Ionis' Strategic Shift Praised by Analysts For Donidalorsen's Global Reach

Monday, Ionis Pharmaceuticals Inc IONS entered into a license agreement with Otsuka Pharmaceutical Co Ltd under which Otsuka obtains exclusive rights in Europe to commercialize donidalorsen, an investigational prophylactic treatment for hereditary angioedema (HAE). 

Ionis will maintain responsibility for the non-clinical and clinical development of donidalorsen, and Otsuka will be responsible for European regulatory filings and commercialization.

Ionis will receive a $65 million upfront payment and milestone payments based on achieving regulatory and sales targets. Ionis can earn tiered royalties ranging from 20% to 30%.

The company plans to report Phase 3 results with donidalorsen for prophylactic treatment of HAE in the first half of 2024.

Needham writes that the deal will ensure no delay to the regulatory filing timelines and patient access in Europe, allowing Ionis to position its global brand strategy before a potential launch. 

The analyst notes Takeda Pharmaceutical Company Limited's TAK Takhzyro is the market leader in HAE prophylactic therapy, and the prophylactic HAE market is largely US-driven. 

Needham models global peak sales for donidalorsen of ~$750 million, of which ~$540 million is in the U.S. 

William Blair writes that Otsuka is an interesting partner but has an established rare disease commercial presence in Europe. Ionis would look to partner with other rare disease assets outside of the U.S. that are currently wholly owned globally.

The analyst sees Ionis' move to focus wholly owned rare disease drug launch efforts in the U.S. and seek partners ex-U.S. as a prudent use of resources and continues to see a sound commercial potential for donidalorsen.

Price Action: IONS shares are down 0.29% at $50.57 on the last check Tuesday.