FDA greenlights Neuren Pharmaceuticals to kick-off next trial for Rett drug

Melbourne-based Neuren Pharmaceuticals (ASX: NEU) reported the US Food and Drug Administration has given the greenlight for the company to start phase three trials for its drug trofinetide, which is used to treat people with Rett syndrome.

Neuren Pharmaceuticals executive chairman Richard Treagus said the FDA meeting provided necessary confirmation on key issues relating to the company’s proposed phase three trial.

“We are now able to progress the final stages of development with full confidence,” he said.

Rett syndrome is a genetic neurological condition found almost exclusively in females. It causes loss of language and motor function. In atypical cases, it can lead to growth retardation and scoliosis.

Onset typically starts between six to 18 months of age and development slows or reverses.

Current treatment options are limited and include: physiotherapy, occupational therapy, horseback riding, music and hydrotherapy.

Neuren Pharmaceuticals’ phase three trial will be double-blind, randomised and placebo-controlled and comprise a six-month treatment time. Outcomes will be measured using the Rett syndrome behaviour questionnaire and the clinical global impression of improvement (CG-I).

In previous human trials, trofinetide use resulted in “meaningful improvement”.

Because Rett syndrome sufferers live well into adulthood and require 24-hour care, the condition has a large economic impact, as well as the emotional cost to families.

Life-long medical care for Rett syndrome sufferers is estimated at US$20,000 annually for each patient.

In animal testing, trofinetide has demonstrated benefits including inhibiting neuroinflammation, normalising the role of microglia, correcting synaptic function and regulating the oxidative stress response.

The drug is also under trial for treating Fragile X syndrome and the FDA has approved fast track designation, with trofinetide also achieving orphan drug designation in the US and Europe.

Neuren Pharmaceuticals is developing new drug therapies for neurodegenerative disorders, brain injury and neurodevelopment.

Currently, no drugs are approved for treating these conditions and few in development.