Rocket Pharmaceuticals, Inc. · NASDAQ:RCKT

Rocket Pharmaceuticals — American Society of Gene and Cell Therapy (ASGCT) 2022

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

  1. Slide 1: Changing the Treatment Paradigm for Pyruvate Kinase Deficiency with Lentiviral Gene Therapy

    Changing the Treatment Paradigm for Pyruvate Kinase Deficiency with Lentiviral Mediated Gene Therapy: Interim Results from an Ongoing Global Phase 1 Study Ami J. Shah, MD Center for Definitive and Curative Medicine Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation

  2. Slide 2: Disclosures

    Disclosures • Advisory Board: Orchard Therapeutics

  3. Slide 3: Introduction

    Introduction • Pyruvate kinase deficiency (PKD) is a rare inherited hemolytic anemia caused by mutations in the PKLR gene which result in decreased red cell pyruvate kinase activity and impaired erythrocyte energy production (glycolysis). • Clinical manifestations include anemia, reticulocytosis, hy

  4. Slide 4: Gene Therapy For PKD: Rationale

    Gene Therapy For PKD: Rationale • Involves insertion of a functional PKLR gene into autologous hematopoietic stem and progenitor cells (HSPCs) with the intent of enabling lifelong red blood cell (RBC) pyruvate kinase enzyme production and normalized RBC function and lifespan • Preclinical studies pe

  5. Slide 5: Global Phase 1 PKD Gene Therapy Study

    RP-L301-0119 NCT#04105166 Global Phase 1 PKD Gene Therapy Study Clinical Sites: Key Eligibility Criteria Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain Stanford University, Palo Alto, California, United States Inclusion: Hospital Infantil Universitario Niño Jesús, Madrid, Spain • PKD d

  6. Slide 6: Study Design

    Study Design CD34+ cell mobilization protocol with G-CSF Schematic diagram of product manufacture and treatment: and plerixafor: HSPC apheresis & immunoselection Minimum cell dose 2×106 CD34+ cells/kg RP-L301 1 Transduction w/ viral vector Infusion PGK-coRPK-WPRE 5 2 3 4 Myeloablative Busulfan Genet

  7. Slide 7: Patient L301-006-1001 Clinical Course

    Patient L301-006-1001 Clinical Course Pre-treatment Characteristics Product Metrics Transfusion Requirement Age (y) & Hemoglobin Bilirubin Erythropoietin Nucleated CD34+ Cells/kg CFCs/kg Mean VCN: PKD Mutation for 2 Years Prior to Gender (g/dL) (mg/dL) (mIU/mL) Cells/kg Liquid Culture Enrollment c.7

  8. Slide 8: Preliminary Efficacy Results: L301-006-1001

    Preliminary Efficacy Results: L301-006-1001 --- Normal Range --- Normal Range (0.3–1.2 mg/dL) (0.5–1.5%) Significant bilirubin decrease from Sustained reduction in reticulocytes 13.4 mg/dL to 1.4 mg/dL --- Normal Range --- Normal Range • Sustained hemoglobin improvement from ~7.4 g/dL to 13.0 g/dL (

  9. Slide 9: Patient L301-001-1002 Clinical Course

    Patient L301-001-1002 Clinical Course Pre-treatment Characteristics Product Metrics Transfusion Requirement Nucleated CD34+ CFCs/kg Mean VCN: Age (y) & Hemoglobin Bilirubin Erythropoietin PKD Mutation for 2 Years Prior to Cells/kg Cells/kg Liquid Culture Gender (g/dL) (mg/dL) (mIU/mL) Enrollment 2.4

  10. Slide 10: Preliminary Efficacy Results: L301-001-1002

    Preliminary Efficacy Results: L301-001-1002 --- Normal Range --- Normal Range (0–1.4 mg/dL) (0.45–2.28%) Normalized bilirubin from 7.4 mg/dL Significant improvement in to 0.9 mg/dL reticulocytes from 25.5% to 4.73% • Hemoglobin normalized to 14.8 g/dL at ~18 months post-rx • No red blood cell transf

  11. Slide 11: Preliminary Results Indicate Improved Quality of Life (FACT-An)

    Preliminary Results Indicate Improved Quality of Life (FACT-An) during 18 months subsequent to RP-L301 therapy Pre-treatment Post-treatment 200 150 L301-006-1001 100 50 0 Screening Pre-Conditioning 6 Months 9 Months 12 Months 18 Months Post-RP-L301 Post-RP-L301 Post-RP-L301 Post-RP-L301 200 150 100

  12. Slide 12: Preliminary Results Indicate Improved Quality of Life (SF-36)

    Preliminary Results Indicate Improved Quality of Life (SF-36) during 18 months subsequent to RP-L301 therapy Pre-treatment Post-treatment 100 90 80 70 60 50 L301-006-1001 40 30 20 10 0 Screening Pre-Conditioning 3 Months 6 Months 9 Months 12 Months 18 Months Post-RP-L301 Post-RP-L301 Post-RP-L301 Po

  13. Slide 13: Safety Profile of RP-L301 appears highly favorable

    Safety Profile of RP-L301 appears highly favorable • Hematopoietic stem and progenitor cell collection appears safe and feasible in the initial adult patients with severe PKD • Hematopoietic reconstitution occurred within 2 weeks post RP-L301 administration - Infusion well tolerated in (N=2) - Patie

  14. Slide 14: Conclusions

    Conclusions • Investigational product RP-L301 was successfully manufactured ◦ Liquid culture VCNs >2.0; CD34+ cell doses 2.4–3.9×106/kg • Robust and sustained efficacy in both patients at 18-months post-RP-L301 infusion demonstrated by: ◦ Normalized hemoglobin ◦ Improved hemolysis parameters ◦ No re

  15. Slide 15: Acknowledgements

    Acknowledgements José Luis Lopez Lorenzo, MD Lucía Llanos, MD, PhD Maria Grazia Roncarolo, MD Bert Glader, MD, PhD Susana Navarro, PhD Begoña Pérez Camino de Gaisse, MD May Chien, MD José-Carlos Segovia, PhD Sol Sanchez, MD Elisabeth Merkel, RN Javier Cornago, MD Eileen Nicoletti, MD Gayatri R. Rao,

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