Syntara Reports 73% Symptom Improvement in Myelofibrosis Phase 2a Trial
Syntara Limited reports encouraging Phase 2a results for amsulostat in myelofibrosis, with significant symptom relief and spleen volume reduction sustained over 12 months, alongside new clinical trial initiations and a solid cash position.
- 73% of patients achieved ≥50% symptom improvement at 6 months+
- 44% of patients showed ≥25% spleen volume reduction with durable effects
- No treatment-related serious adverse events reported up to 12 months
- FDA feedback clarifies clinical development pathway for amsulostat
- Strong cash position of $14.4 million supported by $5.6 million R&D tax incentive
Positive Phase 2a Data Reinforces Amsulostat’s Potential
Syntara Limited (ASX – SNT), a clinical-stage biotech focused on fibrosis and blood cancers, has unveiled promising top-line results from its Phase 2a trial of amsulostat (SNT-5505) in myelofibrosis (MF). The data, covering 12 months of treatment, showed that 73% of evaluable patients experienced at least a 50% reduction in symptom burden, while 44% achieved meaningful spleen volume reduction. These outcomes were accompanied by a favourable safety profile, with no treatment-related serious adverse events reported.
The trial’s durability and tolerability underscore amsulostat’s differentiated dual mechanism; targeting lysyl oxidase inhibition and platelet-derived growth factor receptor pathways; potentially positioning it as a best-in-class oral therapy for MF. CEO Gary Phillips highlighted the sustained clinical benefits and the drug’s potential to redefine treatment paradigms for this challenging bone marrow cancer.
Strategic Advancements and Regulatory Progress
Building on these results, Syntara received constructive feedback from the US Food and Drug Administration (FDA), which has helped clarify the design of upcoming trials. The company has also bolstered its clinical and commercial expertise by appointing globally recognised haematology and biopharmaceutical leaders, including former executives from CTI BioPharma and Celgene Asia, as strategic advisors and clinical board members.
Expanding the amsulostat program, Syntara initiated the AZALOX Phase 1b/2 trial in Germany, targeting myelodysplastic syndromes and chronic myelomonocytic leukemia. This move signals the company’s intent to broaden the drug’s application beyond MF into related myeloid malignancies.
Diversifying Pipeline with Dermatology and Neurology Trials
Beyond hematology, Syntara advanced two Phase 1 trials for topical anti-fibrotic candidates addressing hypertrophic and keloid scars, conditions with significant unmet medical needs and a sizeable market opportunity. The SNT-9465 and SNT-6302 programs aim to offer pharmacological alternatives to current invasive and costly treatments.
Additionally, the company’s SNT-4728 candidate is progressing in a Phase 2 study for isolated REM sleep behaviour disorder, a condition linked to neurodegenerative diseases such as Parkinson’s. This trial, funded by Parkinson’s UK, is on track to provide interim data in the first half of 2026.
Financial Health and Operational Efficiency
Financially, Syntara maintains a robust cash position of $14.4 million, slightly down from $15.1 million last quarter but bolstered by a $5.6 million R&D tax incentive refund. Operating cash outflows have decreased significantly compared to the previous quarter, reflecting improved cost management. The company continues to pursue outstanding payments related to the 2023 sale of its mannitol respiratory business, with some uncertainty remaining around timing and amounts.
Overall, Syntara’s quarterly update paints a picture of a company advancing multiple clinical programs with strategic clarity, supported by a strong balance sheet and expert guidance as it prepares for pivotal late-stage trials.
Bottom Line?
Syntara’s clinical momentum and strategic appointments set the stage for critical trial readouts and potential partnerships in 2026.
Questions in the middle?
- How will the FDA feedback shape the design and endpoints of the next amsulostat trial?
- What are the timelines and expected milestones for the AZALOX and dermatology trials?
- How might ongoing uncertainties around the mannitol business sale impact Syntara’s financial flexibility?
