ARTICLE | Clinical News

Ultragenyx discontinues HIBM therapy

August 22, 2017 11:33 PM UTC

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) lost $6.10 (10%) to $52.75 in after-hours trading Tuesday after the company said it will discontinue development of aceneuramic acid extended release (Ace-ER) after the candidate missed the primary endpoint in a Phase III trial to treat GNE myopathy, a muscle-wasting disorder also known as hereditary inclusion body myopathy (HIBM).

Compared to placebo, Ace-ER failed to significantly improve upper extremity muscle strength composite score from baseline to week 48 (p=0.5387). Ace-ER also missed the secondary endpoints of improving lower extremity muscle strength composite score as measured by hand-held dynamometry (HHD), physical functioning using the Mobility domain of the GNE Myopathy-functional activity scale (GNEM-FAS), and a measure of muscle strength in knee extensors vs. placebo...