Votoplam is a HTT-lowering drug that’s taken as a pill. The idea is that by lowering the disease-causing protein, signs and symptoms of HD will be reduced. #CHDI2025
Amy started by sharing the fantastic news that PTC has an exciting new collaboration with Novartis, a massive drug developer. PTC will complete the ongoing PIVOT-HD trial, but Novartis will be in charge of all future clinical trials, like their planned Phase 3. #CHDI2025
So far, people in the PIVOT-HD trial have been taking votoplam for 12 months. They were really interested in knowing if people who have been on the drug this long have positive changes in biomarkers - biological metrics that track with HD progression. #CHDI2025
The most reliable biomarker we have right now is neurofilament light, or NfL. We know that levels of NfL increase as HD progresses. Excitingly, Amy shows that levels of NfL remain steady for people who have been on votoplam for 12 months. #CHDI2025
Comments