Ionis Pharmaceuticals Stock Price, News & Analysis

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.

Ionis Pharmaceuticals (Nasdaq: IONS), a leader in RNA-targeted therapeutics, will participate in upcoming virtual investor conferences, including the SVB Leerink 10th Annual Global Healthcare Conference on February 26, the Oppenheimer 31st Annual Healthcare Conference on March 17, and the J.P. Morgan 10th Annual Napa Valley Forum on March 29, 2021. Presentation details will be available on the Ionis website. The company is known for its pioneering work in RNA-targeted therapies and has a strong pipeline in neurological and cardiometabolic treatments.

Ionis Pharmaceuticals will host a live webcast on February 24, 2021, at 9:00 a.m. Eastern Time to discuss its 2020 financial results and provide updates on its pipeline and business progress. The webcast will be accessible via the company's website, with a replay available for a limited time. With over 30 years of experience in RNA-targeted therapy, Ionis has developed three marketed medicines and boasts a strong late-stage pipeline.

Ionis Pharmaceuticals (NASDAQ: IONS) announced the appointment of Dr. Eugene Schneider as executive vice president and chief clinical development officer. Since joining Ionis in 2013, he has contributed to over 20 clinical trials globally. Dr. Schneider brings extensive experience from his previous roles, including developing enzyme replacement therapies and overseeing clinical research. His addition is expected to strengthen Ionis' leadership and enhance their portfolio of innovative antisense medicines aimed at treating unmet medical needs. The company boasts a pipeline of over 40 novel therapies.

Ionis Pharmaceuticals (NASDAQ: IONS) announced that founder and executive chairman Stanley T. Crooke will retire from the company and its board effective June 2021. He will shift focus to his scientific interests and the n-Lorem Foundation, which provides free antisense treatments for ultra-rare diseases. Crooke has been instrumental in pioneering RNA-targeted therapeutics, leading to the development of over 20 marketed drugs. He will remain a scientific advisor to Ionis. The ongoing success of the n-Lorem Foundation suggests a continuing impact on patient treatment.

Ionis Pharmaceuticals highlighted its 2020 achievements and future plans during its Investor Day on Dec. 8, 2020. The company outlined a strategy focused on addressing rare diseases in neurology and cardiology while seeking to pioneer new treatment markets. Ionis aims to launch six or more products by 2026, with projections of double-digit revenue and earnings growth. Key highlights included progress in clinical trials, the successful integration of Akcea, and advancements in oral delivery systems.