October 4, 2026

@michaelokun

This late-breaking abstract from the International Congress of Parkinson’s Disease and Movement Disorders in Seoul this weekend is exciting because PSP remains a devastating disease with no proven therapy that meaningfully slows its progression. Here in this data we have a signal that deserves serious attention. In this Phase 2a extension, GV1001 was well tolerated through 72 weeks, with no new worrisome safety signals or drug-related serious adverse events, and the group that started early on 0.56 mg continued to show the most favorable PSP Rating Scale trajectory even after all groups were receiving active treatment. This raises the intriguing possibility of a disease-modifying effect, rather than simply short-term symptomatic benefit. The signal was particularly notable in PSP-Richardson syndrome and in participants with lower baseline NfL biomarker levels (possibly those w/ less severe disease). An external comparison favored the early-treatment group. For persons w/ disease and families waiting for new PSP treatments, this is genuine reason for cautious optimism, but it is not yet proof. The extension study was open-label, included only 67 participants, had 15 dropouts, and the external placebo comparison was not randomized. This was also an abstract and not the fully peer-reviewed paper. The next critical step will be a large, adequately powered Phase 3 randomized trial to determine whether GV1001 can truly slow PSP progression. https://www.mdscongress.org/Abstracts/Late-breaking