Addie

Addie asked whether the safety profile of BMN 333 is expected to largely mirror Voxzogo, or if any meaningful differences are emerging that could differentiate its profile.

Answer

Greg Friberg, Chief R&D Officer, stated that preclinical models, particularly cynomolgus monkey models, have not demonstrated any additional safety concerns for BMN 333 over Voxzogo as a free drug. He noted that BMN 333's profile allows for much higher exposures compared to daily Voxzogo administration, opening a therapeutic window.