Rocket Pharmaceuticals, Inc. · NASDAQ:RCKT
Rocket Pharmaceuticals — American Society of Gene and Cell Therapy (ASGCT) 2022
14 slides from Rocket Pharmaceuticals's conference presentation, May 16, 2022
Slide 1: Gene Therapy for Fanconi Anemia (Group A): Interim Results of RP-L102 Clinical Trials
Gene Therapy for Fanconi Anemia (Group A): Interim Results of RP-L102 Clinical Trials Agnieszka Czechowicz, MD, PhD Center for Definitive and Curative Medicine Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School
Slide 2: Disclosures
Disclosures • Consultant: Beam Therapeutics, GV, Spotlight Therapeutics, Stemodontics • Current Equity: Beam Therapeutics, Decibel Therapeutics, Editas Medicine, Global Blood Therapeutics, GV, Magenta Therapeutics, Spotlight Therapeutics, Stemodontics, Divested Equity in last 24 months: Forty Seven,
Slide 3: Introduction
Introduction Fanconi anemia (FA) is a rare inherited FA represents a significant unmet medical need disorder of defective deoxyribonucleic acid (DNA) repair characterized by: • FA complementation group A (FA-A) accounts for 60–70% of FA; prevalence of ~4,000 cases in US and Europe • Progressive bone
Slide 4: RP-L102 Rationale and Study Design
RP-L102 Rationale and Study Design • Insertion of a functional FANCA gene into Schematic of Product Manufacture and Treatment autologous FA-A CD34+ cells confers resistance to 1. Mobilization of CD34+ cells DNA-damage & provides proliferative advantage (G-CSF + Plerixafor) to modified cells • Enable
Slide 5: Global RP-L102 Studies
Global RP-L102 Studies Key Eligibility Criteria Endpoints Inclusion: Efficacy: • FA complementation group A • Minimum age: 1y Engraftment: Peripheral blood (PB) and BM vector • Maximum age: US Ph 1 (12y); US Ph 2 copy number (VCN) (none); EU Ph 2 (17y) Phenotypic correction: Increased resistance of
Slide 6: Patients Treated: Demographics and Investigational Product Metrics for Initial n=9
12 Patients Treated: Demographics and Investigational Product Metrics for Initial n=9 Mean VCN: Age at Transduction CFC Survival All patients ≤6 years at Enrollment Follow Up Liquid Efficiency MMC enrollment Subject # (years) (months) CD34+ Cells/kg CFCs/kg Culture CFCs (%) 10nM (%) 9 patients have
Slide 7: Sustained Genetic Correction in 6 of 9 Patients ≥1 year post-RP-L102
Sustained Genetic Correction in 6 of 9 Patients ≥1 year post-RP-L102 PBMC VCN BM VCN Progressive increases in gene markings in PB and BM in 6 patients Not shown: PB and BM VCN in Patient 2 (1002), who was withdrawn from the study at 18 months post-RP-L102 infusion 7 Proprietary and Confidential
Slide 8: Increasing Phenotypic Correction over 1-2 years post-RP-L102
Increasing Phenotypic Correction over 1-2 years post-RP-L102 BM MMC-resistance * For 5 pts, increased BM CFC MMC resistance ranging from 51% to 94% observed at 18–24 months post-RP-L102 administration MMC resistance of >20% achieved at two consecutive timepoints ≥12 months for n=5 * BM MMC-res for p
Slide 9: BM CFC MMC Resistance is Associated with Long-term Hematologic Stability
BM CFC MMC Resistance is Associated with Long-term Hematologic Stability FANCOLEN-I (investigator-initiated) long-term follow-up: BM MMC-r leukocytes neutrophils hemoglobin platelets FA-2002 (246,000 cCD34+/Kg) Iron supplement FA-2006 (410,000 cCD34+/Kg) Months Post-Gene Therapy ≥20% BM CFC MMC resi
Slide 10: Genetic Correction Correlates with Phenotypic Improvement
Genetic Correction Correlates with Phenotypic Improvement - PB VCN strongly correlates with BM CFC MMC-Res at 12 months post-RP-L102 (r = 0.83)+* - BM Mononuclear cell VCN strongly correlates with BM CFC MMC-Res at 12 months post-RP-L102 (r = 0.81)** Patient 3 (2004) Patient 4 (2008) Patient 5 (2009
Slide 11: Increased BM CFC MMC Resistance Associated with Hematologic Stabilization
Increased BM CFC MMC Resistance Associated with Hematologic Stabilization at ≥1 year post RP-L102 Leukocytes Neutrophils Hemoglobin Platelets Patient 3 (2004) Patient 4 (2008) Concomitant blood count stabilization over 12–24 months seen in 5/5 Patient 5 (2009) patients with sustained and increasing
Slide 12: RP-L102 Safety Profile Appears Highly Favorable
RP-L102 Safety Profile Appears Highly Favorable • Patients are treated without antecedent conditioning and attendant risks • No signs of dysplasia, clonal dominance or oncogenic integrations • Gene therapy does not preclude subsequent allogeneic HSCT if necessary • Patient 2 (1002) underwent success
Slide 13: Conclusions from Initial 9 Patients with ≥1 Year of Follow-Up
Conclusions from Initial 9 Patients with ≥1 Year of Follow-Up Comprehensive efficacy in multiple patients with >1 year of follow-up • 5 patients have sustained, increasing BM CFC MMC resistance ranging from 51 to 94% at 18–24 months, and ≥20% at two consecutive timepoints • Increasing BM CFC MMC res
Slide 14: Acknowledgements
Acknowledgements Julián Sevilla, MD, PhD Josune Zubicaray, MD Maria Grazia Roncarolo, MD, PhD Elena Sebastian, MD, PhD Rajni Agarwal, MD Juan Bueren, PhD Elisabeth Merkel, RN Paula Río, PhD Susana Navarro, PhD Claire Booth, MBBS, PhD, MSc, FRCPCH John E. Wagner, MD Eileen Nicoletti, MD Philip Anclif
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