PTC Therapeutics to present data from APHENITY clinical trial

PTC Therapeutics 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. Topline 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. The 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 while significantly increasing protein intake, consistent with previous results.