First iadademstat clinical trial in non-malignant hematological indications Aug. 25, 2025 -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, Ticker: ORY), a clinical-stage biopharmaceutical company and a European leader in epigenetics, today announced that the European Medicines Agency (EMA) has approved its Clinical Trial Application (CTA), the European equivalent to an IND, to initiate a Phase Ib trial of iadademstat in sickle cell disease (SCD). This will be the first clinical trial investi

August 26, 2025 Breakthrough Therapy Designation granted to Rina-S for the treatment of adult patients with recurrent or progressive endometrial cancer (EC) who have disease progression on or following prior treatment with a platinum-containing regimen and a PD-(L)1 therapy Regulatory decision supported by data from the Phase 1/2 RAINFOL™-01 trial showing encouraging responses in heavily pretreated EC patientsi Rina-S continues to be evaluated as a single-agent in patients with advanced EC in th

Aug. 25, 2025 -- Arnatar Therapeutics, a biotechnology company pioneering RNA-based therapies for severe and underserved diseases, emerges from stealth today and announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation and Rare Pediatric Disease Designation to ART4, the company’s up-regulating antisense oligonucleotide (ASO) therapeutic candidate for the treatment of Alagille Syndrome (ALGS). “We are excited to emerge from stealth with a strong scientific

Aug. 25, 2025 -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced that the first subjects have been dosed in a Phase 1 trial evaluating SION-451, a first-in-class nucleotide binding domain 1 (NBD1) stabilizer, in proprietary dua

Study met primary endpoint (p-value=0.0068) First global phase 3 study to demonstrate clinically meaningful improvements in disease activity across all three subtypes – MuSK+, LRP4+, triple seronegative Supplemental Biologics License Application (sBLA) to be submitted to U.S. Food and Drug Administration (FDA) by end of 2025 August 25, 2025, 7:00 AM CET – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune

Safety and Pharmacokinetic Data Support Dosing of ART26.12 in Fed or Fasted Conditions Completion of Positive Single Ascending Dose Study with a Preliminary Food Effect Assessment Advances ART26.12 Toward Multiple Ascending Dose Trial Aug. 25, 2025 -- Artelo Biosciences, Inc. (Nasdaq: ARTL), a clinical-stage pharmaceutical company focused on modulating lipid-signaling pathways to develop treatments for people living with cancer, pain, dermatological or neurological conditions, today announce

Median Overall Survival (mOS) of 39.3 months in patients with CPS ≥ 1 – best published result with standard of care pembrolizumab or pembrolizumab + chemotherapy is 17.9 months* VERSATILE-003 Phase 3 trial in progress Aug. 25, 2025 -- PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers, today announced final topline survival data from its VERSATILE-002 Phase 2 c

Aug. 21, 2025 -- Septerna, Inc. (Nasdaq: SEPN), a clinical-stage biotechnology company pioneering a new era of G protein-coupled receptor (GPCR) drug discovery, today announced the dosing of the first participants in its Phase 1 clinical trial of SEP-631, a selective oral small molecule Mas-related G protein-coupled receptor X2 (MRGPRX2) negative allosteric modulator (NAM) being developed for the treatment of chronic spontaneous urticaria (CSU) and other mast cell-driven diseases. The Phase 1 s

Aug. 21, 2025 -- VeonGen Therapeutics, a clinical-stage genetic medicine company developing next-generation gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to VG801, its lead investigational gene therapy for Stargardt disease and other ABCA4 mutation–associated retinal dystrophies. By delivering a full-length, functional ABCA4 gene, VG801 has the potential to address the underlying genetic roo

Aug. 22, 2025 -- BlossomHill Therapeutics, Inc., a privately-held, clinical-stage biotechnology company focused on the design and development of small molecule medicines for treating cancer and autoimmune diseases, today announced the first patient dosed in the expansion cohorts of the SOLARA trial (NCT06706076), the first-in-human trial of BH-30643, a first-in-class, macrocyclic, non-covalent, mutant selective OMNI-EGFRTM inhibitor having sub-nanomolar potency against classical and atypical EG