Ionis unveils positive PhII data for in-house rare disease drug, paving way to blockbuster rivalry with Takeda

One of the mid-stage antisense drugs for rare, non-neurological diseases that Brett Monia boasted about at JP Morgan earlier this year has delivered a solid slate of positive results, boosting the CEO’s promise to turn Ionis into one of the biggest RNA biotechs with its wholly-owned pipeline.

With this particular candidate, he may even be setting the company up for a rivalry with Takeda.