Ultragenyx Presented The Findings of Phase 1 Sialic Acid Extended Release (SA-ER) Trial in Australia

Ultragenyx, a biopharma company presented their findings on phase 1 Sialic Acid Extended Realease (SA-ER)tablets at 17th. International Congress of the World Muscle Society earlier this month in Perth, Australia. SA-ER is being tested for Hereditary Inclusion Body Myopathy (HIBM). I participated in this clinical trial last year, therefore the findings are quite personal for me. I am currently participating in the phase 2 trial which will be completed late in 2013. Here, I am attempting to provide a narrative/summary of what I understand from their presentation in Perth, Australia. I have posted the links for those interested in charts and technicalities. As a disclosure, please note I am not a doctor, I am patient directly experiencing HIBM. SA-ER were tested on 26 participants with HIBM. Eight were males and eighteen females. The races/ethnicity were 6 Asians, 20 white, (1 Hispanic/Latino 25 Non-Hispanic). No mentioned was made of Persian/Jewish patients. SA-ER was well tolerated at all given doses with minimal side effects. Depending on the dose levels. SA-ER was absorbed and showed steady amount of sialic acid between 8-16 hours after dosing. They tested SA-ER on the patients when they fasted and with food. Sialic Acid seemed to be in higher concentration with the group that took the medicine with their meals. The evening dose was given closer to the night dose because it is stated that at night time there is protein synthesis and muscle repair, therefore the need for Sialic acid is greater. Besides all these explanations, the one statement I am quite encouraged about is "SA-ER should achieve levels of free Sialic Acid that are expected to correct Sialic Acid deficiency and improve sialylation in the muscles of HIBM patients." Please feel free to send me or write your comments. P.S. Ultragenyx is still recruiting patients for phase 2 SA-ER trial in Los Angeles, New York, Missouri and Israel. http://globenewswire.com/Tracker?data=d6QAld4vepVP7jLm-NEJ9Kbhc9rUIVakuLOTfLBZAFBQWdVKFU9oWPygz-Xe6MYU7lR2sMmxIoUktEeY9c1uczF8F3y9N1PAlpg0rBRAAcBCm4Gr3Jebqm5hEQwRnPosqNDEM0K.8gfRAz-dV3zoX4LRDSYvtdfsa90dnfChvbK8uTPDaK0Rjq439Mc8jDUxhxXhAJKWGDA6mn1dWJjfQdcebeW3fMwZ6oeAHOupszvc6Ptu0NFn7AGJbgM%3D http://www.reuters.com/article/2012/10/10/idUS186788+10-Oct-2012+GNW20121010 http://www.wms2012.com/