Australian company Pharmaxis (ASX:PXS) has announced results of data analysis from the first of three stages in its phase one trial studying a potential new treatment for the bone marrow cancer myelofibrosis and announced the sale of distribution rights to key products for the Australian and other markets.
The company said that in addition to the good tolerability and consistent pharmacokinetic properties previously announced, the inhibition of two target enzymes, LOX and LOXL2, were assessed with assays and found to be highly statistically significant.
CEO Gary Phillips said, “We are delighted to see that along with excellent tolerability we are achieving levels of LOX and LOXL2 inhibition in myelofibrosis patients that are already exceeding those levels seen in the preclinical models of myelofibrosis.
"In these models, PXS‐5505 caused disease-modifying effects with improvements in blood cell count, diminished spleen size and reduced bone marrow fibrosis. Comparing these results with those achieved in the phase 1 study with healthy volunteers, we are now very confident that we will achieve a level of inhibition of the lysyl oxidase enzyme family that allows us to fully test the clinical relevance of these targets in myelofibrosis patients in the upcoming 6‐month dose-expansion study.”
The company said the second dose cohort of the clinical trial is already fully recruited and dosing of all patients has commenced at participating sites in Australian and South Korean hospitals.
Pharmaxis said the current dose-escalation phase of the study will inform the selection of the optimal dose of PXS‐5505 to be used in the six‐month dose-expansion phase 2a (24 patients) to evaluate safety and efficacy.
The company has also announced the sale of the distribution rights to cystic fibrosis product BRONCHITOL (mannitol) and asthma diagnostic ARIDOL in Australia, New Zealand and several Asian markets.
The company said that under the terms of the sale to Bioimpact, which is a wholly-owned subsidiary of BTC health, it will receive a distributor appointment fee of $2 million within 10 business days.
Pharmaxis said it will continue to manufacture and supply Aridol and Bronchitol to BTC Health from its factory in Sydney, making and exporting the products to the US, European Union, other Asian and Russian markets.
Pharmaxis said the sale will enable it to continued streamlining its mannitol and respiratory business that will deliver ongoing savings in marketing and regulatory expenses.
Australian listed company BTC Health is a Pooled Development Fund that invests in businesses that acquire, develop and distribute innovative medical products in Australia and New Zealand.
Mr Phillips said, "We set out our strategy of generating non‐dilutive cash and cost savings from the mannitol respiratory business late last year when Bronchitol was approved in the United States by the FDA. This agreement with BTC Health is part of that strategy which has also recently seen US$10m in milestone payments received from Chiesi and a A$2m deal with GEN for the Russian Bronchitol rights.
“The initial clinical trials for both Aridol and Bronchito were conducted in Australia and subsequently Australian patients were first to benefit from commercialisation of these two innovative products. Choosing the right distribution partner has therefore been a priority for us and I am delighted to have concluded this agreement with BTC Health who provide first-class support for customers and patients,” said Mr Phillips