PTC Therapeutics Showcases Sephience as Potential New Standard of Care for PKU in Corporate Presentation
Reuters
Nov 05, 2025
PTC <a href="https://laohu8.com/S/LENZ">Therapeutics</a> Showcases Sephience as Potential <a href="https://laohu8.com/S/NSE.AU">New Standard</a> of Care for PKU in Corporate Presentation
PTC Therapeutics Inc. provided an update highlighting the commercial launch and early performance of Sephience, its therapy for phenylketonuria (PKU). The company reported $19.6 million in global revenue from Sephience, with $14.4 million generated in the US and $5.2 million from ex-US markets. As of September 30, 2025, 341 patients were on commercial therapy worldwide, with 521 Patient Start Forms submitted by 141 unique US prescribers. Sephience demonstrated clinically significant reductions in blood phenylalanine (Phe) levels, showing superior Phe lowering compared to sapropterin (BH4) in head-to-head studies. Additional marketing authorization reviews for Sephience are ongoing, including a decision expected in Japan in the fourth quarter of 2025. The company also noted a strong balance sheet and the ability to support future commercial launches and research initiatives without additional capital. You can access the full presentation through the link below.
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