Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis Pharmaceuticals will host a live webcast on February 22 at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2022 financial results. The event aims to provide insights into the company's performance, following its prominent role in RNA-targeted therapies for over 30 years. Ionis is recognized for its antisense technology and is currently advancing its cardiovascular and neurological franchises. Investors can access the webcast via the company’s investor relations page, with a replay available for a limited time.

Ionis Pharmaceuticals has announced the initiation of two Phase 3 studies for bepirovirsen, an investigational antisense drug targeting chronic hepatitis B infection (CHB). This advancement expands Ionis' late-stage pipeline to seven therapies across nine indications. Ionis received a $15 million milestone payment from GSK upon progressing to Phase 3 clinical trials. Bepirovirsen has shown promising Phase 2b results, suggesting potential for a functional cure by reducing viral replication and stimulating immune response. Chronic HBV affects nearly 300 million people globally, emphasizing the need for effective treatments.

Ionis Pharmaceuticals (NASDAQ: IONS) announced that the FDA has granted Fast Track designation to olezarsen for treating familial chylomicronemia syndrome (FCS), a rare genetic disease characterized by dangerously high triglyceride levels and severe pancreatitis risk. This designation aims to expedite the review of drugs addressing unmet medical needs. Olezarsen, which inhibits apoC-III production, has completed enrollment in the Phase 3 BALANCE study, with data expected in the second half of 2023. Currently, there are no approved therapies for FCS in the U.S., highlighting the urgent need for effective treatment options for affected patients.

Ionis Pharmaceuticals announced that its Chief Scientific Officer, C. Frank Bennett, Ph.D., has been awarded the Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research. Dr. Bennett shares this honor with renowned scientists Don W. Cleveland and Timothy M. Miller. This recognition is for their advancements in antisense technology aimed at treating conditions like ALS, Huntington's, and Alzheimer's disease. Notably, their collaboration has led to four investigational medicines, including tofersen, which is under regulatory review with a PDUFA action date of April 25, 2023.

Ionis Pharmaceuticals has announced a significant royalty agreement with Royalty Pharma, involving an upfront payment of $500 million and potential milestones of up to $625 million. This deal allows Royalty Pharma to acquire an interest in Ionis' royalties from SPINRAZA and pelacarsen, while Ionis retains the majority of royalties and milestone payments from Novartis. The investment aims to bolster Ionis' late-stage programs and innovative genetic medicines pipeline, highlighting confidence in the commercial potential of both therapies.

Ionis Pharmaceuticals, based in Carlsbad, California, announced that Brett P. Monia, Ph.D., will present at the 41st Annual J.P. Morgan Healthcare Conference on January 11, 2023, at 9:45 a.m. PT (12:45 p.m. ET). The presentation will include a company overview followed by a Q&A session. A live webcast will be available on the Ionis website, with a replay accessible within 48 hours. Ionis has over 30 years of experience in RNA-targeted therapy and currently offers three marketed medicines alongside a robust late-stage pipeline.

On December 5, 2022, Ionis Pharmaceuticals announced the acceptance of its marketing authorization application (MAA) for tofersen by the European Medicines Agency (EMA) to treat SOD1-ALS. This follows the FDA's acceptance earlier in 2022, with a PDUFA date set for April 25, 2023. If approved, tofersen will be the first therapy targeting a genetic cause of ALS, which affects around 2% of ALS patients globally. The MAA includes data from several studies, and Biogen will maintain its early access program for tofersen across 34 countries.

Ionis Pharmaceuticals partners with Metagenomi to bolster its gene editing capabilities using Metagenomi's advanced systems. This collaboration aims to explore eight genetic targets, with initial research focused on four. Ionis will pay $80 million upfront, with potential milestone payments. The partnership aims to enhance therapeutic options, potentially transforming chronic therapies into curative treatments. A webcast will detail this announcement on Nov. 14, 2022, at 8 a.m. ET.

Ionis Pharmaceuticals (Nasdaq: IONS) reported positive interim results from a Phase 2 open-label extension study of donidalorsen for hereditary angioedema (HAE). After one year of treatment, patients experienced a 95% mean reduction in HAE attacks, with 99.6% of study days being attack-free. No new safety issues were identified, and adverse events did not lead to study discontinuation. The encouraging data were presented at the ACAAI Annual Meeting, reinforcing donidalorsen's potential as a leading HAE treatment.