Ionis Gets FDA Approval for Rare Disease Drug Tryngolza

Shares of Ionis Pharmaceuticals IONS rose nearly 5% in after-market trading on Thursday after the FDA approved its RNA-targeted therapy, olezarsen, in adults with familial chylomicronemia syndrome (FCS). The drug will be marketed under the trade name Tryngolza.The FDA’s nod to Tryngolza makes it the first approved treatment for FCS in the country. The drug’s commercial launch is part of management's broad strategy to deliver a steady flow of wholly-owned medicines to patients. Besides being Ionis’ first wholly-owned drug, Tryngolza marks the company’s first independent commercial launch, expected before this year’s end.The approval is supported by results from the phase III BALANCE study, where FCS patients treated with olezarsen achieved significant triglyceride-lowering and substantial reductions in acute pancreatitis attacks in FCS patients. A regulatory filing for the drug is currently under review by the EMA, also supported by data from this study.FCS is a rare genetic disease marked by extremely elevated triglyceride levels. People living with this condition are at high risk of acute pancreatitis in addition to other chronic health issues such as fatigue and severe, recurrent abdominal pain. Per management, nearly 3,000 patients living in the United States suffer from this disease.Management is also evaluating Tryngolza for the treatment of severe hypertriglyceridemia (sHTG) across three late-stage studies — CORE, CORE2 and ESSENCE. Data from these studies are expected in the second half of 2025. Like FCS, Ionis also has a first-mover advantage in sHTG indication.IONS Stock PerformanceYear to date, shares of Ionis have lost 29.6% compared with the industry’s 11.9% decline.Image Source: Zacks Investment ResearchIonis Boasts a Diverse Revenue StreamIONS enjoys a diverse stream of revenues, including commercial products and royalties and numerous sources of collaborative and R&D revenues. The company has collaboration deals with leading drugmakers/biotech companies, such as AstraZeneca AZN, Biogen BIIB, GSK plc GSK and Novartis, for developing and marketing its medicines.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.Last December, the FDA approved Wainua for treating patients with hereditary transthyretin-mediated amyloid polyneuropathy, commonly called hATTR-PN or ATTRv-PN. The drug has been developed in partnership with AstraZeneca.While Ionis and AstraZeneca will jointly market Wainua for ATTRv-PN in the United States, AZN has exclusive rights to commercialize Wainua outside U.S. markets. The drug was commercially launched in the United States in first-quarter 2024. Ionis and AstraZeneca are also working on expanding the drug’s label in other forms of amyloidosis.Novartis and GSK are its partners for pelacarsen and bepirovirsen, respectively. 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.Independent Product Launches Planned by IONSIonis’ collaboration with the above leading drugmakers/biotech companies provides it with funds in the form of license fees, upfront and milestone payments to invest in its internal pipeline development. Some of its wholly-owned candidates include donidalorsen for hereditary angioedema (HAE), zilganersen for Alexander’s disease and ulefnersen for ALS. These drugs are already being evaluated in late-stage studies.Last month, Ionis announced that the FDA accepted the regulatory filing seeking approval for donidalorsen in HAE indication. A final decision is expected by Aug. 21, 2025. This filing is supported by data from two phase III studies, OASIS-HAE and OASISplus, which showed that treatment with the drug achieved a significant and sustained reduction in the rate of HAE attacks for both monthly and every two-month dosing. Management expects donidalorsen to be its second independent commercial launch.Data from the late-stage study on zilganersen in Alexander’s disease patients are expected in 2025.Earlier this year, Ionis reported that a mid-stage study on MASH/NASH candidate ION224 and a phase I/IIa study on Angelman syndrome drug ION582 achieved their respective primary endpoints. Management is currently in the process of determining the next steps to advance ION224; it plans to start a late-stage study on ION582 in the first half of 2025.Ionis is advancing and expanding its wholly-owned pipeline to drive future revenue growth. While the company does have a broad pipeline of partnered programs with leading drugmakers/biotech companies, its recent decision to increase investments in its internal pipeline seems encouraging, given the recent positive data readouts. The expansion of its internal pipeline should help Ionis diversify its revenue stream and lower its dependence on collaboration partners.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.Zacks Names #1 Semiconductor StockIt's only 1/9,000th the size of NVIDIA which skyrocketed more than +800% since we recommended it. NVIDIA is still strong, but our new top chip stock has much more room to boom.With strong earnings growth and an expanding customer base, it's positioned to feed the rampant demand for Artificial Intelligence, Machine Learning, and Internet of Things. Global semiconductor manufacturing is projected to explode from $452 billion in 2021 to $803 billion by 2028.See This Stock Now for Free >>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 ReportTo read this article on Zacks.com click here.Zacks Investment ResearchWeiter zum vollständigen Artikel bei Zacks Weiter zum vollständigen Artikel bei Zacks