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Australia trial commences for ‘game-changer’ retinal disease eye drop

A clinical trial is under way in Australia for a novel eye drop treatment for retinal vascular diseases that could replace the current approach involving serial intravitreal injections into the back of the eye.

The radical advancement, which could see eye drops become a main form of treatment for diabetic macular oedema and neovascular age-related macular degeneration, stems from research conducted by UNSW Sydney.

Based on initial work carried out at the UNSW School of Chemistry, Professor Jonathan Morris collaborated with ophthalmic biopharmaceutical company Exonate to design the molecule used in the trial called EXN 407.

Professor Jonathan Morris

“The new eye drop treatment is an excellent example of UNSW researchers being able to translate their research into the clinic,” Morris said.

“I’m thrilled to be involved in a collaborative project that could result in a game-changing therapy for retinal vascular diseases. Eye drops offer patients a more effective and accessible treatment that can be self-administered in a painless and convenient way.”

Until now, drug developers have failed to create VEGF inhibitors that could be delivered to back of the eye in high enough concentrations, without injections.

Scientia Professor Fiona Stapleton

UNSW researchers identified novel compounds that modulate the growth factor causing retinal vascular diseases. As they are absorbed in the eye, the compounds allow retinal vascular disease treatment to be given via eye drop rather than an intravitreal injection EXN 407 inhibits serine/arginine-protein kinase 1 (SRPK1). SRPK1 enables production of VEGF, which initiates or inhibits vessel formation depending on alternative splicing.

Exonate, a company focused on retinal diseases, is collaborating on the trial with Janssen Pharmaceuticals, a pharmaceutical company of Johnson & Johnson.

The double-blind, randomised trial is being conducted at centres across Australia, including UNSW’s School of Optometry and Vision Sciences, in 48 diabetic patients with macular oedema.

It consists of a dose escalation phase during which three doses of EXN 407 and a placebo are tested, followed by an expansion phase with a larger cohort of patient volunteers and a longer drug dosing period. The study aims to demonstrate safety and tolerability and an exploratory end point of efficacy through reduction in retinal thickness in a proportion of patients.

With Australia’s effective handling of COVID-19, no major delays are expected in patient recruitment, with topline results anticipated in early 2022.

Dr Catherine Beech, CEO of Exonate, said the first clinical trial is an important step in the validation of the eye drop approach.

“This is a unique opportunity to create a drug that may have the potential to improve the treatment of patients with retinal vascular diseases and transform the lives of those suffering from vision loss,” she said.

Scientia Professor Fiona Stapleton at UNSW’s School of Optometry and Vision Sciences said: “This project reinforces [our] expertise in clinical trials for ocular pharmaceuticals. We are excited to be able to participate in the clinical testing of this novel and much needed treatment for retinal disease.”

Professor Nicholas Fisk, Deputy Vice-Chancellor (Research and Enterprise) at UNSW, commended UNSW research for its potential to improve healthcare worldwide and attract international investment from venture capital, including UniSeed, as well as Big Pharma.

“This is an exemplar of UNSW initiated technology being pivotally commercialised with international partners to deliver healthcare benefits and societal impact,” he said.

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