PTC Therapeutics (PTCT) Corporate presentation summary

Financial position and 2026 priorities

• 2026 product revenue guidance: $960–990M, representing 64–69% YoY growth; total revenue guidance: $1.29–1.32B.

• 2026 non-GAAP OpEx guidance: $680–720M, covering R&D and SG&A and excluding estimated ~$95M non-cash stock-based compensation; GAAP R&D and SG&A guidance: $775–815M.

• Cash balance was $2.1B as of September 30, 2026; target is cash flow breakeven in 2026 and future sustainable profitability.

• Strategic priorities: sustain Sephience launch momentum, advance the Phase 3 votoplam HD program and early-stage R&D, and move toward cash flow breakeven.

• Global commercial infrastructure has achieved regulatory authorization, market access and commercialization of rare-disease therapies in over 50 countries.

Sephience launch and market opportunity

• Sephience generated $458M in global revenue YTD in its first full launch year, with penetration across key market segments.

• Sephience is positioned as a potential new standard of care for PKU, with IP into 2039 and stated global revenue potential above $2B.

• AMPLIPHY crossover data showed mean absolute blood Phe reductions of 437.0 μmol/L with Sephience versus 256.6 μmol/L with BH4; 70.3% greater reduction, p<0.0001.

• In the extension study, 69% of subjects reached age-adjusted protein RDA; reported benefits included improved concentration, slow thinking, irritability and quality of life.

• Potential addressable market spans treatment-naive patients, treatment failures and patients on treatment, totaling 58K patients worldwide across ages and severities.

• Geographic launch footprint expansion is planned for 2026 and 2027 across North America, Europe, Asia and Latin America.

Clinical programs

• ST-920, an AAV gene therapy for Fabry disease, has over 5 years of follow-up, durable α-Gal A activity and reported favorable renal and other disease effects; BLA submission for accelerated approval expected to be completed in Q4 2026.

• In PIVOT-HD, votoplam met the Week 12 primary endpoint of blood HTT protein lowering, with durable dose-dependent lowering at Month 12 and evidence of dose-dependent disease slowing at 24 months in Stage 2 participants.

• At Month 24, cUHDRS progression was 52% lower with votoplam 10 mg and 28% lower with 5 mg versus the natural-history cohort; no new adverse-event signals were identified through 24 months.

• Novartis-sponsored INVEST-HD Phase 3 is enrolling early symptomatic participants worldwide; target enrollment ~770 across >30 countries, with 3:2 randomization to votoplam 10 mg or placebo and treatment up to 36 months.

• Vatiquinone showed 42% slowing on upright stability in the 72-week MOVE-FA trial, which did not meet its primary endpoint of significant change in total mFARS; extension data reported 50% slowing over 3 years versus matched FACOMS natural history.

• PROVE-FA, initiated in Q3 2026, is an open-label Phase 3 study targeting 120 participants with FA ages 7–21; primary endpoint is mFARS change from baseline to 24 months.