Record Quarterly Revenue and Strong YoY Growth
Total revenue of $273.0M and total product revenue of $226.0M in Q1 2026, representing a 47% increase in product revenue versus Q1 2025 (Q1 2025 product revenue $153M).
Sephience Launch Momentum
Sephience global revenue of $125M in Q1 2026 (U.S. $112M; international $13M), representing 36% quarter‑over‑quarter growth versus Q4 2025; company reported a consistent cadence of ~140 new prescription starts per month and plans commercial availability in up to 30 countries by year‑end.
Raised 2026 Guidance
Based on Q1 performance, company raised 2026 full‑year product revenue guidance to $750M–$850M and total revenue guidance to $1.08B–$1.18B.
Positive Votoplam Long‑Term Data
PIVOT‑HD 24‑month interim results showed dose‑dependent slowing of Huntington’s disease progression with an average 52% slowing on cUHDRS at 10 mg in Stage 2 versus a matched natural history cohort; safety profile remained favorable, supporting confidence in the partner‑led Phase III INVEST‑HD (~770 enrollment).
Vatiquinone Regulatory Path Forward
After an FDA Type C meeting, company is initiating an open‑label 24‑month study (target ~120 patients, ages 7–21) using a matched FACOMS natural history control to support NDA resubmission for Friedreich’s ataxia, a design management believes increases probability of success.
Pipeline Progress and New Program Initiation
Expect to initiate Phase I of PTC612 (oral NLRP3 inhibitor) in Q2 2026, and reported continued progress across MSH3 (HD/DM1), PTC844 (DHODH, Phase II‑ready), ferroptosis Parkinson’s program, and NRF2 activator program.
Commercial and Access Execution
Over 90% of U.S. PKU centers have prescribed Sephience; >2,200 prescriptions worldwide generated since initial launches; refill rates strong and discontinuation rates described as 'low double digits'; U.S. payer policies cover >2/3 of population with limited restrictions.
Solid Balance Sheet and Controlled OpEx
Cash, cash equivalents and marketable securities totaled $1.89B as of March 31, 2026; non‑GAAP R&D expense declined to $90M in Q1 2026 from $100M in Q1 2025 (–10%); non‑GAAP SG&A $74M vs $72M prior year (small increase).