Nature Communications· 2026Q1
The conformation-selective PI3Kδ inhibitor roginolisib in patients with advanced or metastatic cancer, including metastatic uveal melanoma: first-in-human clinical trial
- 3citations
- Q1SCImago
- 2026year
Short summary
The conformation-selective PI3Kδ inhibitor roginolisib demonstrated promising efficacy in a first-in-human trial, with a median overall survival of 20.8 months in metastatic uveal melanoma (mUM) patients and a favorable safety profile (6.8% Grade ≥3 toxicities).
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Key points
- Roginolisib, a selective PI3Kδ inhibitor, was tested in a first-in-human dose-escalation study.
- In metastatic uveal melanoma (mUM) patients, median overall survival was 20.8 months.
- Patients with stable disease in the mUM cohort had a median overall survival of 28.5 months.
- The drug showed a good safety profile, with 6.8% of patients experiencing Grade ≥3 toxicities.
- Treatment correlated with reduced T regulatory cells and increased activated CD8+ T cells and IL-15.
AI-generated from the title and abstract; the full text is not read.
Abstract
Abstract Phosphatidylinositol 3-kinase delta (PI3Kδ) promotes tumour cell growth directly or indirectly by activating immune-suppressive cells. Roginolisib is a selective, non-ATP competitive inhibitor, which locks PI3Kδ into an inactive state. In a First-in-Human dose study, continuous daily dosing of roginolisib was investigated in patients with solid and haematologic malignancies. In Part A, 24 patients received escalating doses of roginolisib. In Part B, 20 mostly pre-treated metastatic uveal melanoma (mUM) patients received a dose associated with continuous PI3Kδ inhibition of > 90%. No Dose Limiting Toxicity or drug related toxicities requiring dose modifications were observed (≥ Grade 3 toxicities: 3/44; 6.8%). In mUM, median Overall Survival (mOS) was 20.8 months. mOS in mUM patients with Stable Disease was 28.5 months, and in patients with Progressive Disease was 12.3 months. Roginolisib treatment was associated with reducing T regulatory cell abundance, increasing activated CD8 + T cells and soluble plasma IL-15.
The authors' abstract, as published at the source. Nature Communications, 2026 · DOI ↗
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Field: Ophthalmology
OphthalmologyMedicine