Syntara (ASX:SNT) has launched a new Phase 2 clinical study that could shift the treatment landscape for Australians living with transfusion-dependent Myelodysplastic Syndromes (MDS), a rare blood cancer associated with bone marrow failure and limited therapeutic options.
The MDS05/D3 MESSAGE trial will investigate amsulostat (SNT-5505) in combination with the oral hypomethylating agent ASTX727, aiming to reduce patients’ reliance on regular blood transfusions and improve survival outcomes.
Led by the Australasian Leukaemia and Lymphoma Group (ALLG) and primarily funded through the Australian Government’s Medical Research Future Fund, the multi-centre study will recruit up to 30 patients across 10 hospitals nationwide.
Associate Professor Anoop Enjeti, Principal Investigator for the study, said the combination therapy was inspired by compelling findings from recent pre-clinical research at Heidelberg University. That work showed a marked increase in red blood cell production when amsulostat was combined with a commonly used hypomethylating agent.
“Amsulostat has since been shown to be safe and well tolerated for up to 12 months in patients with myelofibrosis, so we are excited about the potential it might hold for patients with MDS who have very few treatment options,” he said.
MDS is a severe form of blood cancer in which the bone marrow fails to produce enough healthy blood cells, leading to anaemia, infections and bleeding. For patients dependent on fortnightly transfusions, the burden is significant. The disease is associated with five-year survival rates of just 37.3 per cent, and a high likelihood of progression to acute myeloid leukaemia, an aggressive and frequently fatal cancer.
Associate Professor Enjeti said the trial’s accessibility is another important feature.
“Transfusion-dependent myelodysplasia has no approved treatments currently available for Australian patients,” he said. “The MESSAGE trial’s new treatment combination aims to improve survival rates and quality of life by reducing reliance on regular blood transfusions. Another unique aspect will be the combination of two oral medications, making this trial more accessible to patients.”
ALLG Chief Executive Officer Delaine Smith said the trial reflects the collaborative group’s mission to address areas of high unmet need.
“Our independent, clinician-led network enables us to conduct research across all areas of blood cancers, including rarer cancers,” she said. “We’re excited to launch the MESSAGE trial and to work with Syntara, whose original research underpins this new treatment concept.”
The study builds on Syntara’s wider clinical program. The company is already running AZALOX, a Phase 1b/2 study in Germany evaluating amsulostat with 5-Azacitidine in high-risk MDS and Chronic Myelomonocytic Leukemia, with further recruitment and interim data expected in 2026. Amsulostat has also completed a Phase 2a trial in myelofibrosis, where it was dosed alone and in combination with a JAK inhibitor.
Syntara CEO Gary Phillips said the new Australian study offers a significant opportunity to expand the therapeutic potential of amsulostat.
“The MESSAGE trial allows us to prove the potential of amsulostat in another form of blood cancer, complementary to our lead program in myelofibrosis and the AZALOX study in Germany,” he said. “We look forward to initial results in 2026 and, in the longer term, making a difference for these patients with limited treatment options.”