Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis Pharmaceuticals has initiated a pivotal clinical study of its investigational medicine, ION373, targeting Alexander disease, a rare and fatal neurological disorder. This multi-center, double-blind study will involve up to 58 patients over a 60-week period, assessing the safety and efficacy of ION373 in reducing toxic GFAP levels in the brain. Alexander disease, caused by a genetic mutation, affects roughly one in a million births. ION373 has received orphan drug and rare pediatric disease designations from the FDA, highlighting its potential significance in the therapeutic landscape.

Ionis Pharmaceuticals announced the pricing of $550.0 million in 0% Convertible Senior Notes due 2026, increasing from the initial $500.0 million offering. The private placement is set to close on April 12, 2021. The expected net proceeds are around $536.2 million, earmarked for repurchasing existing notes, funding convertible note hedge transactions, and other corporate purposes, including R&D. The conversion rate is 17.2902 shares per $1,000 of notes, with an initial conversion price of $57.84 per share, representing a 32.5% premium over the last sale price.

Ionis Pharmaceuticals intends to offer $500.0 million of Convertible Senior Notes due 2026 in a private placement to qualified institutional buyers. An option for an additional $75.0 million is also available. Proceeds will be used to repurchase certain existing notes, cover convertible note hedge transactions, and support general corporate purposes including R&D and infrastructure expansion. The notes and associated warrants are not registered under the Securities Act and may not be sold in the U.S. without registration or an exemption.

Ionis Pharmaceuticals announced the initiation of a Phase 3 clinical trial for ION363, an investigational antisense medicine targeting FUS mutations in patients with FUS-ALS, a form of amyotrophic lateral sclerosis. The trial aims to demonstrate the efficacy of ION363, which has shown promise in preclinical models by reducing the production of the FUS protein. The global study will enroll up to 64 patients over a 102-week period, marking a significant step towards addressing the urgent need for treatments in this aggressive disease.

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced positive results from a Phase 2 clinical study of IONIS-PKK-LRx for hereditary angioedema (HAE). The treatment demonstrated a 90% mean reduction in monthly HAE attacks compared to placebo, with 92% of patients attack-free in weeks five to 17. IONIS-PKK-LRx, developed using advanced antisense technology, could potentially be a best-in-class prophylactic therapy. The study involved 20 adults, showing mild adverse events. Ionis plans to present full findings at a medical conference later this year.

Ionis Pharmaceuticals (Nasdaq: IONS) announced participation in two upcoming investor conferences:

• 3rd Annual Stifel CNS Day on April 1, 2021
• 20th Annual Needham Healthcare Conference on April 12, 2021

Updates on presentation details will be available on the company's website. A live webcast will be accessible in the Investors & Media section, with replays available within 48 hours. Ionis, a pioneer in RNA-targeted therapies, boasts three marketed medicines and a robust pipeline, especially in neurological and cardiometabolic fields.

Ionis Pharmaceuticals announced that Roche has decided to discontinue dosing in the Phase III GENERATION HD1 study of tominersen for Huntington's disease based on a review of its potential benefit/risk profile. Although no new safety signals were found, the decision reflects concerns about the treatment's efficacy. Participants will still be monitored for safety and clinical outcomes. The open-label GEN-EXTEND study will also pause dosing, while other studies continue. Roche will share further information once the full data is available.

Ionis Pharmaceuticals has launched the Janice Wiesman Young Investigator Grant Program to support U.S. researchers focusing on transthyretin amyloidosis (ATTR). The program commemorates Dr. Janice Wiesman and will award $50,000 annually to two young investigators for up to two years. This initiative aims to enhance understanding, diagnosis, and management of ATTR, a rare and progressive disease affecting various organs. Applications are open until April 2, 2021.

Ionis Pharmaceuticals reported its financial results for Q4 2020, achieving total revenues of $729 million, with net income of $111 million on a non-GAAP basis. The company aims to commercialize its pipeline, driven by strong momentum from marketed products like SPINRAZA, which generated $2 billion in sales. Ionis plans to expand its pipeline, targeting 12 or more products in the market by 2026. The company ended 2020 with a robust cash position of $1.9 billion, which supports further investments in its pipeline.