Hemolysis

Agios Presents Positive Results from Phase 3 ENERGIZE Study of Mitapivat in Non-Transfusion-Dependent Thalassemia in Plenary Session at the European Hematology Association 2024 Hybrid Congress

Retrieved on: 
土曜日, 6月 15, 2024
Head, American University, Iron overload, Patient, Fatigue, Agios Pharmaceuticals, Thalassemia, NTD, Disease, University of Toronto, AGIO, Congress, Hemolysis, University, Master of Science, S104, Pyruvate kinase, Central European, Anemia, American University of Beirut, Arm, Quality of life, BID, FRCPC, Medical device

CAMBRIDGE, Mass., June 15, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today presented detailed results from the global Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia in a plenary session (abstract #S104) at the European Hematology Association 2024 (EHA2024) Hybrid Congress, which is being held June 13-16, 2024, in Madrid, Spain. In a related poster presentation (abstract #P1529), the company presented additional data from the ENERGIZE study highlighting clinically meaningful improvements in health-related quality of life measures and patient-reported outcomes among patients in the mitapivat arm compared to those in the placebo arm.

Key Points: 
  • The ENERGIZE study achieved its primary endpoint, with mitapivat demonstrating a statistically significant increase in hemoglobin response rate compared to placebo.
  • 122 (93.8%) in the mitapivat arm and 62 (96.9%) in the placebo arm completed the 24-week double-blind period of the study.
  • Baseline demographics and characteristics were balanced between mitapivat and placebo arms, and representative of a population of non-transfusion dependent thalassemia patients.
  • Agios will host a virtual investor breakout session tomorrow, June 16, 2024, at 10:00 a.m.

Editas Medicine Reports New Safety and Efficacy Data from the RUBY Trial of Reni-cel in 18 Patients with Sickle Cell Disease, Presented at the European Hematology Association (EHA) Annual Congress

Retrieved on: 
金曜日, 6月 14, 2024
SCD, Adolescence, Division, Patient, EHA, HBG1, Editas Medicine, Șaeș, HB, Livestreaming, European Hematology Association, Caregiver, Clinical trial, Safety, Trial of the century, Sickle cell disease, Hemolysis, EDT, Busulfan, Fetal hemoglobin, Blood, Cleveland Clinic, HBF

CAMBRIDGE, Mass., June 14, 2024 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced new safety and efficacy data in 18 patients living with sickle cell disease (SCD) treated with renizgamglogene autogedtemcel (reni-cel; formerly known as EDIT-301) in the Phase 1/2/3 RUBY clinical trial. Reni-cel, the first investigational AsCas12a gene-edited cell therapy medicine, is being studied in the RUBY trial as a potential one-time, durable medicine for people living with severe SCD. The data will be presented in an oral presentation at the European Hematology Association (EHA) Hybrid Congress in Madrid, Spain and via livestream, on Saturday, June 15 at 11:30 a.m. CEST (5:30 a.m. EDT).

Key Points: 
  • Reni-cel, the first investigational AsCas12a gene-edited cell therapy medicine, is being studied in the RUBY trial as a potential one-time, durable medicine for people living with severe SCD.
  • Since treatment with reni-cel, patients have been free of vaso-occlusive events (VOEs) (N=18) for up to 22.8 months of follow-up.
  • Patients in the RUBY trial underwent a median of 2.0 apheresis and mobilization cycles (min: 1.0, max: 4.0).
  • All patients in the RUBY trial showed sustained high levels of editing in the HBG1 and HBG2 promoter regions.

Samsung Bioepis Presents Post-hoc Analysis of Phase 3 Study for EPYSQLI™ (SB12; Eculizumab Biosimilar), at the European Hematology Association (EHA) Congress 2024

Retrieved on: 
金曜日, 6月 14, 2024
TS2, PNH, Eculizumab, Patient, Pivotal, ECU, Week, European Commission, Biosimilar, Trial of the century, Civil service commission, Physician, RBC, Period, Hemolysis, Anemia, Chelsea Handler, TS1, Medicine, Pharmaceutical industry

The post-hoc analysis of SB12 pivotal Phase 3 study compared the proportion of patients remaining transfusion-free (“transfusion avoidance”) among those treated with SB12 or reference eculizumab (ECU), respectively.

Key Points: 
  • The post-hoc analysis of SB12 pivotal Phase 3 study compared the proportion of patients remaining transfusion-free (“transfusion avoidance”) among those treated with SB12 or reference eculizumab (ECU), respectively.
  • At Week 26, patients were switched to ECU or SB12, respectively, and received infusion until Week 50 (i.e., Period 1 followed by Period 2 from Week 26).
  • This efficacy analysis was conducted in pooled patients treated with SB12 or ECU in either Periods 1 or 2.
  • In March 2024, EPYSQLI was granted an indication expansion for the treatment of adults and children with atypical haemolytic uremic syndrome (aHUS).3

Samsung Bioepis to Present Post-hoc Analysis of Phase 3 Study for EPYSQLI™ (SB12; Eculizumab Biosimilar), at the European Hematology Association (EHA) Congress 2024

Retrieved on: 
木曜日, 5月 30, 2024
PNH, Patient, Pharmacokinetics, Anemia, Hope, Biosimilar, ECU, Trial of the century, Safety, Chelsea Handler, LDH, Eculizumab, European Commission, Hemolysis, Medicine, RBC, Pivotal, Pharmaceutical industry

INCHEON, Korea, May 30, 2024 (GLOBE NEWSWIRE) -- Samsung Bioepis Co., Ltd. today announced that a post-hoc analysis of the Phase 3 clinical study results for EPYSQLI™ (SB12), a biosimilar to Soliris1 (eculizumab), will be presented at the 29th European Hematology Association 2024 (EHA2024) Hybrid Congress held in Madrid, Spain and virtually, from June 13 to 16, 2024.

Key Points: 
  • INCHEON, Korea, May 30, 2024 (GLOBE NEWSWIRE) -- Samsung Bioepis Co., Ltd. today announced that a post-hoc analysis of the Phase 3 clinical study results for EPYSQLI™ (SB12), a biosimilar to Soliris1 (eculizumab), will be presented at the 29th European Hematology Association 2024 (EHA2024) Hybrid Congress held in Madrid, Spain and virtually, from June 13 to 16, 2024.
  • SB12 has shown equivalent efficacy with reference eculizumab (ECU) by lactate dehydrogenase (LDH) and comparable safety, pharmacokinetics, pharmacodynamics, and immunogenicity profiles in paroxysmal nocturnal hemoglobinuria (PNH) through the pivotal Phase 3 study.2
    The post-hoc analysis of SB12 pivotal Phase 3 study presented at EHA2024 compared the proportion of patients remaining transfusion-free (“transfusion avoidance”) among those treated with SB12 or ECU, respectively.
  • Eculizumab is known to significantly reduce hemolysis resulting in improvements in anemia as indicated by increased hemoglobin stabilization and reduced need for red blood cell (RBC) transfusions.3
    “We are excited to present this data at EHA2024, that adds to the growing body of evidence that supports comparable clinical efficacy of SB12 with reference eculizumab in treating PNH patients,” said Hyejin Kim, Vice President and Medical & Lifecycle Safety Team Leader at Samsung Bioepis.
  • In March 2024, EPYSQLI was granted an indication expansion for the treatment of adults and children with atypical haemolytic uremic syndrome (aHUS).4

Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Retrieved on: 
金曜日, 5月 10, 2024
Research, Genetics, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, Oncology, PKD, Fanconi anemia, Patient, Cell, Survival, HSCT, FA, Disorder, Marketing, Food, European Medicines Agency, FDA, Medication, Safety, BMF, Bone marrow, PDUFA, Infection, Hospital, Gene, EMA, Hemolysis, Biologics license application, BLA, Society, MAA, Busulfan, Pyruvate kinase deficiency, Graft-versus-host disease, Quality of life, Pharmaceutical industry

“The positive updates presented at this year’s annual meeting demonstrate the sustained safety and efficacy across the totality of our LV hematology portfolio,” said Jonathan Schwartz, M.D., Chief Medical & Gene Therapy Officer, Rocket Pharmaceuticals.

Key Points: 
  • “The positive updates presented at this year’s annual meeting demonstrate the sustained safety and efficacy across the totality of our LV hematology portfolio,” said Jonathan Schwartz, M.D., Chief Medical & Gene Therapy Officer, Rocket Pharmaceuticals.
  • In our pivotal studies of RP-L102 for Fanconi Anemia, we continue to see maintained genetic and phenotypic correction combined with hematologic stabilization.
  • Additionally, our Phase 1 study of RP-L301 for PKD shows sustained clinically meaningful hemoglobin improvement in all patients.
  • The oral presentation includes positive, updated data (cut-off September 11, 2023) from the global Phase 1/2 pivotal studies of RP-L102, Rocket’s ex vivo LV gene therapy candidate for FA.

The United States FDA Awards Orphan Drug Designation (ODD) to NM5072 for Treating Paroxysmal Nocturnal Hemoglobinuria (PNH)

Retrieved on: 
月曜日, 4月 15, 2024
FDA, Pain, Mortality, Nephrology, Inflammation, Monocyte, Cell, Anemia, Alternative complement pathway, Safety, Ophthalmology, Red blood cell, Food, Hematology, Chelsea Handler, Dermatology, Half-life, Atypical hemolytic uremic syndrome, Blood, AP, Hyperlipidemia, Hemoglobinuria, Disease, Patient, Neurology, Fatigue, Platelet, Diagnosis, Infection, Hemolysis, Immune system, PNH, ODD, Quality of life, Chronic pain, LDH, Paroxysmal nocturnal hemoglobinuria, Pharmaceutical industry

The United States Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NM5072 for the treatment of anemia in PNH patients.

Key Points: 
  • The United States Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NM5072 for the treatment of anemia in PNH patients.
  • CLEVELAND, April 15, 2024 (GLOBE NEWSWIRE) -- NovelMed today announced that the Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to NM5072, an Alternative Pathway (AP) blocker anti-Properdin antibody, for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
  • NM5072 is another drug in our pipeline that has received Orphan Drug Designation in the United States.
  • Among these, Paroxysmal Nocturnal Hemoglobinuria (PNH) emerges as a prominent indication for complement blockers heading towards FDA approval.

BrightSpring Health Services Specialty Pharmacy Onco360® Selected as National Pharmacy Partner for Voydeya®

Retrieved on: 
水曜日, 4月 10, 2024
FDA, Cancer, Pharmacy, Hub, Blood, Eculizumab, Ravulizumab, Insurance, Alexion Pharmaceuticals, Disease, EVH, Patient, Haematopoietic system, Hemolysis, Alexion, Health care, Immune system, PNH, Quality of life, Anemia, Pharmaceutical industry

LOUISVILLE, Ky., April 10, 2024 (GLOBE NEWSWIRE) -- BrightSpring Health Services (“BrightSpring” or “BrightSpring Health Services”) (NASDAQ: BTSG) announced today that its company Onco360®, a leading independent oncology pharmacy, was selected as a national pharmacy partner by Alexion Pharmaceuticals for a first-in-class, oral, Factor D inhibitor, Voydeya® (Danicopan) to treat patients with a rare, life-threatening disease of the blood called paroxysmal nocturnal hemoglobinuria (PNH) who are experiencing extravascular hemolysis (EVH).

Key Points: 
  • LOUISVILLE, Ky., April 10, 2024 (GLOBE NEWSWIRE) -- BrightSpring Health Services (“BrightSpring” or “BrightSpring Health Services”) (NASDAQ: BTSG) announced today that its company Onco360®, a leading independent oncology pharmacy, was selected as a national pharmacy partner by Alexion Pharmaceuticals for a first-in-class, oral, Factor D inhibitor, Voydeya® (Danicopan) to treat patients with a rare, life-threatening disease of the blood called paroxysmal nocturnal hemoglobinuria (PNH) who are experiencing extravascular hemolysis (EVH).
  • Voydeya® is FDA-approved as add-on therapy to Ultomiris® (Ravulizumab) or Soliris® (Eculizumab) for patients living with PNH that experience continued anemia, or EVH.
  • This innovative drug will complement current treatment regimens and help regulate the immune system, providing control and preventing the destruction of healthy blood cells.
  • To learn more about Onco360® and BrightSpring, please visit https://www.brightspringhealth.com/ .

Omeros Corporation Reports Fourth Quarter and Year-End 2023 Financial Results

Retrieved on: 
月曜日, 4月 1, 2024
Mental Health, Research, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, Science, Oncology, PIN, PNH, Patient, Sale, CUD, Graft-versus-host disease, Investment, Clinical trial, Hemolysis, Calcineurin, Emory University, ASH, Risk, L-DOPA, Thumbay Hospital, Behavior informatics, Trial of the century, Myelodysplastic syndrome, Cancer, Safety, Transplant, LID, DRI, BLA, Corporation, Paroxysmal nocturnal hemoglobinuria, Tardive dyskinesia, Parkinson's disease, Publication, DSM-IV codes, Retirement, FDA, PDE7, LDH, Hematology, Mortality, Hematopoietic stem cell transplantation, Bone marrow failure, Abstract, Society, Pharmacology, Pharmaceutical industry

Net loss from continuing operations was $39.3 million for the 2023 fourth quarter compared to $51.7 million for the prior quarter.

Key Points: 
  • Net loss from continuing operations was $39.3 million for the 2023 fourth quarter compared to $51.7 million for the prior quarter.
  • Total operating expenses for the fourth quarter of 2023 were $39.8 million compared to $48.2 million for the third quarter of 2023.
  • Interest expense during the fourth quarter of 2023 was $7.1 million compared to $7.9 million during the prior quarter.
  • During the fourth quarter of 2023, we earned $3.4 million in interest and other income compared to $4.4 million in the third quarter.

Inspira™ Technologies Reports Full Year 2023 Financial Results

Retrieved on: 
月曜日, 3月 25, 2024
ART, OXY, Research, Annual report, Unitary state, IIA, Intubation, Risk, Patent, Patient, Coma, S&M, Hospital, G&A, Coagulation, Insurance, D&O, Carbon, Human, Hemolysis, Marketing, FDA, Total, Food, Pharmaceutical industry

RA'ANANA, Israel, March 25, 2024 /PRNewswire/ -- Inspira Technologies OXY B.H.N. Ltd. (Nasdaq: IINN) (Nasdaq: IINNW) (the "Company" or "Inspira Technologies"), a breakthrough medical technology company, today announced its financial results for the year ended December 31, 2023.

Key Points: 
  • Ltd. (Nasdaq: IINN) (Nasdaq: IINNW) (the "Company" or "Inspira Technologies"), a breakthrough medical technology company, today announced its financial results for the year ended December 31, 2023.
  • With our ground-breaking technologies and products, we are not just looking at the horizon but actively shaping it, leveraging our proprietary technologies to make a meaningful impact."
  • Total operating expenses decreased to US$12,133,000 in of the year ended December 31, 2023, from US$14,893,000 in the same period of 2022.
  • As of December 31, 2023, financial liabilities at fair value totaled US$1,470,000 compared to US$26,000.

Extracorporeal Membrane Oxygenation Machine Market Projected to Reach $1,085.63 million by 2030 - Exclusive Report by 360iResearch

Retrieved on: 
水曜日, 3月 13, 2024
Temperature, Component, Growth, LivaNova, Extracorporeal membrane oxygenation, Getinge Group, Intelligence, Fresenius Medical Care, Incidence, COVID-19, Patient, Pulmonary hypertension, Research, Cannula, Meconium aspiration syndrome, Carbon, ECMO, Heart, Arteriovenous, Periodic breathing, Workforce, Blood, Hemolysis, Medtronic, Nursing care plan, PPHN, Table, Ageing, Medical device

The extracorporeal membrane oxygenation (ECMO) machine, a crucial medical device, offers extended cardiac and respiratory support to patients with insufficient gas exchange capabilities, marking a significant advancement in critical care.

Key Points: 
  • The extracorporeal membrane oxygenation (ECMO) machine, a crucial medical device, offers extended cardiac and respiratory support to patients with insufficient gas exchange capabilities, marking a significant advancement in critical care.
  • Furthermore, market prospects include integrating ECMO with other extracorporeal treatments and concentrated research efforts to expand usage indications.
  • We proudly unveil ThinkMi, a cutting-edge AI product designed to transform how businesses interact with the Extracorporeal Membrane Oxygenation Machine Market.
  • "Dive into the Extracorporeal Membrane Oxygenation Machine Market Landscape: Explore 196 Pages of Insights, 474 Tables, and 26 Figures"