TUSCANY: Promising data for addition of tuspetinib in untreated chemo-ineligible AML

Preliminary data of the TUSCANY study indicated that tuspetinib can be added to standard-of-care venetoclax plus azacitidine in patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for intensive chemotherapy, without adding safety issues to the treatment regimen. So far, the researchers observed promising anti-tumour activity across a diverse patient population, including patients with TP53 mutations.

The phase 2 TUSCANY trial included patients with newly diagnosed AML to investigate the investigational drug tuspetinib [1]. “This agent is a potent oral kinase inhibitor targeting SYK, FLT3, JAK1/2, RSK1/2, and mutant KIT kinases that drive dysregulated proliferation in AML,” explained Dr Gabriel Mannis (Stanford University, CA, USA). Participants received a combination of standard dose azacitidine and venetoclax plus tuspetinib in a range of 40–120 mg daily for 21–28 days per 28-day cycle. The current analysis reported data from 10 participants.

Tuspetinib appeared to be very well-tolerated. There were no non-haematological serious adverse events (AEs), and diarrhoea was the most common non-haematological AE, occurring in approximately 20% of the participants. Grade ≥3 haematologic AEs such as anaemia (57.1%) and neutropenia (42.9%), were frequently observed. Dr Mannis emphasised that the research team had not seen QTc prolongation, CPK elevations, differentiation syndrome, or prolonged myelosuppression in cycle 1 in the absence of AML. Furthermore, the composite complete remission rate was 85.7%. “Next to this, 3 patients were MRD-negative at the time of the analysis, one of whom was a patient with TP53-mutated complex-karyotype disease,” highlighted Dr Mannis.

“Although it is very early, it appears that tuspetinib can be added to standard-of-care dosing of venetoclax and azacitidine without prolonged myelosuppression or undue toxicity,” decided Dr Mannis. “The treatment regimen was associated with encouraging anti-tumour activity, and we look forward to seeing more results in the future.”

1. Daver N, et al. TUSCANY study of safety and efficacy of tuspetinib plus standard of care venetoclax and azacitidine in study participants with newly diagnosed AML ineligible for induction chemotherapy. S139, EHA2025 Congress, 12–15 June, Milan, Italy.

Medical writing support was provided by Robert van den Heuvel.
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Posted on 16 June 2025