Ionis Pharmaceuticals, Inc. develops RNA-targeted medicines and reports news around marketed medicines and a pipeline in neurology, cardiometabolic disease and other high-need areas. Company updates commonly address commercial performance for TRYNGOLZA, financial results, R&D revenue from partnerships, and progress across antisense oligonucleotide programs.
Clinical and regulatory news also covers programs such as olezarsen for severe hypertriglyceridemia, zilganersen for Alexander disease, and partner-developed bepirovirsen for chronic hepatitis B. Governance and investor-relations releases include annual meeting materials, board composition changes, and healthcare conference presentations.
Ionis Pharmaceuticals announced promising results from Pfizer's Phase 2b study of vupanorsen, showing statistically significant reductions in non-HDL cholesterol and triglycerides across all doses. This investigational therapy aims to reduce cardiovascular risks and severe hypertriglyceridemia. The study involved 286 participants and achieved its primary endpoint at 24 weeks. Despite some injection site reactions and increases in liver enzymes, there were no serious adverse events related to treatment. Full results are expected to be presented at a medical meeting next year.
Ionis Pharmaceuticals (NASDAQ: IONS) has announced the initiation of the Phase 3 OASIS-HAE study for donidalorsen, an investigational antisense drug targeting hereditary angioedema (HAE). This registrational trial aims to evaluate the efficacy of donidalorsen in reducing HAE attacks, with a participation of up to 84 patients. Positive Phase 2 data showed that donidalorsen was safe, well-tolerated, and resulted in a significant reduction of moderate to severe attacks. The company emphasizes its commitment to providing transformative treatments to the HAE patient community.
Ionis Pharmaceuticals (NASDAQ: IONS) announced presentations of its cardiovascular pipeline at the AHA Scientific Sessions from Nov. 13-15, 2021. Data from a Phase 1 study of ION449, aimed at lowering LDL-C levels, will feature prominently. ION449 is being developed in collaboration with AstraZeneca. Additionally, olezarsen, which targets triglyceride levels, will be discussed, with evaluations ongoing in two Phase 3 studies. A symposium on the prevalence of ATTR-CM in African American populations will also take place, highlighting health disparities.
Ionis Pharmaceuticals (Nasdaq: IONS) announced its participation in two upcoming virtual investor conferences. The events include the Stifel 2021 Healthcare Conference on November 15, 2021, and the Evercore ISI 4th Annual HealthCONx Conference on November 30, 2021. Details regarding presentation times may change, and updates will be available on their website. A live webcast of the presentations will be accessible in the Investors & Media section of Ionis' site, with replays available within 48 hours. For over 30 years, Ionis has led in RNA-targeted therapy, emphasizing their commitment to innovative treatments.
Ionis Pharmaceuticals announced positive results from the Phase 2 study of donidalorsen, its investigational antisense medicine, for treating hereditary angioedema (HAE). The results indicate that donidalorsen significantly reduced the number of attacks in patients compared to placebo. These findings will be presented at the ACAAI Annual Scientific Meeting in New Orleans from November 4-8. The study's success positions donidalorsen for advancement into Phase 3 development, reinforcing Ionis' commitment to innovative therapies.
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.