South Australia research institute CMAX has announced success with a potential new treatment for retinitis pigmentosa (RP).
RP is a devastating condition that causes progressive vision loss and, ultimately, blindness. There is no cure and limited treatment options.
The Adelaide-based facility said achievement in an early-phase clinical trial it ran would give hope to those sufferers around the world.
CMAX principal investigator Professor Sepehr Shakib said the first-ever human trial of NPI-001, a potential new treatment, showed that photoreceptor loss could be slowed by more than 50% over two years.
The randomised, placebo-controlled, multi-centre trial was conducted at four clinical sites in Australia.
It involved 49 participants and the aim of the trial was to assess the safety, tolerability and efficacy of NPI-001 for RP associated with Usher syndrome.
Media reports said the trial laid the groundwork for what could be a game-changing moment in medical research.“
Patients demonstrated nearly 30% slower loss of visual function when taking NPI-001 compared to those receiving the placebo, said Prof Shakib.
He said 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 have been used to design another trial planned for 2026.
“These promising clinical trial results have the potential to help this under-served patient population still requiring treatment options,” he said.
