Sparks - Percheron Therapeutics

Percheron Therapeutics has broadened the clinical development opportunity for HMBD-002 with the announcement of an investigator-sponsored study in acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS). The exploratory study will be led by Vanderbilt Health, with Percheron’s contribution limited to providing the study drug, in addition to a financial grant and advisory support. Importantly, Percheron has raised A$2.3m through an institutional placement, fully funding its commitments to the study while also providing additional working-capital flexibility.

AML is the most common adult leukaemia, with c 20,000 new cases diagnosed annually in the US and remains an area of substantial unmet need. While 60–80% of patients initially respond to intensive chemotherapy, roughly half subsequently relapse, with outcomes in the relapsed/refractory (r/r) setting remaining poor. The biological rationale for targeting VISTA in AML is also supportive, with VISTA shown to be highly expressed on AML blasts and higher expression associated with poorer survival. Moreover, anti-VISTA treatment has also been shown to reduce tumour growth in preclinical AML models. The two-stage study is expected to enrol 29–38 patients and initiate in H2 CY26, initially in r/r high-risk AML/MDS. HMBD-002 will be evaluated in combination with standard-of-care azacitidine and venetoclax, with the potential to expand into newly diagnosed patients and initial data expected in CY27. In our view, the study offers a capital-efficient route to establish clinical proof-of-concept in a biologically relevant setting. While Percheron has not provided updated guidance on the previously planned solid-tumour basket study, we believe the AML/MDS programme is likely to be prioritised near term. Consequently, we now expect the basket study, previously expected to start in Q4 CY26, to be pushed out into CY27.