Mirum Pharmaceuticals reported on Monday that a drug it recently acquired to treat cerebrotendinous xanthomatosis, a rare and serious disease in which the body doesn’t metabolize cholesterol properly, succeeded in a late-stage trial. The results potentially pave the way for the treatment, already used by physicians, to reach a wider pool of patients.
In the Phase 3 study, dubbed RESTORE, 13 participants all started off on the drug, known as Chenodal, an oral tablet that contains a bile acid patients don’t make enough of. Participants were then either assigned at random to continue to receive the drug for two months or to switch over to a placebo, and that cycle was repeated. The study’s authors found that, when patients were on Chenodal, their level of bile alcohols was more than 20-fold lower than when they were on placebo, which was both the desired result and statistically significant.
Researchers also reported that the most common adverse events, diarrhea and headache, were mild.
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