Category: Clinical Research

Roche has announced the discontinuation of huntingtin-lowering drugs tominersen and RG649 based on independent, data-driven decisions. The Phase 2 study GENERATION HD2 evaluated tominersen in Huntington disease (HD) and enrolled 301 participants across 15 countries, including Canada. The POINT-HD study, a first-in-human study of RG6496 in HD, was open internationally, with planned expansion into Canada. This study will no longer take place. We recognize that this may still be disappointing news and encourage you to access support. Please reach out to your Family Services team member for assistance.

Prilenia and Ferrer have announced the launch of a Phase 3 clinical study of pridopidine in Huntington disease (HD). The first U.S. sites are expected to open for enrollment in June 2026, with sites in Europe, the UK, and Canada to follow. This study will add to a growing body of clinical research on pridopidine in HD; previous trials did not meet their primary endpoints. Canadian study sites have not yet been confirmed, but more information will be provided once available.

UniQure receives FDA guidance for accelerated filing of Huntington disease treatment AMT-130. This decision is a reversal by the FDA, who had indicated late last year that the available data were insufficient to support a filing. Read HDBuzz’s summary article here: https://en.hdbuzz.net/the-other-shoe-drops-uniqure-shares-plans-to-submit-licensing-application-with-the-fda-for-amt-130/

Skyhawk Therapeutics has shared new 12-month results from its Phase 1/2 study of SKY-0515, an investigational oral therapy for Huntington disease (HD). The updated data continue to show that the treatment lowers huntingtin protein, maintains favourable trends in the cUHDRS (a multidimensional scoring metric used in clinical research to track the progression of HD), and remains generally safe and well tolerated.

Novartis presents updated 24-month results from PIVOT-HD, which support the continuation of Novartis’ Phase 3 trial for votoplam.

Roche and Genentech have received feedback from an Independent Data Monitoring Committee (iDMC) regarding the recently reviewed safety data from the Generation HD2 clinical trial. The iDMC have recommended that the study continue, but only the higher dose will move forward. The study is ongoing, and final conclusions are not yet available.

This adds to its existing RMAT, Orphan Drug, and Fast Track designations — all of which support a quicker, more collaborative review process with the agency.

PTC Therapeutics provided an update on their PIVOT-HD study. This study met its main goal by showing that their drug lowers huntingtin protein levels in a dose-dependent way. Early results also suggest potential clinical benefits and good safety in people with Stage 2 Huntington disease.

Novartis shares additional information on their plans following PTC Therapeutics’ communication of the results of the PIVOT-HD Phase 2 study of votoplam (PTC518).

Prilenia and Ferrer provide an update on European regulatory process for pridopidine in Huntington disease.