Sadia Rehman's questions to BioMarin Pharmaceutical Inc (BMRN) leadership • Q4 2024
Question
Sadia Rehman, on for Mohit Bansal, asked if the upcoming DMD data would include biomarkers like splicing levels for comparison with other agents, and whether the decision to escalate to a 9 mg/kg dose was based on the 13-week data.
Answer
Chief R&D Officer Gregory Friberg clarified that the 9 mg/kg dose escalation was a planned step, approved by the independent Data Monitoring Committee based on safety, not a reaction to the 13-week efficacy data. He confirmed they are using multiple assays to measure dystrophin and will be transparent with the data, but did not commit to presenting other specific biomarkers like splicing levels at this time.