Passage Bio, Inc. · NASDAQ:PASG
Passage Bio — Investor Presentation (August 2022)
34 slides from Passage Bio's investor presentation, Aug 4, 2022
Slide 1: Fulfilling the Promise of Genetic Medicines for CNS Disorders
Fulfilling the Promise of Genetic Medicines for Central Nervous System DisordersCorporate PresentationAugust 2022NASDAQ GS: PASG
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)3 programs•GM1 Gangliosidosis•Krabbe Disease•Metachromatic Leukodystrophy3 programs•F
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 PBGM01Initial safety and biomarker data from Cohorts 2 & 3 expected in 2H 2022 Trial DesignPhase 1/2, multi-center, open-label, dose escalation and confirmatory study Route of AdministrationIntra-cisterna magna (ICM)VectorAAVhu68DurationTwo years, with rollo
Slide 12: Key Takeaways from Interim Cohort Data
12 Key Takeaways from Interim Cohort 1 Data*Well tolerated, demonstration of functional transgene expression, and meaningful developmental improvement BIOMARKERSPost treatment CSF and serum β-gal activity for both patients above natural history study (NHS) patient values SAFETYPBGM01 was well tolera
Slide 13: Interim Clinical Results: Patient 1
13 Interim Clinical Results: Patient 1Patient 1 demonstrated meaningful gains in developmental milestones, advancing from baseline to 12-months•Gross motor: progressed from taking a few steps without support toobserved jumping, balancing on 1 foot, and climbing stairs•Expressive language: progressed
Slide 14: Interim Clinical Results: Patient 2
14 Interim Clinical Results: Patient 2At month 4, Patient 2 was clinically observed to have regained previously lost developmental milestones•Gross motor: progressed from standing with support for 5 seconds to standing with support for 1-3 minutes and walking without support•Expressive language: pro
Slide 15: Cohort 1 Interim Biomarker Data
15 •CSF β-gal activity increased above levels seen in NHS following dosing and sustained at upper limit of NHS•Serum β-gal activity increased in both patients; however, over time, post-treatment β-gal activity for Patient 1 was above NHS patient values, while Patient 2 normalized to NHS patient valu
Slide 16: PBKR03 Krabbe Disease
P Bi PBKRO3 oo aati Krabbe Disease
Slide 17: Krabbe Disease: A Devastating Pediatric Disease
17 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 18: PBKR03 Our Approach
18 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 19: GALax-C Global Phase 1/2 Trial with PBKR03
19 COHORT 2> 4m to <9mn = 3 COHORT 3> 1m to <4mn = 3 COHORT 1> 4m to <9mn = 4+GALax-C Global Phase 1/2 Trial with PBKR03Initial safety and biomarker data from subset of Cohort 1 expected by end of 2022 Confirmatory Cohort > 1m to <4mn = 6 Confirmatory Cohort > 4m to <9mn = 6 COHORT 4> 1m to <4mn = 3
Slide 20: PBFT02 Frontotemporal Dementia — GRN
PBFT02Frontotemporal Dementia —GRN
Slide 21: FTD-GRN: A Devastating Adult Disease
21 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 22: PBFT02 Our Approach
22 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 23: upliFT-D: Global Phase 1/2 Trial with PBFT02
23 upliFT-D: Global Phase 1/2 Trial with PBFT02First patient dosing expected in mid-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 escalation s
Slide 24: PBML04 Metachromatic Leukodystrophy
PBML04Metachromatic Leukodystrophy
Slide 25: Metachromatic Leukodystrophy: A Devastating Pediatric Disease
25 Metachromatic Leukodystrophy: A Devastating Pediatric Disease FATAL, PEDIATRIC NEUROLOGICAL LYSOSOMAL STORAGE DISORDER caused by ARSAgene mutations leading to accumulation of sulfatides in the brain and other areas of the body.Disease progression is rapid and highly predictable including muscle w
Slide 26: PBML04 Our Approach
26 PBML04OUR APPROACHNext-generation, proprietary AAVhu68 capsid delivers functional ARSAgene encoding arylsulfatase A (ARSA) to the brain and peripheral tissues via ICM injectionPRECLINICAL EVIDENCECompelling preclinical data in a knock-out mouse model•Dose-related lysosomal correction, improvement
Slide 27: PBML04 Reduced Functional Decline and Increased Survival in MLD Mouse Model
27 Summary•Novel ARSAknockout mouse model to assess effects on survival and clinical impairment•Dose-dependent improvements in survival and reduced clinical impairment observed•All mice in the AAV-treated groups survived while 5/10 mice in the untreated group died prematurely•Rate of clinical impair
Slide 28: Looking Ahead
Looking Ahead i PassageBio
Slide 29: Key Milestones
29 TIMINGMILESTONEGM1Early 2022First patient dosed for Cohorts 2 and 3Feb 2022Late breaker presentation at WORLDSymposium2H 2022Initial safety and biomarker data for Cohorts 2 and 3FTD-GRNMid-2022First patient dosed in Phase 1/2 trial for PBFT02KrabbeEarly 2022First patient dosed in Phase 1/2 trial
Slide 30: Genetic Medicines Company on a Mission to Transform Lives of CNS Patients
30 30Genetic 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 analytic
Slide 31: Thank You
Thank Youpassagebio.com |NASDAQ GS: PASG
Slide 32: Demonstrated Leadership
32 Demonstrated LeadershipLEADERSHIP TEAM Simona KingChief Financial OfficerMaria Törnsén Chief Commercial OfficerMark Forman, M.D., Ph.D.Chief Medical Officer Chip CaleInterim Chief Executive Officer &General CounselAlex Fotopoulos Chief Technology OfficerDeep experience in rare disease, CNS disord
Slide 33: Intra-Cisterna Magna (ICM) Administration
33 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 anesthesi
Slide 34: Pathway to Patient Identification and Trial Recruitment
34 Pathway to Patient Identification and Trial Recruitment Screening / DiagnosisSponsoring 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 counseling o