Vanderbilt Health to lead new HMBD-002 trial in acute myeloid leukaemia
Percheron Therapeutics (ASX: PER) has entered into a clinical research agreement with Vanderbilt Health in Nashville, Tennessee, to conduct an investigator-sponsored trial of its lead asset HMBD-002 in high-risk acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS).
The trial will study HMBD-002 in combination with standard-of-care treatments, with recruitment targeted to begin in 4Q CY2026. The Principal Investigator is Dr Somedeb Ball, an Assistant Professor at Vanderbilt Health.
Vanderbilt Health carries considerable stature in US oncology. It receives over US$1 billion in annual research funding and was designated a National Comprehensive Cancer Center by the US National Cancer Institute in 2001.
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Inside the trial: what Vanderbilt will investigate
The study will be run as a multi-centre investigator-sponsored trial (IST). Under this structure, Vanderbilt Health leads both the design and execution of the study and is primarily responsible for interactions with the US Food and Drug Administration (FDA).
HMBD-002 will be tested in combination with azacitidine and venetoclax, two standard-of-care therapies, in up to 38 patients with recurrent or newly diagnosed AML/MDS. Percheron expects to share additional information regarding the study design around the time of its commencement, so full design details have not yet been disclosed.
Key trial parameters include:
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Sponsor/lead: Vanderbilt Health (VH / VUMC)
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Principal Investigator: Dr Somedeb Ball, Assistant Professor, Division of Hematology and Oncology
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Patient numbers: up to 38
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Combination partners: azacitidine + venetoclax
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Recruitment target: 4Q CY2026
Dr Somedeb Ball, Principal Investigator
“Despite significant advances in the last decade, high-risk AML / MDS remains a challenging diagnosis with substantial unmet medical need. VISTA is expressed primarily on myeloid cells, and previous research has indicated that it may be a relevant target in this disease. My colleagues and I look forward to commencing this clinical trial project and exploring the potential for HMBD-002 to provide benefit to patients with AML / MDS.”
Why Percheron’s role is capital-efficient
The IST structure allows Percheron to generate data in a new indication while a major institution carries the operational burden. Under the agreement, Percheron will provide a financial grant to support the study, along with study drug and technical assistance, while Vanderbilt Health leads design and execution.
HMBD-002 drug supply for upcoming trials was secured following successful completion of a manufacturing campaign in June 2026, with fill-and-finish activities targeted for Q3 CY2026 and a Certificate of Analysis confirming the batch met all product quality specifications.
Importantly, Percheron will have full access to any trial data. The company may use this data to inform future clinical trials, regulatory interactions, and partnering discussions.
Understanding VISTA and why AML is a strong target
HMBD-002 (minperstobart) is a monoclonal antibody targeting VISTA, a novel immune checkpoint regulator.
AML/MDS is a malignancy of myeloid cells, which are found in the blood and function as part of the body’s immune system. AML is the most common form of acute leukaemia in adults, with approximately 20,000 new cases diagnosed each year in the United States.
The scientific rationale is grounded in the biology of the disease. VISTA is highly expressed in AML and is associated with recurrence and with resistance to therapy. Because VISTA sits primarily on myeloid cells, and an experimental VISTA inhibitor has previously shown preclinical activity in mouse models of AML, there is a persuasive basis to explore the target in myeloid malignancies.
Existing treatment regimes are often initially effective in many patients, yet most patients eventually recur, leaving a substantial need for new therapeutic options. This biological context positions HMBD-002 to address a meaningful area of unmet need.
How this fits Percheron’s broader HMBD-002 strategy
The Vanderbilt agreement builds on an established clinical foundation. In April 2026, Percheron presented final data from a Phase I clinical trial in 48 patients, which showed the drug to be safe and well-tolerated and provided signals of potential clinical efficacy.
The Phase I results for HMBD-002 confirmed safety and tolerability across 48 patients at six US clinical centres, with 27.5% of evaluable patients achieving disease stabilisation and biomarker data validating the VISTA blockade mechanism of action.
The company has previously indicated its intention to investigate HMBD-002 in conjunction with existing therapies across several different cancer types. Percheron continues to advance discussions with other clinical research institutions and clinicians to identify additional patient populations in which HMBD-002 may be investigated.
| Milestone | Detail | Timing | Significance for Investors |
|---|---|---|---|
| Phase I complete | 48 patients, safe & well-tolerated | April 2026 | Established safety foundation |
| Vanderbilt IST | Up to 38 patients, AML/MDS | Announced August 2026 | New indication, external validation |
| Recruitment start | Combination with standard-of-care | Targeted 4Q CY2026 | Near-term catalyst |
What comes next for investors
The trial is anticipated to commence start-up activities shortly, with recruitment targeted for 4Q CY2026. Further study design detail is expected around the time of commencement, providing a near-term catalyst for shareholders to monitor.
Beyond this study, Percheron continues to pursue discussions with other institutions and clinicians to broaden the HMBD-002 development programme, and has committed to updating investors as those discussions progress.
Dr James Garner, CEO of Percheron Therapeutics
“We are delighted to be supporting Dr Ball and the VUMC team to conduct this interesting and important clinical trial. AML / MDS is of high strategic interest to the HMBD-002 program, and this study may provide significant insights to guide further clinical development. We hope to see recruitment commence in 4Q CY2026.”
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