PTC Therapeutics posts corporate presentation highlighting Sephience PKU launch momentum

PTCT•2026-07-30

Sephience launch momentum and pipeline priorities

PTC Therapeutics said Sephience generated Q2 2026 global revenue of USD 151 million, with 1,647 patients on commercial therapy worldwide.

The company positioned Sephience as a potential new standard of care for PKU, with intellectual property into 2039 and more than USD 2 billion of global revenue potential.

On the pipeline, PTC said its Phase 3 votoplam Huntington’s trial is targeting about 770 participants, and the PROVE-FA vatiquinone study is expected to start in Q3 2026.

PTC Therapeutics provides 2026 outlook and cash update

PTC Therapeutics guided for 2026 total revenue of USD 1.18-1.28 billion, product revenue of USD 850-950 million, and OpEx of USD 680-720 million.

The company said it had a cash balance of USD 2.2 billion and is targeting 2026 cash flow breakeven.

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PTC Therapeutics posts corporate presentation highlighting Sephience PKU launch momentum

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PTC Therapeutics posts corporate presentation highlighting Sephience PKU launch momentum

Sephience launch momentum and pipeline priorities

PTC Therapeutics provides 2026 outlook and cash update

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