Core Insights - Passage Bio, Inc. reported updated data from the ongoing Phase 1/2 upliFT-D clinical trial for PBFT02, a gene replacement therapy targeting frontotemporal dementia (FTD) with granulin (GRN) mutations, showing promising results in increasing CSF PGRN levels and early signs of reduced plasma NfL levels, a biomarker for disease progression [1][2][4] Clinical Trial Updates - The company is evaluating Dose 2 of PBFT02, which is 50% lower than Dose 1, in FTD-GRN and FTD-C9orf72 patients to explore dosing options and support regulatory strategy [1][4] - Interim data from FTD-GRN patients treated with Dose 1 showed CSF PGRN levels increased from below 3 ng/mL at baseline to 13-27 ng/mL at six months and 22-34 ng/mL at 12 months, with plasma NfL levels averaging 13% lower than baseline at 12 months [4][8] - The company plans to report 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 in the second half of 2025 and seek regulatory feedback on the pivotal trial design in the first half of 2026 [1][6] Manufacturing and Financial Strategy - Passage Bio completed the development and scale-up of a high-productivity, suspension-based manufacturing process for PBFT02, which is expected to improve yield and reduce costs [1][4] - The company extended its cash runway into the first quarter of 2027 by transitioning to an outsourced analytical testing model and reducing operating expenses, including a workforce reduction of approximately 55% [1][4][8] Future Plans - The company is on track to initiate dosing of FTD-C9orf72 patients in the first half of 2025, with an estimated 21,000 patients affected in the U.S. and Europe, and no approved disease-modifying therapies currently available [6][8] - The upliFT-D trial is a global, multi-center, open-label study evaluating the safety and efficacy of PBFT02, with a primary endpoint focused on safety and tolerability [9][10]
Passage Bio Announces Interim Data from upliFT-D Study in FTD-GRN and Provides Business Updates