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) 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...
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...
Seven abstracts, including three late-breaking presentations, will be shared on new IQIRVO® (elafibranor) and Bylvay®/Kayfanda® (odevixibat) data, reinforcing the strong clinical value across Ipsen’s rare liver disease portfolio where unmet need is high and treatments are few In two late-breaking presentations, IQIRVO’s effect on fatigue and its relationship with pruritus in patients...
Paris, France – Ipsen (Euronext: IPN; ADR: IPSEY) announced today that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted regulatory approval for Bylvay® (odevixibat) for the treatment of pruritus associated with progressive familial intrahepatic cholestasis (PFIC). PFIC is a group of rare genetic disorders in which bile acid accumulates in the...
PARIS, FRANCE – Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the conditional marketing authorization of Ojemda® (tovorafenib) as monotherapy for the treatment of patients 6 months of age and...