Ono licenses Ionis rare disease drug for $280M

Ionis Pharmaceuticals has entered into an agreement with Ono Pharmaceutical to license out a promising experimental drug, sapablursen, which targets polycythemia vera, a rare condition characterized by an overproduction of red blood cells that can lead to dangerous blood clots. The deal includes an upfront payment of $280 million and potential additional payments totaling up to $660 million contingent on achieving various development, regulatory, and sales milestones.

Sapablursen is currently undergoing testing in a Phase 2 clinical trial, with Ionis responsible for this stage of development. Upon completion of this trial, Ono will take over the subsequent development phases, as well as manage regulatory submissions and the commercialization process. If sapablursen successfully reaches the market, Ionis will earn royalties ranging in the mid-teen percentages.

Ionis has a longstanding strategy of discovering new medicines and partnering with other companies for their development and commercialization. However, there has been a recent shift in its approach, with Ionis expressing a desire to retain greater control over the development and commercialization of its medications, thereby assuming more financial risk in pursuit of potentially higher profits. The first test of this strategy is the launch of Tryngolza, a drug approved in December for a rare condition that leads to dangerous triglyceride accumulation in the body.

Brett Monia, CEO of Ionis, reiterated the company’s dedication to developing and commercializing its own therapies. He highlighted that agreements such as the one with Ono provide Ionis with the financial flexibility necessary to pursue independent commercial opportunities and enhance revenue growth. Ionis plans to launch three additional independently developed drugs over the next three years.

Sapablursen is an RNA-targeting therapeutic aimed at increasing levels of hepcidin, a hormone crucial for regulating iron in the body. This approach is promising, as evidenced by the success of another hepcidin-targeting drug, rusfertide, developed by Protagonist Therapeutics and Takeda, which recently completed a successful Phase 3 trial. Although sapablursen functions differently from rusfertide, the success of the latter strengthens the rationale for targeting hepcidin. Analyst Myles Minter from William Blair highlighted that sapablursen might offer more convenient dosing than rusfertide, thereby providing a valuable treatment option for patients with polycythemia vera.

Minter emphasized that the Ionis-Ono collaboration is mutually beneficial. It allows Ionis to secure funds for the development of its independently owned drugs while providing Ono with a promising experimental drug poised to be a significant option for healthcare providers and patients dealing with polycythemia vera.