Percheron Therapeutics Ltd (ASX:PER, OTC:PERCF) has entered a clinical research agreement with Vanderbilt Health in the US for a new multi-centre trial of its lead cancer therapy HMBD-002 in acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS).
The investigator-sponsored study will assess HMBD-002 in combination with standard-of-care treatments in patients with high-risk AML/MDS.
Start-up activities are expected shortly and recruitment is targeted to begin in the fourth quarter of 2026.
Trial Summary
Vanderbilt Health will lead the trial's design and execution and will take primary responsibility for interactions with the US Food and Drug Administration (FDA).
Percheron will provide financial support, study drug and technical assistance while retaining full access to the resulting data.
The study proposes to evaluate HMBD-002 alongside azacitidine and venetoclax, two standard-of-care therapies, in up to 38 patients with either recurrent or newly diagnosed AML/MDS.
Principal investigator Dr Somedeb Ball, assistant professor in Vanderbilt Health's Division of Hematology and Oncology, will oversee the study.
Substantial unmet medical need
AML is the most common form of acute leukaemia in adults, with around 20,000 new cases diagnosed annually in the US.
Percheron noted that VISTA, the immune checkpoint targeted by HMBD-002, is highly expressed in AML and has been associated with disease recurrence and resistance to treatment.
Dr Ball said high-risk AML/MDS continued to represent a substantial unmet medical need and that previous research suggested VISTA could be a relevant therapeutic target.
Builds clinical program for HMBD-002
HMBD-002, also known as minperstobart, is a monoclonal antibody targeting VISTA.
Percheron presented final results from a 48-patient Phase 1 study in April 2026, which showed HMBD-002 was generally safe and well tolerated and provided signals of potential clinical efficacy.
“High strategic interest”
The company has indicated it intends to assess the therapy in combination with existing treatments across several cancer types.
Percheron chief executive Dr James Garner described AML/MDS as an area of “high strategic interest” for the HMBD-002 program, with the Vanderbilt study expected to generate information that could help guide further clinical development.
The company is also continuing discussions with other research institutions and clinicians to identify additional patient populations for HMBD-002 and expects to release further details on the Vanderbilt study around the time it begins.