Ionis Pharmaceuticals Stock Price, News & Analysis

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.

Ionis Pharmaceuticals announced that the FDA has accepted its New Drug Application for tofersen, aimed at treating SOD1-ALS, a severe genetic form of ALS affecting around 330 people in the U.S. If approved, tofersen will be the first treatment targeting a genetic cause of this disease. Data from 12-month studies indicate earlier tofersen initiation slowed decline across multiple clinical measures and significantly reduced neurofilament levels, reflecting neurodegeneration. The FDA has granted priority review with a target action date of January 25, 2023.

Ionis Pharmaceuticals has announced the completion of patient enrollment in the pivotal Phase 3 Lp(a) HORIZON study of pelacarsen, with 8,325 participants. Pelacarsen targets elevated lipoprotein(a) levels, a significant risk factor for cardiovascular disease. This study, conducted by Novartis, aims to assess the safety and efficacy of pelacarsen in reducing cardiovascular events in patients with elevated Lp(a). There are currently no approved therapies for effectively lowering Lp(a), and topline results are expected in 2025.

Ionis Pharmaceuticals (Nasdaq: IONS) will participate in a virtual fireside chat at the H.C. Wainwright 1st Annual Hereditary Angioedema Conference on July 20, 2022. Investors can access the live webcast on the Ionis website; a replay will be available within 48 hours post-event. Ionis has been a leader in RNA-targeted therapy for over 30 years, with a strong pipeline and three marketed medicines. The company aims to revolutionize standards of care through innovative therapies.