PBFT02 Phase 1/2 data: durable PGRN boost; new pulmonary embolism SAE, protocol to add anticoagulation

Passage BIO, Inc.

Dose 1 PBFT02 increased CSF PGRN from <3 ng/mL to mean 25.9 ng/mL at 12 months (n=4); Dose 2 patient rose to 7.6 ng/mL at 1 month.
Plasma NfL increased only 4% in treated patients vs expected 28-29% per year in natural history, suggesting slowed disease progression.
Three of eight patients had four SAEs, including new pulmonary embolism in Dose 2 patient (Patient 8) possibly related; responded to anticoagulants.
Company plans to amend upliFT-D protocol to add prophylactic low-dose anticoagulation and restrict enrollment to earlier-stage patients; Patient 9 may proceed with extra monitoring.
Milestones: submit amendment July 2025; report Dose 2 interim data 1H 2026; seek regulatory feedback on registrational trial design 1H 2026.

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