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news.Ultragenyx Pharmaceutical (RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the initiation of the Phase III study of aceneuramic acid extended release (Ace-ER), previously known as sialic acid-extended release (SA-ER; UX001), tablets for the treatment of GNE Myopathy.
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Ace-ER is designed to replace the deficient sialic acid substrate in patients with GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), a rare, progressive muscle-wasting disease.
"This is a significant step forward for our global program to assess treatment with Ace-ER in patients with GNE Myopathy, currently an unmet medical need," said Sunil Agarwal, M.D., Chief Medical Officer of Ultragenyx.
"Our pivotal Phase 3 study is intended to confirm the positive results observed in Phase 2. In parallel, we plan to file for conditional approval in Europe with the possibility of accelerating access for patients with this debilitating disease."
The Phase 3 global, randomized, double-blind, placebo-controlled clinical study in approximately 80 patients will assess the efficacy and safety of six grams per day of Ace-ER over 48 weeks.
The primary endpoint of the study is a composite of upper extremity muscle strength as measured by hand-held dynamometry (HHD).
Key secondary endpoints include the GNE Myopathy-functional activity scale (GNEM-FAS), a disease-specific patient-reported outcome (PRO) that measures mobility and upper-extremity function, and other measures of lower-extremity muscle strength. A Phase 2 randomized, double-blind, placebo-controlled study evaluating the same endpoints was completed in December 2013.
The Phase 3 study design was accepted by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Data from the Phase 3 study are expected in the second half of 2016.
In parallel with the conduct of the Phase 3 study, Ultragenyx intends to file a Marketing Authorization Application (MAA) with the EMA in the second half of 2015.
The company seeks to obtain conditional approval from the EMA for Ace-ER for the stabilization or slowing of decline in upper extremity muscle strength in patients with GNE Myopathy based on the Phase 2 data and Scientific Advice received from the EMA. Once filed, a decision from the EMA is anticipated by the end of 2016.