home page 2 May 21

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)
  • Status: protocols and funding under evaluation 
  • More information here

Topical pan-LOX inhibitor PXS-6302

  • Anti scarring: burns, established scars
  • Status: Phase 1 Investigator Initiated Studies (IIS) commenced Q1 2021 (read more here); IIS patient studies in burns and established scars to commence H2 2021
  • 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 Targets Myelofibrosis in New Clinical Trial

Pharmaxis is advancing a 6-month Phase 2 clinical trial of a new drug (PXS-5505) targeting the rare bone cancer myelofibrosis (MF).

Dosing has been completed in two of three stages of the phase 1c clinical trial and third level dosing commenced in August 2021 following successful  patient recruitment at  Australian and South Korean hospitals.

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.

Pharmaxis used its proprietary assays to examine results from the first 1c trial patient cohort and has concluded the inhibition of the two enzymes being targeted in the trial, LOX and LOXL2, are highly statistically significant.

Read more here.

In the second 1c trial patient cohort the increase in dose lead to a predictable increase in drug blood levels in patients and showed the same good tolerability seen in the first dose cohort.

Read more here.

Watch an interview with CEO Gary Phillips about positive first trial 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 August 2020 the FDA completed a review of the company’s Investigational New Drug (IND) application for PXS-5505 and gave Pharmaxis the green light to proceed with a phase 1c/2a clinical trial for the treatment of myelofibrosis in adults.  The clinical trial protocol incorporates a one-month dose escalation phase followed by six months’ treatment in an open label study of patients who are not on a JAK inhibitor.

The first patient was enrolled in Q1 2021 and the study is expected to conclude in 2022.

Read the media release here.

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

GP and CT

Recent Highlights

Pharmaxis Sells Australian Bronchitol and Aridol Distribution Rights for A$2m

  • Pharmaxis sells distribution rights for Bronchitol and Aridol in Australia to BTC Health Ltd effective July 1st 2021
  • Pharmaxis to receive A$2 million distributor appointment fee
  • Streamlining mannitol respiratory business in line with strategy

Read the media release here

Pharmaxis Investor Briefing

Watch a recording of the Pharmaxis Ltd June Quarterly Shareholder Update by the chief executive officer Mr Gary Phillips on Thursday 29 July 2021. 

Pharmaxis and Charlie Teo Foundation Identify Promising New Drug to Tackle Brain Cancer

An Australian drug discovery will be the focus of a new study to tackle an aggressive and deadly form of brain cancer with a funding grant from the Charlie Teo Foundation. The drug, developed by Sydney-based research company Pharmaxis (ASX: PXS), will enter pre-clinical efficacy testing for glioblastoma (GBM), the most common form of brain cancer with an average survival of only 15 months from diagnosis.

The Charlie Teo Foundation, an Australian charity, has awarded a $AUD 186,837 research grant to Y. Alan Wang, Ph.D., associate professor of Cancer Biology at The University of Texas MD Anderson Cancer Center to enable study of the Pharmaxis drug, known as PXS-5505.

Read the media release here.
See the Channel Nine News story here: https://twitter.com/9NewsSyd/status/1388050205971566593?s=09
Watch an interview with CEO Gary Phillips here

Pharmaxis Sells Bronchitol Russian Distribution Rights for A$2m with Annual A$1m Cost Savings

  • Pharmaxis sells distribution rights for Bronchitol in Russia effective May 1st 2021
  • Pharmaxis to receive a €1.25 million (~A$2m) distributor appointment fee
  • Annual cost savings of approximately A$1m realised from transfer of commercial and product responsibilities
Read the media release here

Pharmaxis Announces World First Clinical Trial of Treatment to Prevent Wound and Burns Scars

A Pharmaxis drug discovery has entered a world first clinical trial aiming to stop scars forming after trauma, particularly following burn injuries. The company is collaborating with renown burns specialist Prof Fiona Wood and a team at the University of Western Australia to tackle the underlying fibrosis causing scar tissue using the promising Pharmaxis compound PXS-6302.

Read the media release here.
Watch an interview with CEO Gary Phillips here

Other recent interviews and articles:  

  • Emerald Financial: Small Caps Watch - Interview with Gary Phillips. Watch the interview here.
  • The Inside Investor: "Pharmaxis flicks the switch to self-funded trial program" (1 June 2021). Read the article here.
  • Bioshares: "Karst Peak Continues Investment in Australia Biotech". (19 April 2021). Read the article here.  
  • The Sentiment:  "Karst backs Aussie small cap following hugely successful pharma strategy" (26 April 2021). Read the article here.