We have received an interesting update from the pharmaceutical company Novartis about the medicine they currently are testing for Huntington’s Disease.
Do you remember the study named PIVOT-HD done a little while ago of a company named PTC Therapeutics? People who took part in this study were later invited to participate in a longer follow-up study – the Long-Term Extension (LTE) study. By continuing treatment over a longer period, researchers are able to better understand how this medicine called votoplam performs over time in terms of both safety and potential benefit.
This medicine helps the body make more of the healthy huntingtin protein and less of the harmful form that leads to disease development, which could help slow or change the course of HD.
The newly shared topline results reflect up to 24 months of data (12 months from the original study – Phase 2 PIVOT-HD parent study – plus 12 months from the extension study – “An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington’s Disease (HD)”.
According to Novartis, these findings strengthen confidence in moving forward to the next phase of development.
This kind of long-term data is especially important in Huntington’s Disease, where understanding how a treatment works over time is key.
It is also important to note that in the LTE study, all participants are receiving the active treatment. This means there is no placebo group within the study, and results are instead compared to external data that reflects how the disease typically progresses.
Building on these results, Novartis has launched a Phase 3 trial called INVEST-HD, which began enrolling participants in 2026.
Phase 3 trials are a critical step. They involve a larger number of participants and are designed to confirm whether a treatment is effective, safe, and well tolerated – often the final stage before potential regulatory approval.
While we should be cautious in jumping to conclusions, this update is indeed an encouraging sign of continued progress. It reflects ongoing commitment to developing new treatment options and building on data collected over time.
It is thanks to the dedication of people living with Huntington’s Disease, along with their families, caregivers, researchers, and clinical teams around the world, that we continue to move forward – building a more informed and connected community, and staying closer to the scientific advances that have the potential to improve quality of life for all.
Novartis has emphasized its commitment to transparency and to sharing updates as development continues. Anyone interested in learning more about ongoing studies or participation opportunities is encouraged to speak with their healthcare provider and consult trusted sources such as ClinicalTrials.gov.
You can view the Phase 3 INVEST-HD study (NCT7326709) information here.
If you would like to learn more about ongoing clinical trials in Europe, you should explore: