Latest News

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.

The Huntington Society of Canada is pleased to announce the recipients of the 2026 Clinical Fellowships. These fellowships will fund a period of advanced training to allow this year’s recipients the opportunity to develop deeper expertise and specialized skills in Huntington disease and neurodegenerative disease care.

Together with our partner, Brain Canada, we are proud to announce the recipients of the 2026 HSC and Brain Canada Undergraduate Student Summer Fellowship. Supported in part by the Canada Brain Research Fund, these fellowships offer undergraduate students across Canada the opportunity to work alongside senior HD scientists and contribute to projects that help advance HD research.

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.

Canadian movement disorders organizations join together to train the next generation of specialists.

Novartis begins patient enrollment in Canada and USA for INVEST-HD Phase 3 trial.

Skyhawk Therapeutics Inc. announced encouraging interim results concerning SKY-0515, an investigational medicine being studied for Huntington disease (HD).

The Huntington Society of Canada (HSC), Brain Canada, and the River Philip Foundation are pleased to announce the recipients of the 2025 Navigator Awards. HSC’s Navigator Research Program is designed to provide funding for Canadian scientific research projects of direct and immediate relevance to Huntington disease.

uniQure released an update on discussions with the FDA about AMT-130, an investigational one-time gene therapy designed to slow Huntington disease (HD) progression.

Canadians affected by HD continue to face significant barriers to receiving the specialized care and support they need. A new multi-stakeholder report commissioned by HSC identifies critical gaps across the healthcare system, including limited access to knowledgeable clinicians, inconsistent care, inadequate caregiver supports, and challenges accessing long-term care. The report also outlines opportunities to improve education, coordination, and services for families across Canada.

The Huntington Society of Canada is pleased to confirm the appointments of its dedicated Board of Directors for the coming year.

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.

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.

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.

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.

We are excited to follow the research of Dr. O’Donoghue, our HSC Research Chair, as he and a fabulously talented team investigate personalized therapies for Huntington disease and other genetic disorders.