Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis Pharmaceuticals (Nasdaq: IONS) has completed enrollment in the pivotal trial of zilganersen (ION373), an investigational RNA-targeted medicine for Alexander disease (AxD). This rare, progressive neurological disorder affects an estimated one in one million people in the U.S. The trial's primary endpoint is percent change from baseline in gait speed, assessed by the 10-Meter Walk Test. Topline data are expected in the second half of 2025.

Zilganersen aims to address the underlying cause of AxD by targeting excess GFAP accumulation resulting from genetic variants in the GFAP gene. This approach could potentially slow or stabilize disease progression in AxD patients, who currently lack treatments that address the root cause of their condition.

Ionis Pharmaceuticals (NASDAQ: IONS) has announced a live webcast scheduled for Monday, July 22, at 8:00 a.m. Eastern Time. This webcast will discuss the results from the Phase 1/2a HALOS study of ION582, a treatment for Angelman syndrome. The findings will also be presented at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium on Wednesday, July 24, in Sandusky, Ohio. Interested parties can access the webcast via the provided link, with a replay available for a time.

Ionis Pharmaceuticals announced the FDA's acceptance of its New Drug Application (NDA) for olezarsen, an investigational RNA-targeted medicine for treating familial chylomicronemia syndrome (FCS). The FDA has granted Priority Review with a PDUFA date set for December 19, 2024. Olezarsen has also received Fast Track, Orphan Drug, and Breakthrough Therapy designations.

Additionally, Ionis completed Phase 3 enrollment for three studies (CORE, CORE2, and ESSENCE) evaluating olezarsen for severe hypertriglyceridemia (sHTG), with results anticipated in the second half of 2025. These studies involve over 2,400 patients and aim to address the life-threatening complications associated with sHTG.

The NDA is supported by positive Phase 3 study results presented at the 2024 ACC Annual Meeting and published in the New England Journal of Medicine.

Ionis Pharmaceuticals has entered an expanded licensing agreement with Otsuka Pharmaceutical for the investigational RNA-targeted medicine donidalorsen, aimed at treating hereditary angioedema (HAE).

Under the agreement, Otsuka gains exclusive rights to commercialize donidalorsen in the Asia-Pacific region while also handling regulatory filings and localized development. Ionis will continue to manage global development and plans to submit a New Drug Application to the FDA this year for U.S. approval, aiming for an independent U.S. launch.

Previously, Otsuka secured licensing for Europe and is preparing a Marketing Authorization Application to the EMA this year. Ionis will receive a $20 million upfront payment and milestone payments from Otsuka, along with tiered royalties.

Recent positive results from Phase 3 trials were presented at the EAACI Annual Congress and published in The New England Journal of Medicine.

Ionis Pharmaceuticals announced that its management will participate in a fireside chat at the TD Cowen Genetic Medicines & RNA Summit on June 20, 2024. The event will be virtual, and a live webcast will be available on the Ionis website. Interested parties can access the webcast on the Investors & Media section of the website, with a replay available within 48 hours for a time.

Ionis Pharmaceuticals revealed positive Phase 3 results for donidalorsen in treating hereditary angioedema (HAE). The OASIS-HAE and OASISplus studies showed significant reductions in mean monthly HAE attack rates, with a >90% reduction over one year. Patients switching from other treatments saw a 62% further reduction, and 84% preferred donidalorsen. Favorable safety and tolerability were reported across all cohorts. Ionis plans to seek regulatory approval based on these results. These findings will be presented at the EAACI Congress 2024, and Ionis aims to launch donidalorsen as part of its independent commercial pipeline.

Ionis Pharmaceuticals (NASDAQ: IONS) announced a live webcast scheduled for May 31, 2024, at 8:00 a.m. Eastern Time to discuss the donidalorsen Phase 3 OASIS-HAE study results. These results will be presented during three late-breaking oral presentations at the 2024 EAACI Annual Meeting in Valencia, Spain. The webcast and a -time replay can be accessed at the Ionis website.

Ionis Pharmaceuticals (Nasdaq: IONS) will present new subgroup analyses from its Phase 3 NEURO-TTRansform study of WAINUA™ (eplontersen) at the 2024 International Symposium on Amyloidosis (ISA). The study highlights consistent benefits in neuropathy impairment and quality of life across various patient segments. WAINUA, approved by the FDA in December 2023 for treating hATTR-PN, will have several notable presentations on May 29, 2024. Additionally, eplontersen is being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This study, with over 1,400 patients, is the largest in this patient population.

Ionis Pharmaceuticals and Biogen announced the discontinuation of BIIB105 (ION541), an investigational antisense oligonucleotide for amyotrophic lateral sclerosis (ALS), based on Phase 1/2 ALSpire study results. Despite demonstrating statistically significant reductions in cerebrospinal fluid ATXN2 protein, BIIB105 did not lower plasma neurofilament light chain (NfL) levels, nor did it impact clinical outcomes such as function, breathing, and strength over a 6-month period. Common adverse events included procedural pain, headache, and fall, with higher discontinuation rates in the BIIB105 group. Further analyses of the data are ongoing, and results will be presented at the European Network to Cure ALS (ENCALS) meeting in June 2024.