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Neuren Pharmaceuticals (ASX:NEU) ends enrolment pause in phase three trial

Health Care
ASX:NEU      MCAP $2.693B
16 June 2020 14:00 (AEST)

Drug developer Neuren Pharmaceuticals’ (NEU) U.S. partner ACADIA has lifted the temporary enrolment pause for the phase three trial of its trofinetide drug.

The LAVENDER trial is testing the therapy in woman and girls with Rett Syndrome, a neurodevelopmental disorder arising from a rare gene mutation.

Virus hiatus

The LAVENDER study was underway before the onset of the COVID-19 pandemic, but had not enrolled the full study cohort.

ACADIA was forced to suspend enrolment in the trofinetide Rett Syndrome study, due to risks associated with clinic visitation and lack of medical resources during the pandemic.

The study is seeking to evaluate the efficacy and safety of trofinetide against a placebo in around 180 girls and young women aged between five and 20 years.

There are still certain difficulties associated with the trial, including site availability and local health guidelines, which may affect enrolments. ACADIA assures any potential enrolees it will be “taking appropriate measures to safely and effectively collect patient data to ensure the integrity of study results.”

Next steps

Enrolment in LAVENDER, and its 40-week LILAC extension study, will now continue where possible across the U.S.

Neuren and ACADIA will also continue the phase two trial of trofinetide in Fragile X syndrome.

Neuren is also undertaking trials of its novel NNZ-2591 drug for Phelan-McDermid, Angelman and Pitt Hopkins syndromes, each of which has received orphan drug designation in the United States.

Neuren Pharmaceuticals is trading 4.7 per cent higher today, at $1.57 per share as at 12:05 pm AEST.

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