PHILADELPHIA, PA — Passage Bio, Inc. (NASDAQ: PASG) reported recently updated clinical data for its PBFT02 therapy in frontotemporal dementia and said it has initiated a strategic review process following feedback from the U.S. Food and Drug Administration on future trial requirements.
The company said interim results from its Phase 1/2 upliFT-D trial showed improvements in disease progression biomarkers, including reduced brain atrophy and stabilization of plasma neurofilament levels in certain patients compared with natural history data.
The data indicated that patients at earlier stages of disease progression experienced slower rates of whole brain and frontotemporal cortex atrophy, while patients with more advanced disease did not show similar benefits.
PBFT02-treated patients with moderate disease showed a 64% reduction in whole brain atrophy and a 54% reduction in frontotemporal cortex atrophy at 12 months compared with untreated patients in comparable cohorts.
Plasma neurofilament levels remained stable in treated patients, while untreated patients showed increases over the same period.
The company also reported increased levels of progranulin, the target protein, in cerebrospinal fluid following treatment, with sustained expression observed through 18 months.
PBFT02 was generally well tolerated, with no new treatment-related serious adverse events reported.
Passage Bio said the FDA advised that a randomized controlled trial would be required for potential approval of PBFT02, rather than a single-arm study.
Chief Executive Officer Will Chou said the company is evaluating next steps for the program following the regulatory feedback.
The company also said it has engaged Wedbush PacGrow as a financial advisor and is reviewing strategic alternatives, including potential mergers, asset sales, or partnerships.
Passage Bio develops genetic therapies for neurodegenerative diseases, including frontotemporal dementia.
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