PHASE2 PRADER WILLI SYNDROME START !!!!, page-43

Although Soleno’s primary endpoint for DCCR in PWS relates to hyperphagia (constant hunger), and it will be approved (or not) on that basis, there is clinical evidence that suggests that DCCR can also positively impact aggression, anxiety, compulsivity/rigidity, depression and disordered thinking.

And although Acadia’s proposed intranasal carbetocin treatment for PWS also targets hyperphagia as its primary endpoint, there is clinical evidence that suggests that carbetocin can also be beneficial for anxiousness and distress.

As for concerns that Soleno’s or Acadia’s drug, or NNZ-2591, might be “abused” by those seeking weight loss – I would think that a high list price will prevent that.

Without insurance, Ozempic costs ~ A$15,000 per year. For wealthy, non-diabetic people seeking substantial weight loss, of which there are many in this world, that’s doable. Hence Jimmy Kimmel’s joke at the Oscars suggesting that most of the audience was using Ozempic.

But if, for example, a 50+kg adult wanted to use DayBue off-label, the cost per annum would be A$1.3m. A significant deterrent!

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10271225/