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Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) is a leading biotechnology company headquartered in Carlsbad, California. Founded in 1989, Ionis has been pioneering the discovery and development of antisense drugs, with an impressive portfolio of over 1300 patents. Ionis focuses on creating innovative RNA-targeted therapies for complex diseases, particularly in the areas of cardiovascular, metabolic, neurological, and rare genetic disorders.
Ionis has successfully brought several groundbreaking medicines to market. Notable achievements include the development of Spinraza, in collaboration with Biogen, which was launched in 2016 to treat spinal muscular atrophy, a rare neuromuscular disorder. In 2023, Biogen also launched Qalsody for ALS, further extending the impact of Ionis' antisense technology.
Through its cardiovascular-focused subsidiary, Akcea Therapeutics, Ionis has introduced drugs like Tegsedi for ATTR amyloidosis and Waylivra in Europe for cardiology indications. The company continues to advance its pipeline with promising candidates such as olezarsen, an investigational RNA-targeted therapy for familial chylomicronemia syndrome (FCS). Ionis' ongoing Phase 3 trials for olezarsen have shown promising results, with significant reductions in triglyceride levels and improvements in related health outcomes.
Ionis' commitment to innovation and patient care is also evident in its robust financial health and strategic partnerships. With a deep understanding of disease biology and cutting-edge technology, Ionis continues to drive advancements in RNA therapies and gene editing, aiming to deliver life-changing medicines for patients around the world.
Recently, Ionis announced positive clinical trial results for several of its investigational drugs and plans to pursue regulatory approvals for these therapies. The company's pipeline is well-positioned to address unmet medical needs in various therapeutic areas, promising a steady flow of new treatments in the coming years.
For more detailed information about Ionis Pharmaceuticals, its products, and career opportunities, visit their official website at www.ionispharma.com.
Ionis Pharmaceuticals highlighted its 2020 achievements and future plans during its Investor Day on Dec. 8, 2020. The company outlined a strategy focused on addressing rare diseases in neurology and cardiology while seeking to pioneer new treatment markets. Ionis aims to launch six or more products by 2026, with projections of double-digit revenue and earnings growth. Key highlights included progress in clinical trials, the successful integration of Akcea, and advancements in oral delivery systems.
Ionis Pharmaceuticals announced the initiation of the Phase 3 BALANCE study for AKCEA-APOCIII-LRx, targeting familial chylomicronemia syndrome (FCS), a serious genetic condition leading to high triglyceride levels and acute pancreatitis risk. The randomized, double-blind study will enroll up to 60 adult patients, assessing the drug's efficacy by measuring fasting triglyceride levels. This milestone underscores Ionis’ commitment to addressing unmet medical needs in the FCS patient community while expanding its pipeline of innovative therapies.
Ionis Pharmaceuticals (NASDAQ: IONS) announced a live investor day webcast on December 7, 2020, at 11:00 a.m. ET. The event will showcase the company’s commercial strategy, focusing on cardiovascular and neurological franchises, along with recent technological advancements. Key presentations will feature CEO Brett Monia, scientific leaders, and financial executives discussing topics such as market opportunities, pipeline valuation, and future strategies. Interested parties can access the webcast at www.ionispharma.com.
Ionis Pharmaceuticals announced that AstraZeneca has initiated a Phase 2b clinical trial of ION449 (AZD8233), aimed at lowering cholesterol levels in patients with dyslipidemia. This investigational antisense medicine targets PCSK9, which regulates LDL cholesterol. The randomized trial will enroll about 108 participants and evaluate safety along with efficacy at different doses. Ionis received a $20 million milestone payment from AstraZeneca for the trial initiation, highlighting the collaboration focused on treatments for various diseases.
Ionis Pharmaceuticals (NASDAQ: IONS) announced that AstraZeneca has licensed ION455, an investigational treatment for nonalcoholic steatohepatitis (NASH). This marks the second partnership on NASH between the two companies, following ION839. Ionis received $30 million upfront and can earn up to $300 million in milestones plus royalties. Currently, there are no FDA-approved treatments for NASH, highlighting the significance of this development. Additionally, Ionis is advancing its wholly-owned NASH program, ION224. The company has over 20 medicines in its pipeline.
On November 13, 2020, Ionis Pharmaceuticals (NASDAQ: IONS) presented new data on ION449, an investigational antisense medicine targeting PCSK9 at the AHA Scientific Sessions. The Phase 1 study showed ION449 significantly reduced plasma PCSK9 protein (>90%) and LDL-C levels (~70%) in subjects. Oral administration feasibility was confirmed in animal studies. ION449 aims to provide additional treatment options for cardiovascular disease, which is still a leading cause of death globally, highlighting its potential as a best-in-class therapy.
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) will present clinical and non-clinical data for its antisense medicines for cardiovascular disease at the AHA Scientific Sessions 2020, Nov 13-17, 2020. Key presentations include data on ION449, an investigational antisense medicine targeting PCSK9, in collaboration with AstraZeneca. The company will also showcase its treatments for apolipoprotein C-III and lipoprotein(a). Notably, Ionis plans to start a Phase 3 study for AKCEA-APOCIII-LRx by year-end. The event emphasizes Ionis' focus on RNA-targeted therapies to address unmet medical needs in cardiovascular health.
Ionis Pharmaceuticals announced participation in the RNA at the Bench and Bedside II Conference, occurring virtually from Nov. 11-13, 2020. The company will showcase advancements in its antisense technology, particularly in clinical trials for cardiovascular treatments such as pelacarsen and AKCEA-TTR-LRx. Presentations will cover drug delivery methods and the effectiveness of RNA-targeted therapies for heart failure and metabolic diseases. Over 30 industry speakers will participate, highlighting collaboration with universities and key advancements in RNA-targeted therapeutics.
Ionis Pharmaceuticals (Nasdaq: IONS) announced its participation in three upcoming virtual investor conferences. Management will present at:
- Stifel 2020 Healthcare Conference on November 16, 2020;
- Evercore ISI 3rd Annual HealthCONx Conference on December 1, 2020;
- Piper Sandler 32nd Annual Healthcare Conference on December 3, 2020.
Live webcasts of each presentation will be available, with replays accessible for a limited time. For updates, visit www.ionispharma.com.
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