34420 Perspective of patients with erythropoietic protoporphyria treated with dersimelagon, a selective melanocortin-1 receptor agonist: Results of the ENDEAVOR study exit questionnaire

Abstract

Background: Erythropoietic protoporphyria (EPP) imposes a significant burden on the quality of life (QoL) of patients. Dersimelagon, a novel synthetic, orally administered, selective melanocortin-1 receptor agonist, has demonstrated statistically and clinically significant improvement from baseline in average daily time to first prodromal symptoms in a phase 2 clinical trial. Methods: ENDEAVOR was a multicenter, phase 2, randomized double-blind, placebo-controlled study with a 16-week treatment and 6-week follow-up period. Here we report the results of a post hoc analysis assessing the clinical benefit of dersimelagon treatment and patient perspective of QoL using an online exit questionnaire composed of 28 close-ended questions completed after week 22 visit or post study completion. Participants rated their perceived change since the start of the study. Results: Of 102 participants, 75 (placebo, n = 24; dersimelagon, 100 mg, n = 28, 300 mg n = 23) completed the questionnaire. More recipients of dersimelagon (100 mg, 57.6%; 300 mg 31.4%) rated their EPP as “very much better” than placebo group participants (5.9%). They were also “much more often” (100 mg, 69.7%; 300 mg 48.6%) able to be outside at the end of the study than patients in the placebo group (8.8%). More participants who received dersimelagon (100 mg, 72.7%; 300 mg 62.9%) reported being “very satisfied” with the study drug vs placebo recipients (14.7%). Conclusions: A greater proportion of patients who received dersimelagon reported improvements in their EPP and associated disease impacts than placebo group participants. The findings from the exit questionnaire support the results from the primary analysis and the potential use of dersimelagon for treatment of patients with EPP.