NEJM

KalVista Pharmaceuticals Announces New England Journal of Medicine Publishes Oral Sebetralstat Phase 3 Data Demonstrating its Potential to Transform the Treatment Landscape for People with Hereditary Angioedema

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金曜日, 5月 31, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Patient, Doctor of Philosophy, KALV, HAE, Safety, IQR, MBA, MD, Q3, NEJM, EAACI, Internal medicine, Attack, Semmelweis University, Pharmaceutical industry

The results of KONFIDENT were simultaneously published in The New England Journal of Medicine.

Key Points: 
  • The results of KONFIDENT were simultaneously published in The New England Journal of Medicine.
  • Subgroup analyses showed a consistency of treatment effect in among attacks regardless of attack severity, attack location, use of long-term prophylaxis, or geography.
  • There were no patient withdrawals due to any adverse events and no treatment-related serious adverse events (SAEs) were observed.
  • Treatment-related adverse event rates were 2.3% for 300 mg sebetralstat, 3.2% for 600 mg sebetralstat, and 4.8% for placebo.

Dupixent® (dupilumab) Late-Breaking Data from NOTUS Confirmatory Phase 3 COPD Trial Presented at ATS and Published in The New England Journal of Medicine

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月曜日, 5月 20, 2024
Patient, Clinical trial, Dupilumab, University, Conference, American Thoracic Society, Birmingham School of Law, NEJM, COPD, Division, ATS, Chronic obstructive pulmonary disease, Regeneron Pharmaceuticals, Critical Care Medicine (journal), Quality of life, Pharmaceutical industry

TARRYTOWN, N.Y. and PARIS, May 20, 2024 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today presented late-breaking data from the NOTUS Phase 3 trial evaluating the investigational use of Dupixent® (dupilumab) as an add-on maintenance treatment in adults with uncontrolled COPD on maximal standard-of-care inhaled therapy (nearly all on triple therapy) and evidence of type 2 inflammation (i.e., blood eosinophils ≥300 cells per μL). The NOTUS trial confirmed the positive results demonstrated in the landmark Phase 3 BOREAS trial, with its data presented at a late-breaking session of the 2024 American Thoracic Society (ATS) International Conference and simultaneously published in the New England Journal of Medicine (NEJM).

Key Points: 
  • The NOTUS trial confirmed the positive results demonstrated in the landmark Phase 3 BOREAS trial, with its data presented at a late-breaking session of the 2024 American Thoracic Society (ATS) International Conference and simultaneously published in the New England Journal of Medicine (NEJM) .
  • “In NOTUS, dupilumab reduced exacerbations by a magnitude never seen before with an investigational biologic in a Phase 3 COPD clinical trial.
  • These comprehensive results reinforce that, if approved, dupilumab could provide a first-of-its-kind medical advancement for the COPD community.”
    As presented and published, the NOTUS trial met its primary and key secondary endpoints.
  • AEs more commonly observed with placebo than Dupixent included COPD (7.8% placebo, 4.9% Dupixent).

Press Release: Dupixent® late-breaking data from NOTUS confirmatory phase 3 COPD study presented at ATS and published in NEJM

Retrieved on: 
月曜日, 5月 20, 2024
Patient, Dupilumab, University, Trial of the century, Conference, American Thoracic Society, Birmingham School of Law, NEJM, COPD, Division, ATS, Chronic obstructive pulmonary disease, Critical Care Medicine (journal), Quality of life, Pharmaceutical industry

The NOTUS study confirmed the positive results demonstrated in the landmark phase 3 BOREAS study, with its data presented at a late-breaking session of the 2024 American Thoracic Society (ATS) International Conference and simultaneously published in the New England Journal of Medicine (NEJM) .

Key Points: 
  • The NOTUS study confirmed the positive results demonstrated in the landmark phase 3 BOREAS study, with its data presented at a late-breaking session of the 2024 American Thoracic Society (ATS) International Conference and simultaneously published in the New England Journal of Medicine (NEJM) .
  • In NOTUS, dupilumab reduced exacerbations by a magnitude never seen before with an investigational biologic in a phase 3 COPD clinical study.
  • These comprehensive results reinforce that, if approved, dupilumab could provide a first-of-its-kind medical advancement for the COPD community.”
    As presented and published, the NOTUS study met its primary and key secondary endpoints.
  • AEs more commonly observed with placebo than Dupixent included COPD (7.8% placebo, 4.9% Dupixent).

Using AI to Improve Diagnosis of Rare Genetic Disorders

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木曜日, 4月 25, 2024
Genetics, Bangladesh Technical Education Board, Undiagnosed Diseases Network, UDN, NRI, Human Genome Sequencing Center, Hope, Hospital, Genetic disorder, DSM-IV codes, AIM, Pediatrics, Jan, Artificial intelligence, Intelligence, Disease, Human, Data, Baylor College of Medicine, Neurology, NEJM, Database, Diagnosis, Algorithm, Baylor University, Gene, DDD, Vaccine

The team developed a machine learning system called AI-MARRVEL (AIM) to help prioritize potentially causative variants for Mendelian disorders.

Key Points: 
  • The team developed a machine learning system called AI-MARRVEL (AIM) to help prioritize potentially causative variants for Mendelian disorders.
  • “The diagnostic rate for rare genetic disorders is only about 30%, and on average, it is six years from the time of symptom onset to diagnosis.
  • AIM is trained using a public database of known variants and genetic analysis called Model organism Aggregated Resources for Rare Variant ExpLoration ( MARRVEL ) previously developed by the Baylor team .
  • “AIM is a major step forward in using AI to diagnose rare diseases.

Clinical Trial by Harvard Pilgrim Health Care Institute, HCA Healthcare, UCI Health and CDC Identifies Strategy to Rapidly Detect and Respond to Hospital Outbreaks Using Algorithm-Driven Technology

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火曜日, 4月 23, 2024
Data Management, Other Health, Technology, Managed Care, General Health, Infectious Diseases, Hospitals, Clinical Trials, Other Technology, Biotechnology, Science, Software, Networks, Other Science, Research, Health, University, Hospital, Risk, CDC, University of California, HCA Healthcare, Patient safety, Fungal infection, Doctor of Science, Patient, MPH, UCI, NEJM, Harvard Medical School, Infection, Harvard Pilgrim Health Care, Doctor of Public Health, Senior advisor, COVID-19, CLUSTER, Vaccine

The large multi-state, real-world study was conducted in 82 hospitals and led by Harvard Pilgrim Health Care Institute, HCA Healthcare, University of California, Irvine (UCI) Health and the Centers for Disease Control and Prevention (CDC).

Key Points: 
  • The large multi-state, real-world study was conducted in 82 hospitals and led by Harvard Pilgrim Health Care Institute, HCA Healthcare, University of California, Irvine (UCI) Health and the Centers for Disease Control and Prevention (CDC).
  • While hospitals and health systems work to prevent infections and reduce the opportunity for outbreaks to occur, there is no standardized approach for detecting transmission.
  • Early detection can lead to a rapid response that reduces the chance for outbreaks to occur.
  • “The CLUSTER trial provides evidence that early detection powered by automation tools and quick action can prevent outbreaks from growing."

Natera Announces Significant Milestone with 200+ Peer-Reviewed Publications

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月曜日, 4月 22, 2024
Research, Genetics, Clinical Trials, Biotechnology, Other Health, Health, Other Science, Science, Oncology, Nucleic acid thermodynamics, NTRA, Investment, Publication, Colorectal cancer, Genetic testing, SMART, DNA, NIPT, Patient, Kidney transplantation, NEJM, Clinical trial, ProActive, Chronic kidney disease, Cohort (statistics), Pharmaceutical industry

Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA (cfDNA) and genetic testing, today announced that it has passed a significant milestone with the publication of more than 200 peer-reviewed papers highlighting Natera’s technology in the scientific literature.

Key Points: 
  • Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA (cfDNA) and genetic testing, today announced that it has passed a significant milestone with the publication of more than 200 peer-reviewed papers highlighting Natera’s technology in the scientific literature.
  • Landmark cutting-edge science and clinical trial data based on Natera’s suite of products have been published in top journals, such as Science, Nature, Nature Medicine, and New England Journal of Medicine (NEJM).
  • “When it comes to transforming patient care, high-quality science and data drive everything,” said Alexey Aleshin, M.D., chief medical officer & general manager of oncology at Natera.
  • We remain committed to generating evidence that supports the real-world impact of our innovations.”

Lumicell’s Cutting-Edge Imaging Platform Receives Historic FDA Approval to Illuminate Residual Breast Cancer

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木曜日, 4月 18, 2024
Medical Devices, Surgery, FDA, Clinical Trials, Health Technology, Other Health, Biotechnology, Pharmaceutical, Health, Oncology, Urine, Society, Cancer, Hospital, Safety, Woman, NDA, New Drug Application, Surgeon, Breast, MD, Doctor of Philosophy, Patient, DVS, Anaphylaxis, Hypersensitivity, NEJM, Harvard Medical School, Multimedia, MD Anderson Cancer Center, Surgical oncology, Neoplasm, Massachusetts General Hospital, PMA, Breast cancer, Medical imaging

The most common side effects with LUMISIGHT are hypersensitivity and an abnormal color in urine.

Key Points: 
  • The most common side effects with LUMISIGHT are hypersensitivity and an abnormal color in urine.
  • Results of the Investigation of Novel Surgical Imaging for Tumor Excision (INSITE) pivotal trial, used to support the efficacy of the system, were published in NEJM Evidence .
  • “We want to thank our clinical investigators and the hundreds of women who participated in our breast program for LUMISIGHT and Lumicell DVS,” said Jorge Ferrer, Chief Scientific Officer, Lumicell.
  • “Due to your important contributions, LUMISIGHT and Lumicell DVS are now approved and will be available in the United States shortly.”
    Please visit www.LumiSystem.com to learn more about LUMISIGHT and Lumicell DVS.

MRM Health Reports Safety and Positive Efficacy Data in Pouchitis in Phase 2a Clinical Study with MH002

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火曜日, 4月 16, 2024
Biotechnology, Other Health, Health, Pharmaceutical, Clinical Trials, Inflammation, Safety, Outline, CNS, IBD, Therapy, Patient, Histology, DSM-IV codes, NEJM, MRM, Inflammatory Bowel Diseases, UC, KU Leuven, CGMP, Pharmaceutical industry

MRM Health NV, a clinical-stage biopharmaceutical company developing innovative therapeutics for inflammatory, CNS and metabolic diseases, today reports positive topline results from its Phase 2a clinical trial with MH002 in the orphan disease indication Pouchitis.

Key Points: 
  • MRM Health NV, a clinical-stage biopharmaceutical company developing innovative therapeutics for inflammatory, CNS and metabolic diseases, today reports positive topline results from its Phase 2a clinical trial with MH002 in the orphan disease indication Pouchitis.
  • MRM Health’s MH002-PC-201 study was a multi-center, open-label clinical trial in 14 acute Pouchitis (PC) patients at multiple clinical sites in Belgium and Italy.
  • The study was designed to evaluate safety (primary objective), initial efficacy, and mechanistic effects of MH002 over 8 weeks.
  • Treatment-Emergent Adverse Events (TEAE) reported were predominantly mild and unrelated, and there was no evidence of adverse reactions related to MH002.

AMBOSS Acquires NEJM Knowledge+ from NEJM Group, Setting a New Standard in Medical Education and Clinical Practice

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木曜日, 4月 25, 2024
Learning, Health care, Entrepreneurship, Acquisition, Physician, NEJM, Evidence-based medicine, Pediatrics, Health, Internal medicine, Student, Family medicine, Nursing

AMBOSS is renowned for its expert medical library, intuitive learning platform, and user interface designed to easily obtain and retain medical knowledge.

Key Points: 
  • AMBOSS is renowned for its expert medical library, intuitive learning platform, and user interface designed to easily obtain and retain medical knowledge.
  • "We are really excited to bring NEJM Knowledge+ into the AMBOSS family," said Dr. Madjid Salimi, Co-Founder and Co-CEO of AMBOSS.
  • "NEJM Group has always been at the forefront of disseminating medical knowledge and improving patient care," said David Sampson, Vice President, Chief Publishing Officer of NEJM Group.
  • Through this acquisition, AMBOSS and NEJM Group reaffirm their commitment to enhancing medical education and clinical practice.

Ionis presents positive results from Phase 3 Balance study of olezarsen for familial chylomicronemia syndrome

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日曜日, 4月 7, 2024
TGS, AP, Ionis, Incidence, Abdominal pain, IONS, Webcast, Patient, NEJM, Fasting, American College, Ionis Pharmaceuticals, FCS, ACC, Triglyceride, Annual general meeting, Medicine, Pharmaceutical industry

CARLSBAD, Calif., April 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced full results from the Phase 3 Balance study of Ionis' lead independent investigational medicine, olezarsen, for the treatment of adults with familial chylomicronemia syndrome (FCS). The olezarsen 80 mg monthly dose met the primary endpoint of significantly reducing triglycerides (TGs) in patients with genetically validated FCS at six months. In addition, olezarsen demonstrated robust and sustained reductions in TGs and serum apolipoprotein C-III (apoC-III) levels. Importantly, olezarsen reduced the incidence of acute pancreatitis (AP) events over the 12-month treatment period compared to placebo. Olezarsen also demonstrated a favorable safety and tolerability profile. These results were presented in an oral presentation at the 2024 American College of Cardiology (ACC) Annual Meeting in Atlanta, Georgia and published simultaneously in The New England Journal of Medicine (NEJM). Based on these data, Ionis is pursuing regulatory approval of olezarsen as a potential breakthrough treatment for adults with FCS.

Key Points: 
  • Based on these data, Ionis is pursuing regulatory approval of olezarsen as a potential breakthrough treatment for adults with FCS.
  • In the study, patients were treated with olezarsen 80 mg (n=22), 50 mg (n=21) or placebo (n=23) once every four weeks.
  • Serious AEs occurred in 14% of patients treated with olezarsen 80 mg, 19% treated with olezarsen 50 mg, and 39% treated with placebo.
  • Ionis will host a webcast to discuss the detailed results from the Balance study on Monday, April 8 at 10:00 am ET.