DAWNZERA demonstrated significant and sustained HAE attack rate reduction and long-term disease control Offers longest dosing option for HAE, with dosing every 4 or 8 weeks Compelling profile supported by recently published switch data Ionis’ second independent launch in just nine months, with potential for two additional launches next year Ionis to host webcast today at 12:15pm ET CARLSBAD, Calif. / Aug 21, 2025 / Business... Read More

TRYNGOLZA TM delivers $19 million in net product sales in the second quarter 2025 Donidalorsen approval in hereditary angioedema (HAE) anticipated next month; Ionis’ second independent launch Phase 3 data from the pivotal CORE and CORE2 studies in severe hypertriglyceridemia (sHTG) expected in September 2025 Increasing 2025 financial guidance based on strong performance and improved outlook CARLSBAD, Calif. / Jul 30, 2025 /... Read More

Recommendation based on Phase 3 Balance results, which showed a significant reduction of triglycerides and substantial reduction of acute pancreatitis events with TRYNGOLZA European Commission decision expected by Q4 2025 CARLSBAD, Calif. / Jul 25, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Sobi® today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European... Read More

Patients switched to donidalorsen without increased breakthrough attacks and experienced further reductions in mean attack rate from baseline 84% of patients surveyed preferred donidalorsen over their prior treatment Donidalorsen is currently under review with the U.S. FDA as a potential first-in-class prophylactic treatment for HAE; PDUFA date August 21 CARLSBAD, Calif. / Jul 21, 2025 / Business Wire / Ionis... Read More

Webcast scheduled for Wednesday, July 30 at 11:30 a.m. Eastern Time CARLSBAD, Calif. / Jul 16, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, July 30 th at 11:30 a.m. Eastern Time to discuss its second quarter 2025 financial results and progress on key programs. The webcast may be accessed at... Read More

Investigational salanersen (ION306/BIIB115) developed using novel Ionis antisense chemistry with the potential to achieve high efficacy and once-yearly dosing Interim Phase 1 data show children with SMA previously treated with gene therapy experienced a substantial slowing of neurodegeneration and clinically meaningful improvements in motor function following initiation of salanersen Biogen is engaging with regulators to... Read More

CARLSBAD, Calif. / Jun 12, 2025 / Business Wire / Ionis Pharmaceuticals, Inc . (Nasdaq: IONS) today announced that Richard Geary, Ph.D., executive vice president and chief development officer, will retire effective January 2026 and that Holly Kordasiewicz, Ph.D., currently senior vice president, neurology, will succeed him in the role. “Richard has made tremendous contributions to Ionis over his 30-year tenure during which... Read More

Completion of enrollment anticipated in 2026 CARLSBAD, Calif. / Jun 11, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the first participant has been dosed in the global Phase 3 REVEAL study, which is designed to evaluate the efficacy and safety of ION582, an investigational medicine for the treatment of people living with Angelman syndrome (AS), a serious and rare neurodevelopmental... Read More

Olezarsen met the primary endpoint with a statistically significant mean reduction in triglycerides versus placebo at 80 mg and 50 mg doses Olezarsen met all key secondary endpoints Olezarsen demonstrated a favorable safety and tolerability profile Nearly 1,500-person Phase 3 study supports the exposure database for olezarsen Data from pivotal Phase 3 CORE and CORE2 studies of olezarsen in severe hypertriglyceridemia (sHTG)... Read More

CARLSBAD, Calif. / May 06, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will conduct its 2025 virtual Annual Meeting of Stockholders followed by a general corporate update on Thursday, June 5, 2025. The agenda for the event is as follows: 5:00 p.m. – 5:15 p.m. ET (2:00 p.m. – 2:15 p.m. PT) – Virtual Annual Meeting of Stockholders All stockholders of record at the close of... Read More

Encouraging start to first independent launch with TRYNGOLZA TM On track for second independent launch with donidalorsen PDUFA August 21, 2025 Increasing 2025 financial guidance by more than 20% CARLSBAD, Calif. / Apr 30, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results for the first quarter ended March 31, 2025. “With an encouraging start to the TRYNGOLZA... Read More

Despite currently available treatment options, the majority of surveyed adults with HAE reported making tradeoffs in their daily lives due to the unexpected and unpredictable nature of HAE attacks Opportunity exists for more consistent use of tools to support transparent communications between patients and their doctors, as HCPs may underestimate the full burden of HAE CARLSBAD, Calif. / Apr 29, 2025 / Business Wire / Ionis... Read More

Webcast scheduled for Wednesday, April 30 at 11:30 a.m. Eastern Time CARLSBAD, Calif. / Apr 16, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, April 30 th at 11:30 a.m. Eastern Time to discuss its first quarter 2025 financial results. The webcast may be accessed at https://ir.ionis.com/events-and-presentations/upcoming-events . A replay will be... Read More

Webinar scheduled for Monday, April 14 at 11:00 a.m. ET CARLSBAD, Calif. / Apr 03, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today it will host a webinar on Monday, April 14 th at 11:00 a.m. ET focused on the risks, clinical presentation and significant unmet medical needs associated with severe hypertriglyceridemia (sHTG). Key thought leaders in lipidology, cardiology and endocrinology will... Read More

CARLSBAD, Calif. / Mar 31, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: 24 th Annual Needham Virtual Healthcare Conference on Monday, April 7, 2025 Bank of America 2025 Health Care Conference on Wednesday, May 14, 2025 2025 RBC Capital Markets Global Healthcare Conference on Wednesday, May 21, 2025 A... Read More

Agreement enables olezarsen to reach people living with conditions associated with elevated triglycerides across the world Ionis will continue to independently commercialize TRYNGOLZA™ (olezarsen) in the U.S. CARLSBAD, Calif. / Mar 26, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it has entered into a license agreement under which Sobi® receives exclusive rights in countries outside... Read More

CARLSBAD, Calif. & OSAKA, Japan / Mar 11, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Ono Pharmaceutical Co., Ltd (Ono), today announced that the two companies have entered into a license agreement in which Ono obtains exclusive global rights for the development and commercialization of sapablursen, an investigational RNA-targeted medicine for polycythemia vera (PV), a rare and potentially... Read More

Second major approval for WAINZUA, which is marketed in the U.S. as WAINUA™ EU approval based on NEURO-TTRansform Phase 3 results showing WAINZUA demonstrated consistent and sustained benefit improving neuropathy impairment and quality of life versus placebo CARLSBAD, Calif. / Mar 10, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that Ionis and AstraZeneca’s WAINZUA (eplontersen) has been... Read More

CARLSBAD, Calif. / Feb 25, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: Raymond James 46 th Annual Institutional Investors Conference on Tuesday, March 4, 2025 TD Cowen 45 th Annual Health Care Conference on Tuesday, March 4, 2025 Leerink Partners Global Healthcare Conference 2025 on Tuesday, March... Read More

Data also show significant and clinically meaningful improvements in quality-of-life measures in new analyses from Phase 3 and Phase 2 clinical program With a PDUFA date of August 21, 2025, donidalorsen positioned to be Ionis’ second independent commercial launch CARLSBAD, Calif. / Feb 20, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it will present additional data from the pivotal... Read More

First independent launch underway following TRYNGOLZA TM approval Second independent launch on track with donidalorsen PDUFA August 21, 2025 WAINUA TM U.S. launch delivering accelerating sequential growth Ionis exceeds 2024 financial guidance and provides full year 2025 guidance CARLSBAD, Calif. / Feb 19, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results for... Read More

Webcast scheduled for Wednesday, February 19 at 11:30 a.m. Eastern Time CARLSBAD, Calif. / Feb 05, 2025 / Business Wire / Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, February 19 th at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2024 financial results. The webcast may be accessed at... Read More

Accelerating value creation with numerous commercial, regulatory and pipeline milestones Ionis outlines clear path to sustained positive cash flow CARLSBAD, Calif. , Jan. 13, 2025 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced highlights from the Company's 2024 achievements and previewed important milestones expected in 2025. Ionis will provide a business update at the 42nd Annual J.P. Morgan... Read More

CARLSBAD, Calif. , Jan. 8, 2025 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Brett P. Monia , Ph.D., chief executive officer, will present a company overview at the 43 rd Annual J.P. Morgan Healthcare Conference at 10:30 a.m. PT on Wednesday, January 15, 2025 . A live webcast of the presentation can be accessed on the Investors & Media section of the Ionis website at... Read More

TRYNGOLZA shown to significantly reduce triglycerides and substantially reduce acute pancreatitis events in adults with FCS; a rare, highly debilitating and life-threatening disease Indicated for adults with FCS regardless of genetically or clinically confirmed diagnosis TRYNGOLZA is the first of four independent launches planned over the next three years, pending approvals Ionis to host webcast today at 6:45pm ET CARLSBAD,... Read More

CARLSBAD, Calif. , Nov. 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: Guggenheim's Inaugural Healthcare Conference on Wednesday, November 13, 2024 UBS Global Healthcare Conference on Wednesday, November 13, 2024 Stifel 2024 Healthcare Conference on Monday, November 18, 2024 Jefferies London... Read More

WAINUA TM U.S. launch progressing well; approved in UK; positive CHMP opinion Olezarsen FCS PDUFA December 19, 2024 Donidalorsen HAE PDUFA August 21, 2025 ; EU regulatory submission in process On track to achieve 2024 P&L financial guidance; increased 2024 cash guidance CARLSBAD, Calif. , Nov. 6, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company") today reported financial results for the third... Read More

Positive End of Phase 2 discussion with FDA, including alignment on Phase 3 design Bayley-4 expressive communication selected as Phase 3 study primary endpoint Initiation of ION582 Phase 3 study planned for H1 2025 Ionis to share ION582 program update at the FAST Global Science Summit in November CARLSBAD, Calif. , Nov. 6, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the pivotal Phase 3... Read More

Donidalorsen will be a first-in-class RNA-targeted medicine for hereditary angioedema, assuming approval Donidalorsen PDUFA date set for August 21, 2025 Donidalorsen has the potential to be Ionis' second independent commercial launch CARLSBAD, Calif. , Nov. 4, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug... Read More

Webcast scheduled for Wednesday, November 6 at 11:30 a.m. Eastern Time CARLSBAD, Calif. , Oct. 23, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, November 6 th at 11:30 a.m. Eastern Time to discuss its third quarter 2024 financial results. The webcast may be accessed at https://ir.ionis.com/events-and-presentations/upcoming-events . A replay will... Read More

Recommendation based on NEURO-TTRansform Phase 3 results showing WAINZUA demonstrated consistent and sustained benefit improving neuropathy impairment and quality of life versus placebo CARLSBAD, Calif. , Oct. 21 , 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that Ionis' and AstraZeneca's WAINZUA (eplontersen) has been recommended for approval by the Committee for Medicinal Products for... Read More

Data continue to demonstrate donidalorsen potential to deliver significant and sustained reductions in HAE attacks up to three years with monthly or every two-month dosing CARLSBAD, Calif. , Oct. 3, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will present new three-year data from the Phase 2 open-label extension (OLE) study as well as additional results from the pivotal Phase 3... Read More

Zilganersen is the first investigational medicine in clinical development for adults and children living with Alexander disease, an ultra-rare neurological condition CARLSBAD, Calif. , Oct. 1, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted zilganersen Fast Track designation for the treatment of children and adults with an... Read More

CARLSBAD, Calif. , Sept. 9, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the pricing of an underwritten public offering of 11,500,000 shares of its common stock. The shares of common stock are being sold at a public offering price of $43.50 per share. The aggregate gross proceeds to Ionis from this offering are expected to be approximately $500.3 million , before deducting underwriting... Read More

CARLSBAD, Calif. , Sept. 9, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced a proposed underwritten public offering of $500.0 million of its common stock. Ionis intends to grant the underwriters a 30-day option to purchase up to an additional $75.0 million of shares of its common stock offered in the public offering on the same terms and conditions. Ionis intends to use the net proceeds from... Read More

CARLSBAD, Calif. , Aug. 28, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: 2024 Wells Fargo Healthcare Conference on Wednesday, September 4, 2024 Morgan Stanley 22 nd Annual Global Healthcare Conference on Thursday, September 5, 2024 A live webcast of these presentations can be accessed on the Investors... Read More

WAINUA TM U.S. launch progressing well; approved in Canada ; EU approval decision expected this year Olezarsen PDUFA December 19, 2024 for FCS Positive Phase 3 donidalorsen data for HAE; preparing U.S. and EU regulatory submissions On track to achieve 2024 financial guidance CARLSBAD, Calif. , Aug. 1, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company"), today reported financial results for the... Read More

ION582 showed robust and consistent benefit in communication, cognition and motor function in a broad patient population evaluated with a comprehensive set of assessment tools that collect input from parents and clinicians 97% of patients in the medium and high dose groups saw improvement in overall Angelman syndrome symptoms as measured by the SAS-CGI-C Improvements on the Bayley-4 in cognition, communication and motor... Read More

Webcast scheduled for Thursday, August 1 at 11:30 a.m. Eastern Time CARLSBAD, Calif. , July 19, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Thursday, August 1 st at 11:30 a.m. Eastern Time to discuss its second quarter 2024 financial results. The webcast may be accessed at https://ir.ionis.com/events-and-presentations/upcoming-events . A replay will be... Read More

Zilganersen is the first investigational medicine in clinical development for people with Alexander disease, a rare, life-threatening neurological condition CARLSBAD, Calif. , July 18, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it has completed enrollment in the pivotal trial of zilganersen (ION373), an investigational RNA-targeted medicine in development for the treatment of... Read More

Webcast scheduled for Monday, July 22 at 8:00 a.m. Eastern Time CARLSBAD, Calif. , July 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Monday, July 22 nd at 8:00 a.m. Eastern Time to discuss results from the Phase 1/2a HALOS study of ION582 for the treatment of people with Angelman syndrome. Results from the HALOS study will also be presented on... Read More

Olezarsen PDUFA date set for December 19, 2024 for treatment of familial chylomicronemia syndrome Phase 3 enrollment completed in CORE, CORE2 and ESSENCE evaluating olezarsen for the treatment of severe hypertriglyceridemia, with results expected in 2H 2025 CARLSBAD, Calif. , June 25, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted... Read More

Otsuka will be responsible for commercialization efforts for donidalorsen across both Asia Pacific and Europe Ionis plans to independently bring donidalorsen to U.S. patients, if approved CARLSBAD, Calif. , June 18 , 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it has entered into a license agreement with Otsuka Pharmaceutical Co., Ltd. (Otsuka) under which Otsuka obtains exclusive... Read More

CARLSBAD, Calif. , June 17, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in a fireside chat at the virtual TD Cowen Genetic Medicines & RNA Summit on Thursday, June 20, 2024 . A live webcast of this presentation can be accessed on the Investors & Media section of the Ionis website at https://ir.ionis.com/events-and-presentations/upcoming-events . A replay... Read More

Donidalorsen delivered significant and sustained reductions in HAE attacks, with high levels of disease control and improvement in quality of life measures with monthly or every two-month dosing; continued attack rate reduction over time In first-of-its-kind prospective analysis, patients switching from prior prophylactic treatment to donidalorsen experienced further reductions in mean monthly HAE attack rates from baseline... Read More

Webcast scheduled for Friday, May 31 at 8:00 a.m. Eastern Time CARLSBAD, Calif. , May 28, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Friday, May 31 th at 8:00 a.m. Eastern Time to discuss the donidalorsen Phase 3 OASIS-HAE and OASISplus (Open-Label Extension and Switch cohorts) study results that will be presented in three late-breaking oral... Read More

– Results across NEURO-TTRansform subgroups show consistent benefit in neuropathy impairment and improved quality of life, regardless of patient segmentation – CARLSBAD, Calif. , May 23, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will present new subgroup analyses from Ionis and AstraZeneca's Phase 3 NEURO-TTRansform study of WAINUA™ (eplontersen) at the 2024 International... Read More

Development of BIIB105, an investigational antisense oligonucleotide for amyotrophic lateral sclerosis (ALS), will be discontinued based on data from the Phase 1/2 ALSpire study Biogen and Ionis continue their long-standing commitment to developing therapies for ALS given the devastating impact of this progressive, fatal neurodegenerative condition CARLSBAD, Calif. and CAMBRIDGE, Mass. , May 16, 2024 /PRNewswire/ -- Ionis... Read More

ION582 demonstrated consistent improvements across multiple functional domains in Angelman syndrome patients ION582 was safe and well tolerated at all dose levels Ionis plans to move ION582 into pivotal trial Detailed ION582 data to be presented at upcoming medical meeting CARLSBAD, Calif. , May 16, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline data from the HALOS Phase 1/2a... Read More

CARLSBAD, Calif. , May 15, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will conduct its 2024 virtual Annual Meeting of Stockholders followed by a general corporate update on Thursday, June 6, 2024 . The agenda for the event is as follows: 5:00 p.m. – 5:15 p.m. ET ( 2:00 p.m. – 2:15 p.m. PT ) – Virtual Annual Meeting of Stockholders All stockholders of record at the close of... Read More

WAINUA TM launch on track; EU and Canada approval decisions expected this year Olezarsen NDA submitted to FDA for FCS; preparing EU regulatory submission Positive Phase 3 donidalorsen data for HAE; preparing regulatory submissions On track to achieve 2024 financial guidance CARLSBAD, Calif. , May 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company"), today reported financial results for the first... Read More

Established new corporate responsibility strategic pillars and actionable goals Received approval for two Ionis-discovered medicines in 2023, and continued to advance a broad pipeline poised to bring a steady cadence of important new medicines to people with serious diseases CARLSBAD, Calif. , April 25, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), today published its 2023 Corporate Responsibility Report:... Read More

Webcast scheduled for Tuesday, May 7 at 11:30 a.m. Eastern Time CARLSBAD, Calif. , April 23, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Tuesday, May 7 th at 11:30 a.m. Eastern Time to discuss its first quarter 2024 financial results. The webcast may be accessed at https://ir.ionispharma.com/events-and-presentations/upcoming-events . A replay will be... Read More

Olezarsen met the primary endpoint with statistically significant reduction of fasting triglycerides and showed substantial, clinically meaningful reduction in acute pancreatitis events Results demonstrate olezarsen may represent a novel treatment option for this rare, life-threatening disease, for which there are no approved treatments in the U.S. Data presented today at ACC 2024 and published in The New England Journal of... Read More

CARLSBAD, Calif. , April 1, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: 23 rd Annual Needham Virtual Healthcare Conference on Monday, April 8, 2024 2024 RBC Capital Markets Global Healthcare Conference on Tuesday, May 14, 2024 BofA Securities Health Care Conference 2024 on Tuesday, May 14, 2024 45 th... Read More

Webcast scheduled for Monday, April 8 at 10:00 a.m. Eastern Time CARLSBAD, Calif. , March 28, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Monday, April 8 th at 10:00 a.m. Eastern Time to discuss the olezarsen Phase 3 Balance study results in patients with familial chylomicronemia syndrome (FCS) presented at the 2024 American College of Cardiology Annual... Read More

First presentation of pivotal results showing significant reduction in triglycerides and substantial reduction in acute pancreatitis events in patients with rare, life-threatening disease, for which there are no approved treatment options in U.S. CARLSBAD, Calif. , March 25, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will present positive Phase 3 Balance results in patients with... Read More

Significant improvement in steatohepatitis with >2 point improvement in NAS score without worsening fibrosis, the primary endpoint of the study Achieved key secondary endpoint of MASH resolution without worsening of fibrosis More ION224 treated patients had an improvement of >1 stage fibrosis compared to placebo ION224 was safe and well-tolerated in this study with once-monthly subcutaneous dosing CARLSBAD, Calif. , March... Read More

Kyle Jenne rejoins company to lead commercial strategy Onaiza Cadoret-Manier departing for new career opportunity With broad commercial capabilities in place, Ionis ready to execute on its first independent launches CARLSBAD, Calif. , Feb. 29, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that Kyle Jenne has rejoined Ionis as executive vice president, chief global product strategy officer,... Read More

CARLSBAD, Calif. , Feb. 28, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: TD Cowen 44 th Annual Health Care Conference on Tuesday, March 5, 2024 Stifel 2024 Virtual CNS Days on Wednesday, March 20, 2024 A live webcast of these presentations can be accessed on the Investors & Media section of the Ionis... Read More

WAINUA™ approved with launch underway; on track for EU and Canada approval decisions this year Positive Phase 3 olezarsen and donidalorsen data, preparing regulatory submissions for FCS and HAE, respectively Olezarsen granted Breakthough Therapy designation by the FDA for FCS Ionis provides full year 2024 financial guidance CARLSBAD, Calif. , Feb. 21, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the... Read More

CARLSBAD, Calif. , Feb. 15, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational medicine olezarsen for the treatment of familial chylomicronemia syndrome (FCS), a rare, genetic disease characterized by extremely elevated triglyceride levels and recurrent acute pancreatitis (AP). The FDA... Read More

CARLSBAD, Calif. , Feb. 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults. The FDA grants development programs Fast Track designation to facilitate the... Read More

Webcast scheduled for Wednesday, February 21 at 11:30 a.m. Eastern Time CARLSBAD, Calif. , Feb. 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, February 21 st at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2023 financial results. The live webcast may be accessed here . A replay will be available for a limited time at the... Read More

Donidalorsen met the primary endpoint with a statistically significant reduction in the rate of HAE attacks in patients treated every 4 weeks or patients treated every 8 weeks Donidalorsen demonstrated a favorable safety and tolerability profile Ionis is preparing to submit a New Drug Application with U.S. FDA Data to be presented at an upcoming medical congress CARLSBAD, Calif. , Jan. 22, 2024 /PRNewswire/ -- Ionis... Read More

Numerous value-driving commercial, regulatory and pipeline milestones anticipated CARLSBAD, Calif. , Jan. 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced highlights from the Company's 2023 achievements and previewed a number of important milestones expected in 2024. Ionis will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10 at 3pm PT ;... Read More

U.S. FDA approval based on Phase 3 NEURO-TTRansform results showing WAINUA demonstrated consistent and sustained benefit halting neuropathy disease progression and improving neuropathy impairment and quality of life Additional regulatory reviews for WAINUA underway in rest of world WAINUA will be available in the U.S. in January 2024 CARLSBAD, Calif. , Dec. 21, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)... Read More

CARLSBAD, Calif. , Dec. 20, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Brett P. Monia , Ph.D., chief executive officer, will present a company overview at the 42 nd Annual J.P. Morgan Healthcare Conference at 3:00 p.m. PT on Wednesday, January 10, 2024 . A live webcast of the presentation can be accessed on the Investors & Media section of the Ionis website at... Read More

Otsuka to leverage strong commercial infrastructure and rare disease experience to reach European HAE patients Ionis plans to independently bring donidalorsen to U.S. patients if approved Donidalorsen Phase 3 results expected in the first half of 2024 CARLSBAD, Calif. , Dec. 18, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it has entered into a license agreement with Otsuka... Read More

CARLSBAD, Calif. , Dec. 14, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the appointment of Michael Yang , an experienced biopharmaceutical executive, to the Ionis Board of Directors. Mr. Yang's appointment expands the total number of Ionis Board members to 10. Mr. Yang has more than 20 years of broad senior level leadership experience in biotech, pharmaceutical and medical device... Read More

Part 1 of HALOS trial evaluating ION582 has completed enrollment with results expected in mid-2024 ION582 has been generally well tolerated at all dose levels, and participants are continuing to Part 2, the long-term extension portion of the study Encouraging EEG and clinical observations support continued development MIAMI , Nov. 11, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the... Read More

Continued treatment over two years with donidalorsen demonstrated sustained reduction in hereditary angioedema attacks and improved quality of life Donidalorsen recently received orphan drug designation in the U.S. Topline Phase 3 results expected in H1 2024 CARLSBAD, Calif. , Nov. 9, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from an ongoing Phase 2 open-label extension... Read More

CARLSBAD, Calif. , Nov. 3, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: Guggenheim 5 th Annual Inflammation, Neurology, & Immunology Conference on Tuesday, November 7, 2023 Stifel 2023 Healthcare Conference on Tuesday, November 14, 2023 Piper Sandler 35 th Annual Healthcare Conference 2023 on Tuesday,... Read More

Olezarsen Phase 3 data showed significant triglyceride lowering, substantial reductions in acute pancreatitis attacks and favorable safety and tolerability in patients with FCS; on track for regulatory filings in early 2024 Eplontersen marketing applications accepted for review in the EU and Canada ; potential U.S. approval in December 2023 On track to achieve 2023 financial guidance CARLSBAD, Calif. , Nov. 2, 2023... Read More

Webcast scheduled for Thursday, November 2 at 11:30 a.m. Eastern Time CARLSBAD, Calif. , Oct. 19, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Thursday, November 2 nd at 11:30 a.m. Eastern Time to discuss its third quarter 2023 financial results. The webcast may be accessed here . A replay will be available for a limited time at the same address. About... Read More

With experienced commercial team in place, Ionis poised to independently bring steady cadence of potentially transformational RNA-targeted medicines to patients beginning in 2024 Plan to add four new wholly owned neurology medicines to clinical pipeline by end of 2024 Innovative technology advances unlocking new opportunities in neurology and cardiology as well as in new therapeutic areas With multiple launches anticipated... Read More

Publication of NEURO-TTRansform study in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed eplontersen halted disease progression and continuously improved quality of life at 35-, 66- and 85-week analyses Data demonstrates consistent and sustained benefit on all co-primary and secondary endpoints CARLSBAD, Calif. , Sept. 28, 2023 /PRNewswire/ -- Ionis Pharmaceuticals , Inc . (Nasdaq:... Read More

Roche gains exclusive worldwide rights to develop, manufacture and commercialize the investigational medicines discovered by Ionis Ionis to receive a $60 million upfront payment CARLSBAD, Calif. , Sept. 27, 2023 /PRNewswire/ -- Ionis Pharmaceuticals , Inc. (Nasdaq: IONS) today announced that it has entered an agreement with Roche for two undisclosed early-stage programs for RNA-targeting investigational medicines for the... Read More

Olezarsen met the primary endpoint with a statistically significant reduction in triglyceride levels versus placebo First study to show an absolute reduction in acute pancreatitis in FCS patients, with olezarsen 80 mg monthly dose reducing events by 100 percent Olezarsen demonstrated a favorable safety and tolerability profile Ionis plans to file a New Drug Application with U.S. FDA in preparation for first potential... Read More

Ionis Innovation Day: Discovering, Developing and Delivering Transformational Medicines Presentations by key members of Ionis leadership and notable KOLs CARLSBAD, Calif. , Sept. 12, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host an investor and analyst day on Wednesday, October 4 . The event will take place at the Westin New York Grand Central from 8:00 a.m. to 3:00 p.m.... Read More

CARLSBAD, Calif. , Aug. 30 , 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: 2023 Wells Fargo Healthcare Conference on Wednesday, September 6, 2023 Citi's 18 th Annual BioPharma Conference on Thursday, September 7, 2023 Morgan Stanley 21 st Annual Global Healthcare Conference on Tuesday, September 12,... Read More

Reported Phase 3 data showing eplontersen continued to show improvement in ATTRv-PN through 85 weeks; December 22, 2023 PDUFA date Completed enrollment in donidalorsen OASIS-HAE study and eplontersen CARDIO-TTRansform ATTR-CM study keeping Phase 3 data readouts on track On track to achieve 2023 financial guidance CARLSBAD, Calif. , Aug. 9, 2023 /PRNewswire/ -- – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company"),... Read More

Builds on existing collaboration with Novartis for pelacarsen, which is currently being evaluated in a Phase 3 cardiovascular outcome study with data expected in 2025 Ionis' new and advanced technologies will be used to create next generation compound targeting Lp(a) Ionis will receive a $60 million upfront payment CARLSBAD, Calif. , Aug. 3, 2023 /PRNewswire/ -- Ionis Pharmaceuticals , Inc. (Nasdaq: IONS) today announced... Read More

CARDIO-TTRansform is the largest, most comprehensive ATTR-CM study with more than 1,400 patients enrolled Eplontersen is currently under U.S. FDA review for ATTRv-polyneuropathy, with ATTR-CM representing a second, larger potential patient population CARLSBAD, Calif. , July 31, 2023 /PRNewswire/ -- Ionis Pharmaceuticals , Inc. (Nasdaq: IONS) today announced the completion of enrollment in the Phase 3 CARDIO-TTRansform... Read More

Companies jointly developing and commercializing eplontersen in the U.S. AstraZeneca adds Latin America as part of its exclusive rights for rest of world CARLSBAD, Calif. , July 28, 2023 /PRNewswire/ -- Ionis Pharmaceuticals , Inc. (Nasdaq: IONS) today announced it has expanded its existing collaboration with AstraZeneca to include exclusive rights for AstraZeneca to commercialize eplontersen in Latin America . Ionis... Read More

Webcast scheduled for Wednesday, August 9 at 11:30 a.m. Eastern Time CARLSBAD, Calif. , July 26, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, August 9 th at 11:30 a.m. Eastern Time to discuss its second quarter 2023 financial results. The webcast may be accessed at https://ir.ionispharma.com/events-and-presentations/upcoming-events . A replay... Read More

Topline Phase 3 NEURO-TTRansform results show eplontersen continued to halt neuropathy disease progression and improve quality of life through 85 weeks Data further strengthen eplontersen's differentiated profile, positioning it to be an important potential treatment for patients with ATTRv-PN CARLSBAD, Calif. , July 10 , 2023 /PRNewswire/ -- Ionis Pharmaceuticals (Nasdaq: IONS) today announced positive topline, 85-week data... Read More

CARLSBAD, Calif. , July 5, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in a fireside chat at TD Cowen's 2nd Annual RNA Therapeutics Summit on Monday, July 10, 2023 . A live webcast of this presentation can be accessed on the Investors & Media section of the Ionis website at www.ionispharma.com . The replay will be available on the Ionis website within 48... Read More

Interim results from RESPOND study show improved motor function in most participants treated with SPINRAZA after Zolgensma ® (onasemnogene abeparvovec) CARLSBAD, Calif. , June 30, 2023 /PRNewswire/ -- Ionis Pharmaceuticals (Nasdaq: IONS) today announced that its partner Biogen presented new data highlighting the potential benefit of SPINRAZA ® (nusinersen) in infants and toddlers living with spinal muscular atrophy (SMA).... Read More

CARLSBAD, Calif. , June 7, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today the pricing of $500.0 million aggregate principal amount of 1.75% Convertible Senior Notes due 2028 (the "notes") in a private placement (the "offering") to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"). Ionis also granted the initial purchasers... Read More

CARLSBAD, Calif. , June 6, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it intends to offer, subject to market conditions and other factors, $500.0 million aggregate principal amount of Convertible Senior Notes due 2028 (the "notes") in a private placement (the "offering") to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the... Read More

Two-year treatment with donidalorsen in the Phase 2 open label study resulted in consistent, sustained protection from HAE attacks Completed enrollment in the Phase 3 OASIS-HAE study Topline Phase 3 results expected in H1 2024 CARLSBAD, Calif. , June 1, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc . (Nasdaq: IONS) today announced positive clinical progress with donidalorsen, its late-stage investigational prophylactic... Read More

CARLSBAD, Calif. , May 10, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will conduct its 2023 virtual Annual Meeting of Stockholders followed by a general corporate update on Thursday, June 1, 2023 . The agenda for the event is as follows: 5:00 p.m. – 5:15 p.m. ET ( 2:00 p.m. – 2:15 p.m. PT ) – Virtual Annual Meeting of Stockholders All stockholders of record at the close of... Read More

CARLSBAD, Calif. , May 9, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: RBC Capital Markets Global Healthcare Conference on Tuesday, May 16, 2023 Goldman Sachs 44 th Annual Global Healthcare Conference on Monday, June 12, 2023 A live webcast of the presentations can be accessed on the Investors & Media... Read More

QALSODY approved for SOD1-ALS; MAA under review in EU Reported positive Phase 3 eplontersen ATTRv-PN data; December 22, 2023 PDUFA date On track to achieve 2023 financial guidance CARLSBAD, Calif. , May 3, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company"), today reported financial results for the first quarter of 2023. Financial results are summarized below: Three months ended March 31, 2023... Read More

Accelerated approval marks a scientific advancement in treatment of superoxide dismutase 1 (SOD1)-amyotrophic lateral sclerosis (ALS) Approval based on reduction of neurofilament, a biomarker associated with neuronal damage in ALS QALSODY joins SPINRAZA ® as Ionis-discovered medicine approved for treatment of a rare and fatal neurodegenerative disease CARLSBAD, Calif. , April 25, 2023 /PRNewswire/ -- Ionis Pharmaceuticals,... Read More

NEURO-TTRansform study met all co-primary and secondary endpoints Positive results to be presented today at AAN 2023 demonstrate eplontersen efficacy, safety and administration profile may provide an important new treatment option in this fatal disease with significant unmet need Ionis to host webcast on Tuesday, April 25 at 1 p.m. ET CARLSBAD, Calif. , April 24, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc . (Nasdaq:... Read More

Webcast scheduled for Wednesday, May 3 at 11:30 a.m. Eastern Time CARLSBAD, Calif. , April 20, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, May 3 rd at 11:30 a.m. Eastern Time to discuss its first quarter 2023 financial results. The webcast may be accessed at https://ir.ionispharma.com/events-and-presentations/upcoming-events . A replay will be... Read More

Phase 1b study showed dose-dependent and sustained reduction of tau protein in cerebrospinal fluid through open-label long-term extension (LTE) IONIS-MAPT Rx reduced aggregated tau pathology across all brain composites assessed starting as early as week 25 through week 100 CARLSBAD, Calif. , March 29, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported that its partner Biogen presented new Phase 1b... Read More

CARLSBAD, Calif. , March 28, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced its participation in fireside chats at the following investor conferences: Guggenheim Virtual Genomic Medicines and Rare Disease Days on Tuesday, April 4, 2023 22 nd Annual Needham Virtual Healthcare Conference on Monday, April 17, 2023 A live webcast of the presentations can be accessed on the Investors & Media... Read More

Eplontersen met co-primary endpoints demonstrating sustained reduction in TTR and benefits in neuropathy and quality of life through 66 weeks 35 and 66-week data to be presented at the American Academy of Neurology (AAN) Annual Meeting in April CARLSBAD, Calif. , March 27, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc . (Nasdaq: IONS) today announced positive topline results from the 66-week analysis of the Phase 3... Read More

If approved, tofersen will be the first treatment targeting a genetic cause of ALS and the next marketed Ionis-discovered antisense medicine FDA decision expected by April 25, 2023 CARLSBAD, Calif. , March 22, 2023 /PRNewswire/ -- Ionis (Nasdaq: IONS) today announced the outcome of the U.S. Food and Drug Administration's (FDA) Peripheral and Central Nervous System Drugs Advisory Committee meeting on tofersen, an... Read More

35-week data included in the filing demonstrated a statistically significant and clinically meaningful change from baseline for co-primary and secondary endpoints compared to external placebo group Eplontersen previously granted Orphan Drug Designation for transthyretin-mediated amyloidosis FDA assigns PDUFA action date of Dec. 22, 2023 CARLSBAD, Calif. , March 7, 2023 /PRNewswire/ -- Ionis Pharmaceuticals (Nasdaq: IONS)... Read More

CARLSBAD, Calif. , Feb. 28, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will present at the following investor conferences: Cowen 43 rd Annual Health Care Conference on Tuesday, March 7, 2023 Oppenheimer 33 rd Annual Virtual Healthcare Conference on Wednesday, March 15, 2023 Stifel 2023 Virtual CNS Days on Wednesday, March 29, 2023 A live webcast of the presentations can be... Read More

Eplontersen NDA submitted to FDA; tofersen under regulatory review for marketing approval in U.S. and EU Phase 3 data planned for eplontersen and olezarsen; robust late-stage pipeline expanding with two new Phase 3 programs Ionis provides full year 2023 financial guidance CARLSBAD, Calif. , Feb. 22, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company"), a genetic medicines company, today reported... Read More

Phase 2 open-label study data reinforce donidalorsen's potential to be a best-in-class prophylactic treatment for patients living with hereditary angioedema Ionis is preparing to launch donidalorsen, one of its three near-term commercial opportunities CARLSBAD, Calif. , Feb. 21 , 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced additional positive interim data from a Phase 2 open-label... Read More

Webcast scheduled for Wednesday, February 22 at 11:30 a.m. Eastern Time CARLSBAD, Calif. , Feb. 8, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, February 22 nd at 11:30 a.m. Eastern Time to discuss its fourth quarter and full year 2022 financial results. The webcast may be accessed at... Read More

Initiation of the Phase 3 program expands Ionis' late-stage pipeline to 7 medicines in 9 indications CARLSBAD, Calif. , Feb. 1, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. ( Nasdaq : IONS) today announced that GSK has initiated two randomized, double-blind, placebo-controlled Phase 3 studies evaluating the safety and efficacy of bepirovirsen (formerly IONIS-HBV Rx ), an investigational antisense medicine for the... Read More

FCS is a rare and debilitating genetic disease often leading to significant risk of acute, potentially fatal pancreatitis If approved, olezarsen would be the first approved treatment for FCS in the U.S. CARLSBAD, Calif. , Jan. 31, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted olezarsen Fast Track designation for the treatment of... Read More

CARLSBAD, Calif. , Jan. 26, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Chief Scientific Officer C. Frank Bennett , Ph.D., has been named a co-recipient of the Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research. Dr. Bennett shares this year's prize with Don W. Cleveland , Ph.D., chair and distinguished professor of cellular and molecular medicine at... Read More

Royalty Pharma to acquire an interest in SPINRAZA ® and pelacarsen royalties -- Ionis retains majority of royalties and all milestones from Novartis for pelacarsen Royalty Pharma to pay Ionis $500 million upfront and up to $625 million in milestones Agreement enables Ionis to achieve commercial readiness for multiple late-stage programs and advance its innovative pipeline of genetic medicines CARLSBAD, Calif. and NEW YORK ,... Read More

CARLSBAD, Calif. , Jan. 4, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Brett P. Monia , Ph.D., chief executive officer, will present a company overview and participate in a Q&A session at the 41 st Annual J.P. Morgan Healthcare Conference at 9:45 a.m. PT ( 12:45 p.m. ET ) on Wednesday, January 11, 2023 . A live webcast of the presentation and Q&A session will be available on the... Read More

SOD1-ALS affects approximately 2% of people living with ALS worldwide 1 If approved, tofersen would be the world's first treatment to target a genetic cause of ALS EMA acceptance follows FDA's acceptance of tofersen NDA earlier this year CARLSBAD, Calif. , Dec. 5, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the European Medicines Agency (EMA) has accepted the marketing authorization... Read More