FY2Q Mar '26 Reports: Takeaways From Our Management Call

研报英文原文证据摘录

FY2Q Mar '26 Reports: Takeaways From Our Management Call

C O M PA N Y N O T E

M a y 1 9 , 2 0 2 6

Regulatory discussions continue to progress steadily following DRUG's positive DEE

data with base case expectation to need two late-stage studies. As a reminder, DRUG read

out impressive results on motor seizure reductions at BMB-101's Ph2 BREAKTHROUGH topline

in January 2026, which served as a key clearing event to advance into FDA discussions on a

pivotal program. As such, mgmt shared that the team has had some informal dialogue with the

agency on potential next steps for the program, where the pre-IND meeting is being worked on

now. Accordingly, DRUG is in full preparation mode where the IND will be a written response. In

fact, the final gating factor before the IND can be finalized is a juvenile toxicology study that has

already completed dosing and has final procedures ongoing. Once completed, DRUG plans to

submit filings in various jurisdictions like EU and Australia to prepare for pivotal DEE program

kick off. Although the final Ph3 DEE program still needs to be aligned upon with the FDA, mgmt

expects to run two studies aligned closely to the design of Ph2 BREAKTHROUGH. That said,

one potential change mgmt expects to propose is a cap on concomitant medications for this

patient population. Nonetheless, we expect to learn more about finalized Ph3 DEE plans for

DRUG after further regulatory interactions take place.

Agency discussions surrounding BMB-101 development in absence epilepsy is also

ongoing where mgmt believes EEG will be the primary endpoint of a future pivotal

study. Additionally, mgmt noted that the regulatory discussions regarding BMB-101 in absence

epilepsy are also ongoing following the positive topline data in Ph2a BREAKTHROUGH. Notably,