Rocket Pharmaceuticals, Inc. · NASDAQ:RCKT
Rocket Pharmaceuticals — Investor Presentation (September 2022)
59 slides from Rocket Pharmaceuticals's investor presentation, Sep 30, 2022
Slide 1: Seeking Gene Therapy Cures Investor Deck
INVESTOR DECK INVESTOR SEEKING DECK CURES GENE THERAPY INVESTOR DECK
Slide 2: Disclaimer
DISCLAIMER Various statements in this release concerning Rocket’s future expectations, plans and prospects, including without limitation, Rocket’s expectations regarding its guidance for 2022, the safety and effectiveness of RP-A501 for the potential treatment of Danon Disease, trends for RP-A501 sa
Slide 3: About Rocket Pharmaceuticals
ABOUT ROCKET PHARMACEUTICALS “For the first time in history, we are discussing not just effective treatments but potential cures at the genetic level, which is the deepest essence of who we are as physical beings.” — GAURAV SHAH, MD | CEO 3
Slide 4: Vision: Seeking Gene Therapy Cures
ABOUT ROCKET PHARMACEUTICALS Vision: Seeking Gene Therapy Cures Values Mission To develop first-in-class and best-in-class curative v Curiosity gene therapies for patients Trust Elevate with devastating diseases Generosity 4
Slide 5: Generating Value-based Gene Therapies
ABOUT ROCKET PHARMACEUTICALS Generating Value-based Gene Therapies Therapeutic area focus: Promising top-line clinical data designed Late-stage Heart and Bone Marrow to facilitate US & European registration & Science and Only company with safety and ???? into launch with potential for expansion effi
Slide 6: Expert Leadership With Proven Track Record
ABOUT ROCKET PHARMACEUTICALS Expert Leadership With Proven Track Record Gaurav Shah, M.D. Chief Executive Officer Spearheaded Kymriah (CART-19) development at Novartis towards approval Kinnari Patel, Pharm.D., MBA Mayo Pujols President and Chief Operating Officer Chief Technical Officer, EVP Led Opd
Slide 7: Strong Science, Carefully-selected Assets and Smart Execution
ABOUT ROCKET PHARMACEUTICALS Strong Science, Carefully-selected Assets and Smart Execution: Four Programs With Compelling Clinical POC Criteria used to select programs Five programs with compelling clinical proof of concept First-, best- and only-in-class On-target MOA; clear endpoints Sizeable mark
Slide 8: Developing First-, Best- and Only-in-Class Therapies for Rare Diseases
ABOUT ROCKET PHARMACEUTICALS Developing First-, Best- and Only-in-Class Therapies for Rare Diseases With Extensive Unmet Needs Strong science, carefully-selected Proven management assets and smart execution expertise • Right technology for the target • Strong drug development track record, successfu
Slide 9: Strategically Building a Leading Gene Therapy Company
ABOUT ROCKET PHARMACEUTICALS Strategically Building a Leading Gene Therapy Company EMA RMAT, FDA Manufacturing PRIME for LAD-I readiness achieved Data updates: Commercial readiness ASGCT, ESGCT in progress for Danon disease EMA PRIME for FA AHA, ASH upcoming launch IND cleared Added clinical sites:
Slide 10: Strong, Strategic Approach to Gene Therapy Manufacturing
ABOUT ROCKET PHARMACEUTICALS Strong, Strategic Approach to Gene Therapy Manufacturing In-house capabilities Streamlined manufacturing AAV cGMP Process capabilities to allow for Development, manufacturing with capabilities to support Analytics and cost-effective commercial products QC testing commerc
Slide 11: World-class Scientific Experts and Partners
ABOUT ROCKET PHARMACEUTICALS World-class Scientific Experts and Partners Stanford Medicine 11 Data on file. Rocket Pharmaceuticals. 2022.
Slide 12: Unmet Needs and Market
UNMET NEEDS AND MARKET “Caring for someone with Danon, while you, yourself have Danon is very hard. Most days we are at clinic appointments or having a procedure done to check on our hearts. The other times we are at home dealing with chest pain, rapid heart rates, muscle pains and learning issues i
Slide 13: Rare Diseases Are Associated With a Reduced Lifespan
UNMET NEEDS AND MARKET Rare Diseases Are Associated With a Reduced Lifespan1 400 million people globally are affected by a rare disease1 Children 80% of rare diseases have monogenic origins1 account for 50% of rare disease patients1 3 of 10 children with a rare disease die before Only about 5% of ra
Slide 14: Market for Rare Disease Treatment Is Rising
UNMET NEEDS AND MARKET Market for Rare Disease Treatment Is Rising Rare disease treatment market by drug type, 2019 (USD million)1 Rare disease treatment market by region, 2015-2026 (USD million)1 • Rare disease treatment market is projected to grow from $161.4 billion in 2020 to $547.5 billion by 2
Slide 15: Costs Associated With Rare Diseases Have Increased Exponentially
UNMET NEEDS AND MARKET Costs Associated With Rare Diseases Have Increased Exponentially1 Economic impact1 Emotional impact4 26-fold increase in average per- Feelings of depression T: 71% UT: 81% 75% patient annual cost for orphan T: 66% UT: 75% 69% drugs* compared to doubled Feelings of anxiety/stre
Slide 16: Pioneering Gene Therapy Clinical Programs
PIONEERING GENE THERAPY CLINICAL PROGRAMS “Due to the high unmet need, there is significant interest within the FA community from both patients and health care providers for an alternative low-toxicity therapy to address and, more specifically, prevent BMF. Overall, the investigational gene therapy
Slide 17: Rocket Offers Multi-platform Gene Therapy Expertise
CLINICAL PROGRAMS Rocket Offers Multi-platform Gene Therapy Expertise IN VIVO platform EX VIVO platform Laboratory- Remove cells Laboratory- produced AAV and isolate produced LV patient HSCs Therapeutic Therapeutic LV AAV Infusion of modified Direct HSCs intravenous Gene-modified injection HSCs RP-L
Slide 18: In Vivo Platform: Adeno-associated Virus (AAV)
CLINICAL PROGRAMS In Vivo Platform: Adeno-associated Virus (AAV) IN VIVO (inside the body) DANON DISEASE AAV gene therapy Multi-system disorder with severe cardiomyopathy • Transduction of non-dividing, terminally differentiated cardiomyocytes Laboratory- • AAV9 serotype has been shown to have a par
Slide 19: Danon Disease: Serious Condition with Unmet Medical Need
RP-A501: Danon Disease Danon Disease (DD): Serious Condition with Unmet Medical Need Disease Etiology Therapeutic Challenges • X-linked, dominant, • Standard of care: monogenic disease o Heart transplant (HTx) • Loss-of-function • Limitations: mutations in LAMP-2B o Considerable morbidity and mortal
Slide 20: RP-A501: Prospect of Direct Benefit
RP-A501: Danon Disease RP-A501: Prospect of Direct Benefit Trajectory of cardiac pathophysiology and heart failure in male Danon disease patients Gene therapy (hypothesized optimal treatment interval) Symptom onset Diagnosis Stabilization or improvement Cardiac Pathophysiology Progression to end-sta
Slide 21: Pathophysiology and Clinical Manifestations of Danon Disease HCM
RP-A501: Danon Disease Pathophysiology and Clinical Manifestations of Danon Disease HCM Pathophysiology Mechanism Patient impact Pathogenic LAMP-2B mutation causing DD Disorganized myocytes disrupt signal conduction Ventricular arrhythmias Sudden cardiac No Cardiac LAMP2B protein expression arrest M
Slide 22: Phase 1 Study: Non-Randomized Open Label
RP-A501: Danon Disease Phase 1 Study: Non-Randomized Open Label Non-randomized open label study in male DD patients INCLUSION CRITERIA • Male PRIMARY OUTCOMES • Confirmed LAMP-2B mutation Adults (& Single Intravenous • Early and long-term safety • Cardiac involvement Adolescents) Dose of RP-A501 con
Slide 23: RP-A501: Safety Monitoring of Phase 1 Patients
RP-A501: Danon Disease RP-A501: Safety Monitoring of Phase 1 Patients Cohort 1: Low-dose adult RP-A501 SAEs of Interest Platelets Pt Age Wt 6.7 x 1013 GC/kg n=2 grade 3 steroid myopathy n=2, 1001a 17.5 52.5 Steroid (prednisone 1mg/kg) Grade 1 1002 20.4 89.1 decrease 1005 18.3 91.8 Tacrolimus (to LLN
Slide 24: RP-A501 Generally Well Tolerated in Pediatric Cohort on Enhanced Immunomodulation
RP-A501: Danon Disease RP-A501 Was Generally Well Tolerated in Pediatric Cohort on Enhanced Immunomodulation All AEs were Transient and Reversible with 6 and 11 month follow up in 1008 and 1009, respectively Pediatric immunomodulation protocol sC5b9 Evaluation Post RP-A501 RP-A501 Daily, real-time 1
Slide 25: Early Pediatric Data are Encouraging and Consistent with Adult Efficacy
RP-A501: Danon Disease Early Pediatric Data are Encouraging and Consistent with Adult Efficacy Patient ID: A501-008-1008 Patient ID: A501-008-1009 Baseline Characteristics Baseline Characteristics AGE AT INFUSION MAX LV WALL THICKNESS AGE AT INFUSION MAX LV WALL THICKNESS 12.3 years 41.9 mm, z-score
Slide 26: Early Pediatric LAMP2 Expression Consistent with Adult Data
RP-A501: Danon Disease Early Pediatric LAMP2 Expression are Encouraging and Consistent with Adult Data Quantified LAMP2 protein expression by immunohistochemistry (IHC) Initial Biopsy Cohort Patient ID Post Infusion 1008 Month 3: 18.5%* Pediatric - Low Dose 1009 Month 3: 34.7% 1001 Month 2: 7.3% Adu
Slide 27: LAMP2 Myocardial Protein Expression and Histologic Improvement in Pediatric Cohort
RP-A501: Danon Disease LAMP2 Myocardial Protein Expression and Histologic Improvement in the Pediatric Cohort A501-008-1008 Endomyocardial Biopsy (EMB) Images Baseline Month 3 Month 6 LAMP2 protein expression assessed (relative to normal Immunohistochemistry human controls) by core lab in a blinded
Slide 28: Pediatric LAMP2 Protein and DNA Suggests Durable Expression
RP-A501: Danon Disease Pediatric LAMP2 Protein and DNA Suggests Durable Expression As Demonstrated in Adult Cohort LAMP2 Protein Expression Cardiac LAMP2 DNA by qPCR (vector copies per diploid nucleus) 1001 4 1002 Patient ID Predose Month 6 Month 12 Month 36 1005 1006 1001a 0 0.384 0.197 0.120 3 100
Slide 29: Restored Autophagy is Sustained Following RP-A501
RP-A501: Danon Disease Restored Autophagy is Sustained Following RP-A501 Restored autophagy indicated by attenuation of vacuolar area Light microscopy images at 20X; M24 Autophagic vacuoles are depicted by yellow arrows. A. Vacuolar Area of Endomyocardial Tissue B. Vacuolar Area Decreases with Treat
Slide 30: Sustained Improvement of Biomarkers of Myocardial Injury and Stress
RP-A501: Danon Disease Sustained Improvement or Stabilization of Biomarkers of Myocardial Injury and Stress Following RP-A501 High Sensitivity Troponin I (hsTnI) Brain Natriuretic Peptide (BNP) 25 25 M36 M30 M12 M18 M24 M30 M12 M18 M24 M30 M18 M12 M18 M24 M18 M24 M30 M36 M12 M18 M24 M30 M18 M30 M12
Slide 31: Sustained Improvement or Stabilization of LV Hypertrophy
RP-A501: Danon Disease Sustained Improvement or Stabilization of LV Hypertrophy Following RP-A501 LV Posterior Wall Thickness LV Mass 25 25 M12 M6 M9 M6 M9 M12 M18 M24 M30 M36 M18 M24 M30 M12 M18 M18 M6 M18 M24 M30 M36 M18 M24 M30 M18 M12 M18 M9 M6 M9 0 0 % Change from Baseline % Change from Baselin
Slide 32: Sustained Improvement of Functional Cardiac Status
RP-A501: Danon Disease Sustained Improvement or Stabilization of Functional Cardiac Status Following RP-A501 New York Heart Association Class Kansas City Cardiomyopathy Questionnaire Overall Score Most Time of Most Patient Month Recent Recent Cohort ID Baseline 12 Follow-up Follow-up 100 1001
Slide 33: Summary of Results and Conclusions
RP-A501: Danon Disease Summary of Results and Conclusions PEDIATRIC COHORT ADULT COHORT • RP-A501 was well tolerated • Low-dose continues to be • No immediate, early or delayed RP- generally well tolerated at 2-3 A501 related SAEs observed to date years post-treatment with enhanced immunomodulation
Slide 34: Connecting Surrogate Endpoints to Functional Outcomes for Pivotal Study
RP-A501: Danon Disease Connecting Surrogate Endpoints to Functional Outcomes for Pivotal Study* Echo Parameters: Wall thickness (LVPWd, MLVWT) Functional parameters: LVEF NYHA Class LV mass KCCQ Overall Score Hemodynamics: PCWp Clinical Status Cardiac output Serology: Troponin-I Cardiac Structure an
Slide 35: RP-A501-0219 Patient Anecdotes
RP-A501: Danon Disease RP-A501-0219 Patient Anecdotes Patient Anecdote He can walk upstairs without being short of breath or having to stop half-way. He doesn’t have chest pain 1005 or fast heart rates like he used too. Another amazing thing we have seen is about 4 months after his therapy trial he
Slide 36: Summary of Results and Conclusions
RP-A501: Danon Disease Summary of Results and Conclusions Phase 1 enrollment and treatment are complete The enhanced immunomodulatory regimen was well tolerated and has effectively mitigated adverse events in the pediatric cohort, who are currently 6 to 11 months post treatment The early LAMP2 e
Slide 37: Development Plan
RP-A501: Danon Disease Development Plan Moving toward pivotal Phase 2 study CURRENT PLANNED • Phase 1 treatment completed in • Expanded natural history study males • End of Phase 1 Regulatory meeting with FDA PLANNED GLOBAL • Orphan Drug, Rare Pediatric and • Initiate Phase 2 Global Pivotal Study Ac
Slide 38: Ex Vivo Platform: Lentiviral Vector (LV)
CLINICAL PROGRAMS Ex Vivo Platform: Lentiviral Vector (LV) EX VIVO (outside the body) Fanconi Anemia, Leukocyte Adhesion Deficiency-I and LV gene therapy Pyruvate Kinase Deficiency Laboratory- Remove cells • HSCs transduced with a lentiviral vector carrying the produced LV and isolate corrected gene
Slide 39: RP-L102 for Fanconi Anemia Complementation Group A (FA-A)
RP-L102: PROGRAM Fanconi 2: RP-L102 forAnemia FA RP-L102 for Fanconi Anemia Complementation Group A (FA-A) Disease etiology Therapeutic challenges • FA-A is an autosomal • Standard of care: recessive disease • Allogeneic HSCT caused by FANCA • Limitations: gene mutations o Significant toxicities, es
Slide 40: Clinical Studies Overview
RP-L102: PROGRAM Fanconi 2: RP-L102 forAnemia FA Clinical Studies Overview Description Autologous HSCs transduced with LV carrying FANCA transgene Conditioning is not required because gene-corrected HSCs display proliferative advantage over time Clinical studies Primary endpoints*: Safety • EU FANCO
Slide 41: Progressive Increase in Peripheral Blood and Bone Marrow VCNs
RP-L102: PROGRAM Fanconi 2: RP-L102 forAnemia FA Progressive Increase in Peripheral Blood and Bone Marrow VCNs Progressive increases in gene markings in peripheral blood and bone marrow cells in 6 patients Peripheral blood VCN Bone marrow VCN VCN in peripheral blood mononuclear cells VCN in bone mar
Slide 42: Strong Evidence for Phenotypic Reversal
RP-L102: PROGRAM Fanconi 2: RP-L102 forAnemia FA Strong Evidence for Phenotypic Reversal Increasing phenotypic correction over 1 to 2 years post RP-L102* in 5 of initial 9 evaluable patients For 5 patients, Patient 1 (1001) Patient 3 (2004) increased BM CFC Patient 4 (2008) MMC resistance Patient 5
Slide 43: Blood Count Stabilization and Sustained Phenotypic Reversal
RP-L102: PROGRAM Fanconi 2: RP-L102 forAnemia FA Blood Count Stabilization and Sustained Phenotypic Reversal Leukocytes Neutrophils Hemoglobin Platelets Increased MMC resistance Patient 3 in BM CFCs associated (2004) with hematologic stabilization at ≥1 year Patient 4 post RP-L102 (2008) Concomitant
Slide 44: Development Plan
RP-L102: PROGRAM Fanconi 2: RP-L102 forAnemia FA Development Plan Moving toward BLA/MAA filing INITIAL EFFICACY AND HIGHLY REGULATORY TOP-LINE DATA READOUT FAVORABLE SAFETY PROFILE DESIGNATIONS ACHIEVED • Initial comprehensive • RMAT, PRIME Rejection of null hypothesis with efficacy in 5/9 evaluable
Slide 45: RP-L201 for LAD-I: ITGB2 Gene Mutation
PROGRAM RP-L201: 2: RP-L102LAD-I for FA RP-L201 for LAD-I: ITGB2 Gene Mutation Disease etiology Therapeutic challenges • ITGB2 gene mutations • Standard of care: (21q22.3), encoding the Allogeneic HSC transplant beta-2-integrin, CD18; • Limitations: essential for leukocyte o Donor availability adhes
Slide 46: Clinical Study Overview
RP-L201: LAD-I Clinical Study Overview Description Autologous HSCs transduced with LV carrying ITGB2 transgene Clinical study Key efficacy data Treatment completed • 100% overall survival Phase 1/2 (N=9) Selected secondary endpoints: Primary endpoints: • CD18 expression • Efficacy evident in 9/9 pat
Slide 47: Sustained CD18 Expression in Peripheral Blood PMNs
RP-L201: LAD-I Sustained CD18 Expression in Peripheral Blood PMNs At 3 to 24 months after infusion, 9/9 patients sustained stable CD18 expression L201-003-1001 (median: 56%) with no therapy-related serious adverse events L201-003-1004 L201-003-2005 L201-003-2006 L201-003-2007 L201-004-2008 L201-004-
Slide 48: Sustained VCN in PBMCs
RP-L201: LAD-I Sustained VCN in PBMCs At 3 to 24 months after infusion, CD18 expression is accompanied by stable PB VCN L201-003-1001 L201-003-1004 (range: 0.5 – 3.9; median: 1.2 copies per PB mononuclear cell) L201-003-2005 L201-003-2006 L201-003-2007 L201-004-2008 L201-004-2009 L201-004-2010 L201-
Slide 49: Significant Reduction in Hospitalizations and 100% Overall Survival
RP-L201: LAD-I Significant Reduction in Hospitalizations and 100% Overall Survival 100% overall survival Kaplan–Meier estimate Significant reduction in incidence of hospitalizations Survival without allogeneic HSCT Post engraftment Primary outcomes • ≥2 years of age AND • ≥1-year post–RP-L201 infusi
Slide 50: Development Plan
RP-L201: LAD-I Development Plan Moving toward BLA/MAA filing ENROLLMENT AND REGULATORY TOP-LINE DATA READOUT INITIAL EFFICACY DESIGNATIONS 2Q 2022 • Enrollment completed; • RMAT, PRIME • Survival for 9/9 patients, ≥2 years 9/9 patients treated • Fast Track and ATMP age and ≥1 year post-treatment • E
Slide 51: RP-L301 for PKD: PKLR Gene Mutation
RP-L301: PROGRAM 4: PKD RP-L301 for PKD RP-L301 for PKD: PKLR Gene Mutation Disease etiology Therapeutic challenges • Autosomal recessive • Standard of care: Chronic inheritance blood transfusions and • Pyruvate kinase splenectomy deficient RBCs cannot • Limitations: synthesize ATP, o Iron overload
Slide 52: Clinical Study Overview
RP-L301: PROGRAM 4: PKD RP-L301 for PKD Clinical Study Overview Description Autologous HSCs transduced with LV containing human PKLR transgene Clinical study N = 4-5 (Phase 1) Phase 1 cohort dosing plan Primary endpoints: • Safety 1st cohort 2nd cohort 3rd cohort • Toxicity Adults (N=2) Adolescent (
Slide 53: Hemoglobin Normalization and Transfusion Independence
RP-L301: PROGRAM 4: PKD RP-L301 for PKD Hemoglobin Normalization and Transfusion Independence Hemoglobin improvement to normal range (from baselines in severe (<8g/dL range) Transfusion independence (extensive transfusion requirements prior to RP-L301) Sustained improvement of hemolysis markers (LDH
Slide 54: Development Plan
RP-L301: PROGRAM 4: PKD RP-L301 for PKD Development Plan Moving toward pivotal Phase 2 study PKD STUDY PROGRESS TO PHASE 2 AND LAUNCH LIFE-CYCLE Key endpoints selected MANAGEMENT • Hemoglobin increase • ↓ 50% transfusions or transfusion independence ANTICIPATED EXPANSION STUDY TO Well-delineated nat
Slide 55: Future Directions
FUTURE DIRECTIONS pharma
Slide 56: Rocket Pharmaceuticals: Elevating Gene Therapy to New Heights
FUTURE DIRECTIONS Rocket Pharmaceuticals: Elevating Gene Therapy to New Heights • Recognized as a premier gene therapy • Commercial company with company initial therapies and revenue build for Danon disease, FA, LAD-I and PKD • Specialized against monogenic diseases • Broad pipeline of additional ne
Slide 57: Anticipated Milestones and Wave 2
FUTURE DIRECTIONS Anticipated Milestones and Wave 2 2022 2023 2024 Q2 2022 Q3 2022 Q4 2022 • LAD-I: BLA/MAA filings in 1H’23 • Label expansion • LAD-I: Top-line • Danon disease: • FDA EOP1 agreement studies for data will be Pediatric and initiation of Phase • FA: BLA/MAA filings Danon disease achiev
Slide 58: Future Therapies: Wave 2 (AAV)
FUTURE DIRECTIONS Future Therapies: Wave 2 (AAV) Focused R&D Strategy for Current Clinical Sustainable Innovation Pipeline First-, best- and only-in-class On-target MOA; clear endpoints Sizeable market to maximize patient impact We continue to build our pipeline based on our core R&D strategy; ident
Slide 59: Thank You!
THANK YOU! rocket
Rocket Pharmaceuticals — Investor Presentation (September 2022) on 6ix