BMN 333 Grows Into Its Own At ENDO

Research evidence excerpt

BMN 333 Grows Into Its Own At ENDO

June 16, 2026

What we know about the phase 2/3. The ongoing study is a multi-regional phase 2/3 study comparing 3 doses of

BMN 333 against Voxzogo in children with achondroplasia (ACH). The phase 2 dose selection portion will evaluate

three doses of BMN 333 (low, medium, and high) against Voxzogo and determine study continuation into the phase 3

portion which will compare the selected dose against Voxzogo. In our conversations with management, they stressed

that every month matters when it comes to getting BMN 333 to market faster considering the expected competitive

landscape. With this and the knowledge that the phase 2 portion must be run in a treatment naive patient population,

BMRN believes its country mix and number of treatment sites can support expedited recruitment. With reference to

BMRN's 1Q26 call, we anticipate the phase 2 data, expected in 2027, to report annualized growth velocity (AGV) at

6-months as well as anthropomorphic, safety, and wellbeing measures. Results from the phase 2 portion of the study

will be used to select which dose will move forward into the phase 3 portion which, again, will be compared against

a Voxzogo control arm. Here, the company has stated that it will be looking for AGV superiority with the thesis that

increased AGV will translate to better improvements in health and wellness measures. Perhaps most importantly, while

BMRN has committed to a 2030 approval, the company has indicated that it intends to pull all available levers, including

using technology and innovation to get BMN 333 to market before 2030.

Three-year phase 2 Voxzogo data in HCH. On Voxzogo, three-year extension data from the phase 2 investigator-