On December 13, 2023 Syntara (ASX:SNT) reported that it has commenced dosing in the final cohort of a phase 2 clinical trial studying its pan-LOX inhibitor SNT-5505 in patients with the bone marrow cancer myelofibrosis (Press release, Syntara, DEC 13, 2023, View Source [SID1234638506]). The trial was cleared to progress after FDA review of the protocol and data from the earlier cohort which demonstrated an excellent safety profile and encouraging signs of efficacy when used in patients who had failed on current standard of care.

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This additional cohort of the phase 2 trial MF-101 aims to demonstrate that SNT-5505, the lead asset in Syntara’s drug discovery pipeline, is safe and effective in myelofibrosis patients who are sub-optimally controlled on the market leading JAK inhibitor, ruxolitinib. Full recruitment of 15 patients is targeted for Q2 2024 from 19 clinical trial sites in Australia, South Korea, Taiwan and the USA. The open label study is expected to report interim data on 6 months of treatment in Q4 2024 and final data from 12 months treatment in Q2 2025.

Data previously announced by the company from the first cohort of MF-101 where SNT-5505 was used for 6 months in patients as a monotherapy was presented this week at the American Society of Haematology (ASH) (Free ASH Whitepaper) 2023 meeting in San Diego. The oral presentation was delivered in the New Therapeutic Frontiers session by Dr. Pankit Vachhani, Assistant Professor of Medicine & Medical Director of the Clinical Research Unit at the University of Alabama at Birmingham.

Commenting on the presentation, Dr Gabriela Hobbs, Assistant Professor, Medicine, Harvard Medical School & Clinical Director, Leukaemia, Massachusetts General Hospital said, "The data presented this week at ASH (Free ASH Whitepaper) demonstrated that when used as a monotherapy in patients who have failed on a JAK inhibitor, SNT-5505 comprehensively inhibits the LOX enzymes, is well tolerated, and in some patients led to improvements in fibrosis and blood counts, which are encouraging signs of efficacy. Treatments like SNT-5505 that are well tolerated and can improve/stabilize blood counts and fibrosis are needed. In particular, SNT-5505 in combination with JAK inhibitor therapy has the potential to enhance the impact of JAK inhibitor treatment on symptoms, which is a vital area for future research. I eagerly anticipate reviewing data from this next study cohort in 2024."

An effective pan-LOX inhibitor for myelofibrosis would open a market that is conservatively estimated at US$1 billion per annum.

Pharmaxis CEO Gary Phillips said, "This study that commenced recruitment today is crucial in establishing the place for SNT-5505 in the treatment regimen of myelofibrosis patients. The open label design enables us to assess the performance of SNT-5505 in real time and we are targeting a major interim data update at ASH (Free ASH Whitepaper) 2024 that will also trigger follow up discussions with the FDA on the pivotal registration study design and support ongoing discussions with strategic partners."

SNT-5505 is a pan-LOX inhibitor that has also demonstrated compelling pre-clinical data when used in combination with standard of care in other haematological malignancies such as myelodysplastic syndrome and solid tumours like those found in hepatocellular carcinoma and pancreatic cancer.