AUSTIN, Texas — IntraBio Inc., a biopharmaceutical company focused on developing therapies for rare neurological diseases, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of AQNEURSA® (levacetylleucine) for the treatment of Niemann-Pick disease type C (NPC)....
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CAMBRIDGE, Mass. — Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today announced the first patient was dosed at Mayo Clinic in the redePHine study evaluating ABO-101, an investigational CRISPR gene editing therapy, in people living with primary hyperoxaluria type 1 (PH1). There...
Results from all 117 patients dosed in the pivotal Phase 2 iMMagine-1 study of anito-cel demonstrated 97% ORR and 68% CR/sCR at a median follow-up of 12.6 months No delayed neurotoxicities including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, and no immune-mediated enterocolitis have been observed to...
REDWOOD CITY, Calif. — Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, today announced that new clinical data from its Phase 1 study of CART-ddBCMA in patients with relapsed or refractory multiple myeloma will...
SAN DIEGO, Calif. — Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and medicines to treat unmet medical needs within liver and respiratory rare diseases, today announced that the European Commission (EC), based on a positive...
AMSTERDAM, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced its plan to advance the clinical development of ARGX-119, a first-in-class agonist antibody to muscle-specific kinase (MuSK), to a registrational study in...
Amsterdam, Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced its plan to continue the development of efgartigimod to Phase 3 in adults with primary Sjögren’s disease (SjD), following the analysis of topline...
Amsterdam, Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved VYVDURA for adults with chronic inflammatory demyelinating polyneuropathy (CIDP). VYVDURA is approved for...
Amsterdam, Netherlands – Argenx SE, a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved VYVDURA® (efgartigimod alfa and hyaluronidase-qvfc) injection for subcutaneous (SC) use for the treatment of adult patients with...
Amsterdam, Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved VYVGART (efgartigimod alfa) for intravenous (IV) use in adults with primary immune thrombocytopenia...