Earning Date, Transcript Summary & Report

Positive Point: Passage Bio is in a strong position at the end of 2022, with a focus on delivering meaningful clinical data from their two lead programs in 2023. This is supported by the completion of dosing of the first four cohorts in the Imagine-1 clinical trial for GM1 gangliosidosis and the presentation of additional data at the WORLDSymposium.

• The company's PBGM01 administration is well tolerated, has a positive safety profile, and exerts a biological effect across all treated infantile GM1 patients.
• Planning to treat additional patients at a higher dose and revising study inclusion criteria to maximize the benefit-risk profile of PBGM01.
• Passage Bio is leveraging its strong in-house analytical capabilities at its CMC lab to support ongoing and future clinical development programs.
• The strong balance sheet provides Passage Bio with the necessary runway to fund operations into the first half of 2025.

Positive Point: Passage Bio's lead program, PBGM01 for GM1 Gangliosidosis, demonstrates promising interim results with a favorable safety profile, dose-dependent increases in CSF beta-galactosidase enzyme activity, and dose-dependent decreases in CSF GM1 ganglioside levels.

• Patients with lower developmental delay at dosing experienced a better response to treatment on developmental scales.
• PBGM01 administration was associated with the stabilization of MRI severity scores in all treated patients, demonstrating potential treatment effects on brain volume and white matter integrity.
• Plans to treat additional patients at a higher dose and revise study inclusion criteria to inform dose and patient selection, indicating a commitment to optimizing the therapeutic benefit of PBGM01.

Positive Point: The upliFT-D trial for frontotemporal dementia (FTD) shows progress in driving patient identification and recruitment initiatives, with expectations to report initial safety and biomarker data from Cohort 1 patients in the second half of 2023.

• Utilizing the AAV1 capsid to deliver a functional copy of the granulin gene via intracisternal magnum (ICM) delivery to overcome the deficiency in granulin gene mutation carriers.
• Focused on activating additional clinical trial sites globally and progressing with momentum to generate meaningful clinical data from the FTD program.
• The streamlining of operations and prioritization of research efforts contribute to managing cash burn judiciously as the company focuses on program execution.

Negative: Decrease in cash equivalents and marketable securities from $315.8 million in 2021 to $189.6 million in 2022. Negative: Rise in R&D expenses from $117.7 million in 2021 to $86.1 million in 2022. Negative: Increase in G&A expenses from $60.1 million in 2021 to $49.3 million in 2022. Negative: Net loss of $136.1 million for the year ended December 31, 2022, compared to $185.4 million in 2021.