Investor Centre

Pharmaceutical research company Pharmaxis Ltd (ASX: PXS) today announced it has enrolled the first patient in a clinical trial studying a potential new treatment for the bone marrow cancer myelofibrosis.

The phase 1c/2a trial cleared by the FDA under the Investigational New Drug (IND) scheme aims to demonstrate that PXS-5505, the lead asset in Pharmaxis’ drug discovery pipeline, is safe and effective as a monotherapy in myelofibrosis patients who are intolerant, unresponsive or ineligible for treatment with approved JAK inhibitor drugs.

Pharmaxis has completed site initiation at several Australian and South Korean hospitals and the first patient has been enrolled. The dose escalation phase of the study that aims to select the optimum dose of PXS-5505.  This first phase, that will recruit up to 18 patients, is expected to conclude and report in 2H 2021 and will be followed by a six-month dose expansion phase (24 patients) to evaluate safety and efficacy. Sites in other countries including the USA will be added for the dose expansion phase.

Pharmaceutical research company Pharmaxis Ltd (ASX: PXS) has announced it has exported the first shipment of its locally developed and manufactured drug Bronchitol®(mannitol) to the USA. The cystic fibrosis (CF) treatment was approved by the US Food and Drug Administration (FDA) on 30 October 2020. Following receipt of an initial payment of US$7 million (~A$10 million) from its exclusive US distributor Chiesi, Pharmaxis will now receive a further US$3 million (~A$4 million) milestone payment.

Pharmaceutical research company Pharmaxis Ltd (ASX: PXS) has today received a US$7 million (~A$9.2 million) milestone payment from its US licensee Chiesi Farmaceutici S.p.A. (Chiesi) following the recent approval by the US Food Drug Administration of Bronchitol®(mannitol) for the treatment of cystic fibrosis.

A further US$3 million is payable by Chiesi on shipment by Pharmaxis of commercial launch stock, scheduled for the first quarter of 2021.

Pharmaxis reported cash funds of A$10 million at 30 September 2020 to which it has since added a R&D tax incentive of $5 million in October and this milestone of A$9 million.

On 2 November 2020 it was announced that the FDA had approved Bronchitol, the drug Pharmaxis developed, for the treatment of adult cystic fibrosis patients in the United States.