Clinical data showing unprecedented remission rates in newly diagnosed AML patients support advancing ICT01 into pivotal trials July 18, 2025, 11:00 am CET – ImCheck Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead program, ICT01, a humanized anti-butyrophilin 3A (BTN3A) monoclonal antibody designed to selectively activate γ9δ2 T cells, for the treatment of acute myeloid leukemia (AML). AML remains a significant

ALK today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) has approved EURneffy® 2 mg in the United Kingdom (UK) for anaphylaxis treatment of adults and children (≥30 kg). The market launch in the UK is expected within the coming months once market access negotiations are completed. EURneffy® (the trade name for neffy® in the EU and the UK) constitutes the first approved adrenaline nasal spray for timely emergency treatment of allergic reactions (anaphylaxis) and h

The Breakthrough Therapy Designation (BTD) was granted based on the highly compelling results from the Phase 2 EMBOLD trial in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) The EMBOLD cohort 2 pivotal trial is on track for topline results in H1 2026 with NDA filing to follow Praxis has recently initiated the EMERALD study investigating relutrigine broadly in DEEs July 17, 2025 -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company t

Additional complete response (CR) observed in patient with multiple tumors Confirmed overall response rate (ORR) of 50% in patients with combined positive score (CPS) ≥1, including 50% ORR in patients with low CPS (1-19) 12-month overall survival of 88% and median overall survival (mOS) of 32 months July 16, 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

July 16, 2025 -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, announced the publication of “Long-term Efficacy and Safety of Arimoclomol in Niemann-Pick Disease Type C: Final Results of the Phase 2/3 NPC-002 48-month Open-label Extension Trial” in the peer-reviewed journal, Molecular Genetics and Metabolism (https://doi-org.sutd.idm.oclc.org/10.1016/j.ymgme.2025.109189). MIPLYFFA® (arimoclomol) is

July 16, 2025 -- Silo Pharma, Inc. (Nasdaq: SILO) (“Silo” or the “Company”), a developmental stage biopharmaceutical company focused on novel therapeutics and drug delivery systems, today announced completion of dosing in a U.S. Food and Drug Administration (FDA)-requested 7-day safety and toxicology large animal study of its lead asset SPC-15, and bioanalytical and safety/toxicology results for such study are expected within 60 days. “Our novel PTSD drug candidate SPC-15 has already shown st

Sanofi’s SAR446597 earns fast track designation in the US for geographic atrophy due to age-related macular degeneration Designation earned for a one-time intravitreal gene therapy designed to inhibit two key complement cascade pathways Geographic atrophy is an advanced form of dry age-related macular degeneration, that can lead to permanent vision loss July 16, 2025. The US Food and Drug Administration (FDA) has granted fast track designation to SAR446597, a one-time intravitreal gene therapy

Biodexa Pharmaceuticals PLC (“Biodexa” or “the Company”), (Nasdaq: BDRX), a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs, announced the filing of a Clinical Trial Application (CTA) with the European Medicines Agency (EMA) for its Serenta trial in patients with familial adenomatous polyposis (FAP), a mostly inherited disease that, if left untreated, almost always leads to colorectal cancer. The only cu

In June 2025, several breakthrough drugs received their first global approvals and market launches, marking significant advances in therapeutic innovation. 1. Genakumab (Firsekibart) Firsekibart (Genakumab), developed by Changchun Genescience Pharmaceutical, is a fully human monoclonal antibody targeting interleukin-1β (IL-1β). It functions as an IL-1β inhibitor and was approved in China on June 30, 2025, for the treatment of gouty arthritis, especially in patients who are intolerant or unresp

In May 2025, a new wave of capital and technological resonance swept across the global biotechnology sector. Industry giants such as CSPC Pharmaceutical Group, Sanofi, and Eli Lilly accelerated their deployment of next-generation therapies through multibillion-dollar deals. From AI-driven targeted protein degradation (TPD) and RNA editing to in vivo CAR-T therapies, and from oral small-molecule drugs to non-opioid analgesics, the industry is fiercely competing around three central themes: “conqu