Efficacy and safety of a novel oral anti-vasculogenic mimicry agent, CVM-1118, in advanced well-differentiated neuroendocrine tumors: a Phase IIa trial
Mené sur 43 patients atteints d'une tumeur neuroendocrine bien différenciée de stade avancé (pulmonaire, gastro-intestinale ou pancréatique), cet essai de phase IIa évalue l'efficacité, du point de vue de la survie sans progression, et la toxicité du foslinanib (un nouvel agent cibant le mimétisme vasculogénique)
Background: Highly vascularised neuroendocrine tumours (NETs) are attractive targets for foslinanib (CVM-1118), which disrupts vasculogenic mimicry and induces apoptosis via tumour necrosis factor receptor-associated protein 1. We evaluated the efficacy and safety of CVM-1118 in advanced NETs.
Methods: Patients with grades 1–2, well-differentiated lung, gastrointestinal, or pancreatic NETs, refractory or intolerant to one or more standard therapies and progressing within 6 months, received CVM-1118 (200–300 mg orally twice daily) in 28-day cycles. Primary endpoint was progression-free survival (PFS); secondary endpoints included objective response rate (ORR), disease control rate (DCR), overall survival (OS), and safety.
Results: Of 43 enroled participants, 35 were efficacy-evaluable; most had grade 2 pancreatic (63%) or gastrointestinal (34%) NETs with two prior therapies. Median PFS was 10.5 months (95% CI, 5.6–22.3). ORR was 3%, DCR was 77%, and median OS was not reached (95% CI, 23.8–NR). Sensitivity analysis in the full analysis set (N = 43) showed a PFS estimate broadly aligned with the primary analysis (median, 8.4 months); patients with prior everolimus, sunitinib, or peptide receptor radionuclide therapy (N = 22; median, 8.3 months) showed similar findings. Treatment-related adverse events occurred in 44%, mostly grades 1–2, with no serious events.
Conclusions: CVM-1118 demonstrates favourable efficacy and safety in advanced NETs.
British Journal of Cancer , résumé, 2026