Many researchers have noted epigenetic changes in AD, but targeting these with a drug has proven difficult (see Nov 2012 conference news; Aug 2014 news; Nov 2014 news). Partly this is because histone deacetylase (HDAC) inhibitors, which derepress genes involved in learning and memory, have broad effects that can lead to toxicity with chronic dosing (see Dec 2008 conference news; Aug 2013 conference news).
In Vienna, César Molinero of Oryzon noted that ORY-2001 has a second mechanism of action as well, inhibiting the mitochondrial membrane protein monoamine oxidase B (MAOB1)….
ORY-2001 can be taken orally, has favorable pharmacokinetics, and crosses the blood-brain barrier, Molinero said.
The researchers are now testing single and multiple doses in Phase 1. Eighty-eight young, healthy volunteers took either 0.2, 0.6, 1, 1.5, 2.5, or 4 mg of ORY-2001. Of the eight people in each cohort, two got placebo, and multiple doses were given over five days. Molinero reported no serious adverse events so far, but with repeated dosing at 2.5 mg, platelet counts dropped by a third after eight days and took a week to rebound. The researchers added the 4 mg dose to explore these effects, and that testing is still ongoing, Molinero said.
The ELP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: Non-Amyloid Treatments: Inflammation, Epigenetics, Regeneration