Organ dysfunction

AdrenoMed Receives FDA Fast Track Designation for Enibarcimab for Treatment of Septic Shock

Retrieved on: 
Wednesday, April 10, 2024
Septic, Infection, Mortality, FDA, Public, CMO, Fast Track, Organ dysfunction, Priority review, Food, Adrenomedullin, Septic shock, Communication, Collection, DPP3, Intensive care medicine, Accelerated approval (FDA), Patient, Death, Biomarker, RCI, Medicine, Sepsis, Pharmaceutical industry

AdrenoMed is now preparing a confirmatory Phase IIb/III clinical trial to confirm the reduced septic shock mortality under enibarcimab treatment employing a precision medicine approach.

Key Points: 
  • AdrenoMed is now preparing a confirmatory Phase IIb/III clinical trial to confirm the reduced septic shock mortality under enibarcimab treatment employing a precision medicine approach.
  • AdrenoMed’s CEO Dr. Richard Jones commented: “We are very pleased that enibarcimab has received Fast Track designation from the FDA, recognizing its potential as an innovative biomarker-guided treatment against septic shock to fill the unmet medical need in this very serious condition with a high death toll.
  • With a mortality rate of 20-30% for sepsis1 and 30%-50% for septic shock in developed countries,2 sepsis represents an enormous public health burden and is responsible for almost 20% of all deaths worldwide.
  • Dr. Stephan Witte, CMO of AdrenoMed, said: “We are very confident that the use of enibarcimab in combination with two biomarkers, Adrenomedullin (bio-ADM) and circulating dipeptidyl peptidase 3 (cDPP3), holds the promise to become the first effective targeted treatment against septic shock.

EQS-News: AdrenoMed Receives FDA Fast Track Designation for Enibarcimab for Treatment of Septic Shock

Retrieved on: 
Wednesday, April 10, 2024
Sepsis, Communication, Patient, Public, Fast Track, Adrenomedullin, Biomarker, Accelerated approval (FDA), Mortality, Septic shock, Death, DPP3, Septic, Medicine, Priority review, Organ dysfunction, Intensive care medicine, CMO, Collection, FDA, Infection, RCI, Food, Pharmaceutical industry

AdrenoMed is now preparing a confirmatory Phase IIb/III clinical trial to confirm the reduced septic shock mortality under enibarcimab treatment employing a precision medicine approach.

Key Points: 
  • AdrenoMed is now preparing a confirmatory Phase IIb/III clinical trial to confirm the reduced septic shock mortality under enibarcimab treatment employing a precision medicine approach.
  • AdrenoMed’s CEO Dr. Richard Jones commented: “We are very pleased that enibarcimab has received Fast Track designation from the FDA, recognizing its potential as an innovative biomarker-guided treatment against septic shock to fill the unmet medical need in this very serious condition with a high death toll.
  • With a mortality rate of 20-30% for sepsis [1] and 30%-50% for septic shock in developed countries, [2] sepsis represents an enormous public health burden and is responsible for almost 20% of all deaths worldwide.
  • Dr. Stephan Witte, CMO of AdrenoMed, said: “We are very confident that the use of enibarcimab in combination with two biomarkers, Adrenomedullin (bio-ADM) and circulating dipeptidyl peptidase 3 (cDPP3), holds the promise to become the first effective targeted treatment against septic shock.

ACTG CROI Presentations Show That Semaglutide Improves Metabolic-Associated Steatotic Liver Disease Among People Living With HIV

Retrieved on: 
Tuesday, March 5, 2024
Infection, Liver, Bangladesh Technical Education Board, Metabolism, Woman, P.T, Fungal infection, NIAID, Prediabetes, CROI, Liver injury, Inflammation, Quality of life, ACTG, ALT, Weight loss, Waist, Weight, Organ dysfunction, University of Southern California, Conference, Health, Glucagon-like peptide-1 receptor, University, SLIM, ART, Triglyceride, BMI, UM1, Insulin resistance, HIV, Non-alcoholic fatty liver disease, Research, University of North Carolina, Dietary supplement

Yesterday, the SLIM LIVER poster “Effects of Semaglutide on Muscle Structure and Function in the SLIM LIVER study” was presented.

Key Points: 
  • Yesterday, the SLIM LIVER poster “Effects of Semaglutide on Muscle Structure and Function in the SLIM LIVER study” was presented.
  • Together, these presentations demonstrate that semaglutide was highly effective in improving, and in some cases, resolving completely, metabolic dysfunction-associated steatotic liver disease (MASLD, formerly known as non-alcoholic fatty liver disease) among people living with HIV.
  • SLIM LIVER is the first study evaluating semaglutide as a treatment for MASLD among people living with HIV.
  • MASLD is common among people living with HIV and likely acts synergistically with HIV to accelerate liver injury and organ dysfunction.

Prothena Reports Fourth Quarter and Full Year 2023 Financial Results, and Provides Financial Guidance and Business Highlights

Retrieved on: 
Thursday, February 15, 2024
Research, FDA, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, Oncology, Therapy, Fast Track, Acquisition, SAD, VITAL, Roche, American Society of Hematology, ASH, Risk, Food, Epitope, AAIC, ID, Novo Nordisk, Exercise, Organ dysfunction, Amyloidosis, Amyloid, Conference, Bristol Myers Squibb, BMS, Society, TTR, European Medicines Agency, Patient, Clinical trial, ATTR, IND, Orphan, Mortality, Failure, Pharmaceutical industry

In addition, the Company provided 2024 financial guidance and business highlights.

Key Points: 
  • In addition, the Company provided 2024 financial guidance and business highlights.
  • “2023 was a year of strong progress for Prothena as we advanced our protein dysregulation portfolio and moved closer to becoming a fully integrated commercial company.
  • Total revenue for the fourth quarter and full year of 2023 included BMS collaboration revenue of $0.3 million and $91.3 million, respectively.
  • As of December 31, 2023, Prothena had $621.0 million in cash, cash equivalents and restricted cash, and no debt.

AnaMar Announces US and EU Orphan Drug Designation for AM1476 for Treating Systemic Sclerosis

Retrieved on: 
Monday, February 5, 2024
Inflammation, Systemic scleroderma, Biomarker, Patient, Diagnosis, FDA, EMA, Marketing, Scleroderma, Interstitial lung disease, Skin, Pulmonary fibrosis, Disability, Disease, Fibrosis, European Medicines Agency, Death, ODD, Autoimmune disease, ILD, Organ dysfunction, Pharmaceutical industry

AM1476 offers a unique dual-action approach to treat both skin and lung manifestations of systemic sclerosis.

Key Points: 
  • AM1476 offers a unique dual-action approach to treat both skin and lung manifestations of systemic sclerosis.
  • Systemic sclerosis is a chronic, progressive, autoimmune disease characterized by inflammation and fibrosis, i.e.
  • AnaMar's Chief Executive Officer, Dr. Ulf Ljungberg, said: “We are delighted with the FDA’s and EMA’s decisions to grant orphan drug designation to AM1476 for SSc.
  • Orphan drug designation provides certain benefits, including the potential for extensive marketing exclusivity following regulatory approval, reduction in regulatory fees and, in the case of EU, a centralized approval process.

Groundbreaking Randomized Controlled Trial Reports Excellent Clinical Outcomes Using CytoSorb® Intraoperatively in Heart Transplant Patients

Retrieved on: 
Wednesday, January 17, 2024
Mortality, Death, Patient, Incidence, ICU, Cytokine adsorbing column, Vasoplegic syndrome, Semmelweis University, Professional corporation, DRS, European Society of Cardiology, RCT, Heart, Standard of care, Pelvic floor dysfunction, Cytosorbents Corporation, Circulatory system, Risk, Organ dysfunction, VS, Multiple organ dysfunction syndrome, Observational study, Shock, Randomized controlled trial, ESC, Dentistry

In particular, vasoplegic syndrome (VS), a form of circulatory failure or shock, is routinely seen in these patients and can cause worsened organ failure.

Key Points: 
  • In particular, vasoplegic syndrome (VS), a form of circulatory failure or shock, is routinely seen in these patients and can cause worsened organ failure.
  • In a prior observational study , CytoSorb was associated with the reduced severity of vasoplegia in heart transplant patients, in addition to other clinical benefits.
  • In this prospective, single-center, open-label RCT, 60 heart transplant recipients were randomly assigned to either receive intraoperative CytoSorb hemoadsorption or standard of care.
  • Now, Nemeth, Soltesz, and colleagues have confirmed many of these observations in this rigorous and well-designed groundbreaking randomized controlled trial in orthotopic heart transplant patients.

Prothena Provides Updates on PRX012, PRX123, Birtamimab and Portfolio Programs

Retrieved on: 
Monday, January 8, 2024
Biotechnology, Health, Clinical Trials, Biology, Failure, Food, Risk, Hand, FDA, Acquisition, Amyloidosis, Roche, Mortality, Organ dysfunction, Orphan, ATTR, VITAL, Novo Nordisk, Epitope, PD, European Medicines Agency, Fast track (FDA), TTR, Amyloid, Alpha-synuclein, Patient, Bristol Myers Squibb, Therapy, Vaccine

Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, provided a business update on portfolio programs.

Key Points: 
  • Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, provided a business update on portfolio programs.
  • The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for PRX012 for the treatment of AD.
  • Update on Financial Position for December 31, 2023
    At December 31, 2023, Prothena had cash, cash equivalents, and restricted cash of approximately $621 million.
  • Cash on hand provides sufficient capital which takes Prothena beyond the completion of its ongoing clinical trials.

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

Retrieved on: 
Monday, January 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.

Ayala Pharmaceuticals Announces AL102 Receives Orphan Drug Designation for Desmoid Tumors

Retrieved on: 
Monday, November 6, 2023
Aggressive fibromatosis, FDA, Organ dysfunction, Incidence, Food, Chronic pain, OTCQX, DT, Head, Plantar fibromatosis, Quality of life, Abdominal wall, Medicine, Patient, Food and Drug Administration, Diagnosis, GSI, Plasma protein binding, Pharmaceutical industry

REHOVOT, Israel and MONMOUTH JUNCTION, N.J., Nov. 06, 2023 (GLOBE NEWSWIRE) -- Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AL102, a Gamma Secretase Inhibitor (GSI), for the treatment of desmoid tumors (DT).

Key Points: 
  • REHOVOT, Israel and MONMOUTH JUNCTION, N.J., Nov. 06, 2023 (GLOBE NEWSWIRE) -- Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AL102, a Gamma Secretase Inhibitor (GSI), for the treatment of desmoid tumors (DT).
  • Orphan drug designation is granted by the FDA to drugs and biologics intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. at the time of designation.
  • Under the FDA’s Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity following drug approval.
  • “Receiving FDA orphan drug status for AL102 underscores the significant unmet need for novel treatment options for people living with desmoid tumors.

Prothena Reports Third Quarter 2023 Financial Results and Business Highlights

Retrieved on: 
Thursday, November 2, 2023
Biotechnology, Pharmaceutical, Health, Amyloid, Survival, FDA, Bristol Myers Squibb, Research, Organ dysfunction, AD, IND, PD, Food, VITAL, ATTR, Therapy, Fast track (FDA), AL, Mortality, G&A, Novo Nordisk, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Acquisition, European Medicines Agency, American Society of Hematology, MAD, SAD, Orphan, R, TTR, ASH, Clinical trial, Biology, Risk, Alpha-synuclein, Epitope, Amyloidosis, Society, SPA, Patient, Failure, Pharmaceutical industry, Vaccine, Fine chemical, Security (finance), Blood

Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, today reported financial results for the third quarter and first nine months of 2023 and provided business highlights.

Key Points: 
  • Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, today reported financial results for the third quarter and first nine months of 2023 and provided business highlights.
  • Robust reduction of brain amyloid plaque has been demonstrated to likely predict clinical benefit for people with early AD.
  • The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for PRX012 for the treatment of AD.
  • As of September 30, 2023, Prothena had $673.1 million in cash, cash equivalents and restricted cash, and no debt.