Ann: Q3 Investor Call Presentation, page-8

Antisense leader, Ionis, is on a roll at the moment, pushing up its market cap by US$1bn over the past month.

Firstly, the company has had success in a late-stage trial of its drug candidate, olezarsen, in a rare autosomal recessive metabolic disorder known as familial chylomicronemia syndrome (FCS). FCS prevents the body from breaking down fats and is characterized by extremely high levels of triglyceride. The condition is estimated to occur in 1-2 million people globally.

Olezarsen demonstrated statistically significant reduction in levels of triglyceride compared to placebo, as well as a 100% reduction in inflammation of the pancreas.

Ionis is planning to file a marketing application with the FDA early next year.

If approved, olezarsen is likely to become the first wholly-owned Ionis drug to be marketed although three other partnered Ionis-developed antisense drugs are already approved and commercially available.

In a second boost, Ionis has also announced a licensing deal with Roche for two drug candidates to target Huntington’s and Alzheimer’s.

Roche is paying US$60m upfront for the exclusive global rights to the pre-clinical programs. Ionis is tasked with completing pre-clinical and Roche with completing all clinical development and commercialization. Development, regulatory and commercial milestones have been negotiated but no details were disclosed.

https://www.reuters.com/business/he...-meets-main-goal-late-stage-study-2023-09-26/

https://www.fiercebiotech.com/biote...rna-targeting-alzheimers-huntingtons-programs