Catégorie : Clinical Research

New four-year results from uniQure provide additional insight into AMT-130, an investigational gene therapy being studied as a potential treatment for Huntington disease (HD). The latest results update on two commonly studied clinical measures in HD: – Total Functional Capacity (TFC): At 48 months, participants who received the high dose of AMT-130 showed a 61% slower decline compared with an external control group. – cUHDRS: The analysis showed a 44% slowing of progression at 48 months compared with the external control group. This result did not reach statistical significance. TFC and cUHDRS measure different aspects of HD progression. TFC focuses on functional abilities and independence, while cUHDRS combines several clinical measures to provide a broader assessment of disease progression. The 48-month results need to be interpreted with caution. The number of participants in the study remains relatively small, and the analysis relies on comparisons with an external control group. uniQure has also noted that substantial missing data and potential survivor bias in the updated external control group may have affected the results. AMT-130 is still an investigational treatment, and these results do not yet establish whether it will ultimately be approved or how it may be used in clinical practice. The HD treatment landscape continues to evolve, and HSC will continue following AMT-130 and other clinical developments closely. We’ll share updates as more information becomes available.

Skyhawk Therapeutics, Inc. today announced final 15-month results from its Phase 1/2 clinical trial evaluating SKY-0515, an investigational oral treatment for Huntington disease (HD).

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 présente des résultats actualisés sur 24 mois de PIVOT-HD, qui soutiennent la poursuite de l'essai de phase 3 de Novartis pour le votoplam.

Roche et Genentech ont reçu un retour d'information d'un comité indépendant de surveillance des données (iDMC) concernant les données de sécurité récemment examinées de l'essai clinique Generation HD2. L'iDMC a recommandé que l'étude se poursuive, mais seule la dose la plus élevée sera maintenue. L'étude est en cours et les conclusions définitives ne sont pas encore disponibles.

Cela s'ajoute à ses désignations existantes RMAT, médicament orphelin et accéléré — qui soutiennent toutes un processus d'examen plus rapide et plus collaboratif avec l'agence.

PTC Therapeutics a fourni une mise à jour sur son étude PIVOT-HD. Cette étude a atteint son objectif principal en montrant que son médicament réduit les niveaux de protéine huntingtine de manière dose-dépendante. Les premiers résultats suggèrent également des bénéfices cliniques potentiels et une bonne sécurité chez les personnes atteintes de la maladie de Huntington de stade 2.