Neuren Pharmaceuticals dips after pediatric Angelman Syndrome Phase 2 trial results fail to impress


Neuren (ASX:NEU) was the biggest faller in lunchtime trades on Friday after announcing some children were “minimally improved” in phase 2 of a trial treating children with Angelman syndrome.

Shares were down -6.2% to just above $16.00ea; Neuren’s results on Friday relate to its experimental oral liquid drug NNZ-2591.

Per assessments by caregivers, 8 out of 12 children showed improvements – that leftover cohort of 4 patients perhaps also being what’s driving risk-off sentiment on Friday, as well as describing improvements as “minimal.”

But investors would have had to open the separate presentation attached to the results to find out the mean score advertised on Friday in the text announcement is classed by the company as “minimally improved” on a slide in its presentation– something pointed out by at least one eagle eyed HotCopper user.

Angelman Syndrome is genetic and causes neurological issues that impede development. Physical characteristics mark the condition; those diagnosed suffer intellectual disability throughout their lives. Seizures and motor issues also define the condition.

“These results provide additional confirmation that NNZ-2591 as an oral liquid dose may address the core symptoms of diverse neurodevelopmental disorders, independent of the origin of the underlying genetics,” Neuren CEO Jon Pilcher said.

The trial ran over 13 weeks in conjunction with three hospitals in Australia. One type of genotype among Angelman Syndrome patients, with ‘mosaicism,’ were not eligible.

Notably – and this is getting off topic – there’s mixed scientific consensus on whether or not children with Angelman Syndrome are more fascinated by water than peers without the condition. At least one study has linked drowning risk with Angelman Syndrome; this is contested elsewhere.

NEU last traded at $16.03ea.


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