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Passage Bio (PASG) investor relations material

Passage Bio Guggenheim Securities 2nd Annual Healthcare Innovation Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Guggenheim Securities 2nd Annual Healthcare Innovation Conference summary12 Nov, 2025

Program focus and differentiation

  • Lead clinical program targets frontotemporal dementia (FTD) with a GRN mutation, with a preclinical Huntington's disease program also in development.

  • Emphasis on AAV gene therapy to increase progranulin levels intracellularly, differing from competitors that block sortilin and may cap efficacy.

  • Study design excludes more severe patients (CDR 2), focusing on early-stage (CDR 0.5–1) to maximize potential clinical benefit.

  • Recruitment has been strong due to patient engagement and growing awareness, with no significant issues reported.

  • Additional FTDC9 patients included due to preclinical evidence and low incremental cost.

Clinical and regulatory strategy

  • FDA's rare disease guidance supports potential for single-arm studies in low-prevalence, fast-progressing diseases with no available treatments.

  • Early discussions with the FDA planned for the first half of next year to align on experimental design and registration path.

  • Immunosuppression protocol stabilized after initial patient, now includes short-term steroids and low-dose Eliquis for safety.

  • Natural history data from large, prospective studies (All FTD, GenFi) will be leveraged for comparison and biomarker validation.

  • Data refresh and FDA guidance expected in the first half of next year, with timing of public data release dependent on regulatory interactions.

Scientific rationale and biomarker strategy

  • Progranulin deficit is the clear proximal cause of neurodegeneration in FTD GRN; increasing levels intracellularly is the therapeutic goal.

  • Early data show CSF progranulin levels near or above the upper limit of normal with current dosing.

  • Plasma neurofilament light chain (NFL) used as a biomarker for neurodegeneration, with observed changes aligning with normal age-related increases.

  • Disease progresses rapidly (about eight years from diagnosis to death), underscoring the need for early intervention.

  • Study aims to slow neurodegeneration, with biomarker and MRI data supporting the approach.

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Frequently asked questions

Passage Bio Inc. is a clinical-stage genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders. The company utilizes a proprietary gene therapy platform in collaboration with the University of Pennsylvania’s Gene Therapy Program to deliver adeno-associated virus (AAV)-based therapeutics directly to the CNS. Passage Bio’s pipeline includes treatments targeting conditions such as frontotemporal dementia, Krabbe disease, and other inherited neurodegenerative and neurodevelopmental disorders. The company is headquartered in Philadelphia, Pennsylvania, and its shares are listed on the NASDAQ.

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