Aptose’s Tuspetinib Triplet Therapy Shows High Response Rates in AML Trial — Aptose Biosciences Inc.

Event summary

Aptose presented Phase 1/2 TUSCANY trial data at EHA 2026, showing 86.2% composite complete response rate in newly diagnosed AML patients treated with tuspetinib (TUS) + venetoclax (VEN) + azacitidine (AZA) triplet therapy.
32 patients were dosed across four TUS dose levels (40 mg, 80 mg, 120 mg, 160 mg), with 29 evaluable for response.
MRD-negativity rate was 86.4% in patients achieving CR/CRh, and 100% composite complete remission in TP53-mutated patients with complex karyotype at the 160 mg dose level.
No treatment-related deaths or serious adverse events were reported, indicating a well-tolerated profile.

The big picture

Aptose’s tuspetinib triplet therapy represents a potential breakthrough in treating newly diagnosed AML patients ineligible for induction chemotherapy. The high response rates across diverse genetic profiles, including historically difficult-to-treat mutations like TP53, position the therapy as a broad-spectrum option in a market increasingly dominated by targeted treatments. The well-tolerated safety profile further strengthens its competitive position as the company advances toward later-stage trials.

What we're watching

Clinical Efficacy: Whether the high response rates and MRD-negativity observed in the TUSCANY trial can be sustained in larger, more diverse patient populations.
Regulatory Pathway: The pace at which Aptose can advance tuspetinib through subsequent clinical trials and potential regulatory approvals for frontline AML therapy.
Competitive Positioning: How Aptose’s mutation-agnostic approach with tuspetinib differentiates it from targeted therapies in the AML treatment landscape.