PBFT02 (AAV1)

April 20, 2026 — Passage Bio (NASDAQ: PASG) reported updated interim results from its ongoing Phase 1/2 upliFT-D trial of PBFT02 in frontotemporal dementia with granulin mutations (FTD‑GRN) and provided regulatory and corporate updates. In early-stage patients (baseline global CDR=1), PBFT02 was associated with reduced brain atrophy versus ALLFTD natural history: whole-brain atrophy 3.1% at 12 months (n=2) vs 8.7% (n=7), a 64% reduction; frontotemporal cortex atrophy 4.6% (n=2) vs 9.9% (n=7), a 54% reduction. Plasma neurofilament light (NfL) stabilized at 12 months (−1.0 pg/mL, n=6) compared with an increase in untreated natural history patients (+13.5 pg/mL, n=7). CSF progranulin rose durably with Dose 1 (mean 22.8 ng/mL at 12 months; 24.2 ng/mL at 18 months) and appeared comparable with Dose 2 in early follow-up.

The FDA indicated in a Type C meeting that a randomized controlled registrational trial will be required, prompting Passage Bio to evaluate next steps. The company also initiated a strategic review (advised by Wedbush PacGrow) to maximize shareholder value.