Otsuka gains global licensing rights from Ionis to ulefnersen

Japanese mid-size drugmaker Otsuka Pharmaceutical (TYO: 4578) announced that it has entered into an exclusive worldwide licensing agreement with Ionis Pharmaceuticals (Nasdaq: IONS) for rights to manufacture and market Ionis' ulefnersen (ION363), a drug candidate under development for the treatment of patients with amyotrophic lateral sclerosis (ALS) caused by mutation of the fused in sarcoma (FUS) gene.

This agreement marks Otsuka's second introduction of an antisense oligonucleotide (ASO) medicine from Ionis, following its acquisition of development and sales rights in Europe and Asia, including Japan, for the hereditary angioedema attack suppressant drug donidalorsen, which provided the US firm with an upfront payment of $20 million.

The global ALS market size was valued at $673.4 million in 2023 and is further anticipated to reach $1,271.6 million by 2033, expanding at a compound annual growth rate (CAGR) of 6.5 % according to Dimension Market Research. The Asia Pacific region is expected to exhibit the fastest growth rate of all regions.