PTC Therapeutics June 2025 Matthew B. Klein, MD CEO Pam and Kelsey living with PKU 1 Corporate Presentation – June 2025 Forward Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the ...

PTC Therapeutics, Inc. PTCT on Monday announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington’s disease (HD) patients.Huntington’s disease is a progressive neurodegenerative disorder caused by mutation of the huntingtin (HTT) gene, leading to the breakdown of brain cells. It is characterized by uncontrolled movements (chorea), cognitive decline, and psychiatric disturbances.The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein leve ...