A new treatment for the deadly bone marrow cancer myelofibrosis is one step closer to a FDA application with clinical stage drug development company Pharmaxis (ASX: PXS) having successfully completed dosing of the first stage of its current trial.

With the first dosing of PXS-5505 confirmed as safe by the study’s safety monitoring committee, Pharmaxis will now progress to the second dose level for trial participants in Australia and South Korea. This second round of dosing is expected to take four weeks while Pharmaxis has already commenced discussions with potential trial sites in the United States in anticipation for the final round of the dose expansion study.

“PXS‐5505 has demonstrated good tolerability and very consistent pharmacokinetic properties in the first dose escalation stage of MF‐101,” said Pharmaxis CEO, Gary Phillips.

“This is very much in line with the results from our phase 1 healthy volunteer study reported last year. Further data from this first dose will be available in coming weeks but based on the pharmacokinetic profile I anticipate that we will see significant inhibition of all lysyl oxidase enzymes even at this lowest level of dose.”

Myelofibrosis – a type of bone cancer which affects 15 in every 1 million Americans. At present, current forms of treatment are classified by the US Food and Drug Administration to be limited in their effectiveness which was one reason PXS-5505 was graded Orphan Drug Designation and approved to commence their Investigative New Drug trials.

Although prevalence of myelofibrosis is rare, current forms of treatment generate more than $1 billion annually while largely masking symptoms of the disease. Comparatively, pre-clinical trials of PXS-5505 have shown the potential to modify the disease which would be a game-changing treatment for a disease that has an average life expectancy of just five years after diagnosis.

Taken orally, PXS‐5505 inhibits the lysyl oxidase family of enzymes which in turn seeks to reverse the bone marrow fibrosis that advances myelofibrosis.

Unlike many clinical stage research companies, Pharmaxis is in the enviable position of having its trials funded by cash flow from its respiratory pharmaceuticals division which already has various FDA-approved products on the market.

“We’re expecting the respiratory business division to be cash flow positive this year and for years going ahead. “In fact, if we look at five years out, we expect a positive cash flow from that business of about $10 million per annum,” said Phillips in a recent investor briefing.

While myelofibrosis remains the primary focus in the development of PXS-5505, its anti-fibrotic properties have been flagged by academics as a potential treatment for other diseases such as liver and pancreatic cancer. Additional research was confirmed last month when the Charlie Teo Foundation committed funding to explore PXS-5505’s application as a treatment for brain cancer.

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