Novartis and PTC Therapeutics have moved their Huntington’s disease candidate, votoplam, into Phase 3 development after results from a mid-stage study indicated a reduction in disease progression. The decision follows an interim analysis from the long-term extension of the Phase 2 PIVOT-HD trial, which was presented earlier this week.
Data from the study showed that patients receiving a 5-mg dose of votoplam experienced a 28% slowing in disease progression over 24 months compared to natural history controls. At a higher 10-mg dose, the effect was more pronounced, with progression reduced by 52%. In addition, treatment with votoplam maintained neurofilament light chain levels below baseline throughout the 24-month observation period, suggesting sustained biological activity over time.
Based on these findings, Novartis announced during a recent earnings call that it has initiated a Phase 3 trial, known as INVEST-HD. This study will evaluate votoplam against a placebo in individuals diagnosed with Huntington’s disease. The trial is expected to enroll approximately 770 participants, with results anticipated in 2030 according to company disclosures.
Under the terms of their collaboration, Novartis will lead late-stage development efforts, while PTC Therapeutics will continue to support discussions regarding the current dataset and outline potential next steps. These discussions are expected to include regulatory considerations tied to the therapy’s future and how the existing evidence may be assessed.
Analysts have noted that the launch of a Phase 3 trial reflects both confidence in the drug’s potential and caution regarding regulatory outcomes. In a note to investors, RBC Capital Markets stated that the initiation of INVEST-HD “conveys continued long-term optimism around the program.” At the same time, the analysts pointed out that the companies have not explicitly referenced plans for accelerated approval, describing their approach as measured.
The analysts further indicated that this positioning could reflect uncertainty about whether the existing data package is sufficient for early regulatory clearance. They suggested that the absence of a clear accelerated approval strategy may indicate that the current evidence is not yet considered definitive.
Votoplam is designed as a small-molecule splicing modifier targeting the huntingtin protein, which plays a central role in Huntington’s disease. The drug works by introducing a stop codon into the messenger RNA responsible for producing the protein, thereby reducing the production of its defective form in patients.
The therapy was originally developed by PTC Therapeutics and later partnered with Novartis in December 2024. The agreement included an upfront payment of $1 billion, along with the potential for up to $1.9 billion in milestone payments. As part of the collaboration, PTC led development through the mid-stage PIVOT-HD trial, after which Novartis assumed responsibility for advancing the program into Phase 3.
Financial terms of the partnership also provide PTC with a 40% share of profits generated from U.S. sales of votoplam, in addition to tiered royalties on sales in international markets. These arrangements remain in place as the program progresses into its next stage of clinical evaluation and further study.
Overview of Novartis and PTC Collaboration
Novartis and PTC have taken a major step forward in neurological drug development as Novartis and PTC advance votoplam into Phase 3 clinical trials. The decision follows promising mid-stage results that demonstrated the drug’s ability to significantly reduce levels of the harmful huntingtin protein associated with Huntington’s disease.
The collaboration between Novartis and PTC reflects a growing commitment to tackling rare neurodegenerative diseases with innovative molecular therapies.
Mid-Stage Data Driving Phase 3 Progress
The advancement by Novartis and PTC is supported by data from the PIVOT-HD Phase 2 study, which showed meaningful reductions in huntingtin protein levels—a key driver of disease progression.
These findings provided strong evidence for Novartis and PTC to initiate a global Phase 3 program aimed at confirming long-term clinical benefits and safety.
Phase 3 INVEST-HD Study by Novartis and PTC
Novartis and PTC have launched the Phase 3 INVEST-HD trial, a large global study evaluating the efficacy and safety of votoplam in patients with early-stage Huntington’s disease.
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