Syntara’s Amsulostat Shows Strong Phase 2a Results in Myelofibrosis Trial
Syntara Limited has reported positive final data from its Phase 2a trial of amsulostat in myelofibrosis, demonstrating significant symptom relief and spleen volume reduction with a favorable safety profile. These results pave the way for advancing clinical development and partnership discussions.
- 73% of patients achieved 50% symptom improvement at 24 weeks or beyond
- 44% of evaluable patients showed meaningful spleen volume reduction
- Amsulostat demonstrated a distinct mode of action targeting bone marrow fibrosis
- Well-funded with A$20.7 million cash runway into 2027
- Global myelofibrosis experts appointed to advisory board
Positive Phase 2a Data Validates Amsulostat’s Potential
Syntara Limited, an Australian clinical-stage biotech, has announced encouraging final results from its Phase 2a trial evaluating amsulostat in patients with myelofibrosis, a rare and debilitating bone marrow cancer. The data reveal that a substantial majority of patients experienced significant and sustained symptom relief, alongside meaningful reductions in spleen size, a key clinical marker of disease burden.
Myelofibrosis disrupts normal blood cell production due to scarring in the bone marrow, leading to fatigue, infections, and bleeding risks. Current standard treatments, primarily JAK inhibitors, offer limited survival benefits and are often poorly tolerated, with many patients discontinuing therapy within five years. Amsulostat’s novel mechanism targets the underlying fibrosis by inhibiting lysyl oxidase enzymes, which contribute to bone marrow stiffening and abnormal cell growth.
Clinical Highlights and Patient Outcomes
In the open-label Phase 2a study, 73% of patients who reached 24 weeks or beyond achieved at least a 50% reduction in their total symptom score, with some patients reporting complete resolution of symptoms by the 52-week mark. Additionally, 44% of evaluable patients demonstrated a spleen volume reduction of 25% or more, sustained through the study period without increasing doses of their background JAK inhibitor therapy.
The trial enrolled a heterogeneous patient population with a high disease burden, many of whom had been on stable ruxolitinib treatment for extended periods without achieving complete response. The safety profile of amsulostat was favorable, with no unexpected adverse events, supporting its potential as a combination therapy to improve outcomes beyond symptom management.
Strategic Positioning and Next Steps
Backed by a strong institutional shareholder base and a cash position of A$20.7 million, Syntara is well-positioned to advance amsulostat’s clinical development. The company has appointed leading global myelofibrosis experts to its advisory board, underscoring its commitment to rigorous clinical strategy and regulatory engagement.
Upcoming milestones include further Phase 1c/2a studies in related blood disorders and ongoing discussions with the FDA, which has granted amsulostat Fast Track designation. The positive Phase 2a results are expected to catalyze partnership opportunities and potentially accelerate the drug’s path toward regulatory approval.
With a patent-protected, first-in-class approach addressing a significant unmet need, amsulostat could represent a meaningful advance in myelofibrosis treatment, offering hope to patients facing limited options.
Bottom Line?
Syntara’s promising Phase 2a results set the stage for pivotal trials and strategic partnerships in a challenging blood cancer market.
Questions in the middle?
- Will upcoming Phase 1c/2a trials confirm amsulostat’s efficacy in related blood disorders?
- How will Syntara navigate regulatory pathways and potential partnerships post-FDA Fast Track designation?
- What competitive advantages will amsulostat hold against emerging myelofibrosis therapies?