Rocket Pharmaceuticals, Inc. · NASDAQ:RCKT

Rocket Pharmaceuticals — American Society of Gene and Cell Therapy 25th Annual Meeting (May 2022)

14 slides from Rocket Pharmaceuticals's conference presentation, May 16, 2022

  1. Slide 1: Extended Results From First-In-Human Clinical Trial Of RP-A501 Gene Therapy For Danon Disease

    Extended Results From First-In-Human Clinical Trial Of RP-A501 (AAV9:LAMP2B) Gene Therapy Treatment For Danon Disease Barry Greenberg1, Joseph Rossano2, Shilpi Epstein3, Pavan Battiprolu3, Marcus A. Urey1, Kimberly N. Hong1, Andrew M. Kahn1, Abigail Waldron2, Emily Eshraghian1, David Ricks3, Paul Ya

  2. Slide 2: Danon Disease

    Danon Disease Autosomal dominant, monogenic X-linked disease • LAMP-2B gene mutation (Lysosomal Associated Membrane Protein 2B) • Impaired autophagy • Prominent sarcoplasmic vacuoles • Myocardial disarray Females Severe Cardiomyopathy (CM 95%) • Mortality secondary to heart failure • Males: Males •

  3. Slide 3: RP-A501: Danon Disease Gene Therapy Clinical Trial Overview

    RP-A501: Danon Disease Gene Therapy Clinical Trial Overview Design Key Patient Entry Criteria** • Male Danon Disease patients Inclusion Non- • Adults (& Adolescents): >15 years • Male Randomized • Pediatric: 8-14 years • Confirmed LAMP2B mutation Open Label • Cardiac involvement confirmed by imaging

  4. Slide 4: RP-A501: Baseline Status of Adult Cohorts

    RP-A501: Baseline Status of Adult Cohorts Age at Clinical Status Cardiac Status Weight Cohort Patient ID Enrollment Six Minute LV Ejection (kg) BNP (years) NYHA Class Walk Fraction* (pg/mL) (meters) (%) 1001 17.5 52.5 II 443 70 62 Adult - Low Dose 1002 20.4 89.1 II 405 942 59 6.7 x 1013 GC/kg 1005 1

  5. Slide 5: LAMP2 Cardiac Protein Expression by Immunohistochemistry and Myofibrillar Structure

    RP-A501: LAMP2 Cardiac Protein Expression by Immunohistochemistry and Myofibrillar Structure by Electron Microscopy Normal Control Baseline* Month 9* Immunohistochemistry Electron Microscopy *Representative images from patient 1005 from endomyocardial biopsy. 5 Proprietary and Confidential

  6. Slide 6: Remodeling of Left Ventricular Hypertrophy in Adult Cohorts

    RP-A501: Remodeling of Left Ventricular Hypertrophy in Adult Cohorts Septum Thickness Posterior WallPosterior ThicknessWall (LVPWd) Thickness Septum Thickness (IVSd) (IVSd) Ejection Fraction (LV EF) (LVPWd) 40 Posterior Wall (End-Diastole) 40 1001 % Change from baseline % Change from baseline Septum

  7. Slide 7: RP-A501: Functional Clinical Status of Adult Cohorts

    RP-A501: Functional Clinical Status of Adult Cohorts Most Recent Cohort Patient ID Variable Baseline Time of Follow-up Follow-up NYHA class II II 1001* 24 Months BNP (pg/mL) 70 30 Adult – NYHA class II I 1002 18 Months Low Dose BNP (pg/mL) 942 200 NYHA class II I 1005 15 Months BNP (pg/mL) 176 44 Ad

  8. Slide 8: Grade 3 and 4 Serious Adverse Events in Adult Cohorts

    RP-A501: Grade 3 and 4 Serious Adverse Events (SAEs) in Adult Cohorts (As Previously Disclosed) RP-A501 Related*: Thrombotic microangiopathy (TMA) [grade 4] associated with Pt 1007 (received highest dose • thrombocytopenia [grade 4] secondary to baseline weight and • acute kidney injury & renal fail

  9. Slide 9: Risk Mitigation Strategies Implemented Prior to Pediatric Enrollment

    RP-A501: Risk Mitigation Strategies Implemented Prior to Pediatric Enrollment Enhanced prophylactic immunosuppressive regimen • Sirolimus to inhibit complement activation • Steroid-sparing regimen to decrease skeletal myopathy • Initial dose 0.5 to 0.8 mg/kg/day (compared to > 1mg/kg/day in adult co

  10. Slide 10: Immunosuppression, Patient Characteristics & Safety in Phase 1

    RP-A501: Immunosuppression, Patient Characteristics & Safety in Phase 1 Cohort 1: Low-dose adult RP-A501 SAEs of Interest Platelets Pt Age Wt 6.7 x 1013 GC/kg n=2 grade 3 steroid myopathy n=2 decreases 1001 17.5 52.5 Steroid (prednisone 1mg/kg) to LLN range 1002 20.4 89.1 1005 18.3 91.8 Tacrolimus C

  11. Slide 11: Inhibition of Complement Activation with Modified Immunosuppression

    RP-A501: Inhibition of Complement Activation with Modified Immunosuppression Low Dose Adult High Dose Adult Low Dose Pediatric Corticosteroids Rituximab + Corticosteroids + Tacrolimus Rituximab + Corticosteroids + Sirolimus 2000 1001 (no data) 2000 1008 2000 1006 sC5b9 (mg/mL) sC59b 1002 1007 1009 s

  12. Slide 12: Summary of Results from Adult and Pediatric Cohorts

    RP-A501: Summary of Results from Adult and Pediatric Cohorts Adult Cohort: Pediatric Cohort: RP-A501 was well tolerated in both • RP-A501 was generally well patients with modified protocol tolerated • T- and B-cell directed immunosuppression associated • Increased LAMP2 protein with markedly reduced

  13. Slide 13: RP-A501: Conclusion and Future Direction

    RP-A501: Conclusion and Future Direction • RP-A501 was generally well tolerated in adult and pediatric patients with Danon disease • Modified immunosuppressive regimen appears well tolerated and effective in pediatric cohort • Phase 1 efficacy signal in age ≥15 cohort: RP-A501 stabilizes and potenti

  14. Slide 14: Acknowledgements

    Acknowledgements For more information: Danonclinicaltrial@rocketpharma.com Jennifer Attias Joseph Rossano, MD, MS, FAAP, FACC Eric Adler, MD Abigail Waldron Quan Bui, MD Danielle Burstein, MD Emily Eshraghian, MPH Lindsey George, MD Kimberly Hong, MD Andrew Glatz, MD Sonia Jain, PhD Kimberly Lin, MD

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