Ono acquires Ionis blood disease drug for $280M upfront

Ono Pharmaceutical, a renowned Japanese pharmaceutical company, has made a strategic move by investing $280 million upfront to acquire the global rights to an innovative drug developed by Ionis Pharmaceuticals. This drug, known as sapablursen, is designed to target RNA and is aimed at treating a rare blood disorder called polycythemia vera. This latest acquisition highlights the growing interest and momentum in the medical field for treatments addressing this rare condition.

Ionis Pharmaceuticals, based in California, stands to gain significantly from this partnership. In addition to the initial payment, the biotech company could potentially receive up to $660 million in milestone payments. Moreover, Ionis is entitled to receive royalties in the mid-teen percentage range, offering a substantial financial incentive linked to the drug's future success.

Sapablursen is currently undergoing a Phase 2 clinical trial named IMPRSSION, focusing on adults diagnosed with polycythemia vera. This disorder is characterized by the overproduction of red blood cells, which can lead to an increased risk of blood clots, iron deficiency, and fatigue. The ongoing trial is fully enrolled, and Ionis is responsible for completing this phase. Following the conclusion of the Phase 2 study, Ono Pharmaceutical will assume the responsibilities of further drug development, regulatory submissions, and eventual commercialization.

The mechanism of action for sapablursen involves the reduction of matriptase-2 production. Matriptase-2 is instrumental in controlling the levels of hepcidin, a protein vital for iron regulation in the body. By decreasing matriptase-2, the expression of hepcidin is expected to increase, thereby promoting improved iron balance in the body.

Polycythemia vera is a relatively rare disease, affecting approximately two to three individuals per 100,000 people. Despite its rarity, the disease has garnered significant attention in the pharmaceutical industry, leading to multiple partnerships and clinical trial developments over the past year. Notably, there was a licensing agreement established in 2024 between Takeda and Protagonist Therapeutics for rusfertide, a promising late-stage candidate. This deal involved a $300 million upfront payment, underscoring the potential value perceived in treatments for this condition.

Furthermore, Protagonist Therapeutics has the option to withdraw from profit-sharing arrangements under specific conditions, although the company has not yet indicated whether it intends to exercise this option. Earlier reports from the company announced that rusfertide successfully met both the primary and secondary endpoints in a Phase 3 trial. The drug acts as a synthetic mimic of hepcidin, similar to the mechanism pursued by Ionis.

July 2024 saw further advancements in the field with Silence Therapeutics revealing encouraging Phase 1 results for its siRNA candidate, divesiran. This study involved a small cohort of individuals suffering from polycythemia vera, adding another potential therapeutic option to the growing list of treatments targeting this rare blood disorder.

In summary, the collaboration between Ono Pharmaceutical and Ionis Pharmaceuticals for the development and commercialization of sapablursen represents a significant step forward in addressing polycythemia vera. The commitment of substantial financial resources and ongoing clinical trials reflects a broader industry trend towards innovative solutions for rare diseases, offering new hope to affected patients worldwide.