Syntara Q2 2025 presentation slides: amsulostat gains momentum with FDA Fast Track

Introduction & Market Context

Syntara Ltd (ASX:SNT) released its quarterly update presentation on August 1, 2025, highlighting significant progress in its clinical pipeline, particularly for its lead drug candidate amsulostat. The biotechnology company, which focuses on developing novel therapeutics for blood cancers and other indications, has seen its stock trade at $0.06 as of July 31, 2025, within its 52-week range of $0.027-$0.095.

The company’s presentation comes amid growing interest in innovative treatments for myelofibrosis, a market valued at approximately $1 billion according to Syntara’s estimates, and myelodysplastic syndrome (MDS), which represents a larger $3.2 billion opportunity. These markets are currently dominated by JAK inhibitors, which often provide suboptimal responses for many patients, creating an opening for novel therapeutic approaches.

Quarterly Performance Highlights

The second quarter of 2025 marked several significant achievements for Syntara, with its lead drug candidate amsulostat (formerly SNT-5505) gaining considerable momentum through regulatory designations and positive clinical data.

As shown in the quarterly highlights:

Key developments for amsulostat include the World Health Organization granting the International Non-Proprietary Name (INN) and the FDA awarding Fast Track designation for the treatment of myelofibrosis. These regulatory milestones potentially accelerate the drug’s path to market and reflect recognition of its potential to address unmet medical needs.

The company also presented positive interim Phase 2 clinical data for amsulostat at the European Hematology Conference and initiated the Phase 1c/2 AZALOX clinical study in myelodysplastic syndrome, expanding the potential applications of its lead compound.

Beyond its blood cancer programs, Syntara advanced its dermatology pipeline with first patients dosed in two clinical trials: a Phase 1a/b trial evaluating SNT-9465 for hypertrophic scars and the Phase 1c SATELLITE trial of SNT-6302 for keloid scars. Additionally, the company reported that its IRBD/Parkinson’s Disease Phase 2 study is 50% recruited and remains on track to report results in the first half of 2026.

Clinical Development Progress

The most significant clinical progress this quarter came from Syntara’s myelofibrosis program, where interim Phase 2 data demonstrated promising results for amsulostat when combined with ruxolitinib, a standard-of-care JAK inhibitor.

The following slide summarizes the key conclusions from the interim data:

The data suggests that amsulostat is safe and well-tolerated when combined with ruxolitinib, even in patients with high disease burden. Despite the relatively small sample size, the company reported encouraging improvements in symptom scores and the number of patients achieving a TSS50 ( Total (EPA:TTEF) Symptom Score reduction of 50% or more).

Notably, the reductions in symptoms and spleen volume appear to improve over time, which Syntara describes as a "novel finding" indicating amsulostat’s potential to provide a differentiated treatment option for patients already on JAK inhibitors. The remaining three patients in the study are scheduled to complete 12 months of treatment in Q3 2025, with FDA guidance on progression to a pivotal study expected in the same quarter.

Pipeline and Market Opportunities

Syntara’s diversified pipeline targets multiple high-value markets with several near-term catalysts expected over the next 12 months. The company’s strategy focuses on indications with significant unmet needs and substantial market opportunities.

The following slide outlines the company’s pipeline and target markets:

Beyond myelofibrosis, Syntara is pursuing opportunities in myelodysplastic syndrome with a market estimated at $3.2 billion. The company’s dermatology programs targeting hypertrophic and keloid scars address a combined market opportunity of approximately $3.5 billion. Additionally, the IRBD/Parkinson’s Disease program represents another potential $3.5 billion market.

The company has structured its development timeline to deliver a steady stream of news flow and potential value-creating events over the coming quarters:

Key upcoming milestones include the final 12-month data from the amsulostat myelofibrosis combination study and the outcome of an FDA review of the Phase 2/3 clinical trial proposal in Q3 2025. In the first half of 2026, Syntara expects to report data from multiple clinical programs, including the hypertrophic skin scarring trial, myelodysplastic syndrome studies, and the IRBD/neuroinflammation study.

Financial Position and Outlook

Syntara reported ending the quarter with a cash position of $15.1 million. This represents a decrease from the $18 million reported in the previous quarter, indicating a burn rate of approximately $2.9 million for the period. Based on the company’s previously disclosed monthly spending of approximately $1 million, the current cash position provides a runway of about 15 months, sufficient to reach several of the upcoming clinical milestones.

The company’s market capitalization was previously reported at just under $100 million, though this may have changed with recent stock price movements. Syntara’s current financial position appears adequate to support its near-term clinical development plans, though additional funding may be required to advance programs through later-stage trials, particularly if the company proceeds with a pivotal Phase 2/3 study for amsulostat following FDA guidance.

Forward-Looking Statements

While Syntara’s presentation highlights significant progress and outlines an ambitious development timeline, investors should note the company’s forward-looking statements disclaimer, which acknowledges that actual results could differ significantly due to various risks and uncertainties. The success of the company’s clinical programs, particularly the amsulostat studies in myelofibrosis and myelodysplastic syndrome, will be critical in determining Syntara’s long-term value proposition.

The upcoming FDA guidance on amsulostat’s clinical development path and the final 12-month data from the myelofibrosis combination study represent particularly important near-term catalysts that could significantly impact the company’s trajectory. Investors will be watching closely for these developments in the third quarter of 2025.

Full presentation:

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