Myotonia

Dyne Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Tuesday, March 5, 2024
Therapy, Myotonia, Central nervous system, Research, DYN, Dystrophin, Safety, DMPK, DMD, Exercise, Standard of care, Conference, DM1, Eteplirsen, Muscular Dystrophy Association, Fatigue, Myotonic dystrophy, Patient, Clinical trial, PMO, Male, G&A, MDA, Episcopal conference, Pharmaceutical industry

WALTHAM, Mass., March 05, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the fourth quarter and full year 2023 and recent business highlights.

Key Points: 
  • (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the fourth quarter and full year 2023 and recent business highlights.
  • Net loss: Net loss for the quarter ended December 31, 2023 was $66.6 million, or $1.09 per basic and diluted share.
  • This compares with a net loss of $38.8 million, or $0.74 per basic and diluted share, for the quarter ended December 31, 2022.
  • Net loss for the year ended December 31, 2023 was $235.9 million, or $3.95 per basic and diluted share.

Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-DM1 for Treatment of Type 1 Myotonic Dystrophy

Retrieved on: 
Friday, March 8, 2024
Pharmaceutical, Genetics, Health, Clinical Trials, Wheelchair, Myotonia, Cataract, DMPK, Wasting, Exercise, Arrowhead, Conference, DM1, RNA, Muscle weakness, Patient, CUG, Primate, MDA, RNA interference, Episcopal conference, Vaccine

Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.

Key Points: 
  • Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.
  • Pathogenesis of DM1 is driven by an expanded CUG trinucleotide repeat in the 3’-untranslated region of DMPK transcripts.
  • These abnormal transcripts cause mis-regulated splicing, known as spliceopathy, for certain messenger RNAs which are directly linked to the clinical manifestations of DM1.
  • Presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

Orphan designation: Humanised IgG1 kappa fragment antibody targeting TfR1 conjugated to P125 oligonucleotide Treatment of myotonic disorders, 22/05/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
Transferrin, Immunoglobulin G, COMP, IRIS, Myotonia, EMA, European Commission, Patient, Committee, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Humanised IgG1 kappa fragment antibody targeting TfR1 conjugated to P125 oligonucleotide
    - Intended use
    - Treatment of myotonic disorders
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2781
    - Date of designation
    - Sponsor
    Pharma Gateway AB
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Avidity Biosciences Announces 2024 Corporate Priorities and Catalysts for Next Stage of Growth

Retrieved on: 
Friday, January 5, 2024
RNA, Marketing, Cardiology, Facioscapulohumeral muscular dystrophy, Research, DM1, Duchenne muscular dystrophy, SAN, Facial muscles, Therapy, FSHD, AOC, University, DSM-IV codes, Myotonia, Growth, Safety, Senior, Nursing

SAN DIEGO, Jan. 5, 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 announced its 2024 corporate priorities and catalysts for the next stage of growth. In mid-2024, Avidity plans to initiate the global Phase 3 HARBOR™ trial of AOC 1001 for adults living with myotonic dystrophy type 1 (DM1). The robust data package of AOC 1001 from the Phase 1/2 MARINA® trial and open-label extension study, MARINA-OLE™, has demonstrated consistent improvements across multiple functional endpoints including myotonia, muscle strength and mobility, and long-term favorable safety and tolerability results in people living with DM1. Avidity also plans to report data in 2024 from all three of its ongoing clinical programs targeting three distinct rare muscle diseases: DM1, Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44) and facioscapulohumeral muscular dystrophy (FSHD) while advancing its cardiology programs toward clinical development. In addition, Avidity announced the appointment of Eric B. Mosbrooker as Chief Strategy Officer.

Key Points: 
  • In mid-2024, Avidity plans to initiate the global Phase 3 HARBOR™ trial of AOC 1001 for adults living with myotonic dystrophy type 1 (DM1).
  • In addition, Avidity announced the appointment of Eric B. Mosbrooker as Chief Strategy Officer.
  • Prior to joining Avidity, Mr. Mosbrooker served as the Chief Operations Officer for Cognoa, where he spearheaded commercial initiatives, program management, product development, and business operations.
  • Mr. Mosbrooker holds a Bachelor of Science in Industrial Engineering from the University of Wisconsin – Madison.

Arrowhead Pharmaceuticals Files for Regulatory Clearance to Initiate Phase 1/2a Study of ARO-DM1 for Type 1 Myotonic Dystrophy

Retrieved on: 
Tuesday, November 28, 2023
Biotechnology, Medical Devices, Health, Pharmaceutical, Clinical Trials, Life expectancy, Safety, Cataract, RNA interference, Exercise, Arrowhead, Clinical trial, DMPK, DM1, Wheelchair, Pharmacokinetics, Muscle weakness, Messenger, Therapy, Ethics committee, MBA, Committee, Wasting, Myotonia, Arrowhead Pharmaceuticals, Patient, Facial muscles, Discovery, Translational medicine, RNA, Pharmaceutical industry

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-DM1, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy.

Key Points: 
  • Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-DM1, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy.
  • ARO-DM1 is designed to reduce expression of the dystrophia myotonica protein kinase (DMPK) gene.
  • Treatments have focused on symptomatic management, including physical therapy, exercise, ankle-foot orthoses, wheelchairs, and other assistive devices.
  • Pending clearance, Arrowhead intends to proceed with ARODM1-1001, a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DM1 in up to 48 subjects with DM1.

HARMONY BIOSCIENCES ANNOUNCES POSITIVE TOPLINE DATA FROM PHASE 2 SIGNAL DETECTION STUDY EVALUATING PITOLISANT IN ADULT PATIENTS WITH MYOTONIC DYSTROPHY TYPE 1

Retrieved on: 
Thursday, December 7, 2023
Harmony, Safety, ESS, EDS, Narcolepsy, Fatigue, Data, FSS, Patient, DM1, OLE, Cataplexy, FDA, Muscle weakness, DSS, Pitolisant, Epworth Sleepiness Scale, Myotonic dystrophy, Myotonia, News, Somnolence, Pharmaceutical industry, Nursing, Medicinal plants

PLYMOUTH MEETING, Pa., Dec. 7, 2023 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (Nasdaq: HRMY) today announced positive topline results from its Phase 2 signal detection study evaluating the safety and efficacy of pitolisant in adult patients with myotonic dystrophy type 1 (DM1).

Key Points: 
  • PLYMOUTH MEETING, Pa., Dec. 7, 2023 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (Nasdaq: HRMY) today announced positive topline results from its Phase 2 signal detection study evaluating the safety and efficacy of pitolisant in adult patients with myotonic dystrophy type 1 (DM1).
  • "In addition, a positive signal for pitolisant has been demonstrated for fatigue, suggesting it could be a potential new treatment option for this symptom as well.
  • "The positive signals from this Phase 2 study are very encouraging, and support pitolisant as a potential treatment option for DM1."
  • This Phase 2 signal detection study was a randomized, double-blind, placebo-controlled study in adults ages 18-65 with DM1.

PepGen Reports Third Quarter 2023 Financial Results and Recent Corporate Developments

Retrieved on: 
Wednesday, November 8, 2023
DM1, FDA, Research, EDO, Clinical trial, Food, Three Months, Therapy, Health Canada, News, Conference, CTA, MAD, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Development, Clinical, BioCompute Object, PGN, Myotonia, Biology, Duchenne muscular dystrophy, Research and development, Letter, Safety, DMD, Patient, Administration, NHP, Investigational New Drug, Neuromuscular disease, Pharmaceutical industry, Cryptocurrency, Rare-earth element

BOSTON, Nov. 08, 2023 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today reported financial results for the third quarter ended September 30, 2023 and highlighted recent corporate developments.

Key Points: 
  • Orphan Drug Designation granted to PGN-EDODM1: In September 2023, the FDA granted Orphan Drug Designation to PGN-EDODM1 for the treatment of DM1.
  • PepGen expects to report initial results from this study in 2024.
  • Financial Results for the Three Months Ended September 30, 2023
    Cash and cash equivalents were $129.5 million as of September 30, 2023, which is anticipated to fund currently planned operations into 2025.
  • PepGen had approximately 23.8 million shares outstanding on September 30, 2023.

Dyne Therapeutics Reports Third Quarter 2023 Financial Results and Provides Update on Significant Progress for ACHIEVE and DELIVER Trials and Upcoming Clinical Milestones

Retrieved on: 
Monday, October 30, 2023
Research, Dystrophin, Therapy, ASO, Facial muscles, DYN, G&A, Safety, Conference, MAD, R, PMO, Pharmacokinetics, OLE, Duchenne muscular dystrophy, DMD, Patient, Cryptocurrency, Copper, Pharmaceutical industry, Medical imaging, Meat, Dietary supplement, DM1, Dyne, Myotonia

WALTHAM, Mass., Oct. 30, 2023 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the third quarter of 2023 and provided an update on progress and upcoming milestones for its Phase 1/2 ACHIEVE and DELIVER clinical trials.

Key Points: 
  • (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the third quarter of 2023 and provided an update on progress and upcoming milestones for its Phase 1/2 ACHIEVE and DELIVER clinical trials.
  • “We’ve made tremendous progress in our ACHIEVE and DELIVER trials with more than 72 patients enrolled and over 300 doses administered thus far.
  • General and administrative (G&A) expenses: G&A expenses were $7.0 million for the quarter ended September 30, 2023, compared to $7.6 million for the quarter ended September 30, 2022.
  • Net loss: Net loss for the quarter ended September 30, 2023 was $60.2 million, or $0.99 per basic and diluted share.

PepGen Inc. Announces FDA has Lifted the Clinical Hold on its Investigational New Drug Application for FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1)

Retrieved on: 
Thursday, October 12, 2023
EDO, Food, DMD, RNA, PGN, Myotonia, IND, Safety, Lifting, Disease, FDA, SAD, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, DM1, Patient, Dietary supplement, Pharmaceutical industry

BOSTON, Oct. 12, 2023 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has lifted the full clinical hold and cleared the Company’s Investigational New Drug Application (IND) to initiate the FREEDOM-DM1 Phase 1 study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1) in the U.S.

Key Points: 
  • FREEDOM-DM1 is a randomized, double-blind, placebo-controlled, single ascending dose (SAD) study, designed to assess PGN-EDODM1 safety and tolerability, correction of mis-splicing of transcripts, and clinical functional outcome measures.
  • Sites in both the U.S. and Canada will evaluate PGN-EDODM1 in 3 cohorts of 5 mg/kg, 10 mg/kg, and 20 mg/kg dose levels.
  • The decision to advance to the next dose level will be contingent upon the evaluation of safety data derived from previous dose cohorts.
  • The Company continues to expect its cash and cash equivalents to be sufficient to fund currently planned operations into 2025.

Myotonic Dystrophy Drug Pipeline Research Report 2023: Comprehensive Insights About 20+ Companies and 22+ Pipeline Drugs - ResearchAndMarkets.com

Retrieved on: 
Thursday, September 14, 2023
General Health, Pharmaceutical, Health, Other Health, Muscular dystrophy, GSK3, Therapy, Tideglusib, DM1, European Medicines Agency, Clinical trial, Histamine, NDA, Shikimate pathway, CNS, Messenger, II, EMA, Discovery, Patient, H3, DMPK, Mergers and acquisitions, ASO, Phase, News, Dyne, Disease, Pharmaceutical industry, Vaccine, Myotonic dystrophy, Myotonia

The "Myotonic Dystrophy - Pipeline Insight, 2023" report offers an extensive overview of the Myotonic Dystrophy pipeline landscape, featuring insights on over 20 companies and 22 pipeline drugs.

Key Points: 
  • The "Myotonic Dystrophy - Pipeline Insight, 2023" report offers an extensive overview of the Myotonic Dystrophy pipeline landscape, featuring insights on over 20 companies and 22 pipeline drugs.
  • For a holistic understanding, it includes a detailed disease overview and Myotonic Dystrophy treatment guidelines.
  • Currently, the drug is being investigated in the Phase II/III stage of clinical trial evaluation for the treatment of Congenital Myotonic Dystrophy.
  • This segment of the report provides insights about the different Myotonic Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:
    There are approx.