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)
  • 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.
  • 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)
    • Pharmaxis Treatment to Prevent Wound and Burns Scars Clears Phase 1 Trial (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 Phase 2 clinical trial of a new drug (PXS-5505) targeting the rare bone marrow cancer myelofibrosis (MF).

The results of the 28 day, phase 1c clinical trial to determine the appropriate phase 2 dose were reviewed by the study safety committee. Having identified no safety signals the safety committee  cleared the study to progress to the phase 2 dose expansion stage where 24 patients will be treated at the highest dose twice a day for 6 months. Trial sites have been established in Australia, Asia and the USA.

The phase 2a stage of the trial commenced dosing in October 2021. Read more here.

Assessment with Pharmaxis’ proprietary assays of the highest dose in the 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.

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.

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 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

2021 Annual General Meeting

The 2021 AGM will be held on 3 November 2021 as a virtual meeting. The notice of meeting is available here.

Pharmaxis Treatment to Prevent Wound and Burns Scars Clears Phase 1 Trial

  • Topical treatment safe and well tolerated in study of healthy volunteers
  • Full inhibition of lysyl oxidase enzymes achieved in skin with minimal systemic exposure
  • Collaboration with research team led by Professor Fiona Wood AM planning for trial in patients with established scars to commence in Q4 2021
Read the media release here

Pharmaxis Grants Aptar Pharma Exclusive Option to Develop and Promote High Payload Dry Powder Inhaler

  • Drug delivery solutions developer Aptar Pharma acquires an exclusive 12-month option for a worldwide license to the “Orbital” technology for $US2.5m plus sales royalties
  • Pharmaxis receives a US$250k upfront fee for the option grant
  • Orbital dry powder respiratory inhaler developed by Pharmaxis delivers large payloads of drug in a convenient easy-to-use format
  • Pharmaxis retains rights to inhaled mannitol products delivered via the Orbital inhaler
Read the media release here

Pharmaxis Cancer Drug Decreases Tumour Burden in Pre Clinical Liver Cancer Model

  • Data presented from collaboration of Pharmaxis and University of Rochester Medical Center at US scientific meeting
  • Combination of PXS-5505 and chemotherapy significantly improves survival, delays tumor growth, and reduces intratumoral pressure.
  • Combination of PXS-5505 and standard of care demonstrates a novel therapeutic strategy for liver cancer
Read the media release here

Pharmaxis Quarterly 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. 
https://youtu.be/HddU8IbnOSY

Other recent interviews and articles:  

  • Gary Phillips’ presentation at the Monsoon Communications and Peak Asset Management Biotech Webinar (Monday 27 September 2021). Watch a recording of the webinar here.
  • Alan Kohler's Eureka Report (22 July 2021) "Pharmaxis: The Business of Making Scars Disappear". Listen to the podcast here.
  • Proactive investors: An in depth interview with Gary Phillips and Proactive Investors (10 August 2021): "Pharmaxis updates on bone marrow cancer trial and talks potential of PXS-5505 in liver cancer."  Watch the interview here
  • Emerald Financial: Small Caps Watch - Interview with Gary Phillips (20 July 2021). Watch the interview here.