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15 October 2021

Institute news

The acquisition of global rights for a novel heart failure drug could help millions of people

Translating pioneering research into the clinicA European biopharma company has acquired the global rights of a novel treatment for people with late-stage heart-failure developed by scientists at the Baker Heart and Diabetes Institute.

The extended-release therapy, developed by leading heart failure researcher, Professor David Kaye and colleagues, has the potential to address the current unmet needs of late-stage heart failure patients.

Moving the discovery from the laboratory through the commercialisation pipeline has been key to this development, with heart failure researcher and investment analyst, Dr Melissa Byrne bringing her unique skillset — honed both here in Australia and the US — to bear in advancing this process, along with the Institute’s Commercialisation Manager, Dr Guy Krippner.

With considerable groundwork already completed, the license agreement now means that the European company can pursue regulatory approval of this drug for clinical use in countries including the US where FDA has agreed to an orphan designation for use in the lead clinical indication to treat patients with left ventricular assist devices (LVAD) that develop right heart failure. Patent claims have been granted in US, Europe Japan and China, with a further claim in Europe pending.

Heart failure is a global pandemic affecting at least 26 million people worldwide and is increasing in prevalence1. Health expenditure on heart failure is considerable and will increase dramatically with an ageing population. Despite the significant advances in therapies and prevention, death and disability are still high and quality of life poor.

The breakthrough offers a patient-friendly and convenient oral treatment of existing drug, Milrinone, with this slow-release oral capsule saving these patients the need for repeated and more invasive IV infusions which often require a hospital stay.

Professor Kaye has witnessed the growing impact of heart failure on the quality of life of his patients — particularly those forced to spend time on the hospital ward away from family — and the development of this novel therapy aims to address these significant quality of life issues.

This advance builds on a successful track record in commercialisation by Professor Kaye, who also heads The Alfred Hospital’s Cardiology Department. The bench to bedside model allowing Professor Kaye to spend his time between the Institute’s labs and the hospital clinics, which are directly linked by a physical walkway, has provided a conducive environment for bringing innovation to an important and growing area of cardiovascular disease where few effective therapies are available.

Professor Kaye says, “Patients with end-stage heart failure — a serious, debilitating disease — experience extreme difficulties breathing, have a very poor quality of life and suffer from multiple, severe co-morbidities”.

“By delivering this treatment in a more convenient oral form, in many cases they are able to avoid frequent hospital stays and time away from their loved ones.

 “Our experience and expertise in translating our research, thanks in part to talented people like Dr Byrne, is helping us to achieve our vision and deliver better health for people globally.”


  1. Savarese G, Lund LH. Global public health burden of heart failure Card Fail Rev. 2017;3(1):7–11

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