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Ionis Pharmaceuticals IONS signed a license agreement with Japan-based Ono Pharmaceutical. Per the terms, Ono has acquired exclusive global rights from IONS to develop and market sapablursen, an investigational RNA-based therapy for polycythemia vera (PV).
A rare blood cancer, PV is marked by the overproduction of red blood cells, which significantly increases the risk of serious blood clots, especially in critical organs like the lungs, heart and brain.
Sapablursen is being evaluated in the ongoing phase II IMPRSSION study in PV patients. While Ionis remains responsible for completing this study, Ono will be solely responsible for subsequent development, regulatory filings and marketing of the drug. The drug was granted fast track and orphan drug designations by the FDA last year in the PV indication.
In consideration of granting these rights, Ionis will receive an upfront payment of $280 million from Ono and be eligible for milestone payments of up to $660 million. It will also be eligible to receive royalties in the mid-teen percentage range on the drug’s annual net sales.
What Will Ionis Do With the Funds?
Ionis intends to use the funds from its Ono deal to support the advancement of its wholly-owned pipeline. This will likely help diversify IONS’ revenue stream and lower its dependence on collaboration partners.
Recently, it achieved a milestone when the FDA approved Tryngolza in familial chylomicronemia syndrome (FCS) indication. Besides being Ionis’ first wholly-owned drug, the drug also marks the company’s first independent commercial launch. A regulatory filing for this FCS drug is under review in the EU.
Some of its other wholly-owned candidates include donidalorsen for hereditary angioedema (HAE), zilganersen for Alexander’s disease and ulefnersen for amyotrophic lateral sclerosis (ALS). These drugs are already being evaluated in late-stage studies. Ionis expects commercial launches for these drugs over the next three years.
IONS Stock Performance
Year to date, shares of Ionis have lost nearly 6% compared with the industry’s 1% decline.
Image Source: Zacks Investment Research
Ionis’ Diverse Revenue Stream
Ionis has collaborations with leading drugmakers/biotech companies, such as AstraZeneca AZN, Biogen BIIB, GSK plc GSK and Novartis. These deals provide the company with funds in the form of license fees, upfront payments and milestone payments to invest in the development of its wholly-owned pipeline.
Ionis earns commercial revenues in the form of royalty payments on net sales of Spinraza, approved in the United States to treat spinal muscular atrophy (SMA) worldwide. Ionis licensed this drug to Biogen, which is responsible for commercializing it. Ionis and Biogen also market Qalsody, which was approved by the FDA in April 2023 for amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations.