PTC Therapeutics (PTCT) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Business performance and financial outlook

• Achieved strong commercial execution in 2026, with SEPHIENCE launch exceeding expectations and setting up for cash flow breakeven and future profitability.

• Maintains a robust balance sheet with over $2 billion in cash, supporting both internal development and strategic acquisitions.

• Raised revenue guidance multiple times, reflecting confidence in multibillion-dollar potential of SEPHIENCE.

• Disciplined approach to business development, with recent acquisition of a BLA-stage gene therapy for Fabry disease for $111 million upfront.

• Positioned for profitability and sustainability, with flexibility to pursue further opportunities.

SEPHIENCE launch and market dynamics

• SEPHIENCE, an oral therapy for PKU, is experiencing broad uptake across all patient segments, including therapy-naive and severe mutation patients.

• Global launch executed rapidly in the U.S., Europe, and Japan, with ongoing expansion into additional regions and reimbursement negotiations.

• Sustained growth expected, with over 2,000 start forms in the U.S. and significant untapped patient segments remaining.

• High adherence rates and diverse patient-reported benefits, including improved mood, diet liberalization, and cognitive function.

• Discontinuation rates stabilizing at 20-25%, consistent with clinical trial data, and strong enthusiasm from patient and prescriber communities.

Pipeline and R&D strategy

• Advancing a robust R&D pipeline, including a gene therapy for Fabry disease, Huntington's disease program (votoplam), and inflammation programs.

• Votoplam for Huntington's disease shows 52% slowing of disease progression at higher doses, with ongoing phase III and multiple regulatory pathways under discussion.

• Splicing platform innovation enables targeting of over 250 RNA sequences, accelerating discovery and development of new therapies.

• MSH3 and PTC-303 programs address additional CNS and triplet repeat disorders, with faster development timelines compared to earlier assets.

• Strategic focus on leveraging validated RNA technology for differentiated, high-value therapies.