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. (NASDAQ: IONS) is a biotechnology company that focuses on RNA-targeted medicines for serious diseases, and its news flow reflects this emphasis on clinical, regulatory and commercial milestones. Company updates frequently highlight progress in neurology and cardiometabolic programs, as well as developments in rare conditions where few or no disease-modifying treatments exist.
Recent Ionis news has featured pivotal clinical trial readouts, such as Phase 3 CORE and CORE2 results for olezarsen in severe hypertriglyceridemia, showing large reductions in triglyceride levels and acute pancreatitis events with favorable safety and tolerability. The company has also reported positive pivotal data for zilganersen in Alexander disease, describing evidence of disease-modifying impact in this rare and often fatal neurological condition.
Regulatory and commercial milestones are another major theme. Ionis has announced FDA approval of TRYNGOLZA (olezarsen) for familial chylomicronemia syndrome and DAWNZERA (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema in adults and adolescents 12 years and older. News items also cover FDA Breakthrough Therapy designations for olezarsen in severe hypertriglyceridemia and zilganersen in Alexander disease, as well as European regulatory opinions and anticipated launches.
Partnered program updates appear regularly, including GSK’s Phase 3 B-Well 1 and B-Well 2 results for bepirovirsen in chronic hepatitis B, Novartis’ Lp(a) HORIZON study of pelacarsen in cardiovascular disease, and AstraZeneca collaborations on WAINUA and eplontersen. Investors can also find coverage of Ionis’ financing activities, such as convertible note offerings, and its participation in healthcare and investor conferences.
For followers of IONS stock, this news page provides a centralized view of Ionis’ clinical trial milestones, FDA and EMA interactions, product launches, collaboration updates and capital markets events, helping readers understand how the company’s RNA-targeted pipeline and marketed medicines are evolving over time.
Ionis Pharmaceuticals (Nasdaq: IONS) announced its participation in three upcoming virtual investor conferences. Management will present at:
- Stifel 2020 Healthcare Conference on November 16, 2020;
- Evercore ISI 3rd Annual HealthCONx Conference on December 1, 2020;
- Piper Sandler 32nd Annual Healthcare Conference on December 3, 2020.
Live webcasts of each presentation will be available, with replays accessible for a limited time. For updates, visit www.ionispharma.com.
Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals (NASDAQ: IONS), has received approval from the Scottish Medicines Consortium (SMC) for volanesorsen, enabling access via the NHS in Scotland for treating familial chylomicronaemia syndrome (FCS). This follows NICE's earlier approval in England. Volanesorsen is the only treatment available for FCS in the UK, aimed at reducing triglyceride levels significantly. The SMC's decision is a milestone for FCS patients, allowing access while additional clinical data is collected over three years.
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Ionis Pharmaceuticals reported its third-quarter 2020 financial results, showing a net income of $5 million (non-GAAP) but a GAAP net loss of $31 million. The company achieved revenue growth, with commercial revenues of $95 million driven by SPINRAZA royalties of $74 million. The recent acquisition of Akcea strengthens Ionis's pipeline, projecting 10 or more marketing applications by 2025. Cash reserves stand at $2.3 billion post-acquisition, maintaining solid financial health.
Ionis Pharmaceuticals and Akcea Therapeutics have won the Prix Galien USA Award for the Best Biotechnology Product for 2020, recognizing TEGSEDI, the first self-administered treatment for the polyneuropathy of hereditary ATTR amyloidosis. Presented in a virtual ceremony on October 29, 2020, this prestigious award highlights significant advancements in biomedicine. Brett P. Monia emphasized TEGSEDI's role in improving the lives of patients affected by this severe disease. TEGSEDI is FDA-approved and offers hope for individuals suffering from hATTR amyloidosis.
Ionis Pharmaceuticals announced the dosing of the first patients with ION541, an investigational therapy for amyotrophic lateral sclerosis (ALS). This milestone is significant as ION541 targets ataxin-2 RNA, potentially reversing TDP-43 toxicity, a common characteristic of ALS. Supported by a $10 million payment from Biogen, this Phase 1/2 trial aims to address both familial and sporadic ALS cases. Ionis continues to advance its ALS pipeline, which includes tofersen and IONIS-C9Rx, enhancing its commitment to developing innovative therapies for this devastating condition.
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) will host a live webcast on November 4, 2020, at 11:30 a.m. ET to discuss its third quarter 2020 financial results and provide updates on its pipeline and business progress. The webcast will be available on their website, www.ionispharma.com, with a replay for a limited time after the event.
Ionis Pharmaceuticals (NASDAQ: IONS) announced that IONIS-PKK-LRx is being tested in a Phase 2 clinical study aimed at reducing respiratory complications in COVID-19 patients. Conducted in Brazil, this study will enroll up to 110 patients across 25 hospitals. The primary endpoint is to monitor the number of days patients are alive and free of oxygen support over 15 days. IONIS-PKK-LRx targets bradykinin signaling, a key factor in severe COVID-19 cases. Additionally, promising data from a related HAE study has been published.
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Ionis Pharmaceuticals (NASDAQ: IONS) announced significant results from a clinical trial of IONIS-ENAC-2.5Rx, an investigational antisense medicine for cystic fibrosis (CF). The trial demonstrated a mean 55.6% reduction in ENaC mRNA expression at a 75 mg dose (p<0.05), marking a breakthrough in aerosol delivery methods. This study lays the groundwork for future developments in treating CF and other lung diseases. The company plans to present additional data at the 2020 North American Cystic Fibrosis Conference from Oct. 21-23 and initiate a study for COPD later this year.