Rocket Pharmaceuticals, Inc. · NASDAQ:RCKT
Rocket Pharmaceuticals — Investor Day (December 2021)
19 slides from Rocket Pharmaceuticals's investor day presentation, Dec 14, 2021
Slide 1: Rocket Program Updates for Fanconi Anemia, Pyruvate Kinase Deficiency and LAD-I
Rocket Program Updates for Fanconi Anemia, Pyruvate Kinase Deficiency and Leukocyte Adhesion Deficiency-I December 14, 2021 SEEKING GENE THERAPY CURES 1 NASDAQ: RCKT
Slide 2: Cautionary Statement Regarding Forward-Looking Statements
Cautionary Statement Regarding Forward-Looking Statements Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2021 in light of COVID-19, the safety, effectiveness and timing of
Slide 3: Summary of Rocket Lentiviral Program Updates and Status
Summary of Rocket Lentiviral Program Updates and Status Fanconi anemia (Pivotal Phase II): • 6 of 8 initial patients with ≥12 months follow-up display evidence of engraftment o Increasing BM MMC-resistance and BM/PB VCN o BM MMC-resistance appears associated with hematologic stability • No condition
Slide 4: Gene Therapy for Fanconi Anemia (Group A): Preliminary Results of Ongoing RP-L102 Clinical Trials
Gene Therapy for Fanconi Anemia (Group A): Preliminary Results of Ongoing RP-L102 Clinical Trials Jonathan Schwartz, M.D. Chief Medical Officer, Senior Vice President 4
Slide 5: Current Status of Patients
Current Status of Patients 11 patients have received RP-L102 8 patients with 12-32 months of follow up 3 patients with < 12 months of follow up* 1 (1001) 9 (2016) *Reflects one additional patient treated after ASH Included in efficacy 3 (2004) 10 (2021) data cutoff 1-NOV-2021 parameter analyses: 11
Slide 6: RP-L102 Study Patients with ≥12m Follow Up Demonstrate Evidence of Genetic and Phenotypic Correction
RP-L102 Study Patients with ≥12m Follow Up Demonstrate Evidence of Genetic and Phenotypic Correction Bone Marrow Patient BM CFC MMC Assessment Subject # Age at Resistance at 10 Performed Treatment nM MMC (%) (months) 1 (1001) 5 24 16† 3 (2004) 3 21 63 4 (2008) 2 12 21 5 (2009) 3 12 29 • Sustained PB
Slide 7: Blood Count Stabilization in at Least 5 Patients with BM CFC MMC Resistance ≥10%
Blood Count Stabilization in at Least 5 Patients with BM CFC MMC Resistance ≥10% Subject 3 (2004) Subject 4 (2008) Subject 1 (1001) • Blood count stabilization in 5/5 patients with BM CFC MMC Resistance ≥ 20% • Patient 1 (1001; MMC-res 16%) had modest decline in blood Subject 5 (2009) Subject 6 (201
Slide 8: Preliminary Associations Between BM & VCN Efficacy Parameters at 12m
Preliminary Associations Between BM & VCN Efficacy Parameters at 12m PB VCN vs. BM VCN at 12 months BM MMC-Resistance vs. PB VCN at 12 months R2 = 0.9634 R2 = 0.6348 L102-001-2010 L102-001-2010 L102-001-2011 L102-001-2011 L102-002-2004 L102-002-2009 L102-002-2004 L102-001-1001 L102-002-2009 L102-001
Slide 9: Fanconi Anemia Summary
Fanconi Anemia Summary • Of 11 patients treated to date: - 6 of 8 patients with ≥12m of follow-up display evidence of engraftment - 1 patient’s course (1002) complicated by Influenza B infection & BMF; required BMT and engrafted without complications • Increasing BM CFC MMC resistance seen in 6 pati
Slide 10: Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric Patients
Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric Patients Kinnari Patel, Pharm.D., M.B.A. Chief Operating Officer, President 10
Slide 11: Preliminary Efficacy Results: L301-006-1001
Preliminary Efficacy Results: L301-006-1001 --- Normal Range (0.3–1.2 mg/dL) --- Normal Range (0.5–1.5%) * 20 pRBC units transfused Transfusion independence over 30m period Significant bilirubin decrease from Sustained reduction in reticulocytes • Sustained hemoglobin improvement from ~7.4 g/dL to 1
Slide 12: 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%) ~7 transfusion events over Transfusion independence 50m period Normalized bilirubin from 7.4 Significant improvement in mg/dL to 1.4 mg/dL reticulocytes from 25.5% to 2.73% • Hemoglobin normalize
Slide 13: Preliminary Results Indicate Improved Quality of Life During One Year Subsequent to RP-L301 therapy
Preliminary Results Indicate Improved Quality of Life During One Year Subsequent to RP-L301 therapy Functional Assessment of Cancer Therapy-Anemia (FACT-An) L301-006-1001 L301-001-1002 Both patients have anecdotally reported improved quality of life (QoL) following RP-L301 administration; improved Q
Slide 14: RP-L301 Efficacy and Safety Summary
RP-L301 Efficacy and Safety Summary • One-year follow-up in both initial adult patients indicates sustained efficacy: • Doubling of pre-treatment baseline hemoglobin to normal-range sustained at 12M and substantial improvement in markers of hemolysis • Improvements in patient-reported QOL assessment
Slide 15: Leukocyte Adhesion Deficiency-1: Interim Results of a Global Phase I/II Pivotal Phase II Study
Leukocyte Adhesion Deficiency-1: Interim Results of a Global Phase I/II Pivotal Phase II Study Gaurav Shah, M.D. Chief Executive Officer 15
Slide 16: RP-L201 Clinical Trial Design & Outcomes Measures
RP-L201 Clinical Trial Design & Outcomes Measures Trial Design Patient Sex Age at Drug Product CD34+ Cell Dose enrollment VCN • Non-Randomized Global Phase 1/2 Study (n=9) 4.2 x 106 L201-003-1001 F 9 yrs. 3.8 Primary Outcomes cells/kg 2.8 x 106 L201-003-1004 F 3 yrs. 2.5 • Phase 1: cell
Slide 17: LAD-1: Clinical Efficacy Overview
LAD-1: Clinical Efficacy Overview % CD18 Expression (PMN) In Peripheral Blood VCN (PBMC) Dim/weak CD18 expression reported at baseline WBC in Peripheral Blood for subject L201-003-1004 in ~63% of cells • As of December 2021: Data reported in 8 of 9 patients with 3-24m follow-up • Final trial subject
Slide 18: RP-L201 Clinical Safety & Efficacy Summary
RP-L201 Clinical Safety & Efficacy Summary • Trial enrollment and dosing complete; 9 of 9 severe LAD-I patients successfully dosed with RP-L201. • Infusion has been well tolerated with no drug product-related SAEs. • Safety profile of RP-L201 appears favorable • 8 of 9 patients have at least 3 month
Slide 19: Question & Answer Session
Question & Answer Session Gaurav Shah, M.D. Chief Executive Officer Kinnari Patel, Pharm.D., M.B.A. Chief Operating Officer, President Jonathan Schwartz, M.D. Chief Medical Officer, Senior Vice President 19
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