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...
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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...
BUFFALO, NY- February 9, 2024 – A new research perspective was published in Oncoscience (Volume 11) entitled, “IPIAD- an augmentation regimen added to standard treatment of pancreatic ductal adenocarcinoma using already-marketed repurposed drugs irbesartan, pyrimethamine, itraconazole, azithromycin, and dapsone.” In this new paper, researcher Richard E. Kast from IIAIGC Study...
PARIS, France and BOSTON, Mass. — Ipsen (Euronext: IPN; ADR: IPSEY) and Skyhawk Therapeutics today announced the signing of an exclusive worldwide collaboration to discover and develop novel small molecules that modulate RNA for rare neurological diseases. The agreement includes an option pursuant to which Ipsen would acquire exclusive license...
Ipsen announced it has completed the acquisition of Albireo Pharma, Inc., a leading innovator in bile-acid modulators to treat rare liver conditions.