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Pharmaxis given FDA green light to start myelofibrosis phase 2 study

The Food and Drug Administration has completed a safety review of the company’s Investigational New Drug application for the pan-LOX inhibitor PXS-5505.

Pharmaxis Ltd - Pharmaxis given FDA green light to start myelofibrosis phase 2 study
The phase 1/2 clinical trial is planned to begin in the December quarter of 2020.

Pharmaxis Ltd (ASX:PXS) (OTCMKTS:PMXSF) has been given the green light by the Food and Drug Administration (FDA) to proceed with a phase 1/2 clinical trial for the treatment of myelofibrosis in adults.

Permission to proceed follows the FDA completing a safety review of the company’s Investigational New Drug (IND) application for the pan-LOX inhibitor PXS-5505.

The study will incorporate a one-month dose-escalation phase followed by 6 months’ treatment in an open-label study of patients with intermediate or high-risk myelofibrosis.

This includes primary myelofibrosis, post-polycythemia vera myelofibrosis and post-essential thrombocythemia myelofibrosis patients.

“Crucial step forward”

Pharmaxis chief executive officer 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 study is planned to commence in the December quarter of 2020 and is expected to conclude in 2022.

Shares higher

Shares have been as much as 28% intra-day to A$0.14 and are up from A$0.066 at the close on June 30.

The CEO said: "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.”

Inhibiting lysyl oxidase family members

PXS-5505 works by inhibiting all of the lysyl oxidase family members that are involved in the bone marrow fibrosis that characterises myelofibrosis.

It is hoped that this will have beneficial effects on blood cell production and consequently other aspects of this fatal disease which has a very high unmet medical need.

Pharmaxis’ strategy for PXS-5505 is to follow this first phase 1/2 study as a monotherapy with further studies to include myelofibrosis patients being treated with JAK inhibitors which are the existing standard of care for many patients.

“Encouraged by support of clinicians”

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.”

Quick facts: Pharmaxis Ltd

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Market: ASX
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