Targeted therapy has seen a threefold increase in the 12-month progression free survival (PFS) rate versus standard of care (SoC) in a Phase II trial of patients with metastatic solid tumours.
The Fondazione per la Medicina Personalizzata (the Foundation of Personalised Medicine; FPM) based in Rome, Italy, presented data from its Phase II trial at the 2024 European Society of Medical Oncology (ESMO) meeting, taking place in Barcelona, Spain, from 13 to 17 September.
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The trial (NCT04591431) is an ongoing Phase II, randomised, 400-patient proof-of-concept study evaluating the efficacy of targeted therapy versus SoC in metastasised solid tumour patients who have received one prior line of therapy. Trial completion is estimated in June 2025.
FPM’s trial used targeted therapy as per the mutational profile of patients’ cancer, most commonly assigning Yervoy (ipilimumab), Opdivo (nivolumab), ipatasertib, and Pemazyre (pemigatinib).
The Phase II trial met its primary endpoint of overall response rate (ORR), achieving a statistical significance of 17% in the targeted therapy arm compared to 9.5% in the SoC arm.
PFS was the trial’s secondary endpoint, achieving statistically significant PFS over 12 months of 22% in the targeted therapy arm compared with 7% for SoC, and a median PFS rate of 3.7 months with targeted therapy compared to 2.8 months in the SoC arm.
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By GlobalDataTargeted therapy demonstrated no statistically significant improvement in overall survival (OS), with a median OS of 9.2 months in the targeted therapy arm versus 7.6 months in the SoC arm.
In total, 1,794 patients across 40 Italian cancer centres were screened using comprehensive genomic profiling (CGP) to identify solid tumours bearing actionable alterations. The most commonly identified genomic alterations were high tumour mutation burden (HTMB) and mutations in the AKT-1, PIK3CA, and ERBB2 genes.
Once a molecular alteration that could be targeted with a drug available in a patient’s own trial centre was identified, the clinical case was reviewed by Italy’s Molecular Tumour Board (MTB) before each of the 400 patients was affirmed for trial participation.
Principal investigator Dr Andrea Botticelli said: “In pretreated metastatic patients, targeted therapy, driven by CGP and MTB consultation, significantly improved ORR and PFS with a long-term PFS benefit at one year and beyond.
“It is clear that the MTB plays a crucial role in the mutational tumour-agnostic approach to cancer treatment and that new trial designs are needed to provide working models to expedite patient access to targeted therapy and to deliver personalised oncology in clinical practice.”
