Ultragenyx Pharmaceutical
Inc. RARE, a biopharmaceutical company focused on the development of
novel products for rare and ultra-rare diseases, today announced the
presentation of results from a Phase 2 extension study of sialic acid
extended-release (SA-ER, UX001) tablets in patients with hereditary inclusion
body myopathy (HIBM; also known as GNE myopathy), a rare, progressive
muscle-wasting disease. SA-ER is designed to replace the deficient sialic acid
substrate in patients with HIBM. The data were presented at the 19^th
International Congress of the World Muscle Society (WMS) in Berlin.
"The data from the extension study of SA-ER support our plans to move forward
with this program," said Sunil Agarwal, M.D., Chief Medical Officer of
Ultragenyx. "While the 12 gram per day dose did not appear to have a clear
advantage over the 6 gram per day dose, it does provide additional evidence of
activity and safety and we are encouraged to see a potential long-term impact
on disease progression in upper extremity muscle strength after approximately
two years of treatment."
Patients in the initial Phase 2 study were randomized to receive placebo, 3
grams/day, or 6 grams/day of SA-ER. After 24 weeks, placebo patients crossed
over to either 3 grams/day or 6 grams/day, on a blinded basis, for an
additional 24 weeks. The 48-week analysis compared change from baseline for
the combined groups at 6 grams/day versus 3 grams/day of SA-ER.
The initial Phase 2 data, which were presented at the American Academy of
Neurology (AAN) Annual Meeting in April 2014, showed a statistically
significant difference in the upper extremity composite (UEC) of muscle
strength at 48 weeks with the higher dose group compared to the lower dose
group. SA-ER appeared to be safe and well-tolerated with no serious adverse
events observed to date. Most adverse events were mild to moderate and most
commonly gastrointestinal in nature.
In the first part of the extension study, all 46 patients from the 48-week
Phase 2 study crossed over to 6 grams/day for a variable period of time that
was on average 24 weeks. In the second part of the extension study, all 46
patients and 13 treatment-naïve patients received 12 grams/day of SA-ER for 24
weeks. The results presented at WMS include the 49 out of 59 patients who had
24 weeks of data at the higher dose. While the 12 grams/day data do not
suggest any clinically meaningful advantage over 6 grams/day, the 12 gram data
do provide additional data that support clinical activity with SA-ER
treatment. The 12 gram daily dose of SA-ER appeared to be generally safe and
well tolerated with no drug-related serious adverse events, but the rate of
mild to moderate gastrointestinal adverse events did appear to be greater with
this dose. Over the entire approximate two-year study, treatment with SA-ER
appeared to slow the progression of upper extremity disease when compared to
the 24-week placebo group extrapolated out to two years.
Based on the 48-week and extension study data, Ultragenyx intends to discuss
with regulatory authorities a potential pivotal study of SA-ER in HIBM
patients. The company will also continue to treat patients in the ongoing
extension study.
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