Passage Bio (PASG) Corporate presentation summary

Strategic focus and pipeline

• Advancing a clinical-stage, one-time gene therapy (PBFT02) for frontotemporal dementia (FTD) with GRN mutations, showing potential best-in-class efficacy.

• Pursuing preclinical development of a gene therapy targeting Huntington's disease by reducing MSH3 to limit CAG repeat expansion.

• Pipeline also includes programs for FTD-C9orf72 and potential expansion into ALS and Alzheimer's disease.

• Cash runway is projected through Q1 2027, with a $46 million balance as of December 31, 2025.

PBFT02 clinical and preclinical data

• PBFT02 uses AAV1 vector delivered via intra-cisterna magna (ICM) for broad CNS distribution and durable elevation of CSF progranulin (PGRN).

• Interim clinical data show robust, sustained increases in CSF PGRN in FTD-GRN patients, with levels reaching or exceeding the healthy adult range.

• Early evidence of reduced plasma neurofilament light chain (NFL) suggests slowed disease progression compared to natural history.

• Preclinical studies in mice and non-human primates demonstrated improved lysosomal function, reduced neuroinflammation, and broad vector biodistribution.

Safety and competitive positioning

• PBFT02 was generally well tolerated; all serious adverse events at Dose 1 were asymptomatic and resolved, with no complications at Dose 2.

• PBFT02 offers a one-time, non-surgical treatment with higher and more durable CSF PGRN levels than competitors.

• Manufacturing milestones include FDA alignment on potency assays and a high-yield process capable of producing over 1,000 doses per lot.