Passage Bio (PASG) Corporate presentation summary

Therapeutic focus and pipeline

• Developing gene therapies for neurodegenerative diseases, targeting FTD-GRN, FTD-C9orf72, and Huntington's disease.

• PBFT02 is a clinical-stage, one-time gene therapy for FTD-GRN, with differentiated approaches for Huntington's disease in preclinical development.

• Pipeline expansion opportunities include ALS and Alzheimer's disease.

• $33.3M in cash resources as of March 31, 2026.

PBFT02 clinical and preclinical data

• PBFT02 uses AAV1 vector for direct CSF delivery, achieving robust and durable increases in CSF progranulin levels in FTD-GRN patients.

• Interim data show 64% reduction in whole brain atrophy and 54% reduction in frontotemporal cortex atrophy at 12 months for CDR 1 patients.

• Plasma NfL biomarker remained stable at 12 months, contrasting with increases in natural history cohorts.

• PBFT02 was generally well tolerated, with all serious adverse events asymptomatic and no evidence of DRG toxicity or complications from the procedure.

• Preclinical models demonstrated PBFT02's ability to ameliorate TDP-43 pathology and improve lysosomal function and neuroinflammation.

Market opportunity and disease background

• Estimated prevalence: 18,000 FTD-GRN, 21,000 FTD-C9orf72, and 70,000 Huntington's disease patients in US/EU.

• FTD is a fatal, rapidly progressive neurodegenerative disease with no approved disease-modifying therapies.

• Progranulin deficiency is central to FTD-GRN pathology, leading to lysosomal dysfunction and neurodegeneration.

• Huntington's disease is caused by CAG repeat expansion in the HTT gene, leading to mutant huntingtin protein and progressive neurological decline.