PTCT revenue doubles on Sephience launch; operating income swings positive to $149M

Filed July 30, 2026 · Period ending June 30, 2026 · Compared to 10-Q Aug 7, 2025 · ~2 min read

Key Changes

• high

  Total revenue rose 101.5% YoY to $360.5M, driven by Sephience's commercial launch ($275.9M in H1 2026 vs. zero in H1 2025). Operating income swung from a $35.0M loss to $149.0M profit as the new product more than offset Translarna and Emflaza declines.

  MD&A: Revenue & Operating Results verify on EDGAR →
• high

  Company withdrew Translarna's U.S. NDA in February 2026 and stated no further U.S. development is planned, closing the domestic regulatory pathway. Translarna sales fell $44.4M YoY following the EC's negative opinion in March 2025.

  MD&A: Translarna NDA Withdrawal verify on EDGAR →
• high

  Novartis initiated votoplam Phase 3 trial in April 2026, triggering a $50M milestone. Company reported 52% slowing of disease progression at 10mg dose in 24-month extension data and plans FDA engagement in H2 2026 to discuss potential accelerated approval.

  MD&A: Votoplam Phase 3 verify on EDGAR →