Dermatomyositis

argenx Reports First Quarter 2024 Financial Results and Provides Business Update

Retrieved on: 
Jeudi, mai 9, 2024
MuSK protein, FDA, ARDA, IND, PDUFA, Medicine, Autoimmunity, Efgartigimod alfa, Glomerulonephritis, CMS, Inflammation, ITP, IIP, ALS, Alfa Romeo, C2, Therapy, Systemic scleroderma, Safety, Euronext, Nektar, DGF, Sjögren syndrome, CIDP, Biology, IL-6, Nektar Therapeutics, GMG, TED, File, MMN, Lupus nephritis, Kidney, AMR, Multifocal motor neuropathy, Patient, Congenital myasthenic syndrome, Immune system, Dermatomyositis, Growth, Amgen, Pharmaceutical industry

“The team at argenx has made significant progress executing across the ambitious plan we set out at the beginning of the year,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

Key Points: 
  • “The team at argenx has made significant progress executing across the ambitious plan we set out at the beginning of the year,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.
  • “We are driven by our commitment to provide patients with the broadest gMG product offering that consistently delivers on safety and efficacy.
  • VYVGART SC played a key role in our growth over the quarter, expanding the breadth of our prescriber base and reaching new patients.
  • In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Treatment of dermatomyositis Withdrawn

Retrieved on: 
Mardi, avril 9, 2024
Eurostat, Civil service commission, Eating, Diagnosis, Azathioprine, Disease, Dermatomyositis, Risk, Rash, Inflammation, Patient, Committee, Knowledge, Tissue, Regulation, EMA, IRIS, Mouth, European, Skin, COMP, Heart, Sponsor, European Commission, Immune system, Safety, Muscle weakness, Skeletal muscle, Stomach, Marketing, Medicine, Pharmaceutical industry

Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Treatment of dermatomyositis Withdrawn

Key Points: 


Orphan designation: (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Treatment of dermatomyositis Withdrawn

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

Retrieved on: 
Jeudi, mars 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.

AMERICAN SKIN ASSOCIATION ANNOUNCES NEW BOARD MEMBER JOSEPH F. MEROLA, MD, MMSc, FAAD, FACR

Retrieved on: 
Jeudi, avril 4, 2024
Cone Health Women's Hospital, Internal medicine, Patient, Skin, FAAD, Connective tissue, DSM-IV codes, Education, ASA, MDS, MMSc, Mass, Harvard Medical School, Associate, National Psoriasis Foundation, Psoriatic arthritis, Hospital, Public health, Atopic dermatitis, Dermatomyositis, Lupus, American Academy, Assessment, Management

NEW YORK, April 4, 2024 /PRNewswire/ -- American Skin Association (ASA) announced today that Joseph F. Merola, MD, MMSc, FAAD, FACR, will join its Board of Directors.

Key Points: 
  • NEW YORK, April 4, 2024 /PRNewswire/ -- American Skin Association (ASA) announced today that Joseph F. Merola, MD, MMSc, FAAD, FACR, will join its Board of Directors.
  • Distinguished Chair in Dermatology and is Professor at the Peter O'Donnell Jr. School of Public Health.
  • "We are proud to announce the addition of Dr. Joseph Merola to ASA's Board of Directors.
  • He was recently elected to the Board of the American Academy of Dermatology.

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

Retrieved on: 
Jeudi, février 29, 2024
DGF, Neonatal Fc receptor, ALS, Congenital myasthenic syndrome, CMS, CIDP, MMN, Alfa Romeo, IIP, IL-6, FDA, Inflammation, MHLW, PDUFA, Euronext, Neurology, MuSK protein, Kidney, ARDA, Health care, C2, GMG, Ministry of Health, Dermatomyositis, Patient, Efgartigimod alfa, MHRA, Medicine, Multifocal motor neuropathy, Immune system, Pharmaceutical industry

“argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

Key Points: 
  • “argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.
  • Clinically, we generated significant data through multiple study readouts, achieving key milestones for both the CIDP and MMN patient communities and importantly advancing our second molecule, empasiprubart.
  • Looking forward to 2024, we will act with a continued sense of purpose to expand our patient reach.
  • In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

Galapagos announces full year 2023 results and outlook for 2024

Retrieved on: 
Jeudi, février 22, 2024
Technology transfer, CD19, Filgotinib, Lupus, Multiple myeloma, DL2, Dermatomyositis, Acceleration, Baltic, BCMA, Mantle cell lymphoma, European Commission, Indolent lymphoma, JAK1, Safety, EBMT, RT, TYK2, CFO, PRAC, MAA, Richter's transformation, Euronext, Executive Committee, JAK, UC, Patient, SLE, Pharmaceutical industry

In addition, we entered into a strategic collaboration with BridGene Biosciences to advance our growing early-stage pipeline in precision oncology.

Key Points: 
  • In addition, we entered into a strategic collaboration with BridGene Biosciences to advance our growing early-stage pipeline in precision oncology.
  • Signed an agreement with Boston-based Landmark Bio and started the technology transfer for the decentralized production of Galapagos’ CAR-T cell therapy candidates.
  • Further streamlined our operations with a reduction of approximately 100 positions across the Galapagos organization to align with the Galapagos’ renewed focus on innovation.
  • Signed a strategic collaboration and license agreement with BridGene Biosciences to further strengthen Galapagos' growing early-stage oncology precision medicine pipeline.

Cabaletta Bio Receives Additional FDA Fast Track Designations for CABA-201 in Dermatomyositis and Systemic Sclerosis

Retrieved on: 
Lundi, janvier 8, 2024
Dermatomyositis, Food, GMG, IND, Research, FDA, SLE, Organ dysfunction, Scleroderma, Investigational New Drug, Systemic scleroderma, Myositis, Patient, Lupus nephritis, Pharmaceutical industry

PHILADELPHIA, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted separate Fast Track Designations to CABA-201, an investigational 4-1BB-containing fully human CD19-CAR T cell therapy, for the treatment of patients with dermatomyositis to improve disease activity and for the treatment of patients with systemic sclerosis (SSc) to improve associated organ dysfunction.

Key Points: 
  • “The additional Fast Track Designations for CABA-201 in both dermatomyositis and systemic sclerosis, the second and third Fast Track Designations for CABA-201, provide the opportunity for expedited development and review of CABA-201 for the treatment of these autoimmune indications where there is a significant unmet need, despite currently available therapies,” said David J. Chang, M.D., Chief Medical Officer of Cabaletta.
  • “We believe these designations potentially accelerate our ability to launch the first targeted, and potentially curative, cell therapy for autoimmune diseases driven by B cells.
  • To date, Cabaletta has received clearance from the FDA for Investigational New Drug (IND) applications for CABA-201 in multiple autoimmune conditions including systemic lupus erythematosus (SLE), myositis, SSc and generalized myasthenia gravis (gMG).
  • Cabaletta is conducting four Phase 1/2 clinical trials with a total of nine cohorts that can advance simultaneously, employing a similar parallel cohort design and starting dose of 1 x 106 cells/kg without a dose escalation requirement.

argenx Highlights 2024 Strategic Priorities

Retrieved on: 
Lundi, janvier 8, 2024
Immunoglobulin therapy, GMG, FDA, PRV, Congenital myasthenic syndrome, ARDA, Research Councils UK, Euronext, Inflammation, Cohort, PGIC, NRDL, Autoimmunity, Efgartigimod alfa, Dermatomyositis, Conference, MuSK protein, IND, CIDP, Pharmacokinetics, MMN, Safety, Therapy, Patient, ALS, Immune system, IL-6, Medicine, International Myeloma Foundation, IIP, FSS, Neonatal Fc receptor, Multifocal motor neuropathy, Growth, Pharmaceutical industry

argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.

Key Points: 
  • argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.
  • In addition, ARGX-119, a muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a studies in congenital myasthenic syndrome and amyotrophic lateral sclerosis in 2024.
  • As of December 31, 2023, argenx had approximately $3.2 billion in cash, cash equivalents and current financial assets*.
  • Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2024 to be less than $2 billion.

Roivant and Priovant Announce Results from Phase 2 Study of Oral Brepocitinib in Systemic Lupus Erythematosus

Retrieved on: 
Lundi, novembre 27, 2023
Dermatomyositis, Uveitis, Hidradenitis suppurativa, Caregiver, Alopecia areata, SLE, Lupus, Calendar, NIU, Patient, Safety, Psoriasis, Ulcerative colitis, Pharmaceutical industry

Priovant plans to disclose the study data at a future date.

Key Points: 
  • Priovant plans to disclose the study data at a future date.
  • “We saw some of the highest SRI-4 responder rates ever observed in a lupus study in the active arm of this trial, along with a favorable safety and tolerability profile.
  • Unfortunately, we also saw the highest placebo response rate observed in any significant SLE study, and as such it was not possible to truly assess the impact of the drug, or to establish sufficient differentiation from other therapies in lupus patients.
  • Oral brepocitinib has demonstrated statistically significant clinically meaningful benefit in six completed placebo-controlled Phase 2 studies in psoriasis, psoriatic arthritis, alopecia areata, hidradenitis suppurativa, ulcerative colitis, and Crohn’s disease.

Northwell Health Pediatric Rheumatologist Recognized for Lifetime Achievement by the American College of Rheumatology

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Mardi, novembre 14, 2023
Children, Biotechnology, Other Consumer, Other Health, Health, General Health, Other Science, Research, Consumer, Science, Teens, Interest group, CCMC, Hope, Pediatrics, Patient, Multimedia, Lupus, American College of Rheumatology, Arthritis, American College, Northwell Health, Rheumatology, Medical research, Dermatomyositis, Mentorship, Credit, MBA, MD, Eberhard, ACR, ARP, Vasculitis, Nursing, Child

For her dedication to research and leadership in rheumatology, Northwell Health’s Cohen Children’s Medical Center (CCMC) pediatric rheumatologist and Feinstein Institutes for Medical Research scientist Barbara Anne Eberhard, MD , has been awarded top honors by the American College of Rheumatology (ACR) and the Association of Rheumatology Professionals (ARP).

Key Points: 
  • For her dedication to research and leadership in rheumatology, Northwell Health’s Cohen Children’s Medical Center (CCMC) pediatric rheumatologist and Feinstein Institutes for Medical Research scientist Barbara Anne Eberhard, MD , has been awarded top honors by the American College of Rheumatology (ACR) and the Association of Rheumatology Professionals (ARP).
  • Dr. Eberhard was recognized, and named an “ACR Master,” during the ACR Convergence 2023 annual conference which is being held November 10-15 in San Diego, California.
  • View the full release here: https://www.businesswire.com/news/home/20231114668794/en/
    Dr. Barbara Anne Eberhard was recognized for lifetime achievement by the American College of Rheumatology.
  • (Credit: Feinstein Institutes)
    The ACR Master recognition is awarded to physician-researchers who have made significant contributions to the field of rheumatology.