Cancer hopeful Pharmaxis (ASX: PXS) is moving ahead with a treatment for a rare bone cancer called myelofibrosis, after a phase 1b study showed it didn’t have any negative effects on the 16 healthy volunteers who took it.

The company is setting up a six-month phase-two study of the oral anti‐fibrotic pan‐Lysyl Oxidase (LOX) inhibitor PXS‐5505 which will study whether it works.

Clinical trials are generally divided into three phases. Phase one focuses on safety, phase two tests for effectiveness and phase three examines whether the new drug is an improvement on existing treatments.

Pharmaxis has received pre-IND (investigational new drug application) feedback from the US Food and Drug Administration (FDA) on the program, as well as discussed the trial outline with key opinion leaders in the US, Europe and Australia.

Key opinion leaders, or KOLs in industry parlance, are essential to building support for a drug or medical device among medical professionals while it is being designed and before it is registered and available for sale. Without this support, companies can find it difficult to jump start interest in a drug or device coming cold into a market.



Prepping for the FDA

Pharmaxis is currently preparing a full IND application for FDA submission mid‐year and plans to start the phase-two trial in the December quarter of this year.

“Pharmaxis believes that the current treatments for myelofibrosis can be augmented by use of a pan‐LOX inhibitor and be disease modifying in a market that is conservatively worth $US1bn per annum,” Pharmaxis CEO Gary Phillips said.

Myelofibrosis is a cancer with a poor prognosis and limited treatment options: stem cell transplants have been shown to work in a small number of patients.

Clinical trials have been shuttered in large numbers over the last two months due to risks associated with COVID19 infections.

But ‘life or death’ trials, where death from the disease is a greater likelihood than any risk from COVID19 infection, are pushing ahead. Most continuing trials are around cancer.


Broad application

The company’s phase 1a and 1b cancer trials data showed the doses at which the drug was delivered strongly inhibited members of the LOX family in tissue and blood.

LOX is an enzyme that stiffens scar tissue. A build up of scar tissue or too much inflammation can lead to fibrosis — a nasty problem in a place like the lungs or liver because it makes it difficult for them to work properly.

Pharmaxis has already had success using the inhibitor treatment in liver condition Non‐alcoholic Steatohepatitis (NASH), diabetic retinopathy, pulmonary fibrosis, and cystic fibrosis.


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Pot stock AusCann (ASX: AC8) has started recruitment and dosing for its first phase I clinical trial to study how its THC/CBD pill moves through the human body. The small study will look at levels of the active ingredients in 28 healthy volunteers.