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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 presented positive Phase 2 results for IONIS-FB-LRx in treating immunoglobulin A nephropathy (IgAN), achieving a 44% mean reduction in proteinuria after 6 months. The study maintained kidney function in all patients and showed robust reductions in plasma complement Factor B and urinary complement fragment Ba. Roche plans to advance IONIS-FB-LRx into Phase 3 development in H1 2023, addressing a significant unmet medical need in IgAN treatment.
Ionis Pharmaceuticals announced positive Phase 2b results for fesomersen presented at Kidney Week 2022. This investigational medicine targets Factor XI reduction to prevent thrombosis in patients on hemodialysis. The study showed no increased risk of major bleeding, achieving median FXI reductions of 53.1%, 72.2%, and 86.6% across different doses. Additionally, incidences of dialysis circuit clotting and AV-access thrombosis decreased significantly. Ionis will regain rights to fesomersen from Bayer and is seeking a new partner for its market delivery.
Ionis Pharmaceuticals (Nasdaq: IONS) will host a live webcast on November 9, 2022, at 11:30 a.m. ET to discuss its financial results for the third quarter of 2022. The webcast can be accessed through Ionis’ investor relations website, and a replay will be available shortly after. With over 30 years in RNA-targeted therapy, Ionis has established itself as a leader in this field and currently markets three medicines while advancing a robust late-stage pipeline featuring key cardiovascular and neurological franchises.
Ionis Pharmaceuticals has entered into a long-term lease agreement with Sudberry Properties to develop a new chemistry and manufacturing facility in Oceanside, California. The new 217,000-square-foot facility will enhance Ionis' production capabilities, expected to be operational by 2025, with active pharmaceutical ingredient manufacturing starting in mid-2026. The facility will incorporate sustainability features, including solar energy generation. Ionis maintains a right of first offer to purchase the property after the lease term.
Ionis Pharmaceuticals announced results from the Phase 2b SOLANO study for ION449 (AZD8233), showing a statistically significant 62.3% reduction in LDL-C after 28 weeks. Despite this success, AstraZeneca will not proceed to Phase 3 due to unmet pre-specified efficacy criteria. The drug was deemed generally safe and well-tolerated. AstraZeneca will analyze the results further for next steps. This study is crucial as hypercholesterolemia poses significant cardiovascular risks, with many patients not achieving LDL-C targets.
Ionis Pharmaceuticals (Nasdaq: IONS) announced positive interim results from the 35-week Phase 3 NEURO-TTRansform study of eplontersen for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The treatment showed an 81.2% reduction in serum transthyretin (TTR) levels and significant improvements in neuropathy impairment and quality of life compared to a placebo group. Eplontersen was well-tolerated with no significant adverse events leading to discontinuation. Following these results, Ionis and AstraZeneca plan to seek regulatory approval.
Ionis Pharmaceuticals (Nasdaq: IONS) announced participation in key investor conferences featuring fireside chats this September 2022. These events include the Wells Fargo Healthcare Conference on September 7, Citi's BioPharma Conference on September 8, and the Morgan Stanley Global Healthcare Conference on September 12. Live webcasts will be available on the Ionis website, with replays accessible within 48 hours post-event.
Ionis leads in RNA-targeted therapies, with three marketed medicines and a strong pipeline in cardiovascular and neurological sectors.
Ionis Pharmaceuticals reported positive Phase 3 data for eplontersen targeting hereditary ATTR polyneuropathy, with an NDA filing expected in H2 2022. The FDA granted priority review for tofersen, with a decision anticipated by January 25, 2023. The company achieved $134 million in Q2 2022 revenues, reflecting a 15% year-over-year increase, although commercial revenue declined 12% due to decreased SPINRAZA royalties. Ionis ended Q2 with $2 billion in cash, maintaining financial stability to support its late-stage pipeline.
Ionis Pharmaceuticals has announced positive topline results from its Phase 2b RE-THINC ESRD study of fesomersen, an investigational therapy targeting patients with end-stage renal disease on hemodialysis. The study achieved its primary goal, with no increase in major bleeding incidents compared to placebo. Fesomersen, demonstrating significant reductions in Factor XI activity levels, was deemed safe and well-tolerated. This outcome underlines its potential as a novel anti-thrombotic treatment, addressing a critical unmet need for patients at high risk for thromboembolic events and bleeding complications.
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced a live webcast on August 9 at 11:30 a.m. Eastern Time to discuss its second quarter financial results and update on its pipeline and business progress. The event underscores Ionis's commitment to transparency and communication with investors regarding its financial performance and future strategies. The webcast will be accessible on their website, and a replay will be available for a limited time.