Rocket Pharmaceuticals, Inc. · NASDAQ:RCKT
Rocket Pharmaceuticals — 64th Annual Meeting of the American Society of Hematology (ASH) 2022
16 slides from Rocket Pharmaceuticals's conference presentation, December 2022
Slide 1: Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe LAD-I: Interim Results
Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an ongoing Phase 1/2 Study Donald B. Kohn, MD, Professor Departments of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Pharmac
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
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) and prevent functional • 60–75% pts with severe LAD-
Slide 4: Gene Therapy for LAD-I: RP-L201
Gene Therapy for LAD-I: RP-L201 Ex-vivo lentiviral vector gene therapy consists HSC ENRICHMENT & 3 TRANSDUCTION of autologous CD34+ cells transduced with with lentiviral vector, a lentiviral vector (Chim-CD18-WPRE LV) Chim-CD18-WPRE encoding the CD18 (β-subunit) component LV (LV-RP-L201) of the β2-i
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 L20
Slide 6: VCN in Peripheral Blood Mononuclear Cells (PBMCs)
VCN in Peripheral Blood Mononuclear Cells (PBMCs) 6 Proprietary and Confidential Data cut-off: April 6, 2022; Preliminary interim results are presented from the ongoing clinical study.
Slide 7: CD18 Expression in PB Polymorphonuclear Cells (PMNs)
CD18 Expression in PB Polymorphonuclear Cells (PMNs) * Re-added marker borders to graph and legends. I think it will help make these be more visible to the ballroom/exhibition hall audience. * Dim/weak CD18 expression reported at baseline for Subject L201-003-1004 in ~63% of cells in conjunction wit
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 * L201-003-2005 L201-003-2007 L201-004-2008 Re-added marker borders to graph and legends. I think it will help make these be more visible to the ballroom/exhibition hall audience. L201-004-2009 L201-003-2011 L201-004-2010
Slide 9: Resolution of LAD-I Related Abnormal Leukocytosis
Resolution of LAD-I Related Abnormal Leukocytosis: A clinical biomarker of a normalized phenotype Age at Enrollment Normal Leukocyte Ranges per Age Group: 0 months to <3 months: 7.20–18.00×1000/μL; ≥3 months to <6 months: 6.70–14.00×1000/μL; ≥ 6months to 12 months: 6.40–13.00×1000/μL; 12 months to <
Slide 10: 100% Overall Survival one-year post-RP-L201 and to 2 years of age
100% Overall Survival one-year post-RP-L201 and to 2 years of age Overall Survival (OS) Kaplan-Meier Estimate Survival without allogeneic HSCT Primary Outcomes • >2 years of age • 1-year post-RP-L201 infusion 10 Proprietary and Confidential Data cut-off: March 9, 2022; Preliminary interim results ar
Slide 11: RP-L201 Clinical Outcome Measures: All Hospitalizations and Infections
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
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) Incidence of Severe Infections Hospitalization 2.5 p: <0.0001 2.0 Exposure Adjusted Event Rate Exposure Adjusted Event Rate p: <0.0012 2.0 1.5 1.5 1.0 1.0 0.5 0.5 p: <0.00
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-RP-L201 P
Slide 14: Clinical Safety Overview
Clinical Safety Overview As of April 6, 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
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
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
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