Biotech Daily
Thursday March 5, 2015
Pharmaust says the second patient in its trial of PPL-1 for cancer has completed the 28-
day treatment at the lowest dose without material adverse events and with stable disease.
Pharmaust said that the Royal Adelaide Hospital patient had bowel cancer and the drug
was “well-tolerated, without material adverse events and the patient ... had stable disease
on completion of the treatment” as determined by computed tomography scan.
The company said that subject to the third patient in the low-dose group completing the
treatment schedule by mid-March, the trial would move to higher doses.
Pharmaust said that in parallel to monitoring patients who had completed the full 28-day
trial period, it was monitoring levels of the biomarker P70S6K in all patients who received
PPL-1 and had stayed on therapy for at least three days.
The company said that a number of patients had to be withdrawn due to reasons
unrelated to the study drug.
Pharmaust said that PPL-1 appeared to define “a new and unexpected mechanism of
action representing a potentially new class of anti-cancer drug”.
The company said that PPL-1 influenced the mammalian target of rapamycin (mTOR)
pathway, but, unlike other mTOR drugs, like Novartis’ Afinitor, or everolimus, and Pfizer’s
Sirolimus or rapamycin, it appeared to have very low toxicity.
Pharmaust said that PPL-1 had not shown an adverse event profile commonly associated
with conventional anti-cancer drugs, the lowest dose of PPL-1 had shown meaningful
reductions in the cancer marker P70S6K and PPL-1 targetted a biochemical pathway that
affected a range of regulatory mediators, without the side effect profile of other mTOR
drugs currently on the market.
Pharmaust was unchanged at 0.8 cents with 4.8 million shares traded.
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