Ionis Envisions Two New Approvals in 2023 in ALS and ATTRv-PN

Courtesy of Ionis Pharmaceuticals

With its partners on the cusp of potentially significant approvals in ALS and hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), Ionis CEO Brett Monia, Ph.D. was upbeat about the company’s commercial prospects in 2023.

During his JPM presentation Wednesday, Monia touted nine positive late- and mid-stage readouts, including one for an investigational antisense oligonucleotide (ASO) licensed to GSK in 2019.

GSK is developing the therapy for chronic hepatitis B virus. After demonstrating sustained clearance of hepatitis B surface antigen and hepatitis B virus DNA in Phase II, GSK plans to launch a Phase III study in 2H, 2023. 

Ionis expects two new products to reach the market in 2023:

• Eplontersen, developed with long-time partner AstraZeneca to treat hereditary transthyretin-mediated amyloid polyneuropathy. The therapy demonstrated promising Phase III safety and efficacy data, reducing serum levels of transthyretin by 81.2%.
• Tofersen for superoxide dismutase 1 (SOD1)-ALS. Tofersen, developed with another long-term partner, Biogen, has a PDUFA date of April 25. The development process has not been without controversy, as tofersen missed its Phase III endpoint. However, 12-month data from the VALOR trial and open-label extension study showed that the therapy decreased declines in clinical function, respiratory function, muscle strength, and quality of life upon earlier initiation.

Monia also discussed Ionis’ move from an R&D organization to a fully integrated biotech company during the past three years.

He highlighted the expansion and diversification of its technology platforms, including a collaboration deal struck with Metagenomi in November 2022 that added DNA editing to the company’s RNA-targeted technology.  

He also emphasized the company’s robust financial position, focusing on the Monday announcement of a $1.1 billion royalty agreement with Royalty Pharma.

The agreement will enable Ionis to monetize some of its royalties from Biogen’s spinal muscular atrophy therapy Spinraza and Novartis’ pelacarsen, an investigational antisense liver therapy, he said.