Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis Pharmaceuticals announced the FDA's acceptance of its New Drug Application (NDA) for eplontersen, an investigational treatment for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The application is set for a Prescription Drug User Fee Act (PDUFA) action date of December 22, 2023. The FDA indicated no review issues and will not hold an advisory committee meeting. The NDA is based on the Phase 3 NEURO-TTRansform study, which showed significant reductions in serum transthyretin (TTR) levels and improved quality of life for patients. Eplontersen has already received Orphan Drug Designation.

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced its upcoming presentations at three investor conferences. These include the Cowen 43rd Annual Health Care Conference on March 7, 2023, the Oppenheimer 33rd Annual Virtual Healthcare Conference on March 15, 2023, and the Stifel 2023 Virtual CNS Days on March 29, 2023. Investors can access a live webcast during the events via the Investors & Media section on the Ionis website, with replays available shortly after. Ionis has been a pioneer in RNA-targeted therapy for over 30 years, focusing on innovative genetic medicine therapies.

Ionis Pharmaceuticals announced its submission of a New Drug Application (NDA) for eplontersen to the FDA, aiming to treat hereditary TTR amyloidosis. The company reported a Q4 2022 revenue of $152 million, down from $440 million in Q4 2021, with a full year revenue of $587 million, compared to $810 million in 2021. Operating expenses rose to $360 million for Q4, up from $219 million the previous year. Ionis expects 2023 revenue to exceed $575 million, while operational losses are projected to be below $425 million. The company also strengthened its financial position through significant transactions and is focusing on advancing its late-stage pipeline.

Ionis Pharmaceuticals has announced positive interim data from a Phase 2 open-label extension study of donidalorsen, an investigational treatment for hereditary angioedema (HAE). The data indicates a 95% reduction in HAE attack rates and a significant 24-point improvement in patients' quality of life after one year of treatment. Ionis is on track to launch donidalorsen, with ongoing enrollment in the Phase 3 OASIS study expected to be completed this year. The findings will be presented at the AAAAI Annual Meeting in San Antonio, Texas, from February 24-27, 2023.

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.