PHILADELPHIA, PA — Passage Bio, Inc. (Nasdaq: PASG) has shared updated findings from its ongoing Phase 1/2 upliFT-D clinical trial, which evaluates PBFT02 as a potential treatment for frontotemporal dementia (FTD) caused by granulin (GRN) mutations. The company also announced strategic changes aimed at advancing its PBFT02 program while optimizing operations and extending its financial runway into early 2027.
Key trial data indicates that Dose 1 of PBFT02 consistently increased cerebrospinal fluid (CSF) progranulin (PGRN) levels from baseline levels below 3 ng/mL to 22-34 ng/mL over 12 months in treated patients. Elevated PGRN levels have remained stable for up to 18 months in some patients. Additionally, the trial demonstrated a reduction in plasma neurofilament light chain (NfL), a biomarker linked to disease progression, providing potential early evidence of PBFT02’s therapeutic effect.
Safety findings remain encouraging, with most treatment-related adverse events reported as mild to moderate. Adjustments to the immunosuppression regimen have further improved safety outcomes, mitigating risks such as asymptomatic venous sinus thrombosis observed in early participants.
Looking ahead, Passage Bio is evaluating a lower dose level, Dose 2, to determine optimal dosing for future regulatory discussions. The company plans to report 12-month data for Dose 1 and interim safety results for Dose 2 in the second half of 2025. Regulatory feedback on pivotal trial design is anticipated in early 2026.
Passage Bio is also advancing its manufacturing capabilities with the completion of a high-productivity suspension-based process for PBFT02, which is expected to reduce production costs and support late-stage trials. To preserve resources, the company will transition to outsourced analytical testing and reduce workforce expenses, supporting its ability to meet critical development milestones.
The upliFT-D trial, designed to evaluate the safety and efficacy of PBFT02 for both FTD-GRN and FTD-C9orf72, is actively enrolling patients and includes plans to expand into FTD-C9orf72 populations by mid-2025.
These developments represent significant progress toward addressing the unmet needs in neurodegenerative diseases, particularly for FTD, for which no cure currently exists.
For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and MSN.