Updated interim data for Adverum’s intravitreal wet age-related macular degeneration gene therapy bolstered the company’s case that the product could compete with less convenient subretinal gene therapies for the indication. Adverum rose $1.81 (18%) to...

FDA qualified a group of kidney injury biomarkers from the Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium (BC) and the Critical Path Institute (C-Path) Predictive Safety Testing Consortium (PSTC), marking the first...

It’s a mystery why investors shaved 10% off gene therapy company AveXis Inc. (NASDAQ:AVXS) following FDA’s approval of Spinraza nusinersen for spinal muscular atrophy from Biogen Inc. (NASDAQ:BIIB) and Ionis Pharmaceuticals Inc. (NASDAQ:IONS). Spinraza has...

Additional data from the double-blind, U.S. Phase IIa TIME-2 trial in 144 patients with DME showed that twice-daily 15 mg subcutaneous AKB-9778 with and without once-monthly Lucentis ranibizumab led to a >=2-step improvement on the...

NephroGenex said an independent DSMB recommended continuation of the double-blind, placebo-controlled, international Phase III PYR-311 trial evaluating 300 mg oral Pyridorin twice daily until ESRD or death occurs. The trial, which has an SPA from...

AB Science completed enrollment of 381 patients in a double-blind, placebo-controlled, European Phase III trial of oral masitinib plus riluzole. AB Science S.A. (Euronext:AB), Paris, France Product: Masitinib ( Masican ) ( AB1010 ) Business: Neurology Molecular target: Stem...

Top-line data from the double-blind, U.S. Phase IIa TIME-2 trial in 144 patients showed that twice-daily 15 mg subcutaneous AKB-9778 plus once-monthly Lucentis ranibizumab significantly reduced CST from baseline to 3 months, the primary endpoint,...

Aerpio Therapeutics Inc. (Cincinnati, Ohio) said a combination of its AKB-9778 and Lucentis ranibizumab was superior to Lucentis alone in the Phase IIa TIME-2 study to treat diabetic macular edema (DME). The combination met the...

Additional data from the open-label, dose-escalation, U.S. Phase Ib/IIa TIME-1 trial in 18 evaluable patients with DME showed that twice-daily subcutaneous AKB-9778 at doses of >=15 mg for 28 days improved BCVA by >=5 letters...

The open-label, international Phase IIIb DIAMOND trial in 893 patients showed that once-daily low-dose (0.15-0.175 mg/kg) Advagraf in combination with Simulect basiliximab and mycophenolate mofetil (MMF) met the primary endpoint of increasing GFR as measured...