PHILADELPHIA, PA — Passage Bio, Inc. (NASDAQ: PASG) announced its 2024 financial results, highlighting progress in its PBFT02 gene therapy program for frontotemporal dementia (FTD) associated with GRN mutations (FTD-GRN) and other neurodegenerative diseases.
Key Clinical Milestones
Interim data from the Phase 1/2 upliFT-D trial demonstrated PBFT02’s potential. A single dose of PBFT02 increased cerebrospinal fluid (CSF) progranulin (PGRN) levels significantly, with patients showing sustained elevations at six and twelve months. Additionally, early evidence suggested a reduction in plasma neurofilament light chain (NfL), a biomarker of disease progression.
Building on this success, Passage Bio has initiated Dose 2, a lower dosage group set at 50% of Dose 1. The first patient in this cohort has been enrolled, with further recruitment underway across trial sites in Brazil, Canada, Portugal, and the United States. The company expects to report comprehensive 12-month Dose 1 data and interim safety and biomarker results from Dose 2 in the second half of 2025.
Passage Bio also plans to expand the trial to include patients with FTD linked to C9orf72 gene mutations by initiating dosing in this subgroup during the first half of 2025.
Extending Financial Runway
The company ended 2024 with cash and equivalents totaling $76.8 million, a decrease from $114.3 million at year-end 2023. However, initiatives such as transitioning to an outsourced analytical testing model have extended the cash runway into the first quarter of 2027.
Research and development expenses for the year totaled $40.2 million, down from $61.4 million in 2023, while general and administrative expenses decreased to $25.0 million from $41.6 million. These cost reductions contributed to a narrowed net loss of $64.8 million for 2024, compared to $102.1 million in 2023.
Innovative Gene Therapy Approach
PBFT02 utilizes an AAV1 viral vector to deliver the GRN gene directly into the central nervous system via intracisternal magna (ICM) administration. This technique aims to increase PGRN levels, offering a potential one-time treatment for FTD-GRN. Preclinical studies have shown promising results, including improved lysosomal function and reduced neuroinflammation in animal models.
Passage Bio’s efforts to support trial recruitment include partnerships for genetic counseling and testing, enhancing accessibility for eligible patients.
With its clinical advancements and sustained financial strategy, Passage Bio is well-positioned to address unmet needs in neurodegenerative diseases and build on its progress in 2025.
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