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    Ryan Mcelroy • Leerink Partners

    Ryan Mcelroy's questions to Regenxbio Inc (RGNX) leadership

    Ryan Mcelroy's questions to Regenxbio Inc (RGNX) leadership • Q2 2025

    Question

    Ryan Mcelroy, on behalf of Mani Faroohar, asked about the expected launch dynamics for RGX-202 in DMD and whether the subretinal approach for wet AMD is viewed as a durable market or simply a precursor to the suprachoroidal delivery method.

    Answer

    President & CEO Curran Simpson clarified that the subretinal approach for wet AMD is viewed as a standalone commercial opportunity, noting strong recruitment in Europe. For the DMD launch, he stated the company is aiming for a broad label and sees an opportunity for a dominant market position, with a current focus on the ambulatory patient population.

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    Ryan Mcelroy's questions to Regenxbio Inc (RGNX) leadership • Q4 2024

    Question

    Ryan Mcelroy, on behalf of Mani Foroohar at Leerink Partners, questioned the pace of enrollment for the RGX-202 pivotal trial in Duchenne muscular dystrophy and whether it's expected to accelerate. He also asked if the trial has begun enrolling younger patients from the 1-to-3-year-old cohort into the pivotal phase.

    Answer

    CEO Curran Simpson confirmed that enrollment is progressing at an encouraging rate and is expected to accelerate as more clinical sites are activated, with the goal of completing enrollment this year. Chief Medical Officer Dr. Steve Pakola added that patient interest grew significantly after the November functional data release. He also confirmed that patients aged 1 and older are now being enrolled directly into the pivotal trial, addressing the significant unmet need in the 1-to-3-year-old population.

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    Ryan Mcelroy's questions to Mirum Pharmaceuticals Inc (MIRM) leadership

    Ryan Mcelroy's questions to Mirum Pharmaceuticals Inc (MIRM) leadership • Q2 2025

    Question

    Ryan Mcelroy of Leerink Partners asked about LIVMARLI's market penetration in Alagille and PFIC, the potential revenue breakdown for its $1B+ target, and what Mirum hopes to learn from Shionogi's upcoming Fragile X data.

    Answer

    COO & President Peter Radovich estimated U.S. Alagille penetration is approaching 50% and noted the PFIC market is expanding beyond initial estimates. Co-Founder & CEO Chris Peetz added that the EXPAND study is a key contributor to the $1B+ potential. Regarding Shionogi, Peetz stated Mirum is confident in its own differentiated candidate regardless of the outcome.

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    Ryan Mcelroy's questions to Mirum Pharmaceuticals Inc (MIRM) leadership • Q1 2025

    Question

    On behalf of Mani Foroohar, Ryan Mcelroy asked about expectations for pruritus reduction in the VISTAS study for PSC and how volixibat would be positioned within the competitive landscape for that indication.

    Answer

    CEO Christopher Peetz indicated that the VANTAGE interim result is a robust benchmark for VISTAS's potential activity level. President and Chief Operating Officer Peter Radovich added that the PSC market is highly unsatisfied with no approved therapies, positioning volixibat to potentially hold a strong, highly preferred market position upon approval.

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