MRCP

NodThera’s NLRP3 Inhibitor NT-0796 Reverses Neuroinflammation in Parkinson’s Disease Phase Ib/IIa Trial

Retrieved on: 
Thursday, March 7, 2024

IL-1β, IL-6, CCL2, CXCL1 and CXCL8) over 28 days compared to baseline to levels approximating those of healthy elderly controls, demonstrating reversal of NLRP3-mediated neuroinflammation.

Key Points: 
  • IL-1β, IL-6, CCL2, CXCL1 and CXCL8) over 28 days compared to baseline to levels approximating those of healthy elderly controls, demonstrating reversal of NLRP3-mediated neuroinflammation.
  • In addition, reductions in neurodegenerative markers were also observed following oral dosing of NT-0796, including NfL and soluble TREM (sTREM2).
  • The correlation between Parkinson’s disease and neuroinflammation is well-documented, with alpha-synuclein fibrils triggering microglial NLRP3 activation, leading to neuroinflammation and subsequent neurodegeneration.
  • This is the inaugural demonstration of an NLRP3 inhibitor’s potential to not only address Parkinson’s disease but also offer a broader impact on neurodegenerative diseases.

The American Diabetes Association Announces the 2024 National Scientific and Health Care Achievement Award Winners

Retrieved on: 
Wednesday, March 6, 2024

ARLINGTON, Va., March 6, 2024 /PRNewswire/ -- The American Diabetes Association® is pleased to announce this year's National Scientific and Health Care Achievement Award recipients.

Key Points: 
  • ARLINGTON, Va., March 6, 2024 /PRNewswire/ -- The American Diabetes Association® is pleased to announce this year's National Scientific and Health Care Achievement Award recipients.
  • The awards honor academics, health care providers, and educators who have contributed to substantial advances in diabetes care and research.
  • ), FMedSci is the recipient of the 2024 Banting Medal for Scientific Achievement, which recognizes significant long-term contributions to the understanding, treatment, or prevention of diabetes.
  • Parker, PhD is the recipient of the 2024 Outstanding Scientific Achievement Award, which recognizes research in diabetes that demonstrates particular independence of thought and originality.

Mallinckrodt Announces U.S. FDA Approval of Supplemental New Drug Application for Acthar® Gel (repository corticotropin injection) Single-Dose Pre-filled SelfJect™ Injector

Retrieved on: 
Friday, March 1, 2024

DUBLIN, March 1, 2024 /PRNewswire/ -- Mallinckrodt plc, a global specialty pharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has approved Mallinckrodt's supplemental New Drug Application (sNDA) for the Acthar® Gel (repository corticotropin injection) Single-Dose Pre-filled SelfJect™ Injector (herein referred to as "SelfJect"), a new delivery device for Acthar Gel for appropriate patients with a range of chronic and acute inflammatory and autoimmune conditions.2 SelfJect is intended to provide the appropriate subcutaneous dose of Acthar Gel, as prescribed by a healthcare professional, and is designed to help give patients control of their administration.1,3

Key Points: 
  • "We're excited to bring this innovation to U.S. patients with chronic and acute inflammatory and autoimmune conditions.
  • Mallinckrodt also offers a team of field-based experts who provide education for healthcare professionals on the reimbursement process as well as tools available for patients.
  • Acthar Gel will continue to be available as an injection with a vial and syringe.
  • Controlled clinical trials have shown Acthar to be effective in speeding the resolution of acute exacerbations of multiple sclerosis.

Takeda Intends to Rapidly Initiate the First Global Phase 3 Trials of TAK-861, an Oral Orexin Agonist, in Narcolepsy Type 1 in First Half of Fiscal Year 2024

Retrieved on: 
Thursday, February 8, 2024

It is currently classified into two different types: narcolepsy type 1 (NT1) caused by significant loss of orexin neurons with resulting lack of orexin, and narcolepsy type 2 (NT2) where orexin levels are generally normal.

Key Points: 
  • It is currently classified into two different types: narcolepsy type 1 (NT1) caused by significant loss of orexin neurons with resulting lack of orexin, and narcolepsy type 2 (NT2) where orexin levels are generally normal.
  • Stimulating the orexin receptor 2 in NT1 patients targets the underlying pathophysiology of the disease to restore orexin signaling.
  • Based on these results, and in consultation with global health authorities, Takeda plans to initiate global Phase 3 trials of TAK-861 in NT1 rapidly in the first half of its fiscal year 2024.
  • Results from the Phase 2b trials have no impact on the full year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023).

Lisata Therapeutics Announces Publication of Case Report on a Complete Response in a Metastatic Gastroesophageal Adenocarcinoma Patient Treated with LSTA1 in Combination with Standard-of-Care Therapy

Retrieved on: 
Thursday, January 4, 2024

The case report, entitled “LSTA1 Potentiates Complete Response in Metastatic Gastroesophageal Adenocarcinoma,” which was co-authored by Kristen K. Buck, M.D., Executive Vice President of R&D and Chief Medical Officer of Lisata, details a patient with metastatic gastroesophageal adenocarcinoma who achieved a complete response when given LSTA1, Lisata’s lead investigational product, in combination with standard of care (“SoC”) FOLFIRINOX + pembrolizumab.

Key Points: 
  • The case report, entitled “LSTA1 Potentiates Complete Response in Metastatic Gastroesophageal Adenocarcinoma,” which was co-authored by Kristen K. Buck, M.D., Executive Vice President of R&D and Chief Medical Officer of Lisata, details a patient with metastatic gastroesophageal adenocarcinoma who achieved a complete response when given LSTA1, Lisata’s lead investigational product, in combination with standard of care (“SoC”) FOLFIRINOX + pembrolizumab.
  • Upon subsequent addition of LSTA1 to such SoC therapeutic regimen, the subject achieved a complete response, confirmed both radiographically and surgically.
  • As detailed in the case report, LSTA1, together with the SoC therapy, demonstrated tumor targeting and penetration-enhancing capability in this patient.
  • “We are increasingly excited to continue investigating LSTA1’s potential.”
    To read the published case report, please visit https://www.lisata.com/research-technology/publications/lsta1-potentiate...

Immodulon Announces Formation of New Scientific Advisory Board to Support Late-stage Clinical Development of IMM-101

Retrieved on: 
Monday, December 11, 2023

UXBRIDGE, United Kingdom, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Immodulon, a late-stage clinical biotechnology company focused on developing a broad spectrum immunomodulator to improve the lives of patients suffering from difficult-to-treat cancers, today announced the formation of a new Scientific Advisory Board comprising leading global experts in the field of clinical oncology. The new SAB will be chaired by Prof. Dr. med. Thomas Seufferlein, Director, Department of Gastroenterology, University of Ulm, Germany, and will complement the experience of Immodulon’s new management team to support the advancement of IMM-101 into a pivotal trial for pancreatic cancer as well as broader pipeline progress.

Key Points: 
  • UXBRIDGE, United Kingdom, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Immodulon, a late-stage clinical biotechnology company focused on developing a broad spectrum immunomodulator to improve the lives of patients suffering from difficult-to-treat cancers, today announced the formation of a new Scientific Advisory Board comprising leading global experts in the field of clinical oncology.
  • The new SAB will be chaired by Prof. Dr. med.
  • My fellow SAB members and I are looking forward to supporting the Immodulon team in their efforts to develop IMM-101 into an effective treatment for patients with metastatic pancreatic cancer,” said Prof. Dr. Thomas Seufferlein, Chair of Immodulon’s Scientific Advisory Board.
  • Thomas Seufferlein (Chairman), University of Ulm, Ulm, Germany
    Dr. Andrew Gaya, BSc, MB SC, MRCP, FRCR, MD, Guy's and St Thomas' Hospital, London, UK

Novartis receives FDA approval for Fabhalta® (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH

Retrieved on: 
Wednesday, December 6, 2023

Approval was also supported by the Phase III APPOINT-PNH study in complement inhibitor-naïve patients1,3.

Key Points: 
  • Approval was also supported by the Phase III APPOINT-PNH study in complement inhibitor-naïve patients1,3.
  • Patients with sustained hemoglobin level ≥ 12 g/dLa in the absence of transfusions: 67.7% of anti-C5-experienced Fabhalta patients responded vs. 0% for anti-C5 (difference of 66.6%b, P
  • Patients avoiding transfusiond,e: Transfusion avoidance rate 95.2% for anti-C5-experienced Fabhalta patients vs. 45.7% for anti-C5 (difference of 49.5%b, P
  • In APPOINT-PNH, serious ARs were reported in two (5%) patients with PNH receiving Fabhalta, which included COVID-19 and bacterial pneumonia1,3.

Imbria Reports Positive Topline Results for Investigational New Drug Ninerafaxstat in Patients with Cardiac Ischemia and Chronic Stable Angina

Retrieved on: 
Sunday, November 12, 2023

BOSTON, Nov. 12, 2023 (GLOBE NEWSWIRE) -- Imbria Pharmaceuticals, Inc., a clinical stage, cardiometabolic company dedicated to developing innovative therapies designed to improve patient symptoms and function by enhancing cellular energetics, today announced positive topline results from the randomized, double-blind, placebo-controlled Phase 2 IMPROVE-ISCHEMIA trial, evaluating ninerafaxstat, a novel cardiac mitotrope and partial fatty acid oxidation (pFOX) inhibitor, in patients with chronic stable angina.

Key Points: 
  • Treatment with ninerafaxstat was well tolerated and no safety signals were observed.
  • At 8-weeks, treatment with ninerafaxstat demonstrated clinically and statistically significant improvements in dobutamine stress-induced ischemic left ventricular (LV) wall motion abnormalities.
  • This robust anti-ischemic effect was not dependent on changes in myocardial blood flow, consistent with a direct, metabolic mechanism of action of ninerafaxstat targeting the cardiomyocyte.
  • “The randomized, placebo-controlled IMPROVE-ISCHEMIA trial provides detailed insights into the anti-ischemic effects of ninerafaxstat in symptomatic patients with stable angina," said Juhani Knuuti, M.D., Ph.D., Professor and Director of Turku PET Centre in Turku, Finland and chief investigator of the IMPROVE-ISCHEMIA trial.

ORIEN to Present Abstracts at 65th Annual ASH Meeting Utilizing Aster Insights' Avatar Platform

Retrieved on: 
Friday, December 8, 2023

 TAMPA, Fla., Dec. 8, 2023 /PRNewswire/ -- Aster Insights, the leading provider of scientific and clinical intelligence for oncology discovery, today announced upcoming research presentations at the American Society of Hematology's (ASH) 65th Annual Meeting, December 9-12 in San Diego, CA.

Key Points: 
  • TAMPA, Fla., Dec. 8, 2023 /PRNewswire/ -- Aster Insights , the leading provider of scientific and clinical intelligence for oncology discovery, today announced upcoming research presentations at the American Society of Hematology's (ASH) 65th Annual Meeting , December 9-12 in San Diego, CA.
  • Three abstracts will be shared at this year's ASH meeting by Moffitt Cancer Center, which co-founded the Oncology Research Information Exchange Network® (ORIEN) in 2006.
  • The investigations all utilize patient data from Aster Avatar, the best-in-class, deepest multimodal dataset for discovery research in oncology.
  • "ASH is a unique opportunity to demonstrate the impactful discoveries resulting from ORIEN and Aster Insights' discovery solutions," said Anand Shah, MD, Aster Insights CEO.

Novartis receives FDA approval for Fabhalta® (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH

Retrieved on: 
Wednesday, December 6, 2023

Patients with sustained hemoglobin level ≥ 12 g/dLa in the absence of transfusions: 67.7% of anti-C5-experienced Fabhalta patients responded vs. 0% for anti-C5 (difference of 66.6%b, P

Key Points: 
  • Patients with sustained hemoglobin level ≥ 12 g/dLa in the absence of transfusions: 67.7% of anti-C5-experienced Fabhalta patients responded vs. 0% for anti-C5 (difference of 66.6%b, P
  • Patients avoiding transfusiond,e: Transfusion avoidance rate 95.2% for anti-C5-experienced Fabhalta patients vs. 45.7% for anti-C5 (difference of 49.5%b, P
  • In APPLY-PNH, serious ARs were reported in two (3%) patients with PNH receiving Fabhalta, which included pyelonephritis, urinary tract infection and COVID-191,2.
  • In APPOINT-PNH, serious ARs were reported in two (5%) patients with PNH receiving Fabhalta, which included COVID-19 and bacterial pneumonia1,3.