Passage Bio, Inc. · NASDAQ:PASG
Passage Bio — Investor Presentation (March 2021)
34 slides from Passage Bio's investor presentation, Mar 3, 2021
Slide 1: Corporate Presentation March 2021
Corporate Presentation March 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: Our vision: 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 CNS1 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 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 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 GTP expertise and insights into research and development Optimization of delivery approaches Disease-specific existing and novel capsid selection Integration of next-gen
Slide 7: 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 8: PBGM01 GM1 Gangliosidosis
" PBGMO1 GM1 Gangliosidosis ‘Passage Bio
Slide 9: GM1 Gangliosidosis: A Devastating Pediatric Disease
GM1 Gangliosidosis: A Devastating Pediatric Disease 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 tone,
Slide 10: PBGM01 Potential Transformative Therapy For Rare, Underserved Disorder
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 ▪ Meaningful transduction of both central nervous system and critical peripheral organs in preclinical
Slide 11: PBGM01 Supportive Preclinical Findings: Biomarker and Pathophysiology
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-/- + AA
Slide 12: 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 –/– (KO
Slide 13: Pathway to Patient Identification and Trial Recruitment
Pathway to Patient Identification and Trial Recruitment 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 Wasserstein I
Slide 14: Goal of Imagine-1 Trial in Early and Late Infantile 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 First patient enrollment expected in 1Q21 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 Onset
Slide 16: PBKR03 Krabbe Disease
F. 4 PBKRO3 Krabbe Disease
Slide 17: Krabbe Lysosomal Storage Disease
Krabbe Lysosomal Storage Disease PBKR03 — A potential transformative therapy for a rare, underserved disorder 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 diagnoses
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 GALC Increase in Brain and Periphery Twitcher Mouse Model: ▪ Increased GALC activity in brain, liver and serum ▪ AAV treated twitcher mouse showed clinica
Slide 19: 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 20: PBKR03 Global Phase 1/2 Trial in Early Infantile Krabbe Disease
COHORT 2 4m to < 9m n = 3 COHORT 3 >1 to < 4m of age n = 3 COHORT 1 >4m to < 9m n = 3 PBKR03 Global Phase 1/2 Trial in Early Infantile Krabbe Disease First patient enrollment expected in 1H21 Trial Design ◼ Phase 1/2, multi-center, open-label, single-arm, dose-escalation and confirmatory study of PB
Slide 21: PBFT02 Frontotemporal Dementia - GRN
PBFT02 Frontotemporal Dementia - GRN
Slide 22: FTD-GRN Frontotemporal Dementia Caused by Progranulin Deficiency
FTD-GRN Frontotemporal Dementia Caused by Progranulin Deficiency PBFT02 – A potential transformative therapy for a rare, underserved disorder 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 mutation, causi
Slide 23: 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 1.2 B
Slide 24: PBFT02 Supportive NHP Preclinical Studies Utilizing ICM
PBFT02 Supportive NHP Preclinical Studies Utilizing ICM PBFT02 Showed Dose-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 PGRN in CSF CSF PGRN (ng/mL) 0 10 20 30 40 CSF 0.1 1 10 100 LLOQ Normal Day RA2981 RA2982 RA
Slide 25: PBFT02 Global Phase 1/2 Trial in Early Symptomatic FTD-GRN
PBFT02 Global Phase 1/2 Trial in Early Symptomatic FTD-GRN First patient enrollment expected in 1H21 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 I
Slide 26: State-of-the-Art Manufacturing
State-of-the-Art Manufacturing
Slide 27: 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 28: Corporate Foundation
Corporate Foundation 28 SOURCE: www.brandywinerealty.com
Slide 29: Financial Summary
Financial Summary Cash balance of $305M at 12/31/20 Raised additional $166M in net proceeds in 1/21 Cash on hand to fund operations for at least 24 months Runway to potentially deliver meaningful catalysts over multiple programs 29
Slide 30: 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 1H21 INITIATE Advance preclinical programs for MLD, ALS CMT2A, and undisclosed adult indication ADVANCE Evaluate and expand pipeline by pursu
Slide 31: 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 32: Thank You
www.passagebio.com NASDAQ GS: PASG THANK YOU
Slide 33: 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 34: 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