Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced a live webcast on August 9 at 11:30 a.m. Eastern Time to discuss its second quarter financial results and update on its pipeline and business progress. The event underscores Ionis's commitment to transparency and communication with investors regarding its financial performance and future strategies. The webcast will be accessible on their website, and a replay will be available for a limited time.

Ionis Pharmaceuticals announced that the FDA has accepted its New Drug Application for tofersen, aimed at treating SOD1-ALS, a severe genetic form of ALS affecting around 330 people in the U.S. If approved, tofersen will be the first treatment targeting a genetic cause of this disease. Data from 12-month studies indicate earlier tofersen initiation slowed decline across multiple clinical measures and significantly reduced neurofilament levels, reflecting neurodegeneration. The FDA has granted priority review with a target action date of January 25, 2023.

Ionis Pharmaceuticals has announced the completion of patient enrollment in the pivotal Phase 3 Lp(a) HORIZON study of pelacarsen, with 8,325 participants. Pelacarsen targets elevated lipoprotein(a) levels, a significant risk factor for cardiovascular disease. This study, conducted by Novartis, aims to assess the safety and efficacy of pelacarsen in reducing cardiovascular events in patients with elevated Lp(a). There are currently no approved therapies for effectively lowering Lp(a), and topline results are expected in 2025.

Ionis Pharmaceuticals (Nasdaq: IONS) will participate in a virtual fireside chat at the H.C. Wainwright 1st Annual Hereditary Angioedema Conference on July 20, 2022. Investors can access the live webcast on the Ionis website; a replay will be available within 48 hours post-event. Ionis has been a leader in RNA-targeted therapy for over 30 years, with a strong pipeline and three marketed medicines. The company aims to revolutionize standards of care through innovative therapies.

Ionis Pharmaceuticals announced that Roche will advance the investigational drug IONIS-FB-L_Rx into a Phase 3 study for treating IgA nephropathy (IgAN), following positive results from a Phase 2 trial. This study showed a significant reduction in 24-hour urinary protein levels after 29 weeks. The Phase 2 study also demonstrated a favorable safety profile. Ionis will receive $55 million for licensing the drug and achieving a milestone. This partnership reinforces Ionis' commitment to addressing unmet needs in kidney diseases.

Ionis Pharmaceuticals announced positive interim results from the Phase 2b B-Clear study of bepirovirsen for chronic hepatitis B treatment, presented at the EASL International Liver Congress 2022. The study showed a virologic response (VR) in 28% of patients on nucleoside analogue treatment and 29% of those not on treatment after 24 weeks. GSK plans to initiate a Phase 3 study in the first half of 2023. Bepirovirsen aims to offer a functional cure for chronic hepatitis B, impacting approximately 300 million people globally.

Ionis Pharmaceuticals (Nasdaq: IONS) announced positive topline results from the Phase 3 NEURO-TTRansform study of eplontersen for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The 35-week interim analysis showed statistically significant improvements in both the modified Neuropathy Impairment Score +7 and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy. Eplontersen also proved to have a favorable safety profile. Ionis and AstraZeneca plan to submit a New Drug Application to the FDA this year.

Ionis Pharmaceuticals announced that the FDA granted orphan drug and rare pediatric disease designations to its investigational drug ION582, targeting Angelman syndrome. This neurogenetic disorder affects 1 in 12,000 to 20,000 people, resulting in severe developmental delays and caregiver dependence. The orphan drug status offers incentives such as tax credits and market exclusivity. ION582 is currently in a Phase 1/2 clinical trial with 44 participants. The company aims to expedite its development process to meet urgent patient needs.

The Phase 3 VALOR study of tofersen, an investigational therapy for SOD1-ALS, revealed that early initiation significantly slowed declines in clinical function, respiratory function, muscle strength, and quality of life. The 12-month data demonstrated a 33% reduction in SOD1 protein levels and a 51% decrease in neurofilament, indicating reduced neurodegeneration. Additionally, early initiation was correlated with a lower risk of death or permanent ventilation. However, the initial six-month study did not meet its primary endpoint.