PHILADELPHIA, PA — Passage Bio, Inc. (Nasdaq: PASG) announced second-quarter 2025 results and reported key advancements in its upliFT-D clinical trial, including completion of dosing in the second cohort of patients with frontotemporal dementia due to progranulin gene mutations (FTD-GRN).
The company said interim data from the study continued to show that its experimental gene therapy, PBFT02, produced sustained increases in cerebrospinal fluid progranulin (PGRN) levels and improvement in plasma neurofilament light chain (NfL), a biomarker of disease progression, compared with natural history benchmarks. Updated results were also presented in July at the Alzheimer’s Association International Conference in Toronto.
In Cohort 2, which included four patients split between two dosing levels, the first patient to receive the lower dose (50% of Dose 1) achieved a substantial PGRN increase one month after treatment, nearing the healthy adult reference range. Patients in Dose 1 maintained elevated PGRN levels for up to 18 months and showed slower NfL increases than typically seen in the disease.
Passage Bio has submitted an amended upliFT-D protocol to global trial sites and regulators. The revisions introduce short-term, low-dose anticoagulation, broaden eligibility to include patients with mild cognitive impairment, and exclude those with advanced disease. Once approved, enrollment will begin for Cohort 3 (FTD-GRN) and Cohort 4 (FTD-C9orf72), both evaluating Dose 2.
CEO Will Chou said the data reinforced PBFT02’s potential as a differentiated therapy for the FTD-GRN community, noting the company remains on track to seek regulatory guidance on a registrational pathway in the first half of 2026.
Financially, Passage Bio ended the quarter with $57.6 million in cash, cash equivalents, and marketable securities, projecting its runway into the first quarter of 2027. R&D expenses fell to $5.8 million from $10.4 million a year earlier, while G&A costs declined to $4.5 million from $6.5 million. Net loss narrowed to $9.4 million, or $2.96 per share (post reverse-split), from $16.0 million, or $5.09 per share, in the prior-year quarter.
PBFT02, delivered via a single injection into the cisterna magna, uses an AAV1 vector to insert a functional GRN gene into the central nervous system. Preclinical data in animal models suggest the therapy can restore lysosomal function, reduce neuroinflammation, and broadly distribute the vector across brain tissue.
The upliFT-D trial is a Phase 1/2, global, open-label study enrolling adults aged 35 to 75 with FTD-GRN or FTD-C9orf72. Primary endpoints focus on safety and tolerability, with secondary measures evaluating biomarkers and clinical outcomes.
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