PTC Therapeutics : PIVOT-HD Results – May 2025

Published: 2025-05-05 09:28:26 ET PTCT

Published on 05/05/2025 at 09:28

Matthew B. Klein, MD CEO

May 2025

1 PIVOT-HD Phase 2 Readout

PTC518 5 mg

PTC518 10 mg

Placebo

12 Weeks

12 Months

PTC518 5 mg

PTC518 10 mg

PTC518 5 mg

PTC518 10 mg

48 Months

PIVOT-HD Study Design

R

Primary Endpoint: Reduction in Blood HTT protein (Week 12)

*Placebo subjects randomized 1:1 to 5 mg or 10 mg for OLE and all subjects remain blinded to initial treatment assignment

Dose-Dependent mHTT Lowering and Safety Confirmed with Signals of Longer-term

Dose-Dependent Biomarker and Clinical Effect

Study met primary endpoint of blood HTT protein lowering at Week 12 with durable dose-dependent lowering at Month 12

Long-term positive clinical trends at Month 24 relative to natural history and dose-dependent NfL lowering

Favorable and dose-dependent trends on clinical scales at Month 12 in Stage 2 subjects

Continued favorable safety and tolerability profile with no treatment-related NfL spikes

5 PIVOT-HD Phase 2 Readout

Disclaimer

PTC Therapeutics Inc. published this content on May 05, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 05, 2025 at 13:22 UTC.