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    Sushila Hernandez • Van Lanschot Kempen Investment Banking

    Sushila Hernandez's questions to Pharming Group NV (PHAR) leadership

    Sushila Hernandez's questions to Pharming Group NV (PHAR) leadership • Q2 2025

    Question

    Sushila Hernandez of Van Lanschot Kempen asked about the expected timeline for converting VUS (variant of uncertain significance) patients to paid Joenja therapy, inquiring about potential bottlenecks and the pace at which 20% of the 1,400 identified patients might be converted. She also requested a geographic breakdown of the 185 APDS patients currently in access programs.

    Answer

    CEO Fabrice Chouraqui and CMO Anurag Relan explained that the VUS reclassification will occur over time as diagnostic labs utilize new data from the Cell publication to re-evaluate patients. They noted that while the process has started, it will take time to fully capture the opportunity. Dr. Relan added that Pharming has prior experience with this reclassification process, which leads to quick reimbursement once a patient is confirmed to have APDS. Mr. Chouraqui declined to provide a geographic breakdown of patients at this stage.

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    Sushila Hernandez's questions to Pharming Group NV (PHAR) leadership • Q2 2024

    Question

    Sushila Hernandez asked for details on the third indication for leniolisib, the source of the 1,200 VUS patients, the expected conversion rate to APDS, the number of identified patients in the UK ahead of the MHRA decision, and for an update on business development activities.

    Answer

    Chief Medical Officer Anurag Relan explained the third indication is another primary immune deficiency with immune dysregulation, larger than the second. He stated the 1,200 VUS patients were identified via databases from large genetic testing companies and a pilot study showed a 20% conversion rate to APDS. CEO Sijmen de Vries added that 61 patients are identified in the UK, with 11 on early access, and noted significant BD activity, including non-binding offers, is ongoing.

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    Sushila Hernandez's questions to Uniqure NV (QURE) leadership

    Sushila Hernandez's questions to Uniqure NV (QURE) leadership • Q2 2025

    Question

    Sushila Hernandez inquired about the expectations for the new fourth cohort of AMT-130 patients with lower striatal volumes and asked about the next steps for the AMT-260 epilepsy program, including site expansion.

    Answer

    Chief Medical Officer Walid Abi-Saab responded that the primary goal for the fourth cohort is to document the safety of the procedure in that population. For AMT-260, he confirmed they have expanded to 14 active sites and, due to strong interest, expect to enroll additional patients before the end of the year.

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    Sushila Hernandez's questions to Verona Pharma PLC (VRNA) leadership

    Sushila Hernandez's questions to Verona Pharma PLC (VRNA) leadership • Q1 2025

    Question

    Sushila Hernandez inquired about the company's latest thinking on its strategy and potential partnerships for Ohtuvayre in the European Union and the United Kingdom as regulatory activities progress.

    Answer

    CEO David Zaccardelli stated that the company is actively engaged in a structured process with both the EMA and MHRA regarding potential applications. He anticipates having better clarity on the regulatory path and agency responses around mid-year 2025. He confirmed that the strategy for partnering in Europe remains the same and that achieving regulatory clarity is an integral part of those partnership discussions, which will progress throughout 2025.

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    Sushila Hernandez's questions to Verona Pharma PLC (VRNA) leadership • Q1 2025

    Question

    Sushila Hernandez from Van Lanschot Kempen inquired about the company's latest strategy and plans for partnering Ohtuvayre in the European Union and the United Kingdom.

    Answer

    David Zaccardelli, Chief Executive Officer, reported that structured discussions with the EMA and MHRA are ongoing. He anticipates having greater clarity on the regulatory path and timelines around mid-year 2025, which will in turn inform and progress discussions with potential European partners, as the core partnering strategy remains unchanged.

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