04117 Results from the BEACON trial: a phase 2, randomized, open-label trial of bitopertin in erythropoietic protoporphyria

g/dL, or concurrent treatment with afamelanotide or dersimelagon.Randomization was stratified by baseline light tolerance (time to prodrome < or !30 minutes), as assessed during a 2week run-in period.The primary endpoint is percent change from baseline in whole blood metal-free PPIX in participants randomized to bitopertin compared to placebo.The key secondary endpoint is the total hours of sunlight exposure to skin on days with no pain from 10:00 to 18:00 hours.Upon completion of the double-blind treatment period, participants may continue in an open-label extension study.Results Unblinded topline safety and efficacy data, including changes from baseline in wholeblood metal-free PPIX and measures of light tolerance, will be presented.Conclusion Bitopertin has been shown to significantly reduce PPIX levels in prior clinical and nonclinical studies of EPP.The AURORA study evaluates whether reductions in PPIX with bitopertin can improve measures of light tolerance in adults with EPP.Topline safety and efficacy data will be presented.