Passage BIO(PASG) - 2025 Q2 - Quarterly Results

Passage Bio Q2 2025 Financial Results and Business Highlights Business Highlights and Outlook Passage Bio advanced its upliFT-D trial for PBFT02, completing dosing for the second cohort and reporting positive interim data, with a cash runway into Q1 2027 - Completed dosing of the second FTD-GRN patient cohort (Cohort 2) in the upliFT-D study in July[1][6] - Reported updated interim data showing PBFT02 robustly increases CSF PGRN levels and reduces plasma NfL, a disease progression biomarker[1][6] - Submitted an amended clinical trial protocol to global authorities, introducing prophylactic anticoagulation and revising inclusion criteria for earlier-stage patients[1][3][6] - Anticipates key milestones including seeking regulatory feedback on manufacturing in 2H 2025, reporting updated interim safety and biomarker data from Dose 2 in 1H 2026, and seeking regulatory feedback on registrational trial design in 1H 2026[5][6] Clinical Program Overview The company's lead program, PBFT02, is an AAV1 gene therapy for FTD, evaluated in the upliFT-D trial, aiming to elevate PGRN levels - upliFT-D (NCT04747431) is a Phase 1/2 global, multi-center, open-label clinical trial of PBFT02 for FTD-GRN or FTD-C9orf72 patients aged 35 to 75[8] - The upliFT-D trial's primary endpoint is safety and tolerability of PBFT02, with secondary endpoints including disease biomarkers and clinical outcome measures[8] - PBFT02 is an AAV1 gene replacement therapy designed to deliver a functional GRN gene, elevating PGRN levels in the CNS to alter neurodegenerative disease progression[10] - Collaboration with InformedDNA provides no-cost genetic counseling and testing for FTD patients, supporting clinical trial recruitment[9] Second Quarter 2025 Financial Results Passage Bio reported a reduced net loss of $9.4 million in Q2 2025, driven by lower operating expenses, with a cash runway into Q1 2027 Q2 2025 Financial Highlights (vs. Q2 2024) | Financial Metric | Q2 2025 (Million USD) | Q2 2024 (Million USD) | Change (%) | | :--- | :--- | :--- | :--- | | Cash, Cash Equivalents & Marketable Securities | $57.6 | $91.8 | -37.2 | | R&D Expenses | $5.8 | $10.4 | -44.1 | | G&A Expenses | $4.5 | $6.5 | -30.8 | | Net Loss | ($9.4) | ($16.0) | +41.3 | | Net Loss Per Share | ($2.96) | ($5.09) | +41.8 | - Current cash, cash equivalents, and marketable securities of $57.6 million are expected to fund operations into the first quarter of 2027[1][12] - Total assets decreased to $79.2 million as of June 30, 2025, from $102.4 million as of December 31, 2024[17] - All share and per-share data reflect a 1-for-20 reverse stock split effected on July 14, 2025[12][17][18] Corporate Information Passage Bio is a clinical-stage genetic medicines company focused on developing PBFT02 for neurodegenerative diseases, with forward-looking statements subject to inherent risks - Passage Bio's mission is to develop one-time therapies for neurodegenerative diseases, targeting underlying pathology to improve patient lives[13] - PBFT02, the lead product candidate, is designed to treat neurodegenerative conditions like frontotemporal dementia by elevating progranulin levels[13] - The press release includes forward-looking statements concerning clinical trial progress, regulatory feedback, and cash runway, subject to risks detailed in SEC filings[15]