Australian Pharma Bags Government Funding to Fight Duchenne Muscular Dystrophy
By T. Chakraborty, Ph.D.
Duchenne muscular dystrophy (DMD) is a rare muscle disorder affecting approximately 1 in 3500 male births globally. DMD is characterized by weakness and atrophy of the muscles in the pelvic area, followed by the shoulder muscles. DMD can be detected between the ages of 2-6 years, and most children end up using a wheelchair by their teens . As of now, there exists no known cure for DMD. However, certain treatments can help control symptoms.
Australian company, Pharmaxis Ltd., has been recently awarded A$1 million by the Australian government to make headway against DMD. The funding provided by the government is under the Biomedical Translation Bridge (BTB) program will propel Pharmaxis to take one of their pipeline drugs, an amine oxidase inhibitor (PXS-4699), to human clinical trials.
Amine oxidases are key enzymes involved in oxidative stress processes. Dysregulation of this critical enzyme leads to reactive oxygen species resulting in muscular degeneration and inflammation in DMD. Hence, the inhibition of amine oxidases is a lucrative drug target. PXS-4699 is a dual amine oxidase inhibitor, which, if successful, has the potential to reduce muscular degeneration and inflammation in DMD patients.
Following a highly competitive and rigorous process conducted by an independent agency and administered by MTPConnect, Pharmaxis emerged victorious. Managing Director and CEO, Dr. Dan Grant of MTPConnect said: “The Pharmaxis research has potential to make a difference to patients with Duchenne Muscular Dystrophy (DMD), a debilitating genetic disorder affecting thousands of Australians. It’s an example of the high-quality research translation being generated in Australia, which will progress to clinical trials.” 
Professor Steve Wilton, Murdoch University Centre for Molecular Medicine & Innovative Therapeutics’ Director, added: “New treatments, including gene and molecular therapies to restore dystrophin are showing promise for DMD. However, DMD and other muscular dystrophies are multisystem disorders with complex pathologies, and there is an urgent need for additional or complementary treatments to reduce the fibrosis and inflammation that are hallmarks of the disease. PXS-4699 is a powerful new tool in the repertoire of potential treatments for DMD and targets the inflammatory pathway and fibrosis that are so detrimental in dystrophies. We see PXS-4699 as an additional or adjunct therapy for muscular dystrophies.” 
This ambitious project, co-funded by BTB, is expected to finish all necessary steps required to progress into Phase 1 clinical trials consisting of healthy volunteers within the time frame of a year. Additionally, it is also expected that the company investigates further studies in DMD patients within 18 months.
The BTB program comprises a $22.3 million Medical Research Future Fund initiative that can provide up to $1 million in matched funding to help bridge the gap in bringing new therapies, medical devices, and technologies from proof of concept to reality.
Gary Phillips, Chief Executive Officer of Pharmaxis, said: “Over recent years Pharmaxis investment in drug discovery has progressed three pipeline drugs through preclinical testing to the commencement of human clinical trials and beyond. Non-dilutive funding sources, such as this one provided by the Australian Government’s BTB grant program, will allow us to similarly progress PXS-4699 in an orphan disease with high unmet need whilst not detracting from the focused investments we are making in a myelofibrosis treatment and as we await the upcoming FDA decision to grant a marketing authorisation of our cystic fibrosis treatment for patients in the United States.” 
Editor: Rajaneesh K. Gopinath, Ph.D.
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