Percheron Therapeutics Ltd (ASX:PER, OTC:PERCF) has presented new phase I clinical data for its cancer therapy candidate HMBD-002 at the American Association for Cancer Research Annual Meeting in San Diego, marking an important step forward for the company’s oncology program, which is “very well positioned” to progress, with $2.2 million in a recent raise expected to drive development work ahead of a targeted phase II study in calendar 2026.
The poster presentation outlined results from a completed phase I study of HMBD-002 in 48 patients with a range of advanced cancers across six US centres, providing the company with early clinical validation as it works towards the next stage of development.
HMBD-002 is designed to target VISTA, an immune checkpoint that tumours use to suppress the body’s immune response. By blocking VISTA, Percheron aims to restore immune activity against cancer cells.
“This is an important read-out for HMBD-002,” Percheron CEO, Dr James Garner said. “Around half of all new cancer therapies fail in phase I, so this study represents the successful completion of a critical hurdle for our drug. As is common in such studies, the patient population was very late-stage, having already failed, in most cases, several prior lines of treatment. Despite that, we see meaningful evidence of pharmacological effect in patients who received higher doses, which is encouraging as we move forward with development. The clinical observations that have previously been shared are supported by biomarker data, presented here for the first time, which further corroborates the intended mechanism of HMBD-002. ”
Phase I data supports safety and mechanism
According to the poster, HMBD-002 was safe and well-tolerated at doses of up to 1,400 milligrams weekly. The most common treatment-related adverse events were fatigue, rash and nausea.
Twenty patients in the trial received HMBD-002 in combination with pembrolizumab, marketed as Keytruda, with no evidence of added toxicity from the combination, an outcome that may support future combination-based development strategies.
The presentation also pointed to signs of anti-tumour activity, with a number of patients remaining on therapy for extended periods or recording material tumour shrinkage.
Biomarker findings added to the significance of the readout, showing HMBD-002 triggered an immunostimulatory response marked by an increase in CD8+ T-lymphocytes and a decrease in monocytes and dendritic cells. Percheron said this was consistent with the drug’s intended mechanism of action.
For the company, the AACR presentation gives external visibility to data that not only supports the safety profile of HMBD-002, but also suggests the therapy is engaging the immune system in the way researchers had hoped.
Next milestone in view
While phase I cancer studies are primarily designed to assess safety and pharmacokinetics, they can also offer early indications of biological activity because they are typically conducted in patients rather than healthy volunteers.
That makes the AACR poster meaningful for Percheron, as it helps position HMBD-002 as a program with both a manageable safety profile and early evidence of pharmacological effect in a difficult, late-stage cancer setting.
The company is now preparing to present new preclinical HMBD-002 data at the American Society of Clinical Oncology Annual Meeting in Chicago from May 29 to June 2, 2026.
Percheron also expects to launch a new clinical trial for HMBD-002 in the second half of calendar 2026, with further details to be provided as development plans progress.