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PTC Therapeutics Presents New Sepiapterin Data from Ongoing Studies
— Results show 97% of phenylalanine (Phe) tolerance study participants were able to increase their dietary Phe intake with a mean increase of 126% — — Genetic
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"— Results show 97% of phenylalanine (Phe) tolerance study participants were able to increase their dietary Phe intake with a mean increase of 126% —\n— Genetic variant analysis of Phase 3 APHENITY trial demonstrates meaningful effect in classical subjects with non-BH4 responsive genotypes —\nWARREN, N.J., March 20, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. These data provide further evidence of the potential meaningful benefits of sepiapterin treatment for the full spectrum of phenylketonuria (PKU) patients, including significant diet liberalization.\n\nKey data presented include:\nOver 97% of subjects participating in the Phe tolerance protocol of the APHENITY open-label extension study demonstrate the ability to liberalize their diet while on sepiapterin treatment, with a mean increase in protein intake of 126%66% of subjects participating in the Phe tolerance sub study reached or exceeded the age-adjusted recommended daily allowance of protein intake for an individual without PKU, while maintaining control of blood Phe levelsGenetic variant analysis of subjects participating in the APHENITY study demonstrates that over 70% had a Genotype-Phenotype Value (GPV) consistent with classical PKU\"These new data provide further evidence of the meaningful benefit sepiapterin can provide for children and adults with PKU, including those with the most severe form of the disease,\" said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. \"Furthermore, the treatment benefit demonstrated in individuals with mutations not responsive to BH4 and consistent with classical disease phenotype support the potential to penetrate all key patient segments.\"\nThe data were presented in a poster titled \"Interim Results From the APHENITY Extension Study: Sepiapterin Reduces Blood Phe with Improved Dietary Phe Tolerance in Participants With Phenylketonuria\" and in a symposium presented by Nicola Longo, M.D., Ph.D., Professor and Chief of Division of Clinical Genetics at the University of California, Los Angeles and Suzanne Hollander, M.S., R.D., L.D.N., Senior Clinical Nutrition Specialist and Associate Di...