Olezarsen Sodium

Ionis Pharmaceuticals previously investigated sapablursen in another blood disorder called beta thalassemia.\n Japan\'s Ono Pharmaceutical is handing over $280 million upfront to acquire Ionis Pharmaceuticals’ phase 2-stage antisense oligonucleotide for a rare type of blood cancer.The RNA-targeted medicine, called sapablursen, is designed to reduce the production of a protein called TMPRSS6. The idea is that this increases the expression of hepcidin, which is the key regulator of iron homeostasis, thereby helping treat various blood diseases.Sapablursen is currently in a phase 2 trial for polycythemia vera (PV), a rare blood condition characterized by the overproduction of red blood cells. As part of the licensing deal with Ono, Ionis will remain responsible for the midstage study.Beyond that, Ono will become solely responsible for sapablursen’s development, regulatory filings and commercialization, according to a post-market release March 11.In return, Ono is handing over $280 million upfront, with potentially up to $660 million to follow in additional development, regulatory and sales milestones, along with mid-teen royalties on annual net sales.Ionis had previously investigated sapablursen in another blood disorder called beta thalassemia before ending those plans in 2023 after looking at midphase efficacy data and instead focusing on PV.At the time, Ionis CEO Brett Monia, Ph.D., said the company had seen “quite remarkable” target engagement in phase 1 plus “very significant elevations in hepcidin levels.” However, “in beta-thalassemia intermedia, we didn\'t see those changes,” the CEO explained back in 2023.In yesterday’s release, Monia said the company was “pleased to entrust sapablursen to Ono, whose unique capabilities will help maximize its value by ensuring broad access for people living with PV.” Ionis recently became a commercial-stage company as a result of the approval of Tryngolza to treat the rare genetic disorder familial chylomicronemia syndrome in December.“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,” Monia added. “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.”Meanwhile, Ono—which codeveloped blockbuster cancer immunotherapy Opdivo with Bristol Myers Squibb—is active in the oncology space, where it is working on a slate of antibodies as well as some multispecific molecules and cell therapies. Last year, the biotech got in on the antibody-drug conjugate action via a $700 million biobucks deal with LigaChem Biosciences.The partnership with Ionis “aligns with our strategy to strengthen our pipeline in hematology,” said Ono President and Chief Operating Officer Toichi Takino, who added that the company “expects sapablursen to become a new treatment option for PV patients worldwide.”The deal comes a week after Takeda and Protagonist Therapeutics posted a phase 3 win for their own PV prospect, an injectable hepcidin mimetic called rusfertide.“While rusfertide is likely to be first to market in the hepcidin-boosting drug class, we see sapablursen as a potential improvement in treatment regimen that will be attractive to patients and healthcare providers,“ William Blair analysts said in a March 12 note, adding that Ono\'s deal with Ionis was “positive for both sides.“