Good news from PTC and uniQure!

On 21 June 2023, positive findings from both PTC Therapeutics and uniQure were shared with the HD community on their respective huntingtin (HTT)-lowering approaches to treating HD. PTC’s interim 12-week data from the global PIVOT-HD trial of PTC518 showed dose-dependent lowering of HTT protein levels, confirmed the intended exposure in cerebrospinal fluid, and indicated a favourable safety profile. These data come from 24 participants exposed to PTC518 (33 total participants, including those assigned to placebo). From uniQure, the AMT-130 trial interim data for up to 24-month treatment in 26 participants showed that this novel gene therapy continues to be generally well tolerated. Preliminary results are encouraging and indicate potential clinical and functional benefits. We look forward to further updates on these encouraging findings.