Percheron Therapeutics has partnered with Vanderbilt Health to initiate a clinical trial testing HMBD-002 in combination with standard treatments for high-risk acute myeloid leukaemia and myelodysplastic syndrome, targeting a recruitment start in late 2026.
- Vanderbilt Health to lead multi-centre HMBD-002 trial in AML/MDS
- Trial combines HMBD-002 with azacitidine and venetoclax
- Investigator-sponsored trial design with FDA interactions led by Vanderbilt
- HMBD-002 targets immune checkpoint VISTA, implicated in therapy resistance
- Recruitment expected to begin in fourth quarter of 2026
Vanderbilt Health to spearhead HMBD-002 trial in aggressive blood cancers
Percheron Therapeutics (ASX:PER) has secured a clinical research agreement with Vanderbilt Health (VH) in Nashville to conduct an investigator-sponsored trial of its monoclonal antibody HMBD-002 in acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS). The study aims to combine HMBD-002 with standard-of-care therapies azacitidine and venetoclax in patients with high-risk or recurrent disease, with recruitment targeted for the fourth quarter of 2026.
Trial leadership and strategic rationale
The trial will be led by Dr Somedeb Ball, Assistant Professor in Hematology and Oncology at VH, who brings extensive research experience in myeloid malignancies. Vanderbilt Health, a National Comprehensive Cancer Center with over US$1 billion in annual research funding, will manage trial design and FDA interactions. Percheron will provide funding, study drug, and technical support, retaining full access to trial data to guide future clinical development and regulatory strategies.
Targeting VISTA to overcome treatment resistance
HMBD-002 targets VISTA, an immune checkpoint inhibitor highly expressed in AML cells and associated with disease recurrence and resistance to therapy. Previous preclinical studies have shown VISTA blockade to be active in mouse models of AML, supporting the rationale for this trial. The drug’s phase I data, presented earlier in 2026, demonstrated safety and early signs of efficacy in advanced cancer patients, bolstering confidence in further investigation.
Addressing a significant unmet medical need
AML is the most common acute leukaemia in adults, with roughly 20,000 new US cases annually. Despite advances, high-risk AML and MDS remain challenging to treat, with most patients eventually relapsing after initial response to standard therapies. This trial represents a strategic push by Percheron to explore HMBD-002’s potential in a disease setting with substantial unmet need.
Next steps and broader clinical ambitions
Percheron expects to release further details on trial design as recruitment approaches. The company is also in discussions with other research institutions to expand HMBD-002’s clinical footprint across additional patient populations. This trial complements Percheron’s ongoing preparations for a pivotal phase II HMBD-002 study planned for later in 2026, reflecting a concerted effort to advance its immuno-oncology pipeline.
Bottom Line?
The Vanderbilt-led trial marks a critical step in validating HMBD-002’s role in AML/MDS, with data from this study poised to influence Percheron’s clinical and regulatory roadmap.
Questions in the middle?
- Will HMBD-002 demonstrate meaningful efficacy when combined with azacitidine and venetoclax in high-risk AML patients?
- How rapidly will patient recruitment progress given the trial’s multi-centre design and investigator sponsorship?
- What additional indications or patient subsets might Percheron target next based on emerging trial data?