Ionis will receive $280 million upfront and could get up to $660 million in future milestone payments. Ono will take charge of late-stage development as well as regulatory and commercialization activities.
Ionis Pharmaceuticals licensed out its investigational antisense oligonucleotide sapablursen to Ono Pharmaceutical on Tuesday, giving the Japanese firm exclusive global rights to develop and commercialize the drug candidate for polycythemia vera, a rare blood disease.
Under the terms of the agreement, Ionis will get $280 million upfront and will be eligible for up to $660 million in certain development, regulatory and commercial milestones. Ionis will also be able to earn mid-teen-percentage royalties from sales of sapablursen, if approved. The companies said the deal is not yet closed and still has to clear anti-trust hurdles.
Polycythemia vera involves the excessive production of red blood cells. Patients have an increased risk of blood clots, which can affect and damage organs, including the lungs, brain and heart. Symptoms also include severe iron deficiency and fatigue. If left unchecked, polycythemia vera can be life-threatening.
Sapablursen is an investigational antisense oligonucleotide therapy designed to lower the expression of the TMPRSS6 gene, in turn boosting the overall production of hepcidin, a protein involved in the regulation of iron homeostasis. In blood disorders such as polycythemia vera, improving hepcidin expression can help ease disease severity.
Sapablursen is currently being assessed in the Phase II IMPRSSION study, which completed enrollment late last year. Topline data are expected this year.
As per Tuesday’s licensing deal, Ionis will complete the IMPRSSION trial, after which Ono will take sole charge over sapablursen’s development, including late-stage trials, regulatory filings and commercialization activities.
The sapablursen deal comes shortly after Ionis signed a similar agreement for its hypertension hopeful tonlamarsen. That drug, which blocks the production of angiotensin to lower blood pressure, was revealed last week to be the star candidate of cardiovascular startup Kardigan, according to reporting from Endpoints News. Ionis had previously completed a mid-stage trial of the asset, testing a monthly dose for uncontrolled hypertension.
Now, Kardigan plans to run another Phase II trial in the same indication, focusing on patients who are unable to achieve their target blood pressure despite taking at least two antihypertensive agents. The study has not yet started to enroll patients, but has a target completion date of December 2027.
