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    Julian PinoStifel Financial Corp.

    Julian Pino's questions to ACADIA Pharmaceuticals Inc (ACAD) leadership

    Julian Pino's questions to ACADIA Pharmaceuticals Inc (ACAD) leadership • Q2 2025

    Question

    Julian Pino, on for Paul Matteis, sought clarification on the ability to modify the statistical analysis plan (SAP) for ACP101 while blinded. He also asked why Daybue guidance was not narrowed given the strong performance and expectation for accelerating growth.

    Answer

    EVP, Head of R&D, Elizabeth Thompson, clarified that while an SAP can technically be changed before unblinding, there are no planned modifications for ACP101. CFO Mark Schneyer explained that the NUPLAZID guidance was narrowed because its initial range was unusually wide, whereas the company will revisit other guidance ranges, including Daybue's, in the third quarter as is typical.

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    Julian Pino's questions to BioMarin Pharmaceutical Inc (BMRN) leadership

    Julian Pino's questions to BioMarin Pharmaceutical Inc (BMRN) leadership • Q4 2024

    Question

    Julian Pino, on for Paul Mattias, asked what was learned from the two early 12-week biopsies in the DMD study and what provided confidence in the 10% dystrophin goal.

    Answer

    Chief R&D Officer Gregory Friberg stated that the two early biopsies were directionally positive. They confirmed that the drug reached the muscle, the novel splice variant was being produced, and near full-length dystrophin was measurable, consistent with their models. He said this removed key 'pertinent negatives' and provides confidence for the more informative 25-week data readout.

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