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...
treatment News
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) today announced that new data in the management of severe hypertriglyceridemia (sHTG) will be presented at the European Society of Cardiology (ESC) Congress 2026, taking place August 28 – 31 in Munich. Presentations include one-year data from the open-label, long-term extension study of TRYNGOLZA® (olezarsen), recently approved in the U.S....
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...
Santa Cruz, Calif. – Lung diseases are attracting renewed attention across drug development and life sciences investment, with idiopathic pulmonary fibrosis (IPF) emerging as a particularly active area. IPF is a chronic, progressive condition marked by irreversible scarring of lung tissue. As fibrosis advances, the lungs become increasingly stiff, making...
HOUSTON, Texas — IPS HEART, Inc., a biopharmaceutical company pioneering induced pluripotent stem cell (iPSC)-derived cell replacement therapies, announced that the FDA has granted Rare Pediatric Drug Designation for ISX9-CPC, for the treatment of cardiomyopathies associated with dystrophinopathies. Redefining the Therapeutic Paradigm: Generating New Human Muscle Duchenne muscular dystrophy...
PARIS – Ipsen (Euronext: IPN; ADR: IPSEY) today announced that its New Drug Application (NDA) for palovarotene, an oral, investigational, selective RARγ agonist for the prevention of heterotopic ossification (new bone formation) as a potential treatment option for people living with the progressive disabling and ultra-rare genetic disorder fibrodysplasia ossificans progressiva...
PARIS, FRANCE, 19 December 2025 – Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the pivotal Phase II FALKON trial did not meet its primary endpoint of reducing new heterotopic ossification (HO) in adults and children living with fibrodysplasia ossificans progressiva (FOP) vs. placebo, as a result the study will be...