Ann: Phase I Trial at RAH Meets Safety & Cancer Marker End Points, page-3

(“PharmAust”) (ASX: PAA & PAAO) is pleased to report on the successful closure of its Phase I (Phase IIa) “first in man” trial at the Royal Adelaide Hospital and confirmation that the last patient, who was treated at the higher dose of PPL-1 (25mg/kg), showed meaningful suppression of key cancer marker p70S6K. Importantly, during the trial, both principal end points were successfully met, namely:
1. PPL-1 demonstrated a very good safety profile as compared with many other established anticancer drugs, and
2. PPL-1 showed activity against cancer through the suppression of a key cancer marker.
In the trial, seven patients were treated with PPL-1 for various time periods and measurements were successfully taken for anticancer activity through marker suppression (p70S6K). Three patients completed the full 28-day treatment period. One patient was not included in the cancer marker results as they only received a single dose of the drug. One patient received the higher dose of PPL-1 (25mg/kg).

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PharmAust’s Executive Chairman Dr Roger Aston said “We have now completed the “first stage” of studies with PPL-1 in humans and canines and we have shown that in both cases PPL-1 is well tolerated and importantly is active against cancer. The suppression of tumour marker, p70S6K, in man was highly significant when the data from 7 patients is combined and analysed (at day 3 of treatment p<0.0004 and at day 7 of treatment p<0.002). We have furthermore initiated the processes to move to the important next stage, which will include the treatment of patients with a combination of “Standard of Care” (chemotherapy drugs) in the presence of PPL-1. For the next stage of human work PharmAust will reformulate the drug into capsules, as the main challenge faced in both canine and human trials with liquid PPL-1 was the poor palatability of the formulation and nausea from the unpleasant taste”.