Ionis licenses olezarsen to Sobi for global reach

CARLSBAD, Calif. - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), a $5.1 billion market cap biotechnology company currently trading near its 52-week low, has announced a licensing agreement granting Sobi® exclusive rights to commercialize olezarsen, a drug for familial chylomicronemia syndrome (FCS) and severely elevated triglycerides, in markets outside the U.S., Canada, and China. According to InvestingPro data, the company maintains a strong liquidity position with current assets significantly exceeding short-term obligations. The agreement follows the U.S. Food and Drug Administration’s approval of olezarsen, branded as TRYNGOLZA™, in December 2024 as the sole treatment for FCS in the U.S. With a robust current ratio of 8.47x, Ionis appears well-positioned to support its commercialization efforts.

Olezarsen is currently under review by the European Medicines Agency for FCS treatment, with approval expected within the year. Sobi, with a commercial presence in over 30 countries, will handle regulatory submissions and commercialization in its territories, building on its experience with Waylivra®, Europe’s only approved FCS medicine. Ionis will receive an upfront payment, milestone-based payments, and up to mid-20% tiered royalties on annual net sales from Sobi.

Ionis CEO Brett P. Monia, Ph.D., expressed enthusiasm for the partnership’s extension and its potential to make olezarsen globally available. Sobi’s CEO Guido Oelkers also welcomed the continued collaboration, emphasizing their commitment to addressing rare and debilitating diseases.

Ionis is further investigating olezarsen for severe hypertriglyceridemia (sHTG) in three Phase 3 clinical trials, with results expected in 2025. FCS, a genetic disorder, leads to extremely high triglyceride levels, increasing the risk of acute pancreatitis and other health issues. sHTG, characterized by triglyceride levels above 500 mg/dL, affects over three million people in the U.S. and can result in acute pancreatitis and cardiovascular diseases.

TRYNGOLZA™, designed to reduce the production of apoC-III, a key regulator of triglyceride metabolism, is the only FDA-approved treatment for FCS. It has not yet been reviewed or approved for sHTG treatment.

Ionis, a leader in RNA-targeted therapies, has six marketed medicines and a robust pipeline focusing on neurology, cardiology, and other areas of significant patient need. The company generated $705 million in revenue over the last twelve months, though it currently operates at a loss with negative EBITDA of $463 million. This press release statement includes forward-looking information regarding the therapeutic and commercial potential of TRYNGOLZA™ and Ionis’ technologies.For deeper insights into Ionis’s financial health and growth potential, including 7 additional key ProTips and comprehensive valuation metrics, visit InvestingPro. Access the detailed Pro Research Report covering what really matters about IONS through intuitive visuals and expert analysis.

In other recent news, Ionis Pharmaceuticals has reported a series of significant developments. The company surpassed financial expectations, with total revenue reaching $705 million, exceeding both Bernstein’s and consensus estimates by 13% and 15%, respectively. Earnings per share also outperformed projections, with a figure of ($0.43) beating expectations by over 50%. Ionis has also secured European Union approval for WAINZUA, a treatment for hereditary transthyretin-mediated amyloidosis, following positive Phase 3 trial results. Additionally, a partnership with Ono Pharmaceuticals has been established to develop sapablursen for polycythemia vera, aiming to strengthen Ionis’s market presence. Oppenheimer has maintained an Outperform rating on Ionis, reflecting confidence in the company’s strategic direction. Meanwhile, Raymond James has adjusted Ionis’s price target to $60 while maintaining a Strong Buy rating. The firm’s analyst highlighted the successful U.S. launch of Wainua and ongoing developments in other therapeutic areas. Lastly, Ionis presented new data on donidalorsen, an investigational drug for hereditary angioedema, which is currently under FDA review.

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