Piqray (alpelisib)

Based on the findings, the patient opted for off-label therapy with alpelisib, an α-specific PI3K inhibitor that selectively targets p110α and has shown promising efficacy in breast cancer patients harboring the identical PIK3CA mutations. However, no clinical response was observed in the patient, and the lack of response may be associated with the specific nature of the PIK3CA mutation, and/or other unfavorable tumor biological factors that override any benefit from alpelisib.

P3, N=19, Active, not recruiting, Novartis Pharmaceuticals | Trial completion date: Jun 2026 --> Feb 2027 | Trial primary completion date: Jun 2026 --> Feb 2027

P=N/A, N=15, Completed, London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's | Active, not recruiting --> Completed

P2, N=19, Completed, Jennifer Lee Caswell-Jin | Suspended --> Completed

Treatment with the PI3Kα-selective inhibitor alpelisib suppressed lesion formation and reversed pro-angiogenic signaling in both mouse models and patient-derived cerebral cavernous malformations organoids. These findings uncover a convergent mechanism involving MAPK and PI3K pathway activation in cerebral cavernous malformations pathogenesis and demonstrate that PI3Kα inhibition may offer a viable therapeutic strategy for a disease that currently lacks effective pharmacological treatment.

In vitro cytotoxicity assays were then performed in MCF-7 and MDA-MB-231 breast cancer cell lines, with alpelisib as a reference compound. Hit 2 reduced cell viability in both cell lines, but its effect was particularly pronounced in MDA-MB-231 cells, a model of triple-negative breast cancer (TNBC). These results suggest that Hit 2 represents a promising natural scaffold for further design and development in breast cancer therapy, with particular relevance for aggressive TNBC.

P2, N=24, Active, not recruiting, University of Miami | Recruiting --> Active, not recruiting | N=500 --> 24

P2, N=24, Active, not recruiting, Novartis Pharmaceuticals | Trial completion date: Jul 2026 --> Jan 2027 | Trial primary completion date: Jun 2026 --> Jan 2027

We provide novel data on the efficacy of putative and established therapies in patient-derived GEP-NEN primary cultures. Our standardized platform for personalized drug screening and risk assessment in GEP-NEN primary cultures enables prediction of individual tumor treatment response in this orphan disease.

In this study, 20 new compounds with thiadiazole structures were synthesized based on Alpelisib...For compounds 3j and 3o, further activity studies, molecular docking, and molecular dynamics studies were performed. The compounds' EGFR and COX-2 inhibitory potential was investigated.

P2, N=44, Active, not recruiting, Marina N Sharifi | Trial completion date: Jan 2026 --> May 2026 | Recruiting --> Active, not recruiting

We report a 79-year-old non-smoking woman with ER-positive/HER2-negative breast cancer who initially underwent breast-conserving surgery and adjuvant anastrozole in December 2023. Following local recurrence in October 2024, she received mastectomy and ribociclib-fulvestrant...Alpelisib was discontinued, and high-dose corticosteroids led to complete clinical and radiological resolution. This case highlights early-onset alpelisib-induced pneumonitis at a reduced dose and underscores the importance of early recognition and prompt management.