PTC Therapeutics (PTCT) Investor presentation summary

Strategic priorities and financial outlook

• Focus on accelerating the global launch of Sephience, advancing Phase 3 votoplam for Huntington's disease, and progressing early-stage R&D programs.

• Targeting 2026 product revenue of $850–950M, representing 45–62% year-over-year growth, and total revenue of $1.18–1.28B.

• Operating expenses for 2026 are projected at $680–720M (non-GAAP), with a cash balance of $2.2B as of June 30, 2026.

• On track to achieve cash flow breakeven and sustainable profitability by 2026.

Product and pipeline highlights

• Sephience, positioned as the new standard of care for PKU, demonstrated superior efficacy and quality of life benefits, with $151M Q2 2026 global revenue and 1,647 patients on therapy.

• Votoplam Phase 2 trial met key efficacy and safety endpoints in Huntington's disease; Phase 3 global trial initiated by Novartis with ~770 participants.

• Vatiquinone showed meaningful slowing of disease progression in Friedreich's ataxia, with a new Phase 3 study to support NDA resubmission starting Q3 2026.

• Innovative R&D pipeline includes programs in RNA splicing, inflammation, and neurodegenerative diseases, with multiple candidates advancing toward clinical trials.

Market expansion and competitive positioning

• Commercial infrastructure enables market access in over 50 countries, with planned expansion of Sephience's geographic footprint in 2026 and 2027.

• Sephience's differentiated profile allows penetration into all key PKU market segments, addressing a total addressable market of 58,000 patients worldwide.

• Proprietary platforms and strong IP position support long-term growth and competitive advantage.