Last month (14 March) the Foundation for Prader-Willi Research...

Last month (14 March) the Foundation for Prader-Willi Research held a webinar for Neuren’s current Phase 2 clinical trial of NNZ-2591 in PWS. The webinar provided a few new insights.

As per the clinical trials website, the PWS trial commenced on 9 January. Unlike the three other Phase 2 trials, this trial is testing sequentially in just two, rather than three age groups.

Interest in the trial is said to be good, especially from the younger 4-7 age group, but the start to the trial wasn’t great due to a series of screening failures. Several more screenings in the older age group were scheduled for mid-April and once safety clearance is given following the first 3 participants being dosed for two weeks, screening in the younger age group can commence. If all goes well, this could happen before the end of June.

There will be 9 sites recruiting for this trial, compared with the 3-5 sites used in the other three trials. Neuren will cover all travel costs and will accept participants from outside the US, although they would need to stay in the US through the course of the trial.

Trial completion is anticipated for the end of this year or Q1 next year and, if there is success in Phase 2 and 3 and NNZ-2591 proceeds to registration,  a rough estimate for time to market is 4-6 years.

Towards the end of the webinar DCCR is mentioned. This is Soleno Therapeutics’ once-daily drug for PWS. Soleno anticipates filing an NDA for DCCR in PWS within the next couple of months. If approved, it will become the first approved drug for hyperphagia in PWS.

Reduction in hyperphagia is the primary outcome for DCCR but the drug has also demonstrated significant benefits in PWS beyond hyperphagia across a number of studies, albeit not in the original pivotal trial that completed in 2020.

Significant change has been seen as measured by the CGI-I. On the PWSP Domain scale, significant improvements have been seen in aggressive behaviours, irritability/rigidity, anxiety, disordered thinking, and compulsivity. On the DBC-2 scale, significant improvements were observed in communication disturbance and social relating.

DCCR has also demonstrated significant change from baseline in body fat mass and in key hormonal and metabolic markers.

While a number of mild-moderate adverse events were reported, the incidence was slightly below those reported in the placebo group.

I am expecting that DCCR will likely be approved by this time next year.

https://www.fpwr.org/blog/pws-clinical-trial-webinar-nnz-2591
https://jneurodevdisorders.biomedcentral.com/articles/10.1186/s11689-024-09536-x
https://investors.soleno.life/