Welcome to our dedicated page for Ionis Pharmaceuticals news (Ticker: IONS), a resource for investors and traders seeking the latest updates and insights on Ionis Pharmaceuticals stock.
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 (Nasdaq: IONS) announced positive topline results from the Phase 3 NEURO-TTRansform study of eplontersen for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The 35-week interim analysis showed statistically significant improvements in both the modified Neuropathy Impairment Score +7 and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy. Eplontersen also proved to have a favorable safety profile. Ionis and AstraZeneca plan to submit a New Drug Application to the FDA this year.
Ionis Pharmaceuticals announced that the FDA granted orphan drug and rare pediatric disease designations to its investigational drug ION582, targeting Angelman syndrome. This neurogenetic disorder affects 1 in 12,000 to 20,000 people, resulting in severe developmental delays and caregiver dependence. The orphan drug status offers incentives such as tax credits and market exclusivity. ION582 is currently in a Phase 1/2 clinical trial with 44 participants. The company aims to expedite its development process to meet urgent patient needs.
The Phase 3 VALOR study of tofersen, an investigational therapy for SOD1-ALS, revealed that early initiation significantly slowed declines in clinical function, respiratory function, muscle strength, and quality of life. The 12-month data demonstrated a 33% reduction in SOD1 protein levels and a 51% decrease in neurofilament, indicating reduced neurodegeneration. Additionally, early initiation was correlated with a lower risk of death or permanent ventilation. However, the initial six-month study did not meet its primary endpoint.
Ionis Pharmaceuticals (Nasdaq: IONS), located in Carlsbad, Calif., will present at the RBC Capital Markets Global Healthcare Conference on May 18, 2022. A live webcast of the presentation will be available on Ionis's website, with a replay accessible within 48 hours after the event. Ionis has over 30 years of experience in RNA-targeted therapy and boasts a strong pipeline focusing on cardiovascular and neurological franchises.
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported a strong start to 2022, achieving $142 million in total revenues for Q1, representing over 25% growth year-over-year. The firm remains on track to meet its 2022 financial guidance. Key highlights include positive data from studies of eplontersen, ION449, and donidalorsen. Operating expenses totaled $173 million on a non-GAAP basis, yielding a net loss of $39 million. Ionis maintains a robust cash position with $2.1 billion available for continued investments in its late-stage pipeline, positioning the company for future growth.
Ionis Pharmaceuticals (Nasdaq: IONS) will hold its 2022 virtual Annual Meeting of Stockholders on June 2, 2022. The schedule includes a brief stockholder meeting at 5:00 p.m. ET, where eligible stockholders can participate via a live webcast. Stockholders must use their proxy cards to access the meeting. Following this, a corporate update will be presented by CEO Brett P. Monia at 5:30 p.m. ET, accessible to all interested parties. Details for participation and additional resources are available on Ionis' website.
Ionis Pharmaceuticals (Nasdaq: IONS) will host a live webcast on May 4, 2022, at 11:30 a.m. Eastern Time to discuss its first quarter financial results and update on pipeline and business progress. This event follows over 30 years of Ionis's leadership in RNA-targeted therapies, with three marketed medicines and a strong late-stage pipeline focusing on cardiovascular and neurological areas. Access the webcast and replay details at ionispharma.com.
Ionis Pharmaceuticals and AstraZeneca announced positive results from the ETESIAN Phase 2b study of ION449 (AZD8233), aimed at reducing cholesterol in patients with hypercholesterolemia. The study met its primary endpoint, achieving a 79% reduction in LDL-C levels, with both treatment doses (50mg and 90mg) showing significant efficacy. The trial also confirmed a reduction in PCSK9 levels by up to 94%. ION449 was well tolerated, and the results support further clinical development as a potential next-generation treatment for patients at high cardiovascular risk.
Biogen and Ionis Pharmaceuticals announced the Phase 1 study results of BIIB078, an investigational drug for C9orf72-associated ALS. The study, involving 106 participants, found that BIIB078 did not demonstrate any clinical benefit and failed to meet secondary efficacy endpoints. As a result, the clinical program for BIIB078 will be discontinued. Although well-tolerated, the study highlighted that the disease mechanism is more complex than initially hypothesized. Both companies remain committed to advancing ALS research and addressing this neurodegenerative disease.
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has announced its participation in several upcoming investor conferences. Key events include:
- Stifel 2022 Virtual CNS Days on March 28, 2022
- Guggenheim Virtual Genomic Medicines and Rare Disease Day on April 1, 2022
- Needham 21st Annual Virtual Healthcare Conference on April 11, 2022
- Bank of America 2022 Healthcare Conference on May 11, 2022, in Las Vegas, NV