Passage Bio, Inc. · NASDAQ:PASG
Passage Bio — Investor Presentation (May 2021)
36 slides from Passage Bio's investor presentation, May 5, 2021
Slide 1: Corporate Presentation May 2021
Corporate Presentation May 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 of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectation about timing and execution of anticipated
Slide 3: Rare CNS diseases and Passage Bio vision
~7,000 Rare diseases 70% of rare genetic diseases produce abnormalities of the CNS1 790+ are rare monogenic CNS diseases with few treatment options2 Passage Bio is currently targeting rare CNS disorders that affect: INFANTS ADULTS 1. Lee, C., Singleton, K., Wallin, M., Faundez, V. (2020). Rare Genet
Slide 4: The Passage Bio Advantage
The Passage Bio Advantage Corporate model designed for success 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-stage th
Slide 5: 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 GTP expertise and insights into R&D Optimization of delivery approaches Disease-specific existing and novel capsid selection Integration of next-generation research adva
Slide 6: A Broad and Robust Pipeline with Global Rights
PROGRAM1 GENE DISCOVERY CANDIDATE SELECTION IND-ENABLING PHASE 1/2 PIVOTAL PEDIATRIC PBGM012 GM1 Gangliosidosis GLB1 PBKR03 Krabbe GALC PBML04 Metachromatic Leukodystrophy ARSA PBCM06 Charcot-Marie-Tooth Type 2A MFN2 ADULT PBFT02 Frontotemporal Dementia-GRN GRN PBAL05 Amyotrophic Lateral Sclerosis C
Slide 7: Intra-Cisterna Magna (ICM) Administration
Intra-Cisterna Magna (ICM) Administration Utilized across lead programs to directly target CNS ▪ Directly deliver vector into the CSF via a single injection to reach both CNS and peripheral tissues - Allows for broad CNS biodistribution - Lower doses compared to IV systemic delivery - Reduced impact
Slide 8: Pathway to Patient Identification and Trial Recruitment
Pathway to Patient Identification and Trial Recruitment Screening / Diagnosis Patient Recruitment and Advocacy Strategies Patient and Caregiver Activities Sponsoring ScreenPlus Pilot Program ▪ GM1 included in the New York newborn screening pilot program led by Dr. Melissa Wasserstein Supporting Invi
Slide 9: PBGM01 GM1 Gangliosidosis
" PBGMO1 GM1 Gangliosidosis ‘Passage Bio
Slide 10: GM1 Gangliosidosis A Devastating Pediatric Disease
GM1 Gangliosidosis A Devastating Pediatric Disease 10 Source: NIH, CHOP, American Journal of Neuroradiology 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 progressiv
Slide 11: PBGM01 Potential Transformative Therapy
PBGM01 Potential Transformative Therapy For Rare, Underserved Disorder ▪ Next-generation, proprietary AAVhu68 capsid delivers functional GLB1 gene encoding β-gal to the brain and peripheral tissues ▪ Compelling preclinical data showing meaningful transduction of both central nervous system and criti
Slide 12: PBGM01 Supportive Preclinical Findings
PBGM01 Supportive Preclinical Findings Improvement in biomarker and pathophysiology in knock-out mouse model Stable, dose-dependent increases in β-gal enzyme activity in brain, cerebrospinal fluid, serum, and critical peripheral tissues Dose-dependent reduction of brain lysosomal storage lesions as
Slide 13: PBGM01 Preclinical Findings: Neurological Function and 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 -/- (KO
Slide 14: Goal of Imagine-1 Trial in GM1
Goal of 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 safety and tolerability of a single intra-cisterna magna dose of PBGM01 2. Demonstrate treatment-
Slide 15: Imagine-1 Global Phase 1/2 Trial with PBGM01
Imagine-1 Global Phase 1/2 Trial with PBGM01 Initial safety and biomarker data expected 4Q21 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
F. 4 PBKRO3 Krabbe Disease
Slide 17: Krabbe Disease A Devastating Pediatric Disease
Krabbe Disease A Devastating Pediatric Disease 17 Source: NIH, CHOP, American Journal of Neuroradiology, Third Party Research FATAL, PEDIATRIC NEUROLOGICAL LYSOSOMAL STORAGE DISORDER caused by GALC gene mutations characterized by demyelination of neurons in the brain and periphery. Disease progressi
Slide 18: PBKR03 Potential Transformative Therapy
PBKR03 Potential Transformative Therapy for Rare, Underserved Disorder 18 ▪ Next-generation, proprietary AAVhu68 capsid delivers functional GALC gene encoding galactosylceramidase (GALC) to the brain and peripheral tissues ▪ PBKR03-treated Krabbe dogs had improved central and peripheral myelination,
Slide 19: 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 Twitcher Mouse Model: ▪ Increased GALC activity in brain, liver and serum ▪ AAV treated twitcher mouse showed clinical scores comparable to wt mice Canine
Slide 20: PBKR03 Supported by Data From Canine Disease Model
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 Supported by Data From Canine Disease Model Survival Extended by AAV Treatment Euthanized due t
Slide 21: GALax-C Global Phase 1/2 Trial with PBKR03
COHORT 2 4m to <9m n = 3 COHORT 3 >1 to <4m n = 3 COHORT 1 >4m to <9m n = 3 GALax-C Global Phase 1/2 Trial with PBKR03 First patient enrollment expected in 3Q21 Trial Design ◼ Phase 1/2, multi-center, open-label, single-arm, dose-escalation and confirmatory study of PBKR03 in patients with early inf
Slide 22: PBFT02 Frontotemporal Dementia — GRN
PBFT02 Frontotemporal Dementia — GRN
Slide 23: FTD-GRN A Devastating Adult Disease
FTD-GRN A Devastating Adult Disease DEVASTATING FORM OF DEMENTIA caused by a GRN gene mutation resulting in progranulin (PGRN) deficiency. Approximately 5–10% of FTD is caused by a GRN mutation. Disease progression is rapid and degenerative including loss of speech, loss of expression, severe behavi
Slide 24: PBFT02 Potential Transformative Therapy
PBFT02 Potential Transformative Therapy for Rare, Underserved Disorder ▪ Proprietary AAV1 capsid delivers function GRN gene encoding progranulin ▪ PBFT02 demonstrated increases in CSF PGRN concentrations in NHP studies to >50-fold normal human CSF PGRN concentrations ▪ Received regulatory clearances
Slide 25: PBFT02 Biomarker Improvement Across Brain Regions
PBFT02 Biomarker Improvement Across Brain Regions Decreases markers of lysosomal function and microgliosis 25 Source: Hinderer et. al, Annals of Clinical and Translational Neurology. 2020 Oct; 7(10):1843–1853 PBFT02 high dose vs. vehicle in GRN-/- mice; post baseline data is 90 days post-dose * = p<
Slide 26: PBFT02 Supportive NHP Preclinical Studies Utilizing ICM
PBFT02 Supportive NHP Preclinical Studies Utilizing ICM A single administration of PBFT02 to NHPs via the optimized AAV1-GRN vector demonstrated transduction broadly across the brain, including a very high transduction of ependymal cells that line the ventricles of the brain and are involved with CS
Slide 27: upliFT-D Global Phase 1/2 Trial with PBFT02
upliFT-D Global Phase 1/2 Trial with PBFT02 First patient enrollment expected in 2Q/3Q21 Trial Design ◼ Phase 1/2, multicenter, open-label, single-arm, dose escalation study of PBFT02 in early symptomatic adult subjects with frontotemporal dementia and mutations in the progranulin gene Intervention
Slide 28: State-of-the-Art Manufacturing
State-of-the-Art Manufacturing
Slide 29: Manufacturing That is Dedicated, Flexible, and Scalable
Manufacturing That is Dedicated, Flexible, and Scalable In-House CMC Capabilities Investment in CMC laboratory with state-of-the-art technologies for analytical and process development, clinical product testing, and clinical and biomarker assay development End-to-End Supply Chain Establishing manufa
Slide 30: Corporate Foundation
Corporate Foundation 30 SOURCE: www.brandywinerealty.com
Slide 31: Financial Summary
Financial Summary 31 * Cash, cash equivalents and marketable securities Cash balance of $438M at 3/31/21* Cash on hand to fund operations for at least 24 months Runway to potentially deliver meaningful catalysts over multiple programs
Slide 32: Anticipated Upcoming Milestones
Anticipated Upcoming Milestones 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 2Q/3Q21 INITIATE Advance preclinical programs for MLD, ALS CMT2A, and undisclosed adult indication ADVANCE Evaluate and expand pipeline by pu
Slide 33: The Passage Bio Advantage
The Passage Bio Advantage Corporate model designed for success 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-stage th
Slide 34: Thank You
www.passagebio.com NASDAQ GS: PASG THANK YOU
Slide 35: Demonstrated Leadership
Demonstrated Leadership Deep experience in rare disease, CNS disorders and gene therapy LEADERSHIP TEAM BOARD OF DIRECTORS Tachi Yamada, M.D. (Chair) Frazier Athena Countouriotis, M.D. Turning Point Bruce Goldsmith, Ph.D. Passage Bio Maxine Gowen, Ph.D. Tamuro Bio Patrick Heron Frazier Saqib Islam,
Slide 36: 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 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 Direc