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    Wing Yip • H.C. Wainwright & Co.

    Wing Yip's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership

    Wing Yip's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q3 2024

    Question

    Wing Yip, on for Ed Arce, asked about the significance of the 12-week primary efficacy period in the Angelman Phase 3 study and inquired about the next development steps for setrusumab (UX143) in 2025.

    Answer

    CMO Eric Crombez explained the Angelman study duration was chosen to allow sufficient time for skills to develop and be detected, based on Phase 1/2 data. CEO Emil Kakkis added that 2025 for setrusumab is focused on the interim analyses of the Orbit study and data from the Cosmic study, with the goal of filing a BLA next year, pending the timing of the final data readout.

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    Wing Yip's questions to Arbutus Biopharma Corp (ABUS) leadership

    Wing Yip's questions to Arbutus Biopharma Corp (ABUS) leadership • Q2 2024

    Question

    Wing Yip, on behalf of Ed Arce, asked if the upcoming IM-PROVE II expansion cohort data would be presented at a major medical conference, if other interim data from IM-PROVE I and II would be released before year-end, and how the operational streamlining would affect other programs.

    Answer

    Interim President and CEO Mike McElhaugh stated they hope to present the IM-PROVE II data at a conference but cannot commit yet. He confirmed more interim data from IM-PROVE I and II is planned for release, with timing to be determined. He also clarified the streamlining focuses resources on imdusiran's late-stage development without impacting other clinical programs.

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