Passage Bio reports promising Phase 1/2 data for PBFT02 in FTD-GRN; shifts strategy to adult neurodegenerative diseases

Passage BIO, Inc.

Dose 1 of PBFT02 achieved 3.6-6.6-fold increase in CSF PGRN at day 30, reaching supraphysiologic levels of 10.7-17.3 ng/mL.
PBFT02 was generally well-tolerated with enhanced steroid regimen; no serious AEs in patients 2 and 3.
Company to focus on PBFT02 in FTD-GRN, FTD-C9orf72, ALS, and Alzheimer's; seeking partnerships for pediatric programs.
Expects to initiate dosing of Cohort 2 in 1H 2024 and report full 6-month data from Cohort 1 in 2H 2024.
Estimates FTD-GRN prevalence at ~18,000 patients in US and Europe; exploring PBFT02 in larger indications.

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