Ionis Pharmaceuticals develops RNA-targeted and antisense medicines for serious diseases, with marketed products and a pipeline concentrated in neurology, cardiometabolic disease and selected high-need areas. Company news regularly covers commercial performance for TRYNGOLZA and DAWNZERA, royalty-generating partnered medicines such as SPINRAZA and WAINUA, and collaboration milestones tied to late-stage programs.
Recurring updates also include FDA and global regulatory reviews, clinical data presentations, and launch preparation for programs such as olezarsen in severe hypertriglyceridemia, zilganersen in Alexander disease and partnered bepirovirsen in chronic hepatitis B. Financial releases typically discuss product sales, royalty revenue, R&D collaboration revenue, operating expenses, cash resources and guidance, alongside governance matters such as annual meeting materials and board transitions.
Ionis Pharmaceuticals has announced a live webcast scheduled for May 3, 2023, at 11:30 a.m. Eastern Time, to discuss its first quarter 2023 financial results. This event will provide insights into the company's financial performance, emphasizing its leadership in RNA-targeted therapies. Ionis has over three decades of experience and currently boasts three marketed medicines alongside a robust late-stage pipeline focused on cardiovascular and neurological treatments. Stakeholders can access the webcast and follow up with a replay through Ionis' investor relations page.
Ionis Pharmaceuticals announced new Phase 1b clinical data for IONIS-MAPTRx (BIIB080), revealing a dose-dependent reduction of tau protein in cerebrospinal fluid (CSF) in early-stage Alzheimer's patients. The study, presented at AD/PD 2023, showed a significant decrease in tau pathology measured by positron emission tomography (PET) at week 25 through week 100. In all dose groups, there was a 60% reduction in baseline CSF tau levels by study's end. The primary focus was on safety, with mild to moderate adverse events reported. The ongoing Phase 2 CELIA study for IONIS-MAPTRx is currently recruiting participants in the U.S.
Ionis Pharmaceuticals (NASDAQ: IONS) announced participation in upcoming investor conferences, enhancing its visibility in the healthcare sector. Notable events include the Guggenheim Virtual Genomic Medicines and Rare Disease Days on April 4, 2023, and the 22nd Annual Needham Virtual Healthcare Conference on April 17, 2023. A live webcast of the presentations will be available on the Ionis website, with replays accessible within 48 hours.
With over 30 years in RNA-targeted therapy, Ionis is a leader in genetic medicine, possessing three marketed medicines and a strong late-stage pipeline focusing on cardiovascular and neurological conditions.
Ionis Pharmaceuticals reported positive topline results from the 66-week analysis of the Phase 3 NEURO-TTRansform study for eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The study met co-primary endpoints, showing significant reductions in TTR levels and improved neuropathy symptoms and quality of life. Results will be shared at the AAN Annual Meeting in April, with U.S. FDA accepting a New Drug Application for eplontersen, targeting a decision by December 22, 2023. The data indicates eplontersen may fulfill a critical need for new treatments for this severe condition.
Ionis Pharmaceuticals announced a favorable outcome from the FDA's Advisory Committee meeting regarding tofersen, a treatment for SOD1 amyotrophic lateral sclerosis (ALS). The committee voted 9-0 that the evidence suggests a reduction in neurofilament light chain (NfL) levels indicates potential clinical benefits. However, there was mixed support for traditional approval, with 3 in favor, 5 against, and 1 abstention. If approved by the FDA, expected by April 25, 2023, tofersen would be the first treatment targeting a genetic cause of ALS. This represents a significant advancement in ALS therapeutics, as there are currently no genetically targeted treatments available.
Ionis Pharmaceuticals announced the FDA's acceptance of its New Drug Application (NDA) for eplontersen, an investigational treatment for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The application is set for a Prescription Drug User Fee Act (PDUFA) action date of December 22, 2023. The FDA indicated no review issues and will not hold an advisory committee meeting. The NDA is based on the Phase 3 NEURO-TTRansform study, which showed significant reductions in serum transthyretin (TTR) levels and improved quality of life for patients. Eplontersen has already received Orphan Drug Designation.
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced its upcoming presentations at three investor conferences. These include the Cowen 43rd Annual Health Care Conference on March 7, 2023, the Oppenheimer 33rd Annual Virtual Healthcare Conference on March 15, 2023, and the Stifel 2023 Virtual CNS Days on March 29, 2023. Investors can access a live webcast during the events via the Investors & Media section on the Ionis website, with replays available shortly after. Ionis has been a pioneer in RNA-targeted therapy for over 30 years, focusing on innovative genetic medicine therapies.
Ionis Pharmaceuticals announced its submission of a New Drug Application (NDA) for eplontersen to the FDA, aiming to treat hereditary TTR amyloidosis. The company reported a Q4 2022 revenue of $152 million, down from $440 million in Q4 2021, with a full year revenue of $587 million, compared to $810 million in 2021. Operating expenses rose to $360 million for Q4, up from $219 million the previous year. Ionis expects 2023 revenue to exceed $575 million, while operational losses are projected to be below $425 million. The company also strengthened its financial position through significant transactions and is focusing on advancing its late-stage pipeline.
Ionis Pharmaceuticals has announced positive interim data from a Phase 2 open-label extension study of donidalorsen, an investigational treatment for hereditary angioedema (HAE). The data indicates a 95% reduction in HAE attack rates and a significant 24-point improvement in patients' quality of life after one year of treatment. Ionis is on track to launch donidalorsen, with ongoing enrollment in the Phase 3 OASIS study expected to be completed this year. The findings will be presented at the AAAAI Annual Meeting in San Antonio, Texas, from February 24-27, 2023.
Ionis Pharmaceuticals will host a live webcast on February 22 at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2022 financial results. The event aims to provide insights into the company's performance, following its prominent role in RNA-targeted therapies for over 30 years. Ionis is recognized for its antisense technology and is currently advancing its cardiovascular and neurological franchises. Investors can access the webcast via the company’s investor relations page, with a replay available for a limited time.