Passage Bio, Inc. · NASDAQ:PASG
Passage Bio — J.P. Morgan 40th Annual Healthcare Conference (January 2022)
30 slides from Passage Bio's conference presentation, Jan 10, 2022
Slide 1: J.P. Morgan 40th Annual Healthcare Conference
J.P. Morgan 40th Annual Healthcare ConferenceBruce Goldsmith, Ph.D.President and Chief Executive OfficerJanuary 10, 2022
Slide 2: Forward-Looking Statement
2 Forward-Looking StatementThis 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: The Passage Bio Advantage
3 The Passage Bio AdvantageOur VisionTo fulfill the promise of genetic medicines by developing groundbreaking therapies that transform the lives of patients with CNS diseasesA corporate model designed for success PENN GTP PARTNERSHIPDEDICATED MANUFACTURING & ANALYTICSBROAD AND ROBUST PIPELINE
Slide 4: Penn GTP Partnership
4 Penn GTP Partnership Leaders in AAV gene therapy World-class Gene Therapy ProgramFounded by renowned innovatorJames M. Wilson, M.D., Ph.D.Chief Scientific Advisor, Passage Bio Cutting-edge research and bioengineering Rigorous preclinical characterization to support product candidate selection Next
Slide 5: A Broad and Robust Pipeline with Global Rights
5 A Broad and Robust Pipeline with Global RightsTransformative therapies for CNS disorders across rare and large patient populations Program* Indication GeneDiscoveryPre-clinicalPhase 1/2PivotalPEDIATRIC PBGM01 † GM1 gangliosidosis GLB1 PBKR03 Krabbe disease GALC PBML04 Metachromatic leukodystrophy
Slide 6: A Diversified Portfolio Strategy
6 A Diversified Portfolio StrategyCreating sustainable value across rare and large, monogenic and non-monogenic CNS indicationsCutting-edge R&D ADULT (RARE, MONOGENIC)ADULT (NON-MONOGENIC)PEDIATRIC (RARE, MONOGENIC)5 programs•GM1 Gangliosidosis•Krabbe Disease•Metachromatic Leukodystrophy•Charcot-Mar
Slide 7: Passage Bio Pilot Manufacturing Suite
7 Passage Bio Pilot Manufacturing SuiteScale-up Capability Supporting R&D Pipeline and Future Development Plans UPenn GTPResearch Analytics and Vector Engineering Catalent Suite and Global Supply ChainDedicated cGMP Manufacturing Capacity and End-to-End Clinical Supply Chain Passage Bio CMC R&D Lab
Slide 8: PBGM01 GM1 Gangliosidosis
P. Bi PBGMOl1 i GM1 Gangliosidosis
Slide 9: GM1 Gangliosidosis: A Devastating Pediatric Disease
9 GM1 Gangliosidosis: A Devastating Pediatric Disease FATAL, PEDIATRIC NEUROLOGICAL LYSOSOMAL STORAGE DISORDER caused by GLB1gene 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 Our Approach
10 PBGM01OUR APPROACHNext-generation, proprietary capsid delivers functional GLB1gene encoding β-gal to the brain and peripheral tissuesPRECLINICAL EVIDENCECompelling preclinical data in knock-out mouse model•Dose-related histological correction, improvements in neurological function, and survival•M
Slide 11: Imagine-1: Global Phase 1/2 Trial with PBGM01
11 Imagine-1: Global Phase 1/2 Trial with PBGM01Currently enrolling Cohort 2 and Cohort 3 Trial DesignPhase 1/2, multi-center, open-label, dose escalation and confirmatory study Route of AdministrationIntra-cisterna magna (ICM)VectorAAVhu68DurationTwo years, with rollover into a separate long-term f
Slide 12: Key Takeaways from Interim Cohort Data
12 Key Takeaways from Interim Cohort 1 DataWell tolerated with demonstration of functional transgene expression at lowest dose BIOMARKERSPost treatment CSF and serum β-gal activity for both patients above natural history study (NHS) patient values•Increases in CSF and serum β-gal activity for Patien
Slide 13: Cohort 1 Interim Biomarker Data
13 •CSF β-gal Activity increased in both patients•Post treatment β-gal activity above NHS patient values (Range 0.30–1.81 nmol/mL/3hr)2Cohort 1 Interim Biomarker Data 01 2 340306090120150180β - Gal Activity, CSF (nmol/mL/3hr) Time (Days)CSF β-gal+0.6+2.7 •Serum β-gal activity increased in both patie
Slide 14: PBKR03 Krabbe Disease
P Bi PBKRO3 alia Krabbe Disease
Slide 15: Krabbe Disease: A Devastating Pediatric Disease
15 Krabbe Disease: A Devastating Pediatric Disease FATAL, PEDIATRIC NEUROLOGICAL LYSOSOMAL STORAGE DISORDER caused by GALCgene mutations characterized by demyelination of neurons in the brain and periphery.Disease progression is rapid and highly predictable including loss of acquired milestones, sta
Slide 16: PBKR03 Our Approach
16 PBKR03OUR APPROACHNext-generation, proprietary capsid delivers functional GALCgene encoding galactosylceramidase (GALC) to the brain and peripheral tissuesPRECLINICAL EVIDENCEPBKR03-treated Krabbe dogs had improved central and peripheral myelination, reduced neuroinflammation and increased surviv
Slide 17: GALax-C Global Phase 1/2 Trial with PBKR03
17 COHORT 24m to <9mn = 3 COHORT 3 > 1 to <4m n = 3 COHORT 1>4m to <9mn = 3GALax-C Global Phase 1/2 Trial with PBKR03First patient dosing expected in early 2022 Confirmatory Cohort > 1 to <4m n = 6 Confirmatory Cohort 4m to <9mn = 6 COHORT 4 > 1 to <4m n = 3DOSE 2 (5.0e11 GC/g)*DOSE 1 (1.5e11 GC/g)*
Slide 18: PBFT02 Frontotemporal Dementia — GRN
PBFT02Frontotemporal Dementia —GRN
Slide 19: FTD-GRN: A Devastating Adult Disease
19 FTD-GRN: A Devastating Adult DiseaseDEVASTATING FORM OF DEMENTIA caused by a GRNgene mutation resulting in progranulin (PGRN) deficiency. Approximately 5–10% of FTD is caused by a GRNmutation.Disease progression is rapid and degenerative including loss of speech, loss of expression, severe behavi
Slide 20: PBFT02 Our Approach
20 PBFT02OUR APPROACHProprietary construct delivers functional GRNgene encoding progranulin (PGRN) with potential therapeutic benefit of a one-time gene therapy approachPRECLINICAL EVIDENCECompelling preclinical evidence from NHP studies•Broad transduction across the brain, including high transducti
Slide 21: upliFT-D: Global Phase 1/2 Trial with PBFT02
21 upliFT-D: Global Phase 1/2 Trial with PBFT02First patient dosing expected in early 2022 OPTIONAL DOSE 3* DOSE 2 (1.1e11 GC/g)* DOSE 1 (3.3e10 GC/g)* 60 days between subject enrollment COHORT 1n = 3 COHORT 2n = 3 OPTIONAL COHORT 3n = 3 Trial DesignPhase 1/2, multicenter, open-label, dose escalatio
Slide 22: Preclinical Pipeline
Preclinical Pipeline
Slide 23: PBML04 – Metachromatic Leukodystrophy
23 DISEASE OVERVIEW•Fatal inherited disease. Mutations in the ARSA(arylsulfatase A) gene reduce enzyme activity•Infantile onset MLD is characterized by muscle weakness, rigidity, gait disorder, developmental delays, and is typically fatal by 5 years of age•Worldwide prevalence ~1 in 100,0001OUR APPR
Slide 24: PBCM06 – Charcot-Marie-Tooth Type 2A
24 DISEASE OVERVIEW•Sensory and motor neuropathy caused by mutations in the gene for mitofusin-2 (MFN2)•Progressive distal limb weakness, muscle atrophy, and loss of sensation1,2•Worldwide prevalence ~1 in 100,0003OUR APPROACH•PBCM06: AAV delivery of miRNA and gene combination to knockdown mutant an
Slide 25: PBAL05 – C9orf72 Amyotrophic Lateral Sclerosis
25 DISEASE OVERVIEW•Fatal neurodegenerative disease characterized by progressive loss of motor function •A hexanucleotide repeat expansion in the C9orf72gene is found in 40% of familial and 8% of sporadic ALS cases (11% of all ALS)1•Estimated ~5,000 cases worldwide in 20202OUR APPROACH•PBAL05: AAV d
Slide 26: Looking Ahead
Looking Ahead i PassageBio
Slide 27: Anticipated Upcoming Milestones
27 Anticipated Upcoming Milestones RESEARCH PIPELINE•Advance programs for ALS, CMT2A, Parkinson’s, Canavan and Huntington’s •Advance target ID research programs for Alzheimer’s Disease and TLE•Evaluate and expand pipeline by pursuing new licenses in partnership with Penn’s GTPBALANCE SHEET•Cash bala
Slide 28: Genetic Medicines Company on a Mission to Transform the Lives of Patients with CNS Diseases
28 Genetic Medicines Company on a Mission to Transform the Lives of Patients with CNS Diseases A diversified rare and large portfolio strategy to ensure sustained value Robust collaboration with GTP, an unmatched leader in cutting-edge AAV gene therapy research Dedicated manufacturing and analytics
Slide 29: Thank You
Thank Youpassagebio.com |NASDAQ GS: PASG
Slide 30: Demonstrated Leadership
30 Demonstrated LeadershipLEADERSHIP TEAM BOARD OF DIRECTORSMaxine Gowen, Ph.D.ChairwomanAthena Countouriotis, M.D.Turning PointBruce Goldsmith, Ph.D.Passage BioSaqib Islam, J.D.SpringWorksSandip KapadiaHarmony BiosciencesDerrell Porter, M.D.Cellevolve BioLiam Ratcliffe, M.D., Ph.D.Access Industries
Passage Bio — J.P. Morgan 40th Annual Healthcare Conference (January 2022) on 6ix