PHILADELPHIA, PA — Passage Bio, Inc. (Nasdaq: PASG) has shared its financial results for the first quarter of 2025, along with updates on its ongoing clinical programs and operational advancements. With a focus on groundbreaking treatments for rare, monogenic central nervous system (CNS) disorders, the company detailed progress in its upliFT-D trial and improvements in its manufacturing capabilities.
Clinical Trial Advances
The upliFT-D trial, Passage Bio’s Phase 1/2 global study of PBFT02, is making strides in its quest to address frontotemporal dementia (FTD) caused by GRN and C9orf72 mutations.
The company has successfully treated its first FTD-GRN patient in Cohort 2 with a reduced dose, marking a critical milestone in the study. This reduction, representing 50% less than Dose 1, follows a robust expression of cerebrospinal fluid (CSF) progranulin (PGRN) at the initial dose. “We made steady progress in executing against our core operational objectives this quarter as we focus on the enrollment of FTD-GRN and FTD-C9orf72 patients in our ongoing global Phase 1/2 upliFT-D trial of PBFT02,” said Dr. Will Chou, president and CEO of Passage Bio. The company expects to report 12-month data from Dose 1 patients and interim biomarker and safety data from Dose 2 in the second half of 2025.
Additionally, enrollment has begun for symptomatic FTD-C9orf72 patients. Overexpression of PGRN has been shown in preclinical studies to slow neurodegeneration and reduce TDP-43 pathology, key contributors to disease progression in this subtype of FTD. Up to five patients will initially receive Dose 2 PBFT02 as part of this protocol expansion.
Breakthrough Manufacturing Development
Passage Bio also announced a breakthrough in manufacturing processes for PBFT02. Transitioning from an adherent-based to a suspension-based system, the company has scaled production to a 200-liter GMP-ready process. This innovation significantly enhances efficiency, yielding over 1,000 doses of Dose 2 with superior capsid purity and content compared to the previous process. Key data was presented this week at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting in New Orleans.
Dr. Chou noted the importance of maintaining engagement with regulatory authorities regarding these advancements. “As we turn towards the second half of the year, we look forward to delivering additional data from upliFT-D to add to our understanding of PBFT02’s safety profile, durability of progranulin expression, dose-response, and impact on plasma neurofilament levels,” he said.
Financial Overview
Passage Bio provided an in-depth overview of its financial health for Q1 2025. The company reported $63.4 million in cash, cash equivalents, and marketable securities as of March 31, 2025, with projections indicating funding sustainability through the first quarter of 2027.
Research and development expenses for the quarter totaled $7.7 million, marking a reduction from $11.5 million in the same period in 2024, reflecting tighter operational controls. General and administrative expenses were similarly reduced year-over-year, from $6.5 million in Q1 2024 to $6.1 million in Q1 2025. Overall, net losses narrowed year-over-year to $15.4 million, or $0.25 per basic and diluted share, compared to $16.7 million, or $0.30 per basic and diluted share, in Q1 2024.
A Forward-Looking Vision
Passage Bio remains firmly focused on the development and refinement of its gene therapy pipeline. The company expects the forthcoming safety and biomarker data from its clinical trials to inform regulatory discussions in early 2026, paving the way for a registrational pathway in FTD-GRN.
With robust advancements in clinical research, significant gains in manufacturing efficiency, and a solid financial runway, Passage Bio is positioned to make a considerable impact in the field of CNS disorders. The upcoming release of additional data later in 2025 will serve as a critical step in validating its therapies and advancing its mission to bring life-changing treatments to underserved patient populations.
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