Novartis recently issued a community letter announcing their plans for a Phase 3 clinical trial for votoplam, a potential new treatment for HD. This next step means they will test how well it works and how safe it is in a larger group of participants, following encouraging results from the Phase 2 trial.
As many of you already know, Novartis entered into a global license and collaboration agreement with PTC Therapeutics several months ago for the development of votoplam. They are now in conversation with the HD community to develop the Phase 3 protocol.
Votoplam, previously called PTC-518, is an oral medicine being developed for Huntington’s Disease. It works by helping the body make more of the healthy huntingtin protein and less of the harmful form that leads to disease development. This could help slow or change the course of HD.Â
The agreement means that PTC Therapeutics granted Novartis the right to develop votoplam, and both companies will work together by combining their expertise, resources, and efforts. This collaboration aims to move the research forward more efficiently and safely, with Novartis leading the next phases and bringing the strength to conduct trials, analyze results, and navigate regulatory steps. If the data continue to be positive, this could eventually support making votoplam available to families worldwide.
Promising data from Phase 2 support moving forward with a Phase 3 trial as soon as possible, to further evaluate the efficacy and safety of votoplam in more participants. While the full analysis of the Phase 2 data is still ongoing, Novartis is actively consulting the HD community. They plan to engage with EHA, HD-CAB, and other patient groups to gather valuable input and expertise from people with lived experience to help shape the next phase. Â
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We are encouraged by Novartis’ renewed involvement with the HD community and look forward to following and supporting the progress of this very exciting program.Â
You can watch a video explaining previous results from the clinical trial here and read the full community letter here.Â
– Article written by Jarelys López