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Overview of Pipeline Opportunities in the Clinic for Fibrosis and Inflammation

Pan-LOX inhibitor PXS-5505

(indications in addition to myelofibrosis) 
  • Targeting myelodysplastic syndrome (MDS) and hepatocellular carcinoma (HCC) - the most common type of primary liver cancer.
  • Combination of PXS-5505 and standard of care demonstrates a novel therapeutic strategy for liver cancer. Data presented from collaboration of Pharmaxis and University of Rochester Medical Center at US Scientific Meeting. Read more here.
  • FDA clears Pharmaxis cancer drug to progress to phase 2 study in liver cancer
    • IND approved to trial PXS-5505 added to current standard of care in newly diagnosed unresectable hepatocellular carcinoma patients
    • University of Rochester Medical Center and Pharmaxis in discussions to commence investigator-led study in 2022
    • Read more here.
  • Status: protocols and funding under evaluation 
  • More information here

Topical pan-LOX inhibitor PXS-6302

  • Anti scarring: burns, established scars
  • Status:
    • Pharmaxis treatment to prevent wound and burns scars clears phase 1 trial (read more here)
    • First patient dosed in phase 1c trial of Pharmaxis scar reduction drug (read more here)
  • More information here

LOXL2 inhibitor PXS-5382

  • Anti fibrotic targeting chronic kidney disease (CKD), pulmonary fibrosis (IPF) and non-alcoholic steatohepatitis (NASH)
  • Status:
    • phase 2 ready program
    • partnering discussions
    • phase 2 protocol and funding discussions with independent investigators
  • More information here

SSAO inhibitor PXS-4728

  • Anti inflammatory: neuro inflammation
  • Status:
    • phase 2 ready program
    • partnering discussions
    • phase 2 protocol and funding discussions with independent investigators
  • More information here
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Pharmaxis Clinical Trial Targets Bone Marrow Cancer Treatment

Pharmaxis is advancing a 6-month clinical trial of a new drug (PXS-5505) targeting the rare bone marrow cancer myelofibrosis (MF). The phase 2a open label trial commenced dosing in October 2021 and will recruit 24 adult patients in an open label study of patients who are not on a JAK inhibitor. Participating trial site locations will include Australia, Asia and the USA. The aim of the phase 2 trial is to show PXS-5505 is safe to be taken longer term with the disease modifying effects seen in the pre-clinical models. The study is proceeding under an Investigational New Drug application cleared by the US FDA and is expected to conclude at the end of 2022. Read more here.

Assessment with Pharmaxis’ proprietary assays of the highest dose in the preceding phase 1c trial showed inhibition of the target enzymes, LOX and LOXL2, at greater than 90% over a 24-hour period at day 7 and day 28. Pre-clinical work has established PXS-5505 causes disease modifying effects with improvements in blood cell count, diminished spleen size and reduced bone marrow fibrosis. Read more here.

Watch an interview with CEO Gary Phillips about the phase 1 results here.

PXS-5505 is an anti‐fibrotic pan-Lysyl Oxidase (pan-LOX) inhibitor that has completed long-term toxicity studies and Phase 1a and 1b clinical trials demonstrating a well-tolerated drug that effectively inhibits all enzymes in the lysyl oxidase family that are involved in fibrosis.

Myelofibrosis is a cancer with a poor prognosis and limited therapeutic options. Pharmaxis believes that the current treatments can be augmented by use of a pan-LOX inhibitor and be disease modifying in a market that is conservatively worth US$1 billion per annum.

PXS-5505 was granted Orphan Drug Designation by the US Food and Drug Administration (FDA) in July 2020. Read the media release.

In this article in The Sentiment, Tim Boreham examines our expertise in fibrotic diseases and how it is being applied to find a new treatment for myelofibrosis - a potential billion dollar-plus market. Read the article here.

A study published by researchers at Boston University School of Medicine has found that the two Pharmaxis drug discoveries PXS-LOX_1 and PXS-LOX_2 are showing promising early results in slowing disease progression in primary myelofibrosis (PMF). Read more here: https://www.eurekalert.org/pub_releases/2021-03/buso-bri032221.php

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Recent Highlights

Pharmaxis R&D Showcase Webinars

Pharmaxis Ltd recently hosted R&D showcase webinars for each of the Company’s two lead drug discoveries. Presentations by globally renowned clinicians and scientists involved in the Company’s clinical programs were  followed by Q&A sessions spearheaded by selected panels of biotech analysts and fund managers.

The first webinar was held on 29 March 2022 and featured the Company’s drug PXS-5505 targeting several cancers

The second webinar was held  on 31 March 2022 and featured the Company's drug PXS-6302 designed to target skin scarring.

Recordings of both webinars and copies of presentations are available here.

First Patients Dosed in Trial of Pharmaxis Scar Reduction Drug

  • Collaboration with research team led by Professor Fiona Wood AM doses first patients in trial of 50 patients with established scars
  • 3-month placebo-controlled study to report safety and tolerability endpoints as well as measures of scar structure and appearance in 2H 2022
  • PXS-6302 is a first in class topical inhibitor of the lysyl oxidase enzymes that are involved in formation and maintenance of scars; a potential breakthrough treatment for patients with problematic scars in a market estimated at US$3.5 billion per year.
Read more here

Pharmaxis Drug Discoveries to be Studied by Respected Scientific Collaborators following Government Grants of $1.4m to Advance Cancer and Skin Disease Treatments

  • Associate Professor Thomas Cox from the Garvan Institute of Medical Research has been awarded an $827,500 NHMRC Development Grant to lead a multidisciplinary team investigating PXS-5505 as a promising new treatment approach for pancreatic cancer.
  • Professor Fiona Wood and Associate Professor Mark Fear of the University of Western Australia have been awarded a $590,200 NHMRC Drug Development Grant to work on a novel small molecule from the Pharmaxis pre-clinical pipeline for tissue repair and inflammatory skin disease.
Read more here.

FDA Clears Pharmaxis Cancer Drug to Progress to Phase 2 Study in Liver Cancer

  • IND approved to trial PXS-5505 added to current standard of care in newly diagnosed unresectable hepatocellular carcinoma patients
  • University of Rochester Medical Center and Pharmaxis in discussions to commence investigator-led study in 2022
Read the media release here
Watch an interview with CEO Gary Phillips discussing the trial here

Other recent interviews and articles:  

  • Proactive Investors: "Gary Phillips speaks to Proactive about the company's phase 1c scar tissue trial, led by world-renowned surgeon Fiona Wood AM. The three-month, placebo-controlled clinical study has already dosed its first patient with Pharmaxis' novel, topical drug treatment for scarring. Fifty patients with established scars will participate in the trial as PXS looks to develop a treatment in a market valued at US$3.5 billion a year." (2 February 2022). Watch the interview here
  • Proactive Investors: “Gary Phillips speaks to Proactive following the news an Investigational New Drug application for a trial of PXS‐5505 in hepatocellular carcinoma (HCC) patients has been cleared by the FDA.”  (9 November 2021). Watch the interview here