Question · Q3 2025
Mark inquired about Mirum's perspective on the optimal endpoint for Fragile X studies, given recent comments from Shionogi, and the implications for Mirum's newly initiated program. He also asked about potential safety risks, specifically GI side effects, for Volixibat in PSC patients who often have inflammatory comorbidities, and whether blinded data showed any material differences compared to PBC or Alagille.
Answer
CEO Chris Peetz deferred to CMO Joanne Quan for details. CMO Joanne Quan expressed confidence in Mirum's Fragile X program, citing strong preclinical data, successful engagement with the community and FDA, and awareness of endpoint validation requirements. Regarding PSC safety, she noted that the data monitoring committee has raised no issues or suggested protocol modifications, indicating comfort with the current safety profile, which is consistent with known IBAT inhibitor effects.
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