Ono Licenses Ionis’ ASO for Rare Blood Disease

Rare Daily Staff

Ono Pharmaceutical agreed to a global license, development, and commercialization agreement with Ionis Pharmaceuticals for the company’s experimental antisense oligonucleotide sapablursen for polycythemia vera, a rare and potentially life-threatening blood disease.

Under the terms of the agreement, Ionis will receive a $280 million upfront payment, with the potential to earn up to $660 million in additional payments based on the achievement of development, regulatory, and sales milestones. Ionis is also eligible to earn royalties in the mid-teen percentage range on annual net sales of sapablursen.

Ionis will be responsible for the completion of the ongoing phase 2 IMPRSSION study, while Ono will be solely responsible for subsequent development, regulatory filings, and commercialization.

Polycythemia vera (PV) is characterized by the overproduction of red blood cells, which significantly increases the risk of serious blood clots, especially in critical organs like the lungs, heart and brain. Patients with PV also experience severe iron deficiency and commonly have symptoms of fatigue.

Sapablursen is designed to reduce the production of TMPRSS6 resulting in increased expression of hepcidin, which is the key regulator of iron homeostasis. By increasing production of hepcidin, sapablursen has the potential to positively impact blood diseases such as PV.

Sapablursen is currently being evaluated in adults living with PV in the fully enrolled phase 2 IMPRSSION study. The U.S. Food and Drug Administration granted sapablursen Fast Track and Orphan Drug designation.

“Ionis remains committed to advancing the wholly owned medicines we choose to commercialize ourselves, which includes our first independent launch currently underway and three additional anticipated launches in the next three years.” said Brett Monia, CEO of Ionis. “Streamlining our Ionis-owned portfolio provides financial flexibility, supporting our commitment to invest in and focus on our near and mid-term commercial opportunities and generate substantial revenue growth.”

Photo: Brett Monia, CEO of Ionis Pharmaceuticals