Core Insights - Passage Bio, Inc. reported strong performance in 2024, advancing its PBFT02 program with promising data in FTD-GRN patients, showing robust and durable progranulin expression and early evidence of improvement in a disease progression biomarker [3][6] - The company has extended its cash runway into Q1 2027 by implementing cost-reduction measures and moving to an outsourced analytical testing model [6][15] - Upcoming milestones include reporting 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 in 2H 2025, and seeking regulatory feedback on the registrational trial design in 1H 2026 [6][5] Clinical Trial Updates - Interim data from the upliFT-D clinical trial demonstrated that Dose 1 PBFT02 increased cerebrospinal fluid (CSF) progranulin (PGRN) levels significantly, from below 3 ng/mL at baseline to 13-27 ng/mL at six months and 22-34 ng/mL at 12 months [6] - Plasma neurofilament light chain (NfL) levels were found to be 13% lower than baseline on average at 12 months post-treatment, contrasting with an expected increase of 29% per year based on natural history data [6] - The first FTD-GRN patient has been enrolled to receive Dose 2 PBFT02, which is 50% lower than Dose 1, to facilitate discussions with health authorities regarding a registrational study design [6][3] Financial Performance - As of December 31, 2024, the company reported cash, cash equivalents, and marketable securities totaling 114.3 million as of December 31, 2023 [15] - Research and Development (R&D) expenses for Q4 2024 were 40.2 million, compared to 61.4 million for the same periods in 2023 [15] - The net loss for Q4 2024 was 0.20 per share, and for the full year, it was 1.07 per share, showing a decrease in losses compared to the previous year [15][21]
Passage Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Recent Business Highlights