Percheron Therapeutics Ltd (ASX:PER, OTC:PERCF) has reported positive final data from its phase I clinical trial of HMBD-002, showing a favourable safety profile and early signs of potential efficacy across several cancer types.
The study — conducted at six sites in the United States under an FDA Investigational New Drug (IND) application — enrolled 48 patients with advanced, metastatic cancers including pancreatic, bowel, breast and lung tumours. All participants had previously received an average of four to five different therapies before entering the trial.
Promising safety results
Percheron said HMBD-002 demonstrated “a very favourable” safety profile at doses up to 1,400 milligrams per week, both as a standalone treatment and in combination with Merck’s Keytruda® (pembrolizumab).
Fewer than 10% of patients experienced treatment-related adverse events of grade 3 or higher, and there were insufficient dose-limiting toxicities to establish a maximum tolerated dose. No cases of cytokine release syndrome were observed.
Only one dose-limiting event — a reversible case of hepatitis — was reported, and the company noted the absence of serious inflammatory responses often seen in early-stage immunotherapies.
“These data are very encouraging as we look toward a Phase II trial next year,” said Percheron CEO Dr James Garner.
“In particular, the data confirms the excellent safety profile of HMBD-002, which has the potential to be best-in-class, and it also provides some tantalising glimpses of potential efficacy,” he added. “We are immensely grateful to the dedicated clinicians who worked on the study, and to the parents and families who participated.”
Early efficacy signals
While the study’s main objective was safety and tolerability, Percheron noted several cases suggesting therapeutic benefit.
One patient with metastatic triple-negative breast cancer recorded a 27% tumour reduction after five weeks on HMBD-002 combined with pembrolizumab — close to the 30% threshold for partial response.
Others maintained stable disease significantly longer than expected for advanced cases, including patients with metastatic liposarcoma, breast, lung, and head and neck cancers.
Next steps
The company plans to publish the full dataset, including pharmacokinetic and pharmacodynamic analyses, in a peer-reviewed journal during calendar year 2026.
Further details on the design of a planned Phase II efficacy trial are expected in the fourth quarter of 2025, with recruitment targeted for 2026.
About HMBD-002
HMBD-002 is a first-in-class inhibitor of VISTA (V-domain immunoglobulin suppressor of T-cell activation), a novel immune checkpoint pathway. The drug was originally developed by Singapore-based Hummingbird Bioscience and licensed to Percheron in June 2025.
Preclinical studies have shown broad anti-tumour activity across animal cancer models, positioning HMBD-002 as a potential new approach in immuno-oncology for patients with limited treatment options.