Passage Bio Announces Positive Feedback from FDA on Expansion of upliFT-D Trial of PBFT02 to Include FTD-C9orf72 Patients

Agency feedback from Type C meeting process supports the company’s proposed clinical development plans to expand the upliFT-D trial to assess PBFT02 in FTD patients with C9orf72 gene mutations

Plan to initiate dosing of FTD-C9orf72 patients in 1H 2025

Excerpt from the Press Release:

PHILADELPHIA, July 16, 2024 (GLOBE NEWSWIRE) — Passage Bio, Inc. (NASDAQ: PASG), a clinical-stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today announced that the company received positive feedback in its Type C meeting process with the U.S. Food and Drug Administration (FDA) on its proposal to evaluate PBFT02 for treating frontotemporal dementia (FTD) patients with mutations in the C9orf72 gene. Compelling preclinical evidence, coupled with available safety and robust progranulin (PGRN) expression data from the first cohort of FTD-GRN patients treated with PBFT02, supported FDA alignment on the proposed trial expansion. The company intends to amend the protocol for the ongoing upliFT-D Phase 1/2 global study for FTD-GRN to introduce a new population of FTD-C9orf72 patients.

“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 is estimated to affect approximately 21,000 individuals in the United States and Europe. Similar to FTD-GRN, FTD-C9orf72 is marked by TDP-43 pathology, where the transactive response DNA binding protein 43 (TDP-43) abnormally accumulates in the cytoplasm of neurons, leading to neuronal dysfunction and degeneration. Third-party preclinical studies have demonstrated that increasing PGRN levels can reduce TDP-43 pathology and slow neurodegeneration.

The company plans to submit the revised trial protocol to health authorities and ethics committees promptly and expects to initiate dosing of FTD-C9orf72 patients in 1H 2025.

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