Well done PYC! A great announcement. Not only restoration of the...

Well done PYC! A great announcement.

Not only restoration of the missing SHANK3 protein in PMS patient-derived brain cells, but achieved using two different RNA chemistries – 2’MOE or PPMO.

With plans to progress this fourth drug candidate into clinic next year, cash needs will inevitably rise. PYC recently extended its cash runway to ~A$100m, but with R&D costs now nudging $20m a quarter, more cash will be needed next year.

It’s my view that the PMS candidate will be licensed out, probably before it enters the clinic, and possibly by US pharma, Acadia, which has previously in-licensed pre-clinical antisense assets from Stoke Therapeutics for the treatment of Rett syndrome and SYNGAP1 syndrome, both rare neurodevelopmental genetic disorders. But the asset will have interest to others as well.

Further examples of pre-clinical antisense licensing deals can be found here and here.