Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis Pharmaceuticals reported total revenues of $133 million for Q3 2021, reflecting a strategic focus on expanding its late-stage pipeline, now featuring seven Phase 3 programs. Despite a net loss of $48 million, the company maintained a robust cash position of $2 billion. Key developments included commencing the Phase 3 CORE study of olezarsen and advancements in the pipeline with multiple catalysts expected in 2022. However, the tofersen Phase 3 VALOR study did not meet its primary endpoint, although secondary endpoints showed favorable trends.

Ionis Pharmaceuticals announces the initiation of the CORE study, a pivotal Phase 3 clinical trial for olezarsen, targeting severe hypertriglyceridemia (triglyceride levels ≥ 500 mg/dL). This life-threatening condition affects millions, leading to pancreatitis and cardiovascular risks. The CORE study aims to evaluate olezarsen's efficacy against placebo, with primary endpoints focused on reducing fasting triglyceride levels over six months. Previous Phase 2 trials demonstrated significant reductions in triglycerides and a favorable safety profile. The broad olezarsen program plans to enroll over 1500 patients.

Ionis Pharmaceuticals has scheduled a live webcast to discuss its Q3 2021 financial results and business progress on November 3, 2021, at 11:30 a.m. ET. This session will provide insights into the company's pipeline developments and financial performance, contributing to its position in the RNA-targeted therapy market. The webcast is accessible to interested parties via Ionis's official website, with a replay available for a limited time afterwards.

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced the topline results from Biogen's Phase 3 VALOR study of tofersen, an antisense medicine for SOD1 amyotrophic lateral sclerosis (ALS). Although the primary endpoint was not met, secondary measures indicated trends favoring tofersen. The study involved 108 participants, showing 38% and 67% differences in key biomarkers for faster and slower progressors, respectively. Biogen plans to engage regulatory bodies for next steps, while expanding its early access program for SOD1-ALS patients.

Ionis Pharmaceuticals (NASDAQ: IONS) will present 14 posters and two symposiums at the 3rd European ATTR Amyloidosis meeting from September 6-8, 2021. Highlighting advancements in its ATTR amyloidosis portfolio, the presentations will focus on the investigational antisense medicine, eplontersen, which is being evaluated in two phase 3 trials for transthyretin amyloid cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN). Ionis aims to enhance treatment options for patients impacted by this disease.

Ionis Pharmaceuticals (Nasdaq: IONS) announced its participation in upcoming virtual investor conferences, including the 2021 Wells Fargo Healthcare Conference on September 10, the Morgan Stanley 19th Annual Global Healthcare Conference on September 13, and the Bank of America Global Healthcare Conference 2021 on September 15. Presentation details may change, with updates available on the company's website. A live webcast will be offered, with replays accessible within 48 hours. Ionis is a leader in RNA-targeted therapy, with three marketed medicines and a strong pipeline.

Ionis Pharmaceuticals reported Q2 2021 financial results, with total revenues of $126 million and a net loss of $36 million (non-GAAP). The company is well capitalized, ending the quarter with $2.1 billion in cash and investments. Key advancements include full enrollment in the Phase 3 NEURO-TTRansform study and ongoing studies for tofersen and pelacarsen. Ionis aims for more than $600 million in revenue for 2021, while operational changes have streamlined costs. The company anticipates significant data readouts in H2 2021, potentially leading to numerous new product launches by 2026.

Ionis Pharmaceuticals announced significant progress for pelacarsen, a potential treatment for lipoprotein(a)-driven cardiovascular disease, as the Lp(a) HORIZON study reached 50% enrollment with 4,000 participants. This Phase 3 study aims to support the drug's indication for reducing cardiovascular risk. Ionis received a $25 million milestone payment from Novartis and is eligible for up to $675 million in additional milestones, along with mid-teens to low 20% royalties on pelacarsen sales. This investigational medicine utilizes LICA technology to target elevated Lp(a) levels, a significant risk factor for cardiovascular diseases.

Ionis Pharmaceuticals (NASDAQ: IONS) has successfully completed enrollment in its pivotal Phase 3 NEURO-TTRansform study, exceeding its target with over 160 patients participating. The study focuses on eplontersen for treating hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). Eplontersen, an investigational drug using Ionis' LICA technology, showed up to 94% reductions in transthyretin (TTR) in Phase 1 trials. An interim analysis is expected by mid-2022, with a potential NDA filing by year-end 2022.