- Sephience Launch Success: Generated $92M in Q4 2025 and $111M since launch, with 80% of US PKU centers of excellence prescribing it and 20–30 international market expansion expected by 2026.
- Full-Year Revenue Growth: Net product and royalty revenue reached $831M in 2025 (exceeding guidance), driven by Sephience, with 2026 guidance of $700M–800M (19–36% YoY growth).
- Strong Cash Position: Ended 2025 with $1.95B in cash after selling the Evrysdi royalty for $240M, enabling R&D investment and strategic business development.
- Cost Discipline: Non-GAAP R&D and SG&A expenses were $728M in 2025 (under guidance), with 2026 guidance of $680M–720M, supporting cash flow breakeven potential.
Revenue Growth and Expense Management
The company's revenue growth was driven by the successful launch of Sephience, with $111 million in revenue since launch. PTC expects Sephience to become a standard of care for PKU, with a potential multibillion-dollar global revenue opportunity. The company's guidance for 2026 product revenue is between $700 million to $800 million, representing 19% to 36% year-over-year growth. Non-GAAP R&D and SG&A expenses are expected to be between $680 million to $720 million.
Valuation Metrics
Analysts estimate next year's revenue growth at 20.8%. The company's current valuation metrics are: P/E Ratio at 8.33, P/B Ratio at -27.71, P/S Ratio at 3.27, EV/EBITDA at 5.81, and Free Cash Flow Yield at 12.35%. These metrics indicate that the market is pricing in a significant growth potential, but also some level of risk given the negative P/B Ratio and relatively low P/E Ratio.
Sephience Launch and Commercial Performance
The launch of Sephience has been successful, with broad uptake across all patient segments and age groups. The company has seen positive feedback from healthcare providers and patients, with 80% of PKU centers of excellence in the US having written prescriptions for one or more patients. As Dr. Matthew Klein mentioned, "adults with PKU still have associations with clinics, and they want to be on therapies," indicating a strong demand for Sephience.
Pipeline Progress and Business Development
PTC is making progress in its pipeline, with the NRF2 activator program showing great activity in kidney and lung studies. The company is also advancing its NLRP3 program, with PTC612 demonstrating strong potency and selectivity. PTC is open to business development opportunities to accelerate top-line growth, with a strong financial position and a focus on maximizing shareholder returns.