In additional to the lucrative royalty stream and milestone payments Neuren Pharmaceuticals (NEU: $14.68) is receiving from its first drug DAYBUE, the company is also conducting four clinical studies in four separate neurodevelopmental disorders with its next drug candidate, NNZ-2591.
The most advanced of these is in Phelan-McDermid syndrome. The Phase II study started in August last year in the US with a trial size of around 20 subjects. That study is now complete with results expected next month.
NNZ-2591 may even be more effective than DAYBUE, having achieved consistently good preclinical data and with an improved bioavailability over DAYBUE, although the company is addressing different indications to Rett syndrome, for which DAYBUE is approved. (Neuren's partner has the rights to NNZ-2591 for the treatment of Rett and Fragile X syndromes).
NNZ-2591 is delivered orally (liquid) twice a day at a significantly lower dose than DAYBUE due to its absorption profile. The endpoint CGI-I score (Clinical Global Impression of Improvement) was used as one of the primary endpoints in the Rett studies and will be one of the endpoints (of approximately 10) used in the current studies with NNZ-2591.
Neuren is applying some learnings from the development of DAYBUE to NNZ-2591. The first of these is that the company will assess the impact of the drug candidate in children from the start, not in adults. Rather than treating patients for just four to six weeks, the Phase II studies have a longer 13-week treatment period to better gauge effect from the treatment. The company is also not including what CEO Jon Pilcher calls an 'underpowered' placebo arm, with only active trial arms. And the company is using just the one dose, with the company confident of the (maximum effective) dose that has been selected.
Neuren’s other programs are in Pitt Hopkins syndrome (also started in August last year in the US), in Angelman syndrome (started in Australia in July last year) and in Prader-Willi syndrome (started in the US in July this year).
If the results are positive in any of the four studies, Neuren intends to proceed to a registration study, which may not need to be as large as Acadia's Phase III Rett syndrome trial (in 187 subjects) if there is a stronger signal from any of the Phase II studies.
The company is well funded to conduct those studies with $230 million in cash at the end of September.
Neuren is capitalised at $1.9 billion.
Bioshares recommendation: Hold
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