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Data from an early-stage study shows that treatment with Ionis' (IONS) ION582 helps improve communication, cognition and motor function in patients with Angelman syndrome, a rare nerve disorder.

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS ) HALOS Study of ION582 in Angelman Syndrome Conference Call July 22, 2024 8:00 AM ET Company Participants Wade Walke - Senior Vice President of Investor Relations Brett Monia - Chief Executive Officer Holly Kordasiewicz - Senior Vice President, Neurology Elizabeth Jalazo - Assistant Professor of Pediatrics, Division of Genetics & Metabolism, University of North Carolina School of Medicine Becky Crean - Executive Director, Neurology Lynne Bird - Professor of Clinical Pediatrics, University of California, San Diego, Rady Children's Hospital San Diego Eugene Schneider - Executive Vice President, Chief Clinical Development and Operations Officer Conference Call Participants Debjit Chattopadhyay - Guggenheim Securities Denis Reznik - Raymond James David Lebowitz - Citi Amy Li - Jefferies Yanan Zhu - Wells Fargo Securities Jessica Fye - JPMorgan Jay Olson - Oppenheimer Gena Wang - Barclays Paul Matteis - Stifel Andy Chen - Wolfe Research Salveen Rich

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 function exceeded those observed in natural history studies Ionis plans to initiate Phase 3 development in H1 2025 Ionis to host webcast on Monday, July 22 at 8:00am ET CARLSBAD, Calif. , July 22, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582 in people with Angelman syndrome (AS) demonstrating consistent and encouraging clinical improvement on measures assessing all functional domains including communication, cognition a

Ionis' (IONS) late-stage study is evaluating zilganersen in a rare neurological disorder called Alexander disease. Top-line data from this study is expected by the end of the next year.

Webcast scheduled for Thursday, August 1 at 11:30 a.m. Eastern Time CARLSBAD, Calif.

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 children and adults with a rare, progressive and ultimately fatal neurological disorder known as Alexander disease (AxD).

Webcast scheduled for Monday, July 22 at 8:00 a.m. Eastern Time CARLSBAD, Calif.

The FDA grants priority review to Ionis' (IONS) filing seeking approval for olezarsen to treat adults with familial chylomicronemia syndrome. A final decision is expected in December 2024.

Ionis Pharmaceuticals (IONS) witnessed a jump in share price last session on above-average trading volume. The latest trend in earnings estimate revisions for the stock suggests that there could be more strength down the road.

–         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 for Priority Review the New Drug Application (NDA) for olezarsen, an investigational RNA-targeted medicine for the treatment of adults with familial chylomicronemia syndrome (FCS).