Expert Interview
A Second Opinion: Evaluating Phase 2 PIVOT-HD Trial Results of PTC Therapeutics' PTC518 in Stage 2 and Stage 3 Huntington’s Disease
Ticker(s): PTCTInstitution: Novant Health Matthews Medical Center
- Outpatient neurologist and Director of Memory Care
- Manages 500 patients with Parkinson's disease and 10 patients with Huntington's Disease
PIVOT-HD met its primary endpoint with significant dose-dependent reductions in blood HTT levels. How meaningful is this biomarker in establishing a disease-modifying effect, and is HTT suppression alone sufficient for regulatory consideration?
Added By: ben_adminStage 2 patients receiving PTC518 showed favorable trends in motor and functional scores over 12 and 24 months. How clinically relevant are these early changes in cUHDRS, TFC, and SDMT when compared to natural history benchmarks?
Added By: ben_adminStage 3 patients showed some inconsistencies in dose response. How should these findings be interpreted in the context of disease stage, and could disease progression stage influence future patient stratification in trials?
Added By: ben_adminPTC518 lowered plasma neurofilament light (NfL) by up to 14% without spikes, suggesting neuronal protection. How reliable is NfL as a predictive biomarker for long-term outcomes in HD, and how does this data compare with other investigational agents?
Added By: ben_adminThe study reported no treatment-related serious adverse events or NfL spikes across 24 months. How critical is this safety consistency in a disease where patients may require lifelong therapy?
Added By: ben_adminPTC518 uses a pseudoexon splicing strategy to reduce mutant HTT mRNA, following the splicing platform used in Evrysdi® for SMA. What are the implications of this splicing-based mechanism in CNS diseases like HD?
Added By: ben_adminThe study leveraged both placebo and ENROLL-HD natural history cohorts. How strong is the scientific rationale for using matched observational data, and can it support future regulatory filings or accelerated approval pathways?
Added By: ben_adminGiven the totality of biomarker and clinical data, what are the critical milestones PTC needs to reach for an accelerated approval filing? Could Stage 2 patients be the primary focus for a confirmatory trial?
Added By: ben_adminAre You Interested In These Questions?
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