Ionis Out-Licenses Rights for Rare Blood Cancer Drug to Ono

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 PerformanceYear to date, shares of Ionis have lost nearly 6% compared with the industry’s 1% decline.Image Source: Zacks Investment ResearchIonis’ Diverse Revenue StreamIonis 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.AstraZeneca, Novartis and GSK are its partners for Wainua, pelacarsen and bepirovirsen, respectively. The FDA approved Wainua in December 2023 to treat adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (ATTRv-PN) in the United States. The drug recently obtained approval in the EU for a similar indication and will be marketed under the name Wainzua.While the GSK-partnered drug is being developed in two late-stage studies for chronic hepatitis B (CHB), the Novartis-partnered drug is being developed in a late-stage study for patients with cardiovascular disease due to elevated Lp(a) levels. Data from the Novartis and GSK-partnered drug studies are expected in 2025 and 2026, respectively.Ionis Pharmaceuticals, Inc. Price Ionis Pharmaceuticals, Inc. price | Ionis Pharmaceuticals, Inc. QuoteIONS’ Zacks RankIonis currently carries a Zacks Rank #3 (Hold).You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.5 Stocks Set to DoubleEach was handpicked by a Zacks expert as the #1 favorite stock to gain +100% or more in 2024. While not all picks can be winners, previous recommendations have soared +143.0%, +175.9%, +498.3% and +673.0%.Most of the stocks in this report are flying under Wall Street radar, which provides a great opportunity to get in on the ground floor.Today, See These 5 Potential Home Runs >>Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report AstraZeneca PLC (AZN): Free Stock Analysis Report GSK PLC Sponsored ADR (GSK): Free Stock Analysis Report Biogen Inc. (BIIB): Free Stock Analysis Report Ionis Pharmaceuticals, Inc. (IONS): Free Stock Analysis ReportThis article originally published on Zacks Investment Research (zacks.com).Zacks Investment ResearchWeiter zum vollständigen Artikel bei Zacks Weiter zum vollständigen Artikel bei Zacks