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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 (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.
Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals (NASDAQ: IONS), has received approval from the Scottish Medicines Consortium (SMC) for volanesorsen, enabling access via the NHS in Scotland for treating familial chylomicronaemia syndrome (FCS). This follows NICE's earlier approval in England. Volanesorsen is the only treatment available for FCS in the UK, aimed at reducing triglyceride levels significantly. The SMC's decision is a milestone for FCS patients, allowing access while additional clinical data is collected over three years.
Ionis Pharmaceuticals reported its third-quarter 2020 financial results, showing a net income of $5 million (non-GAAP) but a GAAP net loss of $31 million. The company achieved revenue growth, with commercial revenues of $95 million driven by SPINRAZA royalties of $74 million. The recent acquisition of Akcea strengthens Ionis's pipeline, projecting 10 or more marketing applications by 2025. Cash reserves stand at $2.3 billion post-acquisition, maintaining solid financial health.
Ionis Pharmaceuticals and Akcea Therapeutics have won the Prix Galien USA Award for the Best Biotechnology Product for 2020, recognizing TEGSEDI, the first self-administered treatment for the polyneuropathy of hereditary ATTR amyloidosis. Presented in a virtual ceremony on October 29, 2020, this prestigious award highlights significant advancements in biomedicine. Brett P. Monia emphasized TEGSEDI's role in improving the lives of patients affected by this severe disease. TEGSEDI is FDA-approved and offers hope for individuals suffering from hATTR amyloidosis.
Ionis Pharmaceuticals announced the dosing of the first patients with ION541, an investigational therapy for amyotrophic lateral sclerosis (ALS). This milestone is significant as ION541 targets ataxin-2 RNA, potentially reversing TDP-43 toxicity, a common characteristic of ALS. Supported by a $10 million payment from Biogen, this Phase 1/2 trial aims to address both familial and sporadic ALS cases. Ionis continues to advance its ALS pipeline, which includes tofersen and IONIS-C9Rx, enhancing its commitment to developing innovative therapies for this devastating condition.
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