Sydney-based Pharmaxis (ASX:PXS) says the US FDA has completed a safety review of the company’s Investigational New Drug application for its pan-LOX inhibitor PXS-5505 and given it permission to proceed with a new clinical trial.
Pharmaxis said PXS-5505 works by inhibiting all of the lysyl oxidase family members that are involved in the bone marrow fibrosis that characterises myelofibrosis.
This could have beneficial effects on blood cell production and consequently other aspects of what is a fatal disease.
Pharmaxis said its strategy for PXS-5505 is to follow this first phase one-two study as a monotherapy with further studies to include myelofibrosis patients being treated with JAK inhibitors, the existing standard of care for many patients.
The company said the phase one-two trial will incorporate a one-month dose-escalation phase followed by six months treatment in an open-label study of patients with intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera myelofibrosis and post-essential thrombocythemia myelofibrosis patients.
The study is planned to commence in late 2020 and expected to conclude in 2022.
CEO Pharmaxis Gary Phillips said, “The positive FDA feedback on our IND is a crucial step forward for Pharmaxis in advancing its pan-LOX anti-cancer program which has already shown compelling evidence of efficacy in a number of pre-clinical cancer models.
"The company is well advanced in its preparations to start this study including production of the drug product, assigning a contract research organisation to manage the study and completing feasibility in a number of countries. We can therefore progress to initiate patient recruitment in Q4 2020 and respond as needed to the rapidly changing availability of trial sites caused by Covid-19 outbreaks.”
Mr Phillips added, “The IND application was a significant body of work containing over 20,000 pages of reports on the phase 1 studies in healthy volunteers, numerous individual pre-clinical studies and manufacture of the drug substance and drug product to be used in human clinical trials.
"We have been very encouraged by the support of clinicians who treat myelofibrosis and the researchers who have conducted many of the pre-clinical studies. We will announce further details of the upcoming trial after completing discussions with our partners and collaborators.”