Neuren Pharmaceuticals (NEU: $19.02) announced top-line results for its phase II trial of drug candidate NNZ-2591 in 16 patients with Pitt-Hopkins Syndrome (PTHS).
Clinical Trial Design
The phase II open-label trial involved 16 children with PTHS aged 3-17, conducted in five hospitals in the US over 13 weeks, excluding four weeks of screening and two of post-trial monitoring. Subjects received NNZ-2591 twice daily as a liquid oral dose, escalated to 12mg/kg during the first 6 weeks.
Primary endpoints involved standard safety, tolerability, and efficacy. Secondary endpoints included four measures specifically created for PTHS, as well as 10 non-specific efficacy metrics used to assess a range of neurodevelopmental conditions.
Pitt-Hopkins Syndrome
PTHS is a neurodevelopmental condition caused by a mutation of the TCF4 gene on chromosome 18, affecting around 1/35,000 people. PTHS primarily causes intellectual disability and behavioural abnormalities.
Safety and tolerability
NNZ-2591's safety profile throughout the trial (NCT05025332) was excellent. All adverse effects (AEs) were mild/moderate. Eleven of the 16 subjects completed the trial. Of the 5 who discontinued, one had an inability to complete safety monitoring procedures, and four discontinued due to adverse events that resolved predictably, although in two cases the events were related to NNZ-2591.
Clinical Data
All four PTHS endpoints were statistically significant (p < 0.05), including those subjects who did not complete the study. Changes from baseline were statistically insignificant for the general neurodevelopmental endpoints. "The consistent results on the PTHS-specific assessment affirms the need for syndrome-specific measurements," said Neuren's VP of Clinical Development, Nancy Jones. It should be noted that no placebo arm was included in this study, with the improvement measured from baseline for each participant.
Next Steps for NNZ-2591
This is the second positive Phase II outcome with NNZ-2591, following the results in Phelan-McDermid syndrome at the end of last year. Topline results from the Angelman syndrome study are expected next quarter. The Phase II trial in Prader-Willi syndrome has been paused until a meeting with the FDA to discuss the most recent results, with a view of potentially simplifying the trial protocol following two positive Phase II studies with the drug candidate.
Neuren Pharmaceuticals is capitalised at $2.45 billion.
Bioshares recommendation: Hold
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