In a potential breakthrough for the field of bone marrow cancer treatment, biotech company Pharmaxis (ASX: PXS) has released the final interim data analysis of their Phase 2 clinical trials for PXS-5505, a promising drug aimed at combating the debilitating effects of myelofibrosis.
The data revealed 60% of trial patients experienced a significant improvement in fibrosis, sparking optimism among medical professionals. At present, existing treatments for myelofibrosis are sadly underwhelming with those available largely just addressing symptoms. The PXS-5505 clinical data, however, has demonstrated an ability to modify the underlying disease where the average life expectancy upon diagnosis is just 5 years under the current standard of care.
This final round of interim data covered 10 patients who completed 6 months’ treatment with PXS‐5505, an oral drug which operates by inhibiting the lysyl oxidase (LOX) enzymes which are responsible for the excessive production of scar tissue in myelofibrosis patients.
Commenting on the data investigator Dr Lucia Masarova, Assistant Professor, Department of Leukemia at MD Anderson Cancer Center, Houston said,
“PXS‐5505 continues to show not only an excellent safety profile but also promising clinical activity. The effect on bone marrow fibrosis is particularly exciting for a disease like myelofibrosis, where despite numerous years of research, we do not have any effective anti‐fibrotic drugs.
“It is encouraging to see that the majority of 10 patients who completed 24 weeks of therapy also had improvements of symptoms and more importantly, stable or improved blood counts; including in those patients with severe thrombocytopenia.”
From biopsies taken from the evaluable patients, 60% had improved bone marrow fibrosis scores while 88% had stable or improved platelet counts from the open-label trial.
Myelofibrosis is a rare bone marrow disorder characterised by the replacement of healthy bone marrow with scar tissue. It has long presented a significant challenge to medical researchers as a disease in need of new treatment options where existing options such as JAK inhibitors already generate USD $1 billion of sales per annum without addressing the underlying disease.
A final presentation of the Phase 2 trial data is expected to be showcased later in 2023 at the American Society of Hematology conference which is attended by global leaders in the field of blood diseases.
In the meantime, Pharmaxis has already submitted a trial protocol to the US Food and Drug Administration (FDA) for an extension arm of the PXS-5505 clinical trials where it will be combined with a JAK inhibitor. The protocol was cited in a meeting with the FDA in April 2023, which had also reviewed all safety and efficacy data available at that time.
Feedback from the FDA is expected by the end of July 2023 with Pharmaxis CEO, Gary Phillips excited for the future of the drug where Pharmaxis is the only company in the world with pan‐LOX inhibitors in clinical development.
“The results from this trial with an oral LOX inhibitor showing significant improvements in fibrosis grade in bone marrow biopsies corroborate the findings of the trial of our topical LOX inhibitor in established skin scars where we saw a 30% reduction in collagen in skin biopsies after only 3 months treatment,” said Phillips.
“The excellent safety profile of PXS‐5505 makes it an ideal candidate to combine with JAK inhibitors, the current standard of care in myelofibrosis. We anticipate that the impact on bone marrow fibrosis and other clinical parameters from the antifibrotic and intracellular effects of LOX inhibition should lead to improved outcomes for patients.
“We look forward to FDA feedback on our protocol and expect to start recruitment of this next cohort later this year.”
Alfred Chan is a Business Reporter at The Sentiment specialising in ASX-listed small cap companies, a bloodstock enthusiast and former equities analyst.
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