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PTC Therapeutics to Present Recent Data for Sepiapterin at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium
- Results from the APHENITY trial in PKU and long-term extension study including Phe tolerance to be highlighted - SOUTH PLAINFIELD, N.J., Aug. 28, 2023
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"- Results from the APHENITY trial in PKU and long-term extension study including Phe tolerance to be highlighted -\nSOUTH PLAINFIELD, N.J., Aug. 28, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that results from the APHENITY clinical trial and long-term extension study will be presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 which is being held from Aug. 29 to Sept. 1 in Jerusalem, Israel.\nTopline results of the APHENITY trial will be presented in an oral presentation, \"Phase 3 APHENITY Study: Oral Sepiapterin for the Treatment of Phenylketonuria.\" The presentation will take place on Thursday, Aug. 31 during the Parallel Session: Phenylketonuria, beginning at 10:45 IST.\nThe poster \"B284: Phase 3 APHENITY Long-Term Study Design: Sepiapterin for Treatment of Phenylketonuria,\" will also be presented on Wednesday, Aug. 30 during the poster walk at 18:35 IST. The poster contains an updated data cut of the ongoing APHENITY long-term extension study. Phe tolerance results continued to demonstrate that sepiapterin treated patients maintain mean blood Phe control (>360 µmol/L) while significantly increasing protein intake, consistent with previous results.\nPTC will also be sponsoring a symposium, \"Advancing the Treatment of PKU: Results of the APHENITY Trial,\" which will be hosted on Aug. 31 at 12:30 IST in Dulcin Hall. The symposium will bring together leading international experts to discuss the latest results from the APHENITY trial, highlight the unique features of sepiapterin, and share findings from the dietary Phe tolerance assessment in the open-label extension study.\nIn addition, PTC will share ongoing research for aromatic l-amino acid decarboxylase (AADC) deficiency during an oral presentation, poster and sponsored symposium. \nMore information about the SSIEM Annual Symposium can be found at ssiem.org.\nAbout APHENITYAPHENITY was a global double-blind, placebo-controlled, registration-directed study which enrolled 156 children and adults with PKU. Participants were randomized to receive sepiapterin or placebo for six weeks with the primary endpoint being reduction in blood phenylalanine levels. The trial consisted of two parts. Part 1 was a run-in phase, during which all screened subjects received sepiapterin for two weeks. Only those s...