The effect is reversed after a few hours and is more or less out of the body after 72 to 96 hours. Because the drug is taken orally it is lowering Huntingtin not just in the brain, but also all other parts of the body. PTC aims to move into a Phase 2 trial later this year in multiple countries.
A newcomer in the HD community was also presenting very exciting news during the conference. Catherine Scart-Grès from SOMBiotech, a Spanish company, told us that they will do a Phase 2b trial with 129 patients. The trial will involve more than 20 European sites and is expected to start by the end of 2021.
The participants will be split in 3 groups or arms as its often called in trials. 1 group will get placebo, the second group will receive a 200 mg dose of the drug and the third group will receive a dose of 300 mg dose. The drug is taken orally twice a day.
The main “target” for this drug is to reduce chorea, but there are also expectations that it may have a positive influence on psychiatric symptoms.