Passage Bio, Inc. · NASDAQ:PASG
Passage Bio — 39th Annual J.P. Morgan Healthcare Conference (January 2021)
33 slides from Passage Bio's conference presentation, Jan 11, 2021
Slide 1: 39th Annual J.P. Morgan Healthcare Conference
39th Annual J.P. Morgan Healthcare Conference Bruce Goldsmith, PhD | CEO January 11, 2021 NASDAQ GS: PASG Fulfilling the Promise of Gene Therapies for Central Nervous System Disorders
Slide 2: Forward-Looking Statement
Forward - Looking Statement This presentation includes “forward - looking statements” within the meaning of, and made pursuant to the safe harbor provisions o f, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectation about timing and execution of antici
Slide 3: Our vision: gene therapies for rare monogenic CNS diseases
~7,000 Rare diseases OUR VISION: To fulfill the promise of gene therapy by developing groundbreaking therapies that transform the lives of patients with rare monogenic CNS diseases 70% of rare genetic diseases produce abnormalities of the CNS 1 790+ are rare monogenic CNS diseases with few treatment
Slide 4: Patients with rare CNS disorders need therapeutic options
Patients with rare CNS disorders need therapeutic options.
Slide 5: The Passage Bio Advantage
The Passage Bio Advantage Corporate model designed for success 5 PENN GTP PARTNERSHIP Led by renowned innovator James M. Wilson, MD, PhD Access to cutting - edge research and development Access to next - generation technologies BROAD AND ROBUST PIPELINE 17 total program license options 3 clinical -
Slide 6: Passage Bio's Differentiated Path for Clinical Success
Passage Bio’s Differentiated Path for Clinical Success 17 program license options from GTP focused on rare monogenic CNS disorders 6 GTP expertise and insights into research and development Optimization of delivery approaches Disease - specific existing and novel capsid selection Integration of next
Slide 7: A Broad and Robust Pipeline with Global Rights
PROGRAM 1 / INDICATION GENE DISCOVERY CANDIDATE SELECTION IND - ENABLING PHASE 1/2 PIVOTAL PEDIATRIC PB GM01 2 Infantile GM1 Gangliosidosis GLB1 PB KR03 Krabbe GALC PBML04 Metachromatic Leukodystrophy ARSA PBCM06 Charcot - Marie - Tooth Type 2A MFN2 ADULT PB FT02 Frontotemporal Dementia - GRN GRN PB
Slide 8: PBGM01 Infantile GM1 Gangliosidosis
PBGM01 Infantile GM1 Gangliosidosis
Slide 9: GM1 Gangliosidosis: A Devastating Pediatric Disease
GM1 Gangliosidosis A D evastating Pediatric Disease 9 FATAL, PEDIATRIC NEUROLOGICAL LYSOSOMAL STORAGE DISORDER caused by GLB1 gene mutations characterized by destruction of neurons in the brain and spinal cord. Characterized by rapidly progressive neurological decline resulting in reduced muscle ton
Slide 10: PBGM01: A Potential Transformative Therapy
PBGM01 A Potential Transformative Therapy For a Rare, Underserved Disorder ▪ Next - generation, proprietary AAVhu68 capsid delivers functional GLB1 gene encoding β - gal to the brain and peripheral tissues ▪ Meaningful transduction of both central nervous system and critical peripheral organs in pre
Slide 11: PBGM01 Supportive Preclinical Findings
11 PBGM01 Supportive Preclinical Findings Improvement in biomarker and pathophysiology in knock - out mouse model p<0.05, p<0.01, NS=not significant. GLB1 +/ - + PBS GLB1 - / - + PBS GLB1 - / - + AAV 10,000 10 1 10.1 CSF 100 1,000 ß - gal activity ( nmol /mL/h) GLB1 +/ - + PBS GLB1 - / - + PBS GLB1
Slide 12: PBGM01 Dose-Related Effect on Survival
Dose - Related Effect on Survival GLB1 - / - (KO) GLB1 +/ - (HET) Dose 4 (highest) Dose 3 Dose 2 Dose 1 (lowest) Vehicle Vehicle Percent Survival 0 Day 100 200 300 0 50 100 Preservation of Neurological Function 0 5 10 15 20 Total score ( + SEM) Days 240 180 120 60 0 Neurological Examinations GLB1 –
Slide 13: Pathway to Patient Identification and Trial Recruitment
Pathway to Patient Identification and Trial Recruitment 13 Newborn Screening / Diagnosis Patient Recruitment and Advocacy Strategies Patient and Caregiver Activities Sponsoring ScreenPlus Pilot Program ▪ GM1 has been added to the New York newborn screening pilot program led by Dr. Melissa Wasserstei
Slide 14: Goal of the Imagine-1 Trial in GM1
Adapted from Regier DS, et al. 2016 GLB - 1 Disorders, In Adam MP, et al. GeneReviews. Goal of the Imagine - 1 Trial in Early and Late Infantile GM1 Elevate β - gal activity to preserve neurological function and improve developmental potential and survival CLINICAL TRIAL OBJECTIVES 1. Assess the saf
Slide 15: Imagine Global Phase 1/2 Trial with PBGM01
Imagine - 1 Global Phase 1/2 Trial with PBGM01 First patient enrollment expected in 1Q21 15 Trial Design Phase 1/2, multi - center, open - label, single - arm, 2+2 dose escalation and confirmatory study Separate cohorts of pediatric subjects with Late Onset Infantile GM1 in cohorts 1 and 2 and Early
Slide 16: PBKR03 Krabbe Disease
"4 PBKRO3 . Krabbe Disease
Slide 17: Krabbe Lysosomal Storage Disease: PBKR03
Krabbe Lysosomal Storage Disease PBKR03 — A potential transformative therapy for a rare, underserved disorder 17 Source: NIH, CHOP, American Journal of Neuroradiology, Third Party Research Severe form of disease ▪ Infantile Krabbe disease is the most severe form, accounting for 60% to 70% of diagnos
Slide 18: PBKR03 Disease Model Data – Twitcher Mouse and Dog Models
PBKR03 Disease Model Data – Twitcher Mouse and Dog Models Meaningful brain and peripheral transduction with improved pathophysiology and function 18 GALC Increase in Brain and Periphery Twitcher Mouse Model: • Increased GALC activity in brain, liver and serum • AAV treated twitcher mouse showed clin
Slide 19: PBKR03 Patient Enrollment Expected in 1H 2021
Naturally occurring Krabbe Dog model study showed substantial benefit with AAV treatment, including full phenotypic recovery and increased survival 0 20 40 60 80 K948 K930 K938 K939 K937 K933 Weeks PBKR03 Patient Enrollment Expected in 1H 2021 Supported by extensive preclinical studies 19 TRIAL DESI
Slide 20: PBFT02 Frontotemporal Dementia - GRN
PBFT02 Frontotemporal Dementia - GRN 20
Slide 21: FTD-GRN: Frontotemporal Dementia Caused by Progranulin Deficiency
FTD - GRN Frontotemporal Dementia Caused by a Progranulin Deficiency PBFT02 – A potential transformative therapy for a rare, underserved disorder 21 Devasting form of dementia ▪ FTD is one of the more common causes of early - onset dementia ▪ Approximately 5 - 10% of FTD is caused by a GRN gene muta
Slide 22: PBFT02 Biomarker Improvement Across Brain Regions
PBFT02 Biomarker Improvement Across Brain Regions Decreases markers of lysosomal function and microgliosis *** * PBFT02 high dose vs vehicle in GRN - / - mice; post baseline data is 90 days post - dose * = p< 0.05*** = p<0.001 0% 20% 40% 60% 80% 100% 120% Baseline Vehicle PBFT02 0 0.2 0.4 0.6 0.8 1
Slide 23: PBFT02 Patient Enrollment Expected in 1H 2021
PBFT02 Patient Enrollment Expected in 1H 2021 Supported by extensive NHP preclinical studies utilizing ICM delivery 23 Phase 1/2 study summary PBFT02 Showed D ose - Related Increase in CSF PGRN at 14d PD 0 2 4 6 8 10 12 High Dose Medium Dose Low Dose Vehicle Human PGRN (ng/ml) Production of Human PG
Slide 24: State-of-the-Art Manufacturing
State - of - the - Art Manufacturing
Slide 25: Manufacturing That is Dedicated, Flexible, and Scalable
Manufacturing That is Dedicated, Flexible, and Scalable 25 CMC Infrastructure Capability Investment in CMC laboratory with state - of - the - art analytical capabilities, clinical assay development and validation, biomarker assay validation, and clinical product testing End - to - End Supply Chain E
Slide 26: Corporate Foundation
Corporate Foundation 26 SOURCE: www.brandywinerealty.com
Slide 27: Financial Summary
Financial Summary 27 Strong cash balance of $335M at September 30, 2020 Cash on hand to fund operations into 2023 Runway to potentially deliver meaningful catalysts over multiple programs
Slide 28: Anticipated Upcoming Milestones
Anticipated Upcoming Milestones 28 Phase 1/2 trial for PBGM01 in patients with infantile GM1 1Q21 INITIATE Phase 1/2 trial for PBFT02 in patients with FTD - GRN in 1H21 INITIATE Advance preclinical programs for MLD, ALS CMT2A ADVANCE Evaluate and expand pipeline by pursuing new licenses in partnersh
Slide 29: The Passage Bio Advantage
The Passage Bio Advantage Corporate model designed for success 29 PENN GTP PARTNERSHIP Led by renowned innovator James M. Wilson, MD, PhD Access to cutting - edge research and development Access to next - generation technologies BROAD AND ROBUST PIPELINE 17 total program license options 3 clinical -
Slide 30: Thank You
www.passagebio.com NASDAQ GS: PASG THANK YOU 30
Slide 31: Appendix
W PassageBio Appendix
Slide 32: Demonstrated Leadership
Demonstrated Leadership Deep experience in rare disease, CNS disorders and gene therapy 32 LEADERSHIP TEAM BOARD OF DIRECTORS Tachi Yamada, M.D. (Chair) Frazier Athena Countouriotis, M.D. Turning Point Bruce Goldsmith, Ph.D. Passage Bio Patrick Heron Frazier Saqib Islam, J.D. SpringWorks Sandip Kapa
Slide 33: Cutting-Edge Gene Therapy R&D Collaboration
Cutting - Edge Gene Therapy R&D Collaboration 17 program license options for Passage Bio focused on rare monogenic CNS disorders 33 Founded by renowned innovator and pioneer James M. Wilson, M.D., Ph.D. Chief Scientific Advisor, Passage Bio Director, Gene Therapy Program Rose H. Weiss Professor and
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