Lancet Neurology

Italfarmaco Receives FDA Approval for Duvyzat™ (givinostat) in Duchenne Muscular Dystrophy

Retrieved on: 
Friday, March 22, 2024
Research, FDA, Genetics, Clinical Trials, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, HDAC, ITF, MAA, The Lancet, Caregiver, Disease, Magnetic resonance imaging, PPMD, Patient, Rehabilitation, Lancet Neurology, University, EMA, Doctor of Philosophy, MD, Duchenne muscular dystrophy, Pediatrics, Therapy, NSAA, DMD, Food, Pharmaceutical industry

“We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval.

Key Points: 
  • “We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval.
  • The FDA approval highlights the dedication of Italfarmaco’s research and clinical teams to achieve this milestone for the company.”
    The approval is based on the results of the pivotal multicentre, randomised, double-blind, placebo-controlled phase 3 EPIDYS trial ( NCT02851797 ).
  • Italfarmaco has significantly expanded its U.S. presence through the formation of a new fully owned subsidiary, ITF Therapeutics LLC.
  • Italfarmaco has a global presence and is also working with other regulatory agencies.

Results from Italfarmaco Pivotal Phase 3 EPIDYS Study of Givinostat in Duchenne Muscular Dystrophy (DMD) Published in The Lancet Neurology

Retrieved on: 
Tuesday, March 19, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, Hospitals, Genetics, Clinical Trials, VL, Civil service commission, Givinostat, Neurology, Catholic higher education, Diarrhea, MAA, The Lancet, Magnetic resonance imaging, Safety, Abdominal pain, NDA, Manuscript, Rehabilitation, Lancet Neurology, University, OLE, EMA, Degenerative disease, Doctor of Philosophy, MD, European Medicines Agency, Duchenne muscular dystrophy, European Commission, NSAA, FDA, DMD, Pediatrics, PDUFA, Pharmaceutical industry

Italfarmaco S.p.A. announced today that the full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology.

Key Points: 
  • Italfarmaco S.p.A. announced today that the full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology.
  • The full manuscript, titled, “Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomized, double-blind, placebo-controlled, phase 3 trial,” is published on The Lancet Neurology website .
  • Participants attended study site visits every 12 weeks for 72 weeks and were evaluated for all endpoints within the study protocol.
  • Upon completion of the 18-month double-blind period, EPIDYS study participants were eligible to enroll in an open-label extension study (OLE study) to receive givinostat on an ongoing basis.

Patient, Research and Industry Leaders Propose Biological Definition and First Biological Staging for Parkinson's Disease

Retrieved on: 
Tuesday, January 23, 2024
NSD, Pathology, Neuroscience, Northwestern University, The Lancet, MJFF, Patient, Dementia, MHSA, Research, Biology, Neurology, MD, DaT scan, Critical Path Institute, Paper, Gait, Knowledge, DLB, Science, Consortium, Protocol, ASAP, Parkinson's disease, PD, Alpha-synuclein, Tremor, Food, Disease, REM, Drug development, Anosmia, Doctor of Philosophy, Deficit spending, Biomarker, Organization, Clinical trial, Bangladesh Technical Education Board, Brain, Motivation, Three-body problem, Lancet Neurology, Pharmaceutical industry

The new research framework stands to accelerate and increase the success of scientific discovery and therapeutic development at all stages of Parkinson's.

Key Points: 
  • The new research framework stands to accelerate and increase the success of scientific discovery and therapeutic development at all stages of Parkinson's.
  • And ultimately, a treatment that targets the biology of the disease — rather than just its symptoms — is the way to reach a cure.
  • (A similar biological framework in Alzheimer's disease resulted in successful trials and new drug approvals, with the first drugs to slow Alzheimer's-related cognitive decline appearing in 2022 and 2023.)
  • This landmark step forward was made possible by the breakthrough discovery of a test — validated in April 2023 by The Michael J.

Patient, Research and Industry Leaders Propose Biological Definition and First Biological Staging for Parkinson's Disease

Retrieved on: 
Tuesday, January 23, 2024
NSD, Pathology, Neuroscience, Northwestern University, The Lancet, MJFF, Patient, Dementia, MHSA, Research, Biology, Neurology, MD, DaT scan, Critical Path Institute, Paper, Gait, Knowledge, DLB, Science, Consortium, Protocol, ASAP, Parkinson's disease, PD, Alpha-synuclein, Tremor, Food, Disease, REM, Drug development, Anosmia, Doctor of Philosophy, Deficit spending, Biomarker, Organization, Clinical trial, Bangladesh Technical Education Board, Brain, Motivation, Three-body problem, Lancet Neurology, Pharmaceutical industry

The new research framework stands to accelerate and increase the success of scientific discovery and therapeutic development at all stages of Parkinson's.

Key Points: 
  • The new research framework stands to accelerate and increase the success of scientific discovery and therapeutic development at all stages of Parkinson's.
  • And ultimately, a treatment that targets the biology of the disease — rather than just its symptoms — is the way to reach a cure.
  • (A similar biological framework in Alzheimer's disease resulted in successful trials and new drug approvals, with the first drugs to slow Alzheimer's-related cognitive decline appearing in 2022 and 2023.)
  • This landmark step forward was made possible by the breakthrough discovery of a test — validated in April 2023 by The Michael J.

Cartesian Therapeutics Highlights Progress and 2024 Strategic Priorities Across Innovative Pipeline of mRNA Cell Therapies for Autoimmunity

Retrieved on: 
Monday, January 8, 2024
Lancet Neurology, RNA, Food, Autoantibody, Autoimmune disease, Risk, IND, Messenger, FDA, Cytokine release syndrome, The Lancet, SLE, Fatigue, Multiple myeloma, BCMA, Muscle weakness, Therapy, Pharmacokinetics, Lupus, Neurotoxicity, Transfection, Myasthenia gravis, Patient, Safety, Autoimmunity, Vaccine

The Company’s RNA-engineering approach is designed to expand the reach of cell therapy to autoimmunity with potent therapies that can be dosed more reliably and safely in an outpatient setting without lymphodepletion.

Key Points: 
  • The Company’s RNA-engineering approach is designed to expand the reach of cell therapy to autoimmunity with potent therapies that can be dosed more reliably and safely in an outpatient setting without lymphodepletion.
  • Cartesian’s proprietary technology platform, RNA Armory®, is designed to enable precision control and optimization of engineered cells for diverse cell therapies leveraging multiple modalities, including autologous, allogeneic, and in situ transfection.
  • Descartes-08 is an autologous anti-B cell maturation antigen (BCMA) mRNA CAR-T.
  • Cartesian continues to anticipate the initiation of Phase 2 basket studies in additional autoimmune indications in the second half of 2024.

Cartesian Therapeutics Announces Positive Long-Term Follow-Up Data from Phase 2a Study of Lead mRNA Cell Therapy Candidate Descartes-08 in Patients with Myasthenia Gravis

Retrieved on: 
Monday, January 8, 2024
Lancet Neurology, Patient, Quality of life, Acetylcholine receptor, Peer review, Food, Autoimmune disease, Risk, IND, Messenger, Cytokine release syndrome, The Lancet, Data, Fatigue, Lupus, BCMA, Muscle weakness, Orphan, Therapy, Clinical trial, Investigational New Drug, Quality, Pharmacokinetics, Neurotoxicity, Manuscript, Month, MedRxiv, Autoimmunity, Pharmaceutical industry

GAITHERSBURG, Md., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Cartesian Therapeutics, Inc. (NASDAQ: RNAC), (“the Company”) a clinical-stage biotechnology company pioneering mRNA cell therapies for autoimmune diseases, today announced positive twelve-month follow-up data from its Phase 2a trial of Descartes-08 in patients with generalized myasthenia gravis (MG), a chronic autoimmune disorder that causes disabling muscle weakness and fatigue. The manuscript titled, “Twelve-Month Follow-Up of Patients With Generalized Myasthenia Gravis Receiving BCMA-Directed mRNA Cell Therapy,” has been submitted for peer-review and can be accessed on the online preprint server, medRxiv.

Key Points: 
  • The manuscript titled, “Twelve-Month Follow-Up of Patients With Generalized Myasthenia Gravis Receiving BCMA-Directed mRNA Cell Therapy,” has been submitted for peer-review and can be accessed on the online preprint server, medRxiv.
  • Descartes-08, Cartesian’s lead mRNA cell therapy candidate and a potential first-in-class mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T), is an autologous anti-B-cell maturation antigen (BCMA) mRNA CAR-T.
  • “Notably, most patients maintained robust, clinically meaningful improvements across all four standard MG severity scores approximately 10 months after the last infusion.
  • Enrollment is ongoing in a Phase 2b randomized, double-blind, placebo-controlled trial (NCT04146051) in patients with MG. Topline results are expected in mid-2024.

Selecta Biosciences Announces Merger with Cartesian Therapeutics

Retrieved on: 
Monday, November 13, 2023
Acquisition, DNA, Entrepreneurship, CVR College of Engineering, Patient, Autoimmune disease, SVB Securities, Lancet Neurology, Webcast, CVR, Food, PIPE, Toxicity, Covington & Burling, Transfection, RNA, GMP, Therapy, Risk, Myasthenia gravis, AAAD, Pharmacokinetics, Mergers and acquisitions, Orphan, Autoimmunity, The Lancet, LLP, Neutrophil extracellular traps, Fatigue, Lupus, Muscle weakness, Event, Vaccine, Mobile phone, Pharmaceutical industry, Management, Security (finance), Selecta, Cartesian, Quality

and GAITHERSBURG, Md., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB) (the Company) today announced that it has merged with Cartesian Therapeutics, Inc., a clinical-stage biotechnology company pioneering RNA cell therapies for autoimmune diseases.

Key Points: 
  • and GAITHERSBURG, Md., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB) (the Company) today announced that it has merged with Cartesian Therapeutics, Inc., a clinical-stage biotechnology company pioneering RNA cell therapies for autoimmune diseases.
  • In connection with the merger, Selecta announced a $60.25 million private financing led by Timothy A. Springer, Ph.D., member of the Selecta Board of Directors.
  • Concurrent with the merger, the combined company has been renamed Cartesian Therapeutics, Inc.
  • Selecta and Cartesian will host a conference call today, Monday, November 13, 2023, at 9:00 am ET to discuss the merger.

Genethon Announces Publication in The Lancet Neurology of Clinical Trial Results of a Gene Therapy for Myotubular Myopathy, a Severe Muscle Disease

Retrieved on: 
Friday, November 17, 2023
Research, Technology, Genetics, Clinical Trials, Nanotechnology, Biotechnology, Pharmaceutical, Health, Science, Other Science, The Lancet, X-linked myotubular myopathy, Acquisition, Therapy, Gene, Pathology, Head, Clinical trial, MTM1, Death, Muscle weakness, Periodic breathing, Muscular Dystrophy Association, Astellas Pharma, Neurology, Myopathy, Knowledge, Lancet Neurology, Pharmaceutical industry, Audentes/Noortekoondis, Inserm

Myotubular myopathy is caused by mutations in the MTM1 gene encoding myotubularin, a protein involved in muscle cell function.

Key Points: 
  • Myotubular myopathy is caused by mutations in the MTM1 gene encoding myotubularin, a protein involved in muscle cell function.
  • Characterized by extreme muscle weakness and severe respiratory distress, 50% of affected children die before age 18 months and 75% die before age 10.
  • The gene therapy uses an adeno-associated viral vector (AAV8) to deliver a copy of the MTM1 gene.
  • It took years of research to imagine, design and demonstrate the efficacy of the gene therapy for this very severe and complex disease.

UCB announces U.S. FDA approval of ZILBRYSQ® (zilucoplan) for the treatment of adults with generalized myasthenia gravis

Retrieved on: 
Tuesday, October 17, 2023
Neonatal Fc receptor, UCB, MuSK protein, Food, MG, University of North Carolina at Chapel Hill, MAC, System, Disease, Immunoglobulin G, Hospital, Plasmapheresis, Oklahoma Office of the Chief Medical Examiner, Brussels Stock Exchange, NMJ, GMG, Diarrhea, Physician, RAISE, U.S. FDA, Neuromuscular junction, University, Safety, Lancet Neurology, Patient, The Lancet, Week, MD, Allied health professions, Myasthenia, Quality of life, Muscle weakness, Acetylcholine receptor, Travel, QMG, Neuromuscular disease, Fatigue, FDA, Upper respiratory tract infection, Pathology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Priority review, ACHR, Weakness, Pharmaceutical industry, Vaccine, Medicine, Neurology, C5

It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG.

Key Points: 
  • It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG.
  • Patients were randomized in a 1:1 ratio to receive daily subcutaneous injections of 0.3 mg/kg ZILBRYSQ or placebo for 12 weeks.
  • The most common adverse reactions (≥10%) in patients with gMG were injection site reactions, upper respiratory tract infection, and diarrhea.
  • The primary endpoint for the RAISE study was change from baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score.

argenx Announces VYVGART (efgartigimod alfa) Authorized for Sale by Health Canada for Generalized Myasthenia Gravis

Retrieved on: 
Thursday, September 21, 2023
Hair, University, Lancet Neurology, FC, Muscular dystrophy, GMG, Efgartigimod alfa, Netherlands, Sale, Muscular Dystrophy Canada, Euronext, Patient, University of Toronto, Division, ADAPT, Immunoglobulin G, SE, University Health Network, Acetylcholine receptor, Neonatal Fc receptor, Health Canada, The Lancet, Pharmaceutical industry, Neurology

With this regulatory milestone, VYVGART is the first-and-only neonatal Fc receptor (FcRn) blocker authorized for sale in Canada.

Key Points: 
  • With this regulatory milestone, VYVGART is the first-and-only neonatal Fc receptor (FcRn) blocker authorized for sale in Canada.
  • “There continues to be a significant unmet medical need for people living with gMG, who face debilitating muscle weakness and mobility issues.
  • The gMG community in Canada has long awaited new effective treatment advancements for this rare condition.
  • “Today is an important day for us as we deliver on our commitment to the gMG community to make VYVGART available to patients in Canada,” said John Haslam, General Manager argenx Canada.