PHILADELPHIA, PA — Passage Bio, Inc. (NASDAQ: PASG) announced positive feedback from the U.S. Food and Drug Administration (FDA) on its proposal to evaluate PBFT02 for treating frontotemporal dementia (FTD) in patients with C9orf72 gene mutations. The FDA’s alignment on the proposed trial expansion is backed by compelling preclinical evidence and promising safety and progranulin (PGRN) expression data from the first cohort of FTD-GRN patients treated with PBFT02.
“Securing FDA alignment on amending our upliFT-D protocol to include FTD-C9orf72 patients is a critical milestone in expanding access to those who could benefit from PBFT02,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “Given the limited clinical trials for FTD-C9orf72 patients, we believe PBFT02 could fill a significant unmet need and bring new hope to this underserved patient community. We look forward to further advancing the clinical development of PBFT02 and building upon the encouraging data we’ve seen from the upliFT-D trial so far.”
FTD-C9orf72 affects about 21,000 individuals in the United States and Europe. Similar to FTD-GRN, FTD-C9orf72 is characterized by the abnormal accumulation of the transactive response DNA binding protein 43 (TDP-43) in the cytoplasm of neurons, leading to neuronal dysfunction and degeneration. Studies have shown that increasing PGRN levels can reduce TDP-43 pathology and slow neurodegeneration.
Passage Bio plans to submit the revised trial protocol to health authorities and ethics committees promptly, aiming to start dosing FTD-C9orf72 patients in the first half of 2025.
The FDA has granted PBFT02 Fast Track and Orphan Drug designations, and the European Commission has also awarded it an Orphan designation. This regulatory support highlights the potential of PBFT02 to address significant unmet needs in the treatment of FTD.
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