PBFT02 shows robust CSF progranulin increase; well-tolerated with enhanced immunosuppression

Passage BIO, Inc.

CSF PGRN up to 6-fold at month 1 (10.7-17.3 ng/mL) and up to 10-fold at month 6 (21.7-27.3 ng/mL), exceeding healthy controls (3.3-8.2 ng/mL).
No serious adverse events in patients 2-5 with enhanced immunosuppression; first patient had SAEs with lower steroid regimen.
CSF PGRN remained elevated at 12 months (34.2 ng/mL), with rate of increase slowing from 58% to 26% between months 1-6 and 6-12.
Cohort 2 currently enrolling; Cohort 4 anticipated to open in first half of 2025 for FTD-C9orf72.
Plasma PGRN expression remained below healthy reference levels across all five patients.

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