Passage Bio, Inc. · NASDAQ:PASG
Passage Bio — Investor Presentation (October 2021)
35 slides from Passage Bio's investor presentation, Oct 4, 2021
Slide 1: Fulfilling the Promise of Genetic Medicines for Central Nervous System Disorders
Fulfilling the Promise of Genetic Medicines for Central Nervous System Disorders Corporate Presentation October 2021 NASDAQ GS: PASG
Slide 2: Forward-Looking Statement
2 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 anticipate
Slide 3: Passage Bio Overview
3 Passage Bio Overview Committed to serving patients with rare and large CNS diseases OUR VISION: To fulfill the promise of genetic medicines by developing groundbreaking therapies that transform the lives of patients with CNS diseases Significant unmet medical need with few treatment options Infant
Slide 4: The Passage Bio Advantage
4 The Passage Bio Advantage PENN GTP PARTNERSHIP Led by renowned innovator James M. Wilson, MD, PhD Access to cutting-edge research and development & next-generation technologies CNS disorders affecting both rare and large populations BROAD AND ROBUST PIPELINE 17 total program license options 3 clin
Slide 5: Passage Bio's Differentiated Path for Clinical Success
5 Passage Bio’s Differentiated Path for Clinical Success GTP expertise and insights into R&D Optimization of delivery approaches Disease-specific existing and novel capsid selection Integration of next-generation research advances Patient need Optimal capsid, transgene and promoter Preclinical safet
Slide 6: A Broad and Robust Pipeline with Global Rights
6 A Broad and Robust Pipeline with Global Rights *10 additional CNS pipeline license options. † Program includes ongoing natural history study of infantile and juvenile GM1 gangliosidosis patients Transformative therapies for CNS disorders across rare and large patient populations Program* Indicatio
Slide 7: Intra-Cisterna Magna (ICM) Administration
7 Intra-Cisterna Magna (ICM) Administration • 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 of neutralizing antibodies • Administered under anes
Slide 8: Pathway to Patient Identification and Trial Recruitment
8 Pathway to Patient Identification and Trial Recruitment Screening / Diagnosis Sponsoring ScreenPlus Pilot Program • GM1 included in the New York newborn screening pilot program led by Dr. Melissa Wasserstein Supporting Invitae Detect LSD and InformedDNA Programs • Free genetic testing and counseli
Slide 9: PBGM01 GM1 Gangliosidosis
PBGMO1 y Passadaae GM1 Gangliosidosis
Slide 10: GM1 Gangliosidosis: A Devastating Pediatric Disease
10 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 to
Slide 11: PBGM01 Program Summary
11 PBGM01 → 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 critical peripheral organs → Received multiple global regulatory c
Slide 12: PBGM01: Supportive Preclinical Findings
12 PBGM01: Supportive Preclinical Findings • Dose-related improvements in neurological function demonstrated • Top two doses similar to GLB +/- vehicle • Treated mice demonstrated increased survival at all doses tested • Top 2 doses achieved 100% survival to study endpoint Source: Hinderer et. al, H
Slide 13: Goal of Imagine-1 Trial in Early and Late Infantile GM1
13 Goal of Imagine-1 Trial in Early and Late Infantile GM1 GM1 Gangliosidosis is a Continuum Disease Severity Residual Enzyme Activity Imagine-1 Trial will include Type I (Early Infantile) and Type IIa (Late Infantile) patients Negligible to 5% ~ 1– 5% ~ 3 – 10% Type I (Early Infantile) • Onset <6 m
Slide 14: Imagine-1: Global Phase 1/2 Trial with PBGM01
14 Imagine-1: Global Phase 1/2 Trial with PBGM01 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 Infantile GM1 in cohorts 3 and 4 Inter
Slide 15: PBKR03 Krabbe Disease
PBKRO3 Cad Krabbe Disease
Slide 16: Krabbe Disease: A Devastating Pediatric Disease
16 Krabbe Disease: A Devastating Pediatric Disease 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 progress
Slide 17: PBKR03 Program Summary
17 PBKR03 → 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, reduced neuroinflammation and increased survival rates with ful
Slide 18: PBKR03: Disease Model Data — Twitcher Mouse and Dog Models
18 PBKR03: Disease Model Data — Twitcher Mouse and Dog Models Twitcher Mouse Model: • Increased GALC activity in brain, liver and serum • AAV treated twitcher mouse showed clinical scores comparable to wt mice Canine Model: • Increased GALC activity in CSF • Decreased psychosine levels in CSF GALC I
Slide 19: PBKR03: Supported by Data From Canine Disease Model
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 Survival Extended by AAV Treatment Euthanized d
Slide 20: GALax-C Global Phase 1/2 Trial with PBKR03
20 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 Trial Design • Phase 1/2, multi-center, open-label, single-arm, dose- escalation and confirmatory study of PBKR03 in patients with early infantile Krabbe disease who are >1 to 9
Slide 21: PBFT02 Frontotemporal Dementia — GRN
PBFT02 Frontotemporal Dementia — GRN
Slide 22: FTD-GRN: A Devastating Adult Disease
22 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 be
Slide 23: PBFT02 Program Summary
23 PBFT02 → 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 from FDA and Health Canada for global Phase 1/2 clinical tri
Slide 24: PBFT02: Biomarker Improvement Across Brain Regions
24 PBFT02: Biomarker Improvement Across Brain Regions *** * 0% 20% 40% 60% 80% 100% 120% Baseline Vehicle PBFT02 0 0.2 0.4 0.6 0.8 1 1.2 Baseline Vehicle PBFT02 Lipofuscin Accumulation Stopped in Thalamus Hexosaminidase Activity Normalization in Cortex Reduced CD68 in Hippocampus • Lipofuscin accumu
Slide 25: PBFT02: Supportive NHP Preclinical Studies Utilizing ICM
25 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 wit
Slide 26: upliFT-D: Global Phase 1/2 Trial with PBFT02
26 upliFT-D: Global Phase 1/2 Trial with PBFT02 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 • Single ICM dose of AAVh1.hPGRN (PBFT02)
Slide 27: State-of-the-Art Manufacturing
27 State-of-the-Art Manufacturing
Slide 28: Manufacturing That is Dedicated, Flexible, and Scalable
28 Manufacturing That is Dedicated, Flexible, and Scalable In-House CMC Capabilities Princeton West CMC laboratory with state-of-the-art technologies open for analytical and process development, clinical product testing, and clinical and biomarker assay development End-to-End Supply Chain Establishi
Slide 29: Corporate Foundation
29 Corporate Foundation SOURCE: www.brandywinerealty.com
Slide 30: Financial Summary
30 Financial Summary * Cash, cash equivalents and marketable securities Cash balance of $408M at 6/30/21* Cash on hand to fund operations for at least 24 months Runway to potentially deliver meaningful catalysts over multiple programs
Slide 31: Anticipated Upcoming Milestones
31 Anticipated Upcoming Milestones Phase 1/2 trial for PBGM01 in patients with infantile GM1 in 1Q21 INITIATE Phase 1/2 trial for PBFT02 in patients with FTD-GRN in 4Q21 INITIATE Advance programs for MLD, ALS, CMT2A, and undisclosed adult indication Advance target ID research programs for Alzheimer’
Slide 32: The Passage Bio Advantage
32 The Passage Bio Advantage PENN GTP PARTNERSHIP Led by renowned innovator James M. Wilson, MD, PhD Access to cutting-edge research and development & next-generation technologies CNS disorders affecting both rare and large populations BROAD AND ROBUST PIPELINE 17 total program license options 3 cli
Slide 33: Thank You
Thank You www.passagebio.com NASDAQ GS: PASG
Slide 34: Demonstrated Leadership
34 Demonstrated Leadership LEADERSHIP TEAM BOARD OF DIRECTORS Maxine Gowen, Ph.D. (Chair) Tamuro Bio Athena Countouriotis, M.D. Turning Point Bruce Goldsmith, Ph.D. Passage Bio Saqib Islam, J.D. SpringWorks Sandip Kapadia Harmony Biosciences Derrell Porter, M.D. Cellevolve Bio Liam Ratcliffe, M.D.,
Slide 35: Cutting-Edge Gene Therapy R&D Collaboration
35 Cutting-Edge Gene Therapy R&D Collaboration 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 Director, Orphan Disease Center Professor of Medicine and Pediatrics, Department of M