Novartis development of Votoplam in HD: INVEST-HD Phase 3 trial information now available

February 3rd 2026

Dear Huntington’s disease Community,

We are delighted to share an update on the development of votoplam (formerly PTC518). Following the review of the results of the Phase 2 study, Novartis is now planning a Phase 3 clinical trial evaluating votoplam in people living with Huntington’s disease (HD). The study name for this trial is INVEST-HD. Information about the votoplam Phase 3 clinical study, INVEST-HD, has now been posted on ClinicalTrials.gov. This is an important step in clinical research, and it allows the HD community, healthcare professionals, and advocacy organizations to access key information about the upcoming study in a publicly available place.

About the INVEST-HD study

INVEST-HD is a global. pivotal Phase 3 clinical trial designed to evaluate the safety and efficacy of votoplam, an investigational oral medicine for Huntington’s disease. The aim of the study is to see whether votoplam helps slow the progression of Huntington’s disease in people in the early stages of the condition. The study builds on findings from earlier research, including the Phase 2 PIVOT-HD study. While current treatments can help manage symptoms of HD, there remains a high unmet need for therapies that slow the progression of Huntington’s disease. As with all clinical studies, participant safety is a top priority, and their health will be carefully monitored throughout the duration of this study.

What does it mean that the study is listed on ClinicalTrials.gov?

Posting the study on ClinicalTrials.gov means that important details (such as the study’s purpose, design, and general participation criteria) are now publicly available. As the study progresses, the listing may be updated with additional information. Being listed does not mean that the study is enrolling at all locations yet. You can view the INVEST-HD study information here: https://clinicaltrials.gov/study/NCT07326709

We understand the significant impact Huntington’s disease has on individuals, families, and caregivers. Research studies like INVEST-HD are only possible because of the commitment and partnership of the HD community. We are deeply grateful to everyone who contributes to advancing research, whether through participation, advocacy, or support.

Novartis remains committed to transparency, collaboration with the HD community, and sharing updates as the development continues. We encourage anyone who is interested to review the study information posted on ClinicalTrials.gov and to speak to their healthcare provider. If you have any questions about this information, you can also contact us through the email below.

Kind regards, Votoplam Clinical Development Team at Novartis Novartis Pharmaceuticals novartis.email@novartis.com