Shares declined 1.1% following the quarter as investors appeared cautious despite solid early revenue growth, likely reflecting concerns over modest prescription uptake and a subdued near-term outlook for pipeline progress.

• Total net revenue reached $36.2 million, up 78% year-over-year, driven by continued adoption of MIPLYFFA in Niemann-Pick disease type C (NPC).
• Prescription enrollment rose to 170 since launch, with only nine new enrollments during the quarter, highlighting a slower pace of recent patient additions.
• The diagnosed U.S. NPC patient pool is approximately 300-350, with around half reached to date, suggesting substantial runway but early-stage commercial traction.
• The Phase III DiSCOVER trial for Celiprolol in Vascular Ehlers-Danlos Syndrome (VES) enrolled 62 patients, with just 10 added this quarter, indicating gradual recruitment progress in a challenging rare disease.
• Despite a strong cash position ($236.8 million) and no debt, investors likely remain cautious about near-term catalysts given limited near-term visibility on regulatory milestones and clinical readouts.