Rocket Pharmaceuticals, Inc. · NASDAQ:RCKT

Rocket Pharmaceuticals — American Society of Gene & Cell Therapy 25th Annual Meeting 2022

22 slides from Rocket Pharmaceuticals's conference presentation, May 19, 2022

  1. Slide 1: Interim Results From an Ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for LAD-I

    Interim Results From an Ongoing Phase 1/2 Study of Lentiviral- Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I) Donald B. Kohn, M.D., Professor Departments of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Ph

  2. Slide 2: Disclosures

    Disclosures I am a paid member of the Scientific Advisory Boards of: • Allogene Therapeutics • Pluto Therapeutics • ImmunoVec • MyoGeneBio 2 Proprietary and Confidential

  3. Slide 3: Leukocyte Adhesion Deficiency-I (LAD-I)

    Leukocyte Adhesion Deficiency-I (LAD-I) LAD-I Clinical Prognosis Kaplan-Meier Survival • Mutations affect the common chain • Patients suffer from recurrent infections; (CD18) of the beta2-integrin family fatal in majority Estimates by (ITGB2 gene) & prevent functional • 60–75% pts with severe LAD-I:

  4. Slide 4: Gene Therapy for LAD-I: RP-L201

    Gene Therapy for LAD-I: RP-L201 Ex-vivo lentiviral vector gene therapy consists 3 HSC ENRICHMENT & TRANSDUCTION of autologous CD34+ cells transduced with with lentiviral vector, a lentiviral vector (Chim-CD18-WPRE LV) Chim-CD18-WPRE LV (LV-RP-L201) encoding the CD18 (β-subunit) component of the β2-i

  5. Slide 5: RP-L201 Clinical Trial Design, Patient and Drug Product Characteristics

    RP-L201 Clinical Trial Design, Patient and Drug Product Characteristics Trial Design Drug • Non-Randomized Global Phase 1/2 Study (n=9) Age at Patient Sex​ Product CD34+ Cell Dose​ enrollment​ VCN​ Key Eligibility Criteria • Severe LAD-I; CD18 expression <2% PMNs, or CD11a/b <2% with documented L201

  6. Slide 6: Vector Copy Number

    VCN in Peripheral Blood Mononuclear Cells 4.0 L201-003-1001 Vector Copy Number L201-003-1004 3.0 L201-003-2006 L201-003-2005 2.0 L201-003-2007 L201-004-2008 1.0 L201-004-2009 L201-003-2011 0.0 L201-002-2010 Screening 0 3 6 9 12 15 18 21 24 Months Post-RP-L201 6 Proprietary and Confidential

  7. Slide 7: CD18+ Expression in Peripheral Blood PMNs

    CD18+ Expression in PB Polymorphonuclear Cells (PMNs) 100 L201-003-1001 %CD18+ PB PMNs 80 L201-003-1004 L201-003-2006 * 60 L201-003-2005 L201-003-2007 Re-added marker borders to graph and legends. I think it will help make these be more visible to the ballroom/exhibition 40 hall audience. L201-004-2

  8. Slide 8: CD18+ and CD11a+ Expression in PB PMNs

    CD18+ and CD11a+ Expression in PB PMNs L201-003-1001 L201-003-1004 L201-003-2006 100 %CD18+/CD18+ PB PMNs 100 %CD18+/CD18+ PB PMNs 100 %CD18+/CD18+ PB PMNs 80 80 80 60 60 * 60 40 40 40 %CD18+ PB PMNs 20 20 20 %CD11a+ PB PMNs 0 0 0 Screening 0 3 6 9 12 15 18 21 24 Screening 0 3 6 9 12 15 18 Screening

  9. Slide 9: Resolution of LAD-I Related Abnormal Leukocytosis

    Resolution of LAD-I Related Abnormal Leukocytosis: A clinical biomarker of a normalized phenotype 28 Age at Leukocytes (×1000/μL) Enrollment 24 L201-003-1001 (9y) 20 L201-003-1004 (3y) * L201-003-2006 (7mo) 16 L201-003-2005 (3y) 12 L201-003-2007 (3mo) 8 L201-004-2008 (5mo) L201-004-2009 (3y) 4 L201-

  10. Slide 10: Overall Survival

    Overall Survival Overall Survival (OS) Kaplan-Meier Estimate Survival without 1.0 Survival Probability allogeneic HSCT 0.8 Primary Outcomes • >2 years of age 0.6 • 1-year post-RP-L201 infusion 0.4 0.2 Censored 0.0 0 3 6 9 12 15 18 21 24 Months Post-RP-L201 10 Proprietary and Confidential

  11. Slide 11: RP-L201 Clinical Outcome Measures: Hospitalizations

    RP-L201 Clinical Outcome Measures Incidence of All Hospitalizations and Infection and Inflammatory Related Hospitalizations Incidence of Hospitalizations Incidence of Infection and Inflammatory Related Hospitalizations 4 p: <0.0001 3 p: <0.0001 Exposure Adjusted Event Rate Exposure Adjusted Event Ra

  12. Slide 12: RP-L201 Clinical Outcome Measures: Prolonged Hospitalizations and Severe Infections

    RP-L201 Clinical Outcome Measures Incidence of Prolonged Hospitalizations and Severe Infections Incidence of Prolonged (>7 days) Infection Incidence of Severe Infections and Inflammatory Related Hospitalization 2.5 p: <0.0001 2.0 Exposure Adjusted Event Rate Exposure Adjusted Event Rate p: <0.0012 2

  13. Slide 13: Spontaneous LAD-I Related Skin Rash Resolution and Restoration of Wound Repair

    Spontaneous LAD-I Related Skin Rash Resolution and Restoration of Wound Repair Capabilities after RP-L201 Spontaneous resolution of abdominal lesion Wound repair at thoracotomy site after surgical correction of double aortic arch L201-003-1001 L201-004-2008 3 months 6 months 12 months Post-IP Post-I

  14. Slide 14: Clinical Safety Overview

    Clinical Safety Overview As of March 9, 2022, nine severe LAD-I patients have received RP-L201. Data is available from 9/9 patients with 3–24 m follow-up. • To date, no RP-L201 related adverse events have been reported. • Neutrophil engraftment achieved in 9/9 subjects (<34 days post-infusion) • Adv

  15. Slide 15: RP-L201 Clinical Safety & Efficacy Overview

    RP-L201 Clinical Safety & Efficacy Overview • All (9/9) severe LAD-I patients have successfully received RP-L201; currently with 3–24 months follow-up • Infusion has been well tolerated; no drug product-related SAEs • Safety profile of RP-L201 appears favorable • Initial ISA indicates highly polyclo

  16. Slide 16: Acknowledgements

    Acknowledgements Inquiries or questions, please email: ladclinicaltrial@rocketpharma.com Julián Sevilla, MD, PhD Josune Zubicaray, MD Claire Booth, MBBS, PhD, FRCPCH Marta González-Vicent, MD, PhD Philip Ancliff, MA, MRCP, MRCPath Elena Sebastián, MD, PhD Adrian J. Thrasher, MBBS, PhD, FMedSci Camil

  17. Slide 17: Appendix

    Appendix Interim Results: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I) 17 Proprietary and Confidential

  18. Slide 18: CD18+ Expression and Vector Copy Number in PB and BM

    CD18+ Expression and Vector Copy Number in PB and BM L201-003-1001 L201-003-1004 L201-003-2006 * L201-003-2005 L201-003-2007 L201-004-2008 L201-004-2009 L201-003-2011 L201-004-2010 *Dim/weak CD18+ expression reported at baseline for Subject L201-003-1004 in ~63% of cells 18 Proprietary and Confident

  19. Slide 19: Efficacy as Demonstrated by VCN in Peripheral Blood Subpopulations

    Efficacy as Demonstrated by VCN in Peripheral Blood Subpopulations Peripheral Blood Granulocytes (CD15+ Cells) CD3+ Cells CD19+ Cells 19 Proprietary and Confidential

  20. Slide 20: Integration Site Analysis (ISA) – Top Integration Sites

    Integration Site Analysis (ISA) – Top 10 Integration Sites L201-003-1001 – 24 months – PB WBC L201-003-2005 – 12 months – PB WBC L201-003-1004 – 18 months – PB WBC PB WBC: White Blood Cells in Peripheral Blood 20 Proprietary and Confidential

  21. Slide 21: Integration Site Analysis (ISA) – Top Integration Sites

    Integration Site Analysis (ISA) – Top 10 Integration Sites L201-003-2006 – 9 months – PB WBC L201-004-2009 – 3 months – PB WBC L201-003-2007 – 6 months – PB WBC PB WBC: White Blood Cells in Peripheral Blood 21 Proprietary and Confidential

  22. Slide 22: Integration Site Analysis (ISA) – Top Integration Sites

    Integration Site Analysis (ISA) – Top 10 Integration Sites L201-004-2008 – 3 months – PB WBC L201-003-2011 – 3 months – BM WBC PB WBC: White Blood Cells in Peripheral Blood BM WBC: White Blood Cells in Bone Marrow 22 Proprietary and Confidential

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