The EMA had its second busiest year ever in 2025, approving 104 drugs, of which 38 had a new active substance. Sixteen of these are for treating rare diseases, and four are advanced therapy medicinal products.

Microbiome specialist Enterobiotix Ltd. is in the throes of raising a new round of funding for a phase IIb trial after reporting positive results from the phase IIa study of EBX-102-02, an oral therapy for irritable bowel syndrome (IBS).

Immunofoco Biotechnology Co. Ltd. reported encouraging early clinical data for its CLDN18.2-targeted CAR T therapy, IMC-002, in heavily pretreated gastric and gastroesophageal junction cancer, adding momentum to the push to extend cellular immunotherapy into solid tumors.

Lexeo Therapeutics Inc.’s phase I/II data – characterized by Oppenheimer analyst Leland Gershell as “heroic” – this week with gene therapy LX-2020 in PKP2-associated arrhythmogenic cardiomyopathy pushed the New York-based firm’s shares (NASDAQ:LXEO) down 35% to a low of $6.90 Jan. 12, following a $10.54 close the previous trading day.

Takeda Pharmaceuticals Co. Ltd.’s oral tyrosine kinase 2 inhibitor, zasocitinib (TAK-279), met the co-primary endpoints and all ranked secondary endpoints in two pivotal phase III studies in patients with moderate to severe plaque psoriasis.

Stoke Therapeutics Inc.’s speeded-up timeline for zorevunersen, the antisense oligonucleotide in development with Biogen Inc. as a first-in-class potential disease-modifying treatment for Dravet syndrome, put the rare, severe form of lifelong epilepsy in the spotlight. The news involved completion of enrollment and a phase III data readout from the Emperor study, as officials said signups of 150 patients are expected in the second quarter of the year, which puts the study on track for data in mid-2027.

Ollin Biosciences Inc. is gearing up for global phase III trials this year after disclosing favorable top-line results from its randomized, head-to-head phase Ib Jade study comparing OLN-324, a next-generation VEGF/Ang2 bispecific antibody, to Vabysmo (faricimab, Roche AG), which takes aim at the same targets. The drugs were tested in more than 160 patients with diabetic macular edema (DME) or wet age-related macular degeneration (AMD).

Takeda Pharmaceuticals Co. Ltd.’s oral tyrosine kinase 2 inhibitor, zasocitinib (TAK-279), met the co-primary endpoints and all ranked secondary endpoints in two pivotal phase III studies in patients with moderate to severe plaque psoriasis.