PTC Therapeutics (PTCT) investor relations material

Executive summary

• Q3 2026 revenue was $357.8M, including record net product revenue of $275.9M; Sephience growth and higher Evrysdi royalties drove the increase.

• Reported net loss was $51.7M versus net income of $15.9M, including $111.0M of ST-920 acquired IPR&D and a $19.3M debt-extinguishment loss.

• Raised FY 2026 product revenue guidance to $960M–$990M and total revenue guidance to $1.29B–$1.32B; cash flow breakeven remains targeted for 2026.

• More than 2,000 patients were active on Sephience at quarter-end, with continued U.S. demand and international expansion.

Financial highlights

• Q3 net product revenue was $275.9M versus $131.0M; royalty revenue was $81.7M versus $70.8M, and collaboration and license revenue was $0.2M versus $9.3M.

• Q3 operating income was $25.1M versus $3.0M; basic and diluted EPS were both $(0.62) versus $0.20.

• GAAP R&D was $105.4M and SG&A was $80.1M; adjusted non-GAAP R&D was $94.9M and SG&A was $66.7M, excluding stock-based compensation.

• 9M revenue was $990.9M versus $1.566B, reflecting the prior-year $1.0B Novartis upfront payment; 9M net income was $29.0M versus $817.6M.

Outlook and guidance

• FY 2026 product revenue guidance increased to $960M–$990M from $850M–$950M; total revenue guidance is $1.29B–$1.32B, implying 64%–69% product revenue growth.

• FY 2026 GAAP R&D and SG&A guidance remains $775M–$815M; non-GAAP guidance remains $680M–$720M, excluding estimated $95M stock compensation.

• Cash flow breakeven remains the 2026 goal; ST-920 may be accretive in 2027, while commercialization and clinical-development expenses are expected to continue.

• ST-920 rolling BLA submission is targeted for Q4 2026, with potential U.S. approval and launch in 2027; regulatory approval outside the U.S. is also planned.

• Votoplam FDA Type C meeting is planned for Q1 2027; Phase 3 remains the base-case approval path. PROVE-FA is expected to enroll ~120 patients, ages 7–21.