Nine reports of tumors in children treated with Novartis’ spinal muscular atrophy (SMA) gene therapy Zolgensma raise new questions about its long-term safety. In two cases, viral vector DNA was detected in the patients’ genomes. In 2022, two children had already died of acute liver failure following treatment. The cases illustrate why assessing the safety of gene and cell therapies remains a particular challenge.
French pharmaceutical group Servier has suffered a major setback just four months after completing its $1.55 billion upfront acquisition of Edgewise Therapeutics’ muscular dystrophy business. Sevasemten, the centerpiece of the deal, has failed its pivotal trial in Becker muscular dystrophy, despite encouraging results from earlier studies.
Germany-based cancer immunotherapy specialist Immatics presented updated clinical data for two PRAME-targeted drug candidates at the ESMO Congress 2026 in Madrid. Particularly striking is the survival signal for the bispecific T-cell engager IMA402: In a subgroup of patients with advanced melanoma and high PRAME expression, 91% were still alive after one year. Median overall survival had not yet been reached at the time of analysis.
French biotech OSE Immunotherapeutics has reported higher one-year survival rates with two combinations of its cancer vaccine Tedopi compared with chemotherapy alone in a Phase 2 lung cancer trial. However, the results are similar to those previously achieved with Tedopi alone, raising questions about the added benefit of combination treatment.
French-Belgian biotech Osivax has reported disappointing Phase 2b results for its broad-spectrum influenza vaccine OVX836, which achieved a nonsignificant efficacy estimate of 24% against a predefined target of 50%. The company is now emphasizing a strategy to combine its candidate with existing seasonal flu vaccines.
BioNTech’s BNT113, combined with Keytruda, failed to significantly improve progression-free survival in a Phase 2/3 head and neck cancer trial. The study will continue to assess whether the combination helps patients live longer.
Swedish biotech Elicera Therapeutics reported complete metabolic responses in eight of 12 patients treated with its experimental CAR-T therapy ELC-301 in the Phase 1 portion of its CARMA trial. Six remained in complete response at their latest assessment.
Zealand Pharma shares fell about 4% in Thursday trading after new Phase 2 results for its Roche-partnered obesity drug petrelintide showed encouraging weight loss but a modest improvement in blood sugar control. It’s the second positive trial readout that has failed to reassure investors in the course of a week.
Argenx is stopping its Phase 3 UNITY trial of efgartigimod in Sjögren’s disease after an interim analysis showed the study could not meet its primary endpoint. Shares fell about 18% in Euronext Brussels trading following the announcement, overshadowing positive Phase 2 results for its recently acquired celiac disease drug FB102.
Genmab’s rinatabart sesutecan (Rina-S) delivered durable responses in a Phase 2 trial in platinum-resistant ovarian cancer, providing encouraging validation for the centerpiece of its $1.8 billion acquisition of ProfoundBio.