MD Anderson to Launch Preclinical Study of Investigational Product Targeting Brain Cancer (Glioblastoma)

MD Anderson to Launch Preclinical Study of Investigational Product Targeting Brain Cancer (Glioblastoma)

Thanks to a grant from an Australian foundation called the Charlie Teo Foundation, a local drug discovery venture will now progress an investigational product to pre-clinical efficacy testing for glioblastoma (GBM), the most common form of brain cancer with an average survival rate of only 15 months from diagnosis. Now Pharmaxis (ASX:PXS) will receive a $AUD 186,837 research grant, which will be distributed to Alan Y. Wang, PhD, associate professor of Cancer Biology at the University of Texas, MD Anderson Cancer, to enable study of PXS-5505. Worldwide, it’s estimated there are more than 300,000 cases of brain and nervous system tumours  each year with GBM being the most common form.

But What is PXS-5505?

As recently shared in the company’s press release, Pharmaxis shared that Lysyl oxidase enzymes play a crucial role in GBM because they attract inflammatory cells that accelerate tumour growth and reduce survival.   

PXS5505 is a potent inhibitor of lysyl oxidases and  should stop these processes. Pharmaxis’ drug has demonstrated a good safety profile in healthy volunteer clinical studies in  Australia. Previous research has already shown the importance of lysyl oxidases to tumour growth in  GBM, and the current research will seek to validate the effectiveness of the Pharmaxis drug in brain cancer models.

As it turns out, the grant helps the company and investigational site to access the expertise and funding to extend potential uses of the drug, which is already entering efficacy trials in myelofibrosis, a rare form of bone marrow cancer, now enrolling patients in a Phase 1c/2 clinical trial.

The Preclinical Study

What comes next is a preclinical study, which typically involves study in various animal models. The hope is, according to the company’s CEO Gary Phillips, that the group could be ready for an investigational new drug application (IND) and in human clinical trials by 2022.

Charlie Teo Foundation

Charlie Teo Foundation is an Australian charity dedicated to funding brain cancer research. Brain cancer kills more children in Australia than any other disease and more young people under 40 than any other cancer. The charity was officially launched by Professor Charlie Teo in March 2018, on the  premise of running a low‐cost charity and funding research differently. To date, Charlie Teo  Foundation has raised over $14 million for brain cancer research.  

The Sponsor: Pharmaxis

Founded in 1998, Pharmaxis Ltd is an Australian pharmaceutical research company with expertise developing drugs for inflammatory and fibrotic diseases and focused on myelofibrosis. Publicly traded on the Australian Securities Exchange since 2003 under the symbol “PXS”, the company has a highly productive drug discovery engine, drug candidates in clinical trials and two respiratory products approved in global markets generating ongoing revenue. Their head office along with R&D is based in Sydney, Australia.

Pharmaxis’ lead drug candidate (PXS-5505) is for the treatment of the bone marrow cancer myelofibrosis, which affects 1 in 500,000 people. In myelofibrosis patients, normal bone marrow tissue is gradually replaced with a fibrous scar-like material, destroying the normal bone marrow environment and preventing the production of adequate numbers of red and white blood cells and platelets. The US Food and Drug Administration (FDA) has granted PXS-5505 orphan drug designation for the treatment of myelofibrosis and has approved an Investigational New Drug application so that clinical trials in myelofibrosis patients can commence.

PXS‐5505 is also being investigated as a potential treatment for other cancers such as liver and pancreatic cancer. PXS-5505 was developed from the Company’s product pipeline, which is founded on its expertise in the chemistry of amine oxidase inhibitors. Drug candidates being developed from the pipeline, which includes a number of candidates.

Lead Research/Investigator

Y. Alan Wang, PhD, associate professor of Cancer Biology at the University of Texas, MD Anderson Cancer