John Wang's questions to BioMarin Pharmaceutical Inc (BMRN) leadership • Q3 2024
Question
An analyst from Barclays, identified as John Wang, questioned the potential for BMN 333 to achieve superior efficacy, given that Voxzogo's dose-escalation studies suggested a ceiling effect. He also asked about the importance of dosing frequency in the new drug's profile.
Answer
EVP, Worldwide R&D Greg Freiberg clarified that BMN 333 aims to alter the PK curve shape, not just the peak concentration, to increase the time above a therapeutic threshold and total AUC for free CNP. He compared it to other peptides like insulin where different constructs yield different biologic effects, and noted this hypothesis will be tested in the clinic early next year.