CARLSBAD, Calif. – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced new long-term data for DAWNZERA™ (donidalorsen), the first and only RNA-targeted prophylactic medicine for hereditary angioedema (HAE), to be presented at the 2025 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting in Orlando, Florida. Results demonstrate the long-term durability and safety...
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CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc.(Nasdaq: IONS) today announced full results from the Phase 3 Balance study of Ionis’ lead independent investigational medicine, olezarsen, for the treatment of adults with familial chylomicronemia syndrome (FCS). The olezarsen 80 mg monthly dose met the primary endpoint of significantly reducing triglycerides (TGs) in...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from an ongoing Phase 2 open-label extension (OLE) study evaluating the safety and efficacy of its investigational prophylactic medicine, donidalorsen, in patients with hereditary angioedema (HAE), a rare and life-threatening genetic disease. Over the two years, patients treated...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to ION582 for the treatment of Angelman syndrome (AS), a rare neurological disease that often presents in infancy and is characterized by profound intellectual disability, impaired communication, motor impairment and...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to zilganersen for the treatment of Alexander disease (AxD), a rare, progressive and often fatal neurological condition. Over time, it can lead to loss of mobility and independence, along with...
CARLSBAD, Calif. – Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced that data from a Phase 2 clinical study of IONIS-PKK-LRx met its primary and secondary endpoints, achieving significant reductions in the number of attacks suffered by patients with hereditary angioedema (HAE) compared to placebo. The study demonstrated a mean reduction of...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. today announced positive topline results for the Phase 3 OASIS-HAE study of donidalorsen in people with hereditary angioedema (HAE). The trial met its primary endpoint of reduction in rate of angioedema attacks in patients treated with donidalorsen (80mg) via subcutaneous injection dosed every 4...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced positive results from a Phase 2 study of ION224, an investigational DGAT2 antisense inhibitor in development for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), previously referred to as nonalcoholic steatohepatitis (NASH). The study met its primary endpoint at both doses (120 mg...
SAN CARLOS, Calif. — Iovance Biotherapeutics, Inc., a biotechnology company focused on innovating, developing and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, today announced a clinical program update for LN-145 TIL therapy in non-small lung cancer (NSCLC). The U.S. Food and Drug Administration (FDA) placed...
SAN CARLOS, Calif. — Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, today announced interim data from its registrational Phase 2 IOV-LUN-202 trial of lifileucel monotherapy in patients with previously treated advanced...