By Dr Joe Milton, the Australian Science Media Centre

Today, a new drug called Cobenfy (formerly KarXT), which was developed based on a discovery by Aussie researchers, has been approved by the US Food and Drug Administration (FDA) for the treatment of schizophrenia.

Professor Brian Dean from The Florey and the University of Melbourne told the AusSMC that research he and his colleagues conducted in the 1980s and 90s, which showed that muscarinic receptors in the brain are involved in schizophrenia, paved the way for the drug’s development.

“In 2000, we proposed that a drug that activated the muscarinic M1 and M4 receptors would alleviate the symptoms of schizophrenia,” he said. “A drug with that exact mechanism of action has now validated our proposal.”

Cobenfy is a combination of two drugs - xanomeline and trospium – and differs from the drugs that have previously been used to treat schizophrenia, explained Monash University’s Professor Arthur Christopoulos.

“Until now, all available medicines for the treatment of schizophrenia have predominantly been based on science that essentially dates back to the 1950s,” he said.

“Cobenfy represents a whole new class of more targeted medicines.”

Professor Murray Cairns from the University of Newcastle told the AusSMC that one of the drugs in Cobenfy treats the condition, while the other helps prevent side effects.

“Xanomeline… crosses the blood brain barrier… trospium chloride blocks the unpleasant side effects in the rest of the body,” he said.

Xanomeline alone has been trialled before for schizophrenia, he added, but despite reducing symptoms, “the side effects made it unacceptable”.

“The combination is a great step forward in a field that has been difficult to progress,” he said.

Dr Paul Joyce from the University of South Australia said older schizophrenia drugs target the brain’s dopamine receptors, but these can frequently lead to poor treatment outcomes or negative side effects, leading many patients to abandon treatment.

“By targeting cholinergic receptors in the brain, rather than dopamine receptors, as is the case for existing anti-psychotics, [Cobenfy] offers promise to schizophrenia patients who have exhausted the current standard of care or have suffered from poor treatment outcomes.”

Prof Dean said he believes the drug based on his earlier work will help people with schizophrenia.

“The hope is this drug… will be effective in people resistant to treatment with existing drugs and it will alleviate negative and cognitive symptoms of the disorder that do not respond to current drug treatments,” he said.

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This article originally appeared in Science Deadline, a weekly newsletter from the AusSMC. You are free to republish this story, in full, with appropriate credit.

Contact: Joseph Milton

Phone: +61 8 7120 8666

Email: info@smc.org.au