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 clinical trial of AAV-GAD, a one-time infusion, demonstrated significant and clinically meaningful improvements in key efficacy endpoints. The study of 14 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. The company’s stock (NASDAQ:MGTX) had risen 15% at midday to $5.34 a share.

Topas’ platform validated with top-line celiac disease data

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. The nanoparticle platform was touted as a new approach for autoimmune disease when it was spun out into Topas more than eight years ago by Evotec GmbH. Topas began its phase IIa trial, an international, multicenter, double-blind, randomized and placebo-controlled study, in 2023. It intends to submit full data for publication in a peer-reviewed journal and for presentation at an upcoming scientific conference.

Amid shifting MASH landscape, Inventiva adds €348M financing

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. The company sealed the deal just as it was about to reach the end of its cash runway in mid-October, and after a voluntary halt to the Nativ3 phase III trial of lanifibranor in February caused a nine-month delay in completing recruitment.

Artiva, Century, others extend natural-killer autoimmune push

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.

Lung macrophages put invasive breast cancer cells to sleep

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 (M-like) 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.

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Astrazeneca, Avacta, Benitec, Commit, Curasen, Cycle, Dizal, Dong-A, Exelixis, Galecto, GSK, Inventiva, Invo, Jasper, Kintara, Klotho, Lilly, Merck, Molecular Templates, Naya, Nicox, Orion, Perspective, Pfizer, Redhill, Roche, Sernova, Soleus, Tevogen, Transpire, Tuhura, Vanda