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Pharmaxis awarded A$1 million to progress Duchenne Muscular Dystrophy drug into the clinic

The Australian Government matched funding under the Biomedical Translation Bridge program will allow the company to take amine oxidase inhibitor PXS-4699 through to the start of human clinical trials.

Pharmaxis Ltd - Pharmaxis awarded A$1 million to progress Duchenne Muscular Dystrophy drug into the clinic
PXS-4699 is expected to protect muscle and reduce inflammation as well as organ fibrosis in DMD

Pharmaxis Ltd (ASX:PXS) (OTCMKTS:PXSLY) (FRA:UUD) has been awarded A$1 million in Australian Government funding to advance work on the company’s drug discovery for the treatment of the devastating genetic disorder Duchenne Muscular Dystrophy (DMD).

The government matched funding under the Biomedical Translation Bridge (BTB) program will allow Pharmaxis to take another one of its pipeline of amine oxidase inhibitors (PXS-4699) through to the start of human clinical trials.

Dual amine oxidase inhibitor

PXS-4699 is a dual amine oxidase inhibitor which is expected to protect muscle and reduce inflammation as well as organ fibrosis in DMD.

Pharmaxis was selected following a highly competitive review conducted by an independent expert evaluation committee as part of the BTB program, which is administered by MTPConnect.

“Potential to make a difference”

MTPConnect managing director and CEO Dr Dan Grant 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.”

A planned comprehensive program of pre-clinical studies will build on the pioneering work already conducted on Pharmaxis compounds by independent international researchers focused on DMD.

It is hoped this could result in better daily functioning for patients, improved quality of life and longer life expectancy.

“Urgent need” for treatments

Murdoch University Centre for Molecular Medicine & Innovative Therapeutics director Professor Steve Wilton said: “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 BTB co-funded project is expected to complete all the steps necessary to commence phase 1 healthy volunteer studies within 12 months and a further study in DMD patients scheduled to start within 18 months.

BTB program

The BTB program is a $22.3 million Medical Research Future Fund initiative that provides up to $1 million in matched funding to nurture the translation of new therapies, technologies and medical devices through to proof of concept to turn innovative medical ideas into reality.

Pharmaxis chief executive officer Gary Phillips said: “Over recent years Pharmaxis investment in drug discovery has progressed three pipeline drugs through pre-clinical 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.”

What is DMD?

Duchenne muscular dystrophy is a genetic condition that affects the muscles, leading to muscle wasting that gets worse over time. It occurs primarily in males, though in rare cases may affect females.

The symptoms of DMD include progressive weakness and loss (atrophy) of skeletal and heart muscles.

Early signs of DMD may include delayed ability to sit, stand, or walk and difficulties learning to speak.

Muscle weakness is usually noticeable in early childhood. Most children with DMD use a wheelchair by their early teens.

Heart and breathing problems also begin in the teen years and lead to serious, life-threatening complications.

While there is no known cure for DMD, there are treatments that can help control symptoms.

DMD is estimated to occur in about 16 live male births per 100,000 in the USA and about 20 live male births per 100,000 in the United Kingdom.

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