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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.

New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy

Retrieved on: 
Wednesday, March 6, 2024

* The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA over a 2-year period in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Zolgensma® (onasemnogene abeparvovec).

Key Points: 
  • * The Phase 4 study evaluates clinical outcomes and safety following treatment with SPINRAZA over a 2-year period in infants and toddlers with spinal muscular atrophy (SMA) who have unmet clinical needs after treatment with Zolgensma® (onasemnogene abeparvovec).
  • The new data show that plasma neurofilament light chain (NfL) levels, an objective biomarker of axonal injury and neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA.
  • These data will be presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 3-6, 2024).
  • “Our evolving understanding of gene therapy indicates there may be an opportunity for better outcomes,” said Crystal Proud, M.D., Pediatric Neurologist at Children’s Hospital of the King’s Daughters.

Lantheus Announces the FDA Approval of DEFINITY® (Perflutren Lipid Microsphere) for Pediatric Patients

Retrieved on: 
Monday, March 4, 2024

This approval represents a significant step forward in pediatric medicine, providing healthcare professionals with a valuable tool to opacify the left ventricular chamber and better identify the left ventricular endocardial border.

Key Points: 
  • This approval represents a significant step forward in pediatric medicine, providing healthcare professionals with a valuable tool to opacify the left ventricular chamber and better identify the left ventricular endocardial border.
  • Currently, DEFINITY is the most utilized, extensively studied, and a trusted diagnostic ultrasound enhancing agent in the U.S.1-3
    “The expanded indication for DEFINITY is a testament to the rigorous research and development efforts undertaken by Lantheus to address unmet medical needs of pediatric patients,” said Jean-Claude Provost, MD, Chief Medical Officer.
  • “We are proud that this approval will extend the benefits of DEFINITY to healthcare professionals who work with these young individuals.”
    Clinical studies have substantiated the efficacy and safety of DEFINITY in pediatric patients.
  • The Kutty and Fine studies each reported successful left ventricular opacification in all participants.

SpringWorks Therapeutics Initiates Rolling Submission of New Drug Application to the FDA for Mirdametinib for the Treatment of Children and Adults with NF1-PN

Retrieved on: 
Monday, March 4, 2024

Mirdametinib treatment showed deep and durable responses and demonstrated significant improvements in key secondary patient-reported outcome measures.

Key Points: 
  • Mirdametinib treatment showed deep and durable responses and demonstrated significant improvements in key secondary patient-reported outcome measures.
  • The FDA and the European Commission have granted Orphan Drug designation for mirdametinib for the treatment of NF1.
  • In July 2023, the FDA granted mirdametinib Rare Pediatric Disease designation for the treatment of NF1, which provides eligibility for a priority review voucher upon FDA approval.
  • SpringWorks expects to complete the NDA submission in the second quarter of 2024.

SELLAS Life Sciences Delivers Oral Presentation of SLS009 Phase 1 Data for Acute Myeloid Leukemia Patients at 2024 European School of Haematology (ESH) Conference

Retrieved on: 
Friday, March 1, 2024

NEW YORK, March 01, 2024 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS” or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced the delivery of an oral presentation of data for the cohort of patients with acute myeloid leukemia (AML) from the Phase 1 dose-escalation study of SLS009 (formerly GFH009) by Dr. Tapan Kadia, Professor at MD Anderson Cancer Center and the study’s primary investigator, at the 2024 European School of Haematology Acute Leukaemias (ESH) Conference: How to Diagnose and Treat Acute Leukaemias, taking place March 1-3, 2024, in Stockholm, Sweden.

Key Points: 
  • Positive topline data for the heavily pretreated AML patients showed evidence of anti-tumor activity increasing with higher dose levels and no significant safety issues.
  • The CR lasted eight months with the patient achieving one year survival at the latest assessment.
  • Strong inhibitory activity against key biomarkers with a dose-proportional response and universal decrease of MYC and MCL-1 in evaluable patients.
  • “Its potential strong synergy with the standard regimen of venetoclax and hypomethylating agents could open up new avenues in the treatment of acute myeloid leukemia.

Sensei Biotherapeutics Reports Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Wednesday, February 28, 2024

BOSTON, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), a clinical stage immuno-oncology company focused on the discovery and development of next-generation therapeutics for cancer patients, today reported financial results for the full year 2023, and provided corporate updates.

Key Points: 
  • “2023 saw the entry of our differentiated anti-VISTA antibody, SNS-101, into clinical development,” said John Celebi, President and Chief Executive Officer.
  • Recent updates include:
    A total of 33 patients have been treated with SNS-101 +/- Libtayo in the dose escalation phase of this study.
  • On October 23, 2023 , Sensei presented a trial-in-progress poster from the Phase 1/2 clinical trial for SNS-101 at the European Society for Medical Oncology Congress (ESMO) 2023.
  • On November 1, 2023, Sensei announced the appointment of Stephanie Krebs, MS, MBA, as Chief Business Officer.

Purple Biotech Presents Data of its Phase 1 Head & Neck Cancer of NT219 in combination with Cetuximab at ESMO TAT Congress 2024

Retrieved on: 
Tuesday, February 27, 2024

REHOVOT, Israel, Feb. 27, 2024 (GLOBE NEWSWIRE) --   Purple Biotech Ltd. ("Purple Biotech" or "the Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that harness the power of the tumor microenvironment to overcome tumor immune evasion and drug resistance, today announced clinical results from its Phase 1/2 dose escalation study of NT219 in combination with cetuximab in the treatment of patients with recurrent/metastatic head and neck cancer (R/M SCCHN).

Key Points: 
  • The Phase 1/2 dose escalation study (NCT04474470) evaluated NT219 as a monotherapy in various indications and in combination with cetuximab in the treatment of R/M SCCHN and colorectal cancer.
  • As of cut-off date of January 25, 2024:
    Seventeen patients with R/M SCCHN were enrolled in the combination arm of NT219 + cetuximab.
  • The median number of prior lines of therapy was 2 and 94% of the patients received prior immunotherapy.
  • “We were encouraged to see anti-tumor activity in HPV negative patients,” said Dr. Michael Schickler, Purple Biotech’s Head of Clinical and Regulatory Affairs.

SpringWorks Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Business Updates

Retrieved on: 
Tuesday, February 27, 2024

Launched OGSIVEO in the U.S. and achieved net product revenue of $5.4 million in the first partial quarter of the launch.

Key Points: 
  • Launched OGSIVEO in the U.S. and achieved net product revenue of $5.4 million in the first partial quarter of the launch.
  • Presented additional patient-reported outcome data from the Phase 3 DeFi trial at the 2023 Connective Tissue Oncology Society Annual Meeting.
  • Revenues: OGSIVEO net product revenues were $5.4 million in the fourth quarter of 2023, the first partial quarter of the U.S. launch.
  • Cash Position: Cash, cash equivalents and marketable securities were $662.6 million as of December 31, 2023.

ORYZON Reports Financial Results and Corporate Update for Quarter Ended December 31, 2023

Retrieved on: 
Monday, February 26, 2024

Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the fourth quarter.

Key Points: 
  • Dr Carlos Buesa, Oryzon’s Chief Executive Officer, said: “Oryzon continued with a strong path in its clinical programs in the fourth quarter.
  • Research and development (R&D) expenses were $3.9 and $16.6 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $5.0 and $18.1 million for the quarter and twelve months ended December 31, 2022.
  • General and administrative expenses were $1.2 and $4.2 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $1.2 and $4.8 million for the quarter and twelve months ended December 31, 2022.
  • Net losses were $1.4 and $5.0 million for the quarter and twelve months ended December 31, 2023, respectively, compared to $1.6 and $5.9 million for the quarter and twelve months ended December 31, 2022.

PolarityTE Announces First Subject Screened in Phase III Pivotal Study Evaluating Investigational New Drug SkinTE® in Diabetic Foot Ulcers

Retrieved on: 
Monday, February 26, 2024

Subjects will be randomized to one of two treatment groups, receiving either SkinTE plus the standard of care (SOC) or the SOC alone.

Key Points: 
  • Subjects will be randomized to one of two treatment groups, receiving either SkinTE plus the standard of care (SOC) or the SOC alone.
  • The primary endpoint is the incidence of DFUs closed at 12 weeks.
  • COVER DFUS II is a pivotal study that PolarityTE will conduct under its open IND for SkinTE.
  • We are excited to evaluate how the optimized healthy cells from the patient in SkinTE can facilitate closure in these difficult-to-treat ulcers.