Ionis Pharmaceuticals (IONS) PT Raised to $64 at Needham & Company as ENDEAR Study Halted Early for Efficacy

Go back to Ionis Pharmaceuticals (IONS) PT Raised to $64 at Needham & Company as ENDEAR Study Halted Early for Efficacy

Biogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset Spinal Muscular Atrophy

August 1, 2016 7:30 AM EDT

Biogen Intends to File Marketing Applications for Nusinersen with Regulatory Authorities in the Coming Months

Biogen Exercises Option to Develop and Commercialize Nusinersen Globally

Companies to Host Webcast Today at 9:00 a.m. EDT to Provide Program Update

CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)-- Biogen (NASDAQ: BIIB) and Ionis Pharmaceuticals (NASDAQ: IONS) today announced that nusinersen, their investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint pre-specified for the interim analysis of ENDEAR, the Phase 3 trial evaluating nusinersen in... More

Akcea Therapeutics Receives Orphan Designation in Europe for Volanesorsen for the Treatment of Familial Partial Lipodystrophy (FPL)

July 28, 2016 4:00 PM EDT

CAMBRIDGE, Mass., July 28, 2016 /PRNewswire/ -- Akcea Therapeutics, a wholly-owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the European Commission (EC) has designated volanesorsen as an orphan medicinal product for the treatment of familial partial lipodystrophy (FPL).  FPL is a rare lipid disorder characterized by abnormal fat distribution across the body and a range of metabolic abnormalities, including severe type 2 diabetes, high triglycerides, and accumulation of fat in the liver. The EC's approval follows a positive opinion in June from the European Medicine Agency's (EMA) Committee for Orphan Medicinal... More