The trial will assess the safety and effectiveness of a gene therapy designed to temporarily reduce cholesterol levels.
The Victorian Heart Hospital will shortly begin recruiting patients for the first-in-human trial of STX-1150, a new genetic therapy designed to lower cholesterol in people with an increased risk of cardiovascular disease.
LDL cholesterol, the “bad” kind of cholesterol, is a key driver behind atherosclerotic cardiovascular disease such as heart attacks and strokes. STX-1150 has been designed to reduce LDL levels by targeting the PCSK9 gene, known to regulate cholesterol levels and cardiovascular risk.
The gene therapy, delivered as a once-off infusion, will last for an unspecified period of time but will not permanently alter the DNA of the person being treated.
Up to 64 patients will be enrolled in the phase 1 study, which will test the safety, tolerability and biological effects of STX-1150 over a 12-month period. Recruitment will initially occur at the VHH before expanding to other sites.
Professor Stephen Nicholls, director of the VHH and principal investigator on the new study, described the trial as an important step forward in finding more long-term ways to prevent cardiovascular disease.
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“The best way to treat heart disease, the leading cause of death globally, is to prevent it,” he told media.
“While existing cholesterol-lowering therapies are highly effective, many people still struggle to maintain long-term treatment due to cost, access, side effects or the burden of ongoing medication.
“This new investigational therapy is designed to provide sustained cholesterol reduction following a single treatment, which could significantly change how we manage cardiovascular risk in the future. This represents the new frontier of cardiovascular medicine.
“It is incredibly exciting that Victorians and Australians will again play a leading role in the development of next-generation genetic therapies for heart disease.”
STX-1150 was developed by Scribe Therapeutics.
