Ono pays $280M for Ionis' antisense drug for rare blood cancer

In a significant transaction within the pharmaceutical industry, Japan's Ono Pharmaceutical has agreed to acquire Ionis Pharmaceuticals' phase 2-stage antisense oligonucleotide, sapablursen, for a substantial upfront payment of $280 million. This RNA-targeted medicine is designed specifically to address a rare type of blood cancer by reducing the production of the TMPRSS6 protein, thus increasing the expression of hepcidin, a crucial regulator of iron homeostasis. This mechanism aims to provide a therapeutic approach to various blood disorders.

Currently, sapablursen is being tested in a phase 2 trial for polycythemia vera (PV), a rare condition characterized by the excessive production of red blood cells. According to the terms of the licensing agreement between Ono and Ionis, Ionis will oversee the ongoing midstage study of the drug. After this phase, Ono will take full responsibility for further development, regulatory submissions, and commercialization of sapablursen. In addition to the initial payment, Ionis stands to earn up to $660 million in future development, regulatory, and sales milestones, alongside mid-teen royalties based on annual net sales.

Previously, Ionis had explored the potential of sapablursen in treating another blood disorder known as beta thalassemia but decided to shift its focus entirely to PV in 2023 following an evaluation of midphase efficacy data. Ionis CEO Brett Monia, Ph.D., highlighted the company’s promising findings in phase 1 trials, where there was significant engagement with the target and notable increases in hepcidin levels. However, the anticipated changes were not observed in beta-thalassemia intermedia, leading to a strategic pivot towards PV.

Monia expressed confidence in handing over the development of sapablursen to Ono, citing the company's impressive capabilities which would ensure expansive access for individuals affected by PV. Ionis recently transitioned to a commercial-stage company following the approval of Tryngolza, a treatment for the rare genetic disorder familial chylomicronemia syndrome, in December. Monia emphasized Ionis's ongoing commitment to advancing its independently owned medicines and the company's plans to launch three additional products over the next three years. Streamlining its portfolio allows Ionis to maintain financial flexibility, support its strategic investments, and focus on near and mid-term commercial opportunities to drive significant revenue growth.

Ono Pharmaceutical, known for its collaboration with Bristol Myers Squibb on the cancer immunotherapy drug Opdivo, continues to expand its presence in the oncology sector by developing antibodies, multispecific molecules, and cell therapies. Last year, Ono entered into a $700 million partnership with LigaChem Biosciences for the development of antibody-drug conjugates. The acquisition of sapablursen aligns with Ono's strategy to enhance its hematology pipeline. Ono President and COO Toichi Takino expressed optimism about sapablursen's potential to serve as a new treatment for PV patients globally.

This deal between Ionis and Ono coincides with the recent success of Takeda and Protagonist Therapeutics, who announced a phase 3 win for their own PV treatment candidate, rusfertide, an injectable hepcidin mimetic. While rusfertide may be the first hepcidin-boosting drug to reach the market, analysts from William Blair believe sapablursen presents a promising alternative, potentially offering an improved treatment regime that could appeal to both patients and healthcare providers. The partnership with Ono is seen as mutually beneficial for both companies.