brax FORTITUDE™ study for FSHD; pursuing a potential accelerated approval path for del-brax Reported positive del-zota data from Phase 1/2 EXPLORE44™ trial for DMD44 Avidity to provide a first look at precision cardiology candidates and a glimpse at next-generation technology innovations via webcast event November 12, 2024 SAN DIEGO, Nov. 7, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today reported financial results for the third quarter ended September 30, 2024, and highlighted recent corporate progress. "We are executing three potentially registrational clinical trials in three rare diseases where there are limited or no therapeutic options available. We reported positive del-zota data from the 5 mg/kg cohort of our Phase 1/2 EXPLORE44™ trial for DMD44 and have initiated enrollment in the EXPLORE44-OLE™. In addition, enrollment is on track for del-desiran's global Phase 3 HARBOR™ trial in DM1 and we initiated the biomarker cohort of our FORTITUDE™ study in FSHD, marking a key step in our strategy to pursue a potential accelerated approval path for del-brax," said Sarah Boyce, president and chief executive officer at Avidity. "As we build for the future, we look forward to sharing our exciting innovations in precision cardiology and a glimpse at our next-generation technology. We continue to build our global commercial infrastructure to provide potential new therapies to people living with these serious rare diseases as quickly as possible." "We were pleased to complete an additional upsized public offering in August following positive clinical data from our EXPLORE44 program. With a strong cash position of approximately $1.6 billion, we remain focused on advancing our programs, bringing forward additional candidates from our DMD pipeline, executing on our precision cardiology programs and building additional capabilities, including commercial functions, as well as expanding to countries outside of the US," said Mike MacLean, chief financial officer and chief business officer at Avidity. Recent Highlights Del-zota (AOC 1044) for DMD44 In August, Avidity reported positive initial del-zota data from the 5 mg/kg cohort of the Phase 1/2 EXPLORE44™ trial in people living with DMD44 demonstrating unsurpassed delivery to skeletal muscle, unprecedented, unadjusted increase of 25% in near full-length dystrophin production with a profound reduction in creatine kinase levels to near normal, and robust exon 44 skipping. Del-zota demonstrated favorable safety and tolerability with most treatment emergent adverse events mild or moderate. The Phase 1/2 EXPLORE44 trial is fully enrolled and ongoing. In addition to the participants rolling over from the Phase 1/2 EXPLORE44 trial, Avidity has begun enrolling 10-15 new participants in the EXPLORE44 Open-label Extension study (OLE). Del-brax (AOC 1020) for FSHD In October, Avidity announced the initiation of the biomarker cohort in the Phase 1/2 FORTITUDE™ trial of del-brax. 2 mg/kg of del-brax will be administered every six weeks, designed to ensure continuous suppression of DUX4. In June of this year, Avidity reported positive initial del-brax 2 mg/kg data at four months from the Phase 1/2 FORTITUDE trial demonstrating unprecedented and consistent reductions of greater than 50% in DUX4 regulated genes, mean reductions of 25% or greater in novel circulating biomarker and creatine kinase, trends of functional improvement, and favorable safety and tolerability in people living with FSHD. Avidity remains on track to initiate the functional cohort in the FORTITUDE study in the first half of 2025. In July, the first participants from FORTITUDE began to roll over to the FORTITUDE Open-Label Extension (OLE) trial. All participants that complete FORTITUDE are eligible to enroll in the FORTITUDE-OLE™ trial. Del-desiran (AOC 1001) for DM1 Enrollment for the global Phase 3 HARBOR™ trial is ongoing and on track. In October, the U.S. Food and Drug Administration (FDA) removed the partial clinical hold on del-desiran. Pipeline Advancements and Organizational Highlights In November, Avidity plans to provide a first look at precision cardiology candidates. In addition, Avidity plans to share a glimpse at next-generation technology innovations. In August, Avidity announced it plans to advance additional candidates from its DMD franchise following robust del-zota data; Exon 45 is currently in IND-enabling studies. Kathleen Gallagher was promoted to chief program officer in September 2024. Third Quarter 2024 Financial Results Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $1.6 billion as of September 30, 2024, which reflects a gross $345.1 million raise from a public offering. Collaboration Revenue: Collaboration revenues of $2.3 million for the third quarter of 2024 and $7.9 million for the first nine months of 2024 primarily relate to Avidity's research collaboration and license partnership with Bristol Myers Squibb. Collaboration revenues of $2.8 million for the third quarter of 2023 and $7.4 million for the first nine months of 2023 primarily related to Avidity's research collaboration and license partnership with Eli Lilly and Company. Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $77.2 million for the third quarter of 2024 compared with $47.7 million for the third quarter of 2023, and $208.0 million for the first nine months of 2024 compared with $138.2 million for the first nine months of 2023. The increases were primarily driven by the advancement of del-desiran, del-brax and del-zota, as well as internal and external costs related to the expansion of the company's overall research capabilities. Gener al and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were $23.3 million for the third quarter of 2024 compared with $13.7 million for the third quarter of 2023, and $57.9 million for the first nine months of 2024 compared with $38.1 million for the first nine months of 2023. The increases were primarily due to higher personnel costs to support the company's expanded operations. About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through internal discovery efforts and key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit and engage with us on LinkedIn and X. Forward-Looking Statements Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: a potential accelerated pathway for registration for del-brax, and the potential for all of Avidity's current clinical trials to be registrational; Avidity's next-generational technology innovations and the timing of its announcement; the anticipated announcement of lead precision cardiology program targets, including the timing thereof; Avidity's plans to build a global commercial infrastructure and capabilities; plans for the advancement of DMD programs beyond DMD44; plans for the initiation of the functional cohort in the FORTITUDE™ trial and the timing thereof; the characterization of data associated with Avidity's product candidates in their respective clinical trials, the conclusions drawn therefrom, and the impact of such data on the advancement of the respective product candidates; enrollment statuses of Avidity's clinical programs; the status of the HARBOR™ study; Avidity's platform, planned operations and programs; and Avidity's cash position and runway. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: preliminary results of a clinical trial are not necessarily indicative of final results; further analysis of existing clinical data and analysis of new data may lead to conclusions different from those established as of the respective data cutoff dates in Avidity's clinical trials, and such data may not meet Avidity's expectations; Avidity's biomarker and planned functional cohorts in the FORTITUDE study may not support the registration of del-brax; unexpected adverse side effects to, or inadequate efficacy of, Avidity's product candidates that may delay or limit their development, regulatory approval and/or commercialization; later developments with the FDA and other global regulators that could be inconsistent with the feedback received to date regarding Avidity's clinical trials; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven and may not produce any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of preclinical studies or clinical trials; the success of its preclinical studies and clinical trials for the company's product candidates; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; Avidity may not realize the expected benefits of its collaborations; legislative, judicial and regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2023 and subsequent filings with the SEC. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Contact: Mike MacLean (619) 837-5014 [email protected] Media Contact: Navjot Rai (619) 837-5016 [email protected] SOURCE Avidity Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED