Gene therapy specialist Meiragtx Holdings plc got a market bump courtesy of newly released top-line data from its phase II bridging study in Parkinson’s disease. The six-month, three-arm randomized, double-blind, sham controlled trial of AAV-GAD, a one-time infusion, demonstrated significant and clinically meaningful improvements in key efficacy endpoints. The primary objective was evaluating the therapy’s safety and tolerability. The study of participants with idiopathic disease showed the therapy was safe and well-tolerated with no serious adverse events. Meiragtx is pursuing approvals in the U.S., Europe and Japan. Read More
Securing clinical proof of concept for its nanoparticle platform, Topas Therapeutics GmbH reported promising top-line phase IIa results of TPM-502 for celiac disease. Results show a clear statistically significant dose response for antigen-specific markers of tolerance. The effects persisted throughout the follow-up period, and the candidate was safe at all doses tested, according to the Hamburg, Germany-based company. Read More
Inventiva SA is back on course to complete phase III development of its metabolic dysfunction-associated steatohepatitis (MASH) drug, lanifibranor, after putting in place new financing of up to €348 million (US$379.4 million), including an immediate cash injection of €94.1 million. Read More
This summer’s IPO by Artiva Biotherapeutics Inc. highlighted early stage efforts with natural killer cells in autoimmune disease, where a handful of companies are advancing programs. The firm raised $167 million through an upsized financing, with funds aimed at the development of its lead AlloNK program for systemic lupus erythematosus and other indications in the same category. Read More
Breast cancer cells, when disseminated to other secondary organs such as the lungs, may stay in a dormant state for years, even decades. But the mechanisms that limit their expansion are not well understood. This is what researchers call a dormant mesenchymal-like phenotype before metastasis to the lungs. Now, scientists have shown in a study published Oct. 7, 2024, in Cell, that the limiting of disseminated breast cancer cells (DCCs) to metastasize in the lungs is due to alveolar macrophages, which activate signals that make DCCs stay dormant. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Benitec, Eli Lilly, Genentech, GSK, Jasper, Perspective. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Cycle, Exelixis, Invo, Kintara, Merck & Co., Molecular Templates, Naya, Redhill, Tuhura, Vanda. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Astrazeneca, Bayer, Daiichi Sankyo, Dizal, Pfizer. Read More
