Sanofi’s rilzabrutinib earns orphan designation in the EU for IgG4-related disease Data from the positive Phase 2 study evaluating rilzabrutinib for the treatment of IgG4-related disease presented at EULAR 2025 Additional orphan designation underscores Sanofi commitment to advancing new medicines in immune-mediated rare diseases August 14, 2025. The European Medicines Agency has granted orphan designation to rilzabrutinib, a reversible covalent Bruton’s tyrosine kinase (BTK) inhibitor, for IgG4

Aug. 14, 2025 -- 247marketnews.com, a pioneer in digital media dedicated to the swift distribution of financial market news and corporate information, spotlights LIXTE Biotechnology Holdings (Nasdaq: LIXT), a clinical-stage pharmaceutical company focused on developing novel cancer therapies by targeting the epigenetic modulation of key cellular pathways. 2025 is shaping up to be a defining year for LIXTE. With a focus on its lead compound LB-100, a first-in-class small molecule inhibitor of pr

Webinar to Review Results from Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Allocetra™ in Patients with Moderate to Severe Knee Osteoarthritis Aug. 14, 2025 -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the “Company”), a clinical-stage macrophage reprogramming immunotherapy company, today announced it will host a webinar on Monday, August 18, 2025, at 8:00 AM Eastern Time to present and discuss 3-month topline results from the Phase IIa stage of its Phase I/IIa EN

The Phase 1/2 study (up to 141 participants) will evaluate the safety, tolerability and efficacy of GRWD0715 in healthy volunteers and participants with axial spondyloarthritis (axSpA) GRWD0715 is an investigational small molecule designed to interrupt T-cell activation by inhibiting the peptide processing enzyme ERAP1 (Endoplasmic Reticulum Aminopeptidase 1) AxSpA has no cure and affects an estimated 0.5–1.4% of the global population - millions worldwide - leading to chronic inflammatory back p

First patient dosed with CUE-102 for recurrent glioblastoma multiforme (rGBM) at Dana-Farber Cancer Institute (DFCI) Aug. 13, 2025 -- Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively engage and modulate disease-specific T cells for the treatment of autoimmune disease and cancer, announced today the initiation of an investigator sponsored trial (IST) in rGBM at the DFCI with the first patient in the t

Phase 3 results position telitacicept as potential best-in-disease profile in primary Sjögren's disease Telitacicept demonstrated a favorable safety profile Vor evaluating timing of global Phase 3 clinical study in primary Sjögren's disease Data anticipated to be presented at an upcoming medical conference Aug. 13, 2025 -- Vor Bio (Nasdaq: VOR), a clinical-stage biotechnology company transforming the treatment of autoimmune diseases, today announced that its collaborator, RemeGen Co., Ltd (HK

Aug. 13, 2025 -- Creative Medical Technology Holdings, Inc. (NASDAQ: CELZ), a clinical-stage biotechnology company focused on regenerative and immune cellular therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead investigational therapy, CELZ-201-DDT, for the treatment of degenerative disc disease (DDD), a major cause of chronic lower back pain and disability. This designation positions CELZ-201-DDT among a select group of th

At extended median follow-up of 19.7 months, median overall survival (“OS”) increased from 16.33 to 28.94 months Clinical efficacy correlated with the magnitude of T cell responses specific to mutant-KRAS (“mKRAS”) induced by ELI-002 77% reduction in the risk of death and 88% reduction in the risk of relapse, associated with T cell responses above the threshold for anti-tumor efficacy ELI-002 induced both CD4+ and CD8+ mKRAS-specific T cell responses in most patients, and evidence of antigen-spr

In a gold-standard model of obesity, treatment with ARD-201 resulted in approximately 19% body weight reduction after 30 days of treatment. ARD-201 was also associated with attenuated weight regain after discontinuation of tirzepatide. In combination with low-dose tirzepatide, ARD-201 improved weight loss compared to high dose tirzepatide alone. Aardvark plans two separate Phase 2 clinical trials for ARD-201: Phase 2 POWER trial to focus on weight rebound in patients discontinuing GLP-1RA therap

Aug. 12, 2025 -- AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company developing next-generation gene therapies for inherited retinal diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for AAVB-039, the company’s gene therapy program for Stargardt disease secondary to biallelic mutation in ABCA4. The Investigational New Drug (IND) application for AAVB-039 was cleared to proceed by the FDA. Stargardt disease is the most