Adelaide trial brings global hope for vision-loss patients
CMAX researchers pioneer world-first human trial for Retinitis Pigmentosa treatment.
An early-phase clinical trial achievement at CMAX is bringing fresh hope to patients around the world.
In 2017, CMAX principal investigator, Professor Sepehr Shakib, and his team conducted the first-ever human trial of NPI-001, a potential new treatment for Retinitis Pigmentosa (RP) – a devastating condition that causes progressive vision loss and, ultimately, blindness.
Retinitis Pigmentosa is a group of inherited retinal diseases that cause progressive vision loss due to the degeneration of photoreceptor cells in the retina.
Symptoms include a gradual loss of night vision, tunnel vision (loss of peripheral vision), sensitivity to light and eventual loss of central vision in advanced stages.
There is currently no universal cure for RP. Treatment options are limited and usually only benefit a small fraction of patients.
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“RP is genetic, so it affects people from a young age – which is devastating as patients know that they are slowly and progressively going to go blind,” Professor Shakib says.
“The condition affects daily functioning, independence and quality of life.”
The early-phase trial focused on RP associated with Usher Syndrome (USH) – a genetic condition causing combined hearing and vision loss.
The randomised, placebo-controlled, multi-centre trial was conducted at four clinical sites in Australia, involving 49 participants over a two-year period. The aim of the trial was to assess the safety, tolerability and efficacy of NPI-001 for RP associated with Usher Syndrome.
The trial – which was database locked in June 2025 – laid the essential groundwork for what has now become a game-changing moment in medical research.
“We discovered that NPI-001 slowed photoreceptor loss by more than 50 per cent over two years,” Professor Shakib says.
Patients demonstrated nearly 30 per cent slower loss of visual function when taking NPI-001 compared to those receiving the placebo.
The effects were noticeable as early as six months and maintained over 24 months, showing the potential of NPI-001 to slow the progression of RP, giving patients more years of useful vision.
The results, recently announced by Nacuity Pharmaceuticals, were leveraged to design a confirmatory registration trial planned for 2026 and offer new hope to individuals and families affected by the condition.
“These promising clinical trial results have the potential to help this underserved patient population still requiring treatment options,” Professor Shakib says.
In addition to the scientific milestone, the success of the NPI-001 trial highlights the strength of Australia’s clinical research ecosystem. With its robust regulatory framework, diverse participant base and globally respected research standards, Australia continues to attract pioneering studies from around the world.
For CMAX – one of Australia’s largest and most experienced early phase clinical trial units – this achievement reinforces its reputation as a trusted partner in advancing new therapies from the laboratory to real-world use.
The facility delivers successful world-leading research for local and international clients, specialising in a range of early-phase trials and first-time-in-human studies (FTIH).
Trials undertaken at CMAX have resulted in the advancement of global medicines and technologies, from cancer treatment and pain control to early warning bio-devices and hormone replacement therapies.
“Clinical trials are the fastest and safest way to advance medical innovations into safe, effective treatments that have potential to change and save lives,” Professor Shakib says.
