Myopathy

IGM Biosciences Announces First Quarter 2024 Financial Results and Provides Corporate Update

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星期三, 五月 8, 2024
PFS, Myopathy, Patient, Research and development, Investment, Colorectal cancer, Progression-free survival, Research, Immunoglobulin M, CD38, Bevacizumab, Trial of the century, Lupus, Administration, Rheumatoid arthritis, IGM, Myositis, SLE, Clinical trial, FOLFIRI, Rheumatism, GAAP, G&A

MOUNTAIN VIEW, Calif., May 08, 2024 (GLOBE NEWSWIRE) -- IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company creating and developing engineered IgM antibodies, today announced its financial results for the fiscal quarter ended March 31, 2024 and provided an update on recent developments.

Key Points: 
  • MOUNTAIN VIEW, Calif., May 08, 2024 (GLOBE NEWSWIRE) -- IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company creating and developing engineered IgM antibodies, today announced its financial results for the fiscal quarter ended March 31, 2024 and provided an update on recent developments.
  • Collaboration Revenue: For the first quarter of 2024 and 2023, collaboration revenues were $0.5 million in each quarter.
  • Research and Development (R&D) Expenses: For the first quarter of 2024, R&D expenses were $43.8 million, compared to $50.9 million for the first quarter of 2023.
  • General and Administrative (G&A) Expenses: For the first quarter of 2024, G&A expenses were $10.5 million, compared to $13.0 million for the first quarter of 2023.

Edgewise Therapeutics Reports First Quarter 2024 Financial Results and Recent Business Highlights

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星期四, 五月 9, 2024
Health, Communications, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Public Relations, Investor Relations, TNNI2, Safety, Pharmacokinetics, DUNE, Duchenne, McArdle, Ageing, Exercise, Creatine kinase, Patient, MRI, CK, Duchenne muscular dystrophy, Myopathy, Biomarker, Therapy, ARCH, Muscular dystrophy

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2024 and recent business highlights.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2024 and recent business highlights.
  • The Company expects to report CANYON data in the fourth quarter of 2024.
  • Research and development (R&D) expenses were $27.7 million for both the first quarter of 2024 and for the immediately preceding quarter.
  • General and Administrative (G&A) expenses were $7.1 million for the first quarter of 2024, compared to $6.2 million for the immediately preceding quarter.

New US product, ORMALVI™ (dichlorphenamide) tablets, launches for Primary Periodic Paralysis (PPP) treatment

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星期三, 五月 8, 2024
FDA, General Health, Pharmaceutical, Health, Fetus, Cardiomyopathy, Torrent, Acidosis, Respiratory acidosis, Full, Diclofenamide, Risk, Pregnancy, Cognitive disorder, Milk, Aspirin, Metabolic acidosis, Dysgeusia, Hyperkalemia, Aplastic anemia, Partnership, PPP, Agranulocytosis, Anorexia nervosa, Human, Carbonic anhydrase inhibitor, SVP, Tablet, Incidence, Patient, Nursing, Lethargy, Serum, Hyperkalemic periodic paralysis, Diagnosis, Myopathy, Hypokalemic periodic paralysis, Paralysis, Paresthesia, Cycle, Stevens–Johnson syndrome, Hypokalemia, Quality of life, Argyreia nervosa, Sulfonamide, Coma, Pharmaceutical industry

We want to provide them with the support that they require to effectively manage their PPP.

Key Points: 
  • We want to provide them with the support that they require to effectively manage their PPP.
  • ORMALVI™ is an oral carbonic anhydrase inhibitor indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants.
  • Simply Stated: Research Updates in Periodic Paralysis - Quest | Muscular Dystrophy Association [online] Available at: https://mdaquest.org/simply-stated-research-updates-in-periodic-paralysis Accessed 23 Nov. 2023.
  • The impact of permanent muscle weakness on quality of life in periodic paralysis: a survey of 66 patients.

Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Delpacibart Etedesiran (AOC 1001) for Treatment of Myotonic Dystrophy Type 1

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星期三, 五月 8, 2024
FDA, Food, RNA, QMT, Initiation, Facial muscles, Natural history, AOC, Therapy, Myopathy, European Medicines Agency, EMA, DM1, Avidity, Breakthrough therapy, SAN, Pharmaceutical industry

SAN DIEGO, May 8, 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 that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1 (DM1). Delpacibart etedesiran, abbreviated as del-desiran, is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.

Key Points: 
  • Delpacibart etedesiran, abbreviated as del-desiran, is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.
  • "We are pleased that the FDA has granted Breakthrough Therapy designation to del-desiran for myotonic dystrophy type 1, underscoring the potential of del-desiran to be an effective treatment and the urgency of bringing this treatment to people living with DM1," said Sarah Boyce, president and chief executive officer at Avidity.
  • Avidity is initiating the global pivotal HARBOR™ study of del-desiran this quarter.
  • In addition to receiving FDA Breakthrough Therapy designation, del-desiran has previously been granted Orphan Drug and Fast Track designations by the FDA and Orphan designation by the European Medicines Agency (EMA) for the treatment of DM1.

Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker)

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星期一, 四月 15, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, TNNI2, Neuromuscular disease, University, Safety, Pharmacokinetics, MDA, Muscle weakness, NSAA, Duchenne, Patient, Muscular dystrophy, University of Florida, Multimedia, CK, Duchenne muscular dystrophy, Myopathy, Biomarker, Therapy, ARCH, Pharmaceutical industry

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced positive two-year topline results from the ARCH trial.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced positive two-year topline results from the ARCH trial.
  • ARCH is an open label, single-center study assessing safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics (PK) and functional measures with sevasemten (EDG-5506) in adults with Becker.
  • Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Becker and Duchenne muscular dystrophy (Duchenne).
  • ET to discuss the ARCH two-year data, and will be joined by Dr. Byrne, who will share his perspective of sevasemten and Becker.

Cabaletta Bio Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

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星期四, 三月 21, 2024
Research, MPV, IIM, MuSK protein, Toxicity, Conference, Disease, Dermatomyositis, Juvenile dermatomyositis, Federation, Patient, NOL, Observation, CRS, SLE, Health Canada, IND, Immunoglobulin therapy, Pemphigus, Investment, Investigational New Drug, Myopathy, Systemic scleroderma, Marketing, FDA, Clinical trial, Cytokine release syndrome, Cyclophosphamide, CD19, Food, Autoimmune disease, Pharmaceutical industry

In October 2023, Cabaletta received Investigational New Drug (IND) application clearance from the U.S. Food and Drug Administration (FDA) for the Phase 1/2 RESET-SSc trial.

Key Points: 
  • In October 2023, Cabaletta received Investigational New Drug (IND) application clearance from the U.S. Food and Drug Administration (FDA) for the Phase 1/2 RESET-SSc trial.
  • In November 2023, Cabaletta announced that its IND application for CABA-201 was allowed to proceed by the FDA for the Phase 1/2 RESET-MG trial.
  • Cabaletta anticipates reporting initial clinical data from the Phase 1/2 RESET-MG trial in the second half of 2024.
  • As of December 31, 2023, Cabaletta had cash, cash equivalents and short-term investments of $241.2 million, compared to $106.5 million as of December 31, 2022.

MetrioPharm Establishes Scientific Advisory Board of Seasoned Immunology and Pediatric Experts

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星期三, 三月 13, 2024
Patient, Child, Laboratory, Duchenne muscular dystrophy, University of Oxford, School, Research, Inflammation, Electrophysiology, National Science Foundation, Immunology, Rockefeller University, Research fellow, Neuromuscular disease, Pediatrics, Pathology, SAB, IRB, CNRS, Neurology, Inflammation Research, Surgery, University, Neuro, MD, Senior, Consultant, Chemokine, Medical school, Retirement, CSO, Myopathy, Signal transduction, Doctor of Philosophy, Nursing

Ferdinando Nicoletti, MD, PhD, has been Full Professor of General Pathology and Immunology since 2011 at the University of Catania (Italy).

Key Points: 
  • Ferdinando Nicoletti, MD, PhD, has been Full Professor of General Pathology and Immunology since 2011 at the University of Catania (Italy).
  • “We welcome the members of MetrioPharm’s newly established scientific advisory board,” said Thomas Christély, CEO of MetrioPharm.
  • “We are happy to now formally establish and introduce this panel of experts for an even closer collaboration.
  • Every aspect of our research development program - from preclinical research to clinical trials - will benefit from the unparalleled expertise of our new scientific advisors.”

Study shows improved hand function using Carbonhand

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星期三, 三月 13, 2024
Jamar, Conference, Face, Perception, IBM, Glass, Facial muscles, Person, Myopathy, Pain, TMA, Hand, Autoimmune disease, Personal protective equipment

STOCKHOLM, March 13, 2024 /PRNewswire/ -- Today, a study on Improved physical function using a power-enhancing glove in persons with Inclusion Body Myositis was presented at the Global Conference on Myositis in Pittsburgh, USA.

Key Points: 
  • STOCKHOLM, March 13, 2024 /PRNewswire/ -- Today, a study on Improved physical function using a power-enhancing glove in persons with Inclusion Body Myositis was presented at the Global Conference on Myositis in Pittsburgh, USA.
  • The preliminary results show increased hand function and improved physical function for people with impaired hand function during use of Carbonhand.
  • The study was open to people with idiopathic inflammatory myopathies who perceived hand weakness.
  • The study included 40 persons with IBM that had reduced grip strength (kg) (md;range) (3.75;1.3-10.7) and physical function (IBM-FRS 20;2-38, IBM-PRO 23;1-44).

IGM Biosciences Announces Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update

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星期四, 三月 7, 2024
CD20, Progression-free survival, PFS, Myopathy, Research, Immunoglobulin M, Investment, FDA, Research and development, IGM, FOLFIRI, Myositis, Bevacizumab, Lupus, GAAP, Rheumatoid arthritis, Administration, Patient, Clinical trial, Survival, CD38, SLE, G&A, IND, Colorectal cancer, Pharmaceutical industry

MOUNTAIN VIEW, Calif., March 07, 2024 (GLOBE NEWSWIRE) -- IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company creating and developing engineered IgM antibodies, today announced its financial results for the fourth quarter and full year ended December 31, 2023 and provided an update on recent developments.

Key Points: 
  • MOUNTAIN VIEW, Calif., March 07, 2024 (GLOBE NEWSWIRE) -- IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company creating and developing engineered IgM antibodies, today announced its financial results for the fourth quarter and full year ended December 31, 2023 and provided an update on recent developments.
  • Collaboration Revenue: For the fourth quarter and year ended 2023, collaboration revenues were $0.7 million and $2.1 million, respectively, compared to $0.4 million and $1.1 million for the fourth quarter and year ended 2022, respectively.
  • Research and Development (R&D) Expenses: For the fourth quarter and year ended 2023, R&D expenses were $54.2 million and $215.5 million, respectively, compared to $45.0 million and $179.3 million for the fourth quarter and year ended 2022, respectively.
  • General and Administrative (G&A) Expenses: For the fourth quarter and year ended 2023, G&A expenses were $11.6 million and $50.1 million, respectively, compared to $11.6 million and $49.7 million for the fourth quarter and year ended 2022, respectively.

Satellos Presents Positive Preclinical Efficacy Data for SAT-3247 at the 2024 MDA Clinical & Scientific Conference

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星期一, 三月 4, 2024
Biotechnology, Health, Science, Pharmaceutical, Research, Molecule, OTCQB, Regeneration, MSCL, TSX, Animal, DMD, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, Skeletal muscle, Conference, Event, FSHD, Poster, Trauma, Myopathy, Episcopal conference, Face

Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preclinical data showing SAT-3247 can improve skeletal muscle function in multiple mouse models of muscle degeneration.

Key Points: 
  • Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preclinical data showing SAT-3247 can improve skeletal muscle function in multiple mouse models of muscle degeneration.
  • In all instances, treatment with SAT-3247 over a three-to-four-week period resulted in a statistically significant improvement in muscle force versus animals receiving placebo.
  • We believe the muscle injury data further expand and broaden the potential for SAT-3247 into non-dystrophy disease indications as well as multiple forms of muscle injury or trauma in otherwise healthy individuals.
  • We continue on our development plan to advance SAT-3247 into first-in-human clinical trials mid-year.”
    These data are being presented in a poster at the 2024 MDA Clinical & Scientific Conference being held March 3-6 in Orlando.