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    Rudy Li • Chardan Capital Markets

    Rudy Li's questions to Stoke Therapeutics Inc (STOK) leadership

    Rudy Li's questions to Stoke Therapeutics Inc (STOK) leadership • Q2 2025

    Question

    Rudy Li from Chardan Capital Markets questioned the higher incidence of CSF protein elevations in the OLE study and asked about any specific concerns, especially for the patients classified as having treatment-emergent adverse events.

    Answer

    CMO Barry Ticho clarified that elevated CSF protein is a known class effect for intrathecally administered oligonucleotides and is a laboratory finding without any associated clinical manifestations in the 81 patients dosed. He emphasized that the long-term safety profile over four years remains favorable and does not present a concern for the Phase 3 study.

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    Rudy Li's questions to Praxis Precision Medicines Inc (PRAX) leadership

    Rudy Li's questions to Praxis Precision Medicines Inc (PRAX) leadership • Q2 2025

    Question

    Rudy Li from Chardan Capital Markets asked about the impact of background therapies on patient selection for pivotal trials and what additional data might be needed to support combination use with other sodium channel blockers.

    Answer

    President & CEO Marcio Souza stated the RADIANT population is representative of real-world practice. He expressed no concern about combinations, noting vormetragene had the lowest side effect rate despite aggressive background regimens. No major changes to enrollment criteria are expected beyond clearer instructions on tapering background ASMs.

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    Rudy Li's questions to Prothena Corporation PLC (PRTA) leadership

    Rudy Li's questions to Prothena Corporation PLC (PRTA) leadership • Q4 2024

    Question

    Rudy Li of Chardan asked about the number of actively treated AL amyloidosis patients, changes in market dynamics since daratumumab's approval, and the rationale for the 200mg expansion cohort in the PRX012 ASCENT-2 trial.

    Answer

    COO Brandon Smith stated that the quoted 16,000 U.S. patients are diagnosed and treated, up from 15,000 since daratumumab's 2021 approval. Chief Development Officer Chad Swanson explained the PRX012 expansion cohort was an opportunistic way to utilize patients who screened for but were not eligible for the rarer APOE homozygous cohorts, thereby building a larger dataset to better characterize the drug.

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