Having delivered impressive interim trial data from their Phase 2 clinical trials for a treatment targeting incurable bone marrow cancer, the US Food & Drug Administration (FDA) could soon be the next stop for biopharmaceuticals company Pharmaxis (ASX: PXS) fresh off their poster presentation at the American Society of Hematology (ASH) conference.
Hosted in New Orleans it is deemed one of the premier gatherings of scientists and clinicians in this field of research. This year’s conference was attended by more than 20,000 people where Pharmaxis presented clinical evidence on the disease modifying effect of its pan LOX inhibitor PXS‐5505 in the bone marrow cancer myelofibrosis.
The presentation materials primarily focused on interim data recorded from their MF-101 trial which has dosed 6 myelofibrosis patients with PXS-5505. The data proved the drug to be well tolerated and inhibited the target enzyme in 5 of the 6 patients which reported improved bone marrow fibrosis scores, as well as stable or improved platelet or haemoglobin scores.
Gary Phillips, Pharmaxis CEO said, “It is very encouraging to see the many positive responses at ASH to the pioneering work Pharmaxis has been conducting in the role of lysyl oxidase in a variety of diseases.
“For our pan‐LOX inhibitor, PXS‐5505, there is increasing data that we have a safe and well tolerated drug achieving high target engagement and with the potential to make a real difference to patients, not only in myelofibrosis but also other myeloid neoplasms.”
With there being no cure for myelofibrosis, existing treatments primarily mask the symptoms of patients which are typically given less than five years to live once diagnosed with the disease. Still, these treatments generate more than USD $1 billion per year.
Although the interim data published reflects the first six patients dosed in the Phase 2 trials, more have since entered the 24-week trial which is scheduled to report full results data around June 2023.
As Pharmaxis moves closer to reporting the full data for MF-101, Phillips is set to resume dialogue with the US FDA as part of Pharmaxis’ process to commercialise their drug, or unlock additional opportunities to realise shareholder value.
“Based on the ongoing data from MF‐101 we will schedule discussions with the FDA in Q1 2023 on the next steps of clinical development for PXS‐5505 in myelofibrosis.”
With further Phase 2 trials on the agenda the commercial opportunities for Pharmaxis continue to grow given the myelofibrosis treatment market is tightly held by existing drug manufacturers that have limited new entrants into the market.
This was evidenced in 2021 when Constellation Pharmaceuticals was acquired for $1.7 billion after completing Phase 2 trials of their myelofibrosis treatment .
Beyond interest in Pharmaxis from the scientific field at the ASH conference in New Orleans, institutional investment has also followed the impending results release. Fund management firm Platinum Asset Management recently entered Pharmaxis’ share register for the first time in October 2022 after acquiring a 5.06% equity stake for $1.9 million which has since increased to 7.63% for $3.3 million.
With more than $17 billion in funds under management and their success available to all to see, Platinum Asset Management was founded in 1994 and delivered returns of 11.2% per annum since 1995.
The Pharmaxis presentation materials at the ASH conference are available here.
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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