Alnylam's updated lumasiran data points to PhIII short track; Genentech snatches fourth approval for Rituxan
→ Alnylam $ALNY came out with some updated data from a Phase I/II study Friday, showing the company’s rare disease drug lumasiran is on the short track for a Phase III trial. The trial — testing the drug in 20 patients with the ultra-rare primary hyperoxaluria type 1 (PH1) — has a primary endpoint of reducing the urinary oxalate at six months. Preliminary results, announced last year, suggested that lumasiran led to a mean maximal reduction in urinary oxalate of 66% in an unblinded group of four patients. In this Part B study, lumasiran demonstrated a mean maximal reduction in urinary oxalate of 64% in patients. “Based upon our recent discussions with the FDA, we are on track to advance this program into Phase III development at mid-year, with the goal of bringing lumasiran to patients around the world as rapidly as possible,” said Alnylam’s VP and GM of the lumasiran program Pritesh Gandhi in a statement.
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