Ionis Pharmaceuticals, Inc. - IONS STOCK NEWS

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

Ionis Pharmaceuticals has published positive Phase 2 results for donidalorsen, an investigational treatment for hereditary angioedema (HAE). The study showed a 90% reduction in angioedema attacks with 80 mg monthly doses compared to placebo, alongside significant improvement in quality of life. The treatment was well-tolerated with no serious adverse events noted. These findings support the ongoing Phase 3 study for donidalorsen, emphasizing Ionis' commitment to addressing unmet medical needs in HAE patients.

Ionis Pharmaceuticals (Nasdaq: IONS) announced it is accepting applications for the Janice Wiesman Young Investigator Grant Program, honoring Dr. Wiesman for her contributions to understanding transthyretin amyloidosis (ATTR). The program offers $50,000 annually for two years to U.S. researchers advancing ATTR knowledge, with applications due by April 4, 2022. The inaugural grants were awarded to Dr. Trejeeve Martyn and Dr. Jeremy Slivnick for their research on cardiac amyloidosis. March is also recognized as Amyloidosis Awareness Month, highlighting the disease's significance.

Ionis Pharmaceuticals (Nasdaq: IONS) will engage in a panel discussion focused on orphan neurological disease therapeutics at the upcoming Cowen 42nd Annual Health Care Conference. The event is scheduled for 9:10 a.m. ET on March 7, 2022. Interested parties can access a live webcast on the company's Investors & Media section of their website, with a replay available for a limited time. Ionis boasts over 30 years in RNA-targeted therapy, including three marketed medicines and a robust late-stage pipeline that emphasizes neurological and cardiometabolic franchises.

Ionis Pharmaceuticals reported strong financial results for 2021, exceeding guidance with total revenues of $810 million, driven by significant partner payments. The company has a solid cash position of $2.1 billion, enabling continued investment in R&D. Key achievements include collaborations with AstraZeneca on eplontersen and ongoing Phase 3 studies for multiple therapies. Positive Phase 2 results for olezarsen and donidalorsen were also announced. For 2022, Ionis expects revenue of over $575 million and a net loss of less than $275 million.

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) will host a live webcast on February 24 at 11:30 a.m. ET to discuss its fourth quarter and full year 2021 financial results. The webcast will also cover the company's pipeline and business progress. Access the webcast and replay through the company's investor relations page. Ionis is a leader in RNA-targeted therapy, offering three marketed medicines and a robust late-stage pipeline focused on neurological and cardiometabolic diseases.

Pfizer and Ionis Pharmaceuticals announced the termination of the clinical development program for vupanorsen (PF-07285557), an investigational antisense therapy aimed at cardiovascular risk reduction and severe hypertriglyceridemia. Despite meeting its primary endpoint of reducing non-HDL cholesterol and triglycerides in a Phase 2b study, the results were insufficient to justify further development. Additionally, vupanorsen was linked to dose-dependent liver fat increases and elevated liver enzymes. Pfizer will return development rights to Ionis, which licensed vupanorsen in November 2019.

Ionis Pharmaceuticals has received orphan drug designation from the FDA for eplontersen, an investigational antisense therapy aimed at treating transthyretin-mediated amyloidosis. This designation provides Ionis with key incentives, including tax credits and seven years of market exclusivity after approval. Eplontersen, currently in Phase 3 trials, targets both hereditary and non-hereditary forms of the disease, which can lead to severe complications such as heart failure and nerve damage. The company's collaboration with AstraZeneca further supports the development of this promising treatment.