Biopharma Neuren has received initial approval from the US government to proceed with a new trial of a drug to treat the incurable genetic disorder Rett syndrome.

Rett is found almost exclusively in females and gradually causes developmental delays — often with symptoms of a loss of control of hands or ability to vocalise sounds or words.

Neuren (ASX:NEU) has so far completed successful Phase 2 trials. The next trial  would move its trofinetide drug to one stage away from commercialisation.

Neuren shares rose 8 per cent to 8.5c on Friday, close to the 12-month high achieved after when the company announced its Phase 2 success in March.

In most patients, the onset of the disorder starts between six and 18 months of age when head growth stops. Symptoms then become more aggressive such as losing any purposeful use of hands and habitually wringing or rubbing hands together.

Rett Syndrome affects 350,000 females

There are about 200 girls in Australia who have been diagnosed with this condition that affects approximately 1 in 15,000 females.

Globally, some 350,000 girls and women are affected including 15,000 in the United States.

Treatment to this point has focussed on the management of symptoms – often described as similar to autism – but Neuren’s earlier study showed signs of potential biological change.

“The results provided evidence of biological activity across multiple symptom areas, indicating the potential for disease modification rather than simply addressing isolated symptoms,” Neuren told investors in its half-yearly report.

“Improvements from baseline were considered by the leading physicians to be clinically meaningful.”

The phase three trials will be conducted over six months. Chairman Richard Treagus said the latest approval allowed the company to progress to final development with confidence.

Rett Australia President Bill Callaghan was optimistic of the latest development: ” In Australia the disease affects roughly 500-600 families, it is a rare condition and often these tend to be left on the back burner so we are so happy to hear Neuren are one step closer. We’re confident there will be a wider treatment field as the research continues.”

Neuren raised $11.5 million in a July placement in preparation for the phase three study but will have to consider options, including partnership, to fund and execute the next stage.

In the last half-year the company burned through $3.6 million, leaving $1.3 million in the bank at the end of June.

The company has been contacted for comment.

More information:

Rett Syndrome Association of Australia –
Grace For Rett Syndrome –