Syntara Accelerates Clinical Trials and Secures Key EMA Orphan Drug Status
Syntara Limited has made significant strides in its clinical pipeline, launching new trials and receiving key regulatory designations, positioning 2026 as a pivotal year for the company.
- Initiation of Phase 2 MESSAGE trial for amsulostat in transfusion-dependent MDS
- Positive European Medicines Agency opinion for orphan drug designation in myelofibrosis
- Completion of recruitment in Phase 2 iRBD trial with milestone payment triggered
- Progression of topical anti-fibrotic SNT-9465 into Phase 1b hypertrophic scar study
- Proforma cash balance of $12.3 million at 31 December 2025
Clinical Pipeline Momentum
Syntara Limited (ASX, SNT), a clinical-stage drug developer focused on extracellular matrix dysfunction, has reported robust progress across its drug pipeline for the quarter ended 31 December 2025. The company’s lead candidate, amsulostat, continues to gain traction with the launch of a Phase 2 clinical trial in Australia targeting transfusion-dependent myelodysplastic syndromes (MDS). This MESSAGE trial, supported by the Australasian Leukaemia & Lymphoma Group and funded by the Medical Research Future Fund, aims to evaluate amsulostat in combination with ASTX727 in a patient population with limited treatment options.
Alongside this, Syntara received a positive opinion from the European Medicines Agency (EMA) granting orphan drug designation for amsulostat in myelofibrosis (MF), a regulatory milestone that offers market exclusivity and development incentives in the EU. This complements the existing orphan drug status granted by the US FDA, underscoring amsulostat’s potential in treating serious blood cancers.
Upcoming Data Catalysts and Collaborations
The company has also completed recruitment in its Phase 2 trial of SNT-4728 for isolated REM Sleep Behaviour Disorder (iRBD), a condition linked to high risk of neurodegenerative diseases such as Parkinson’s. This milestone triggered a $1.8 million payment expected in early 2026, with top-line results anticipated in the second quarter. SNT-4728’s potential to reduce neuroinflammation could mark a significant advance in early intervention for neurodegeneration.
In the anti-fibrotic space, Syntara’s topical candidate SNT-9465 has progressed into a Phase 1b study for hypertrophic scars following promising Phase 1a safety and target engagement data. This trial will assess the drug’s ability to improve scar appearance and function, leveraging advanced imaging techniques. The company also continues its keloid scarring program in collaboration with Professor Fiona Wood, with results expected later this year.
Financial Position and Strategic Outlook
Financially, Syntara ended the quarter with a proforma cash balance of $12.3 million after accounting for the milestone payment from Parkinson’s UK. Operating cash outflows of $3.8 million were primarily driven by research and development and staff costs, reflecting the company’s investment in advancing its clinical programs. The company is also pursuing outstanding payments related to the sale of its mannitol respiratory business, though timing and amounts remain uncertain.
CEO Gary Phillips highlighted 2026 as a defining year, with multiple data readouts expected that could materially enhance the company’s value and strategic options. The combination of regulatory progress, clinical trial advancements, and collaborative funding support positions Syntara to potentially reshape treatment landscapes in blood cancers, neurodegeneration, and fibrotic skin diseases.
Bottom Line?
With multiple clinical milestones on the horizon, Syntara’s 2026 progress will be closely watched by investors and industry alike.
Questions in the middle?
- Will the Phase 2 MESSAGE trial data confirm amsulostat’s efficacy in transfusion-dependent MDS?
- How might the EMA orphan drug designation impact Syntara’s commercial partnerships and market access in Europe?
- What insights will the Phase 2 iRBD trial provide on SNT-4728’s potential to modify neurodegenerative disease progression?
