Ultragenyx is buying its Angelman syndrome partner GeneTx for $75m on the heels of an interim Phase I/II readout that shows signs of clinical activity and appears to resolve earlier safety worries.

Axsome’s CRL was not the first from the US FDA for the nearly insoluble NSAID as sponsors try different technologies for delivering its pain relief quickly.

Depending on financing, the company could start a Phase III trial of CBM588 as early as this year, but it’s looking to develop the microbiome therapeutic for a potentially broad array of cancers.

Trofinetide could be the first approved drug for Rett patients after hitting both co-primary endpoints in a Phase III trial, but high rates of diarrhea and vomiting raise questions.