Slide 1: 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 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to
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 living 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: Four Programs With Compelling Clinical POC
ABOUT ROCKET PHARMACEUTICALS Strong Science, Carefully-selected Assets and Smart Execution: Four Programs With Compelling Clinical POC Criteria used to select programs Four programs with compelling clinical proof of concept PHASE 2 US BLA/ DISCOVERY PRECLINICAL PHASE 1 (Pivotal) EU MAA Filing DESIGN
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: RP-A501 for Danon Disease: LAMP2B Gene Mutation
RP-A501: Danon Disease RP-A501 for Danon Disease: LAMP2B Gene Mutation Disease etiology Therapeutic challenges • Standard of care: • LAMP2 mutation o Heart transplant • Autosomal • Limitations: dominant, o Considerable morbidity and monogenic mortality X-linked disease o Not curative o Available to
Slide 20: First AAV Program in History to Address Monogenic Cardiomyopathy
RP-A501: Danon Disease First AAV Program in History to Address Monogenic Cardiomyopathy Description Recombinant AAV9 containing the human LAMP2B transgene Clinical study Key efficacy data N=7 (Phase 1) Efficacy in low-dose adult cohort: Primary endpoints: • Robust LAMP2B cardiac expression • Safety
Slide 21: Reduction in Heart Wall Thickness Indicates Cardiac Remodeling
RP-A501: Danon Disease Reduction in Heart Wall Thickness Indicates Cardiac Remodeling Treated patients show stabilization or improvement in LV wall and septal wall thickness compared to untreated† males with Danon disease Posterior wall thickness in RP-A501–treated patients* Septal thickness in RP-A
Slide 22: Improved Cardiac Phenotype Across Dose Levels
RP-A501: Danon Disease Improved Cardiac Phenotype Across Dose Levels Stabilization or improvement of cardiac biomarkers and functional status is seen across treatment cohorts Time of Most recent Cohort Patient ID Variable Baseline follow-up follow-up (months) NYHA class II II 1001* BNP (pg/mL) 70 30
Slide 23: Improved Protein Expression Across Dose Levels
RP-A501: Danon Disease Improved Protein Expression Across Dose Levels Endomyocardial LAMP2B protein expression is seen across dose levels LAMP2B protein expression LAMP2B protein expression Cohort Patient ID (by IHC)* (by Western Blot) Month 12 Month 5-18 1001† 2.5% (previously <15%)a 17.9%d Adult –
Slide 24: Manageable Safety Profile
RP-A501: Danon Disease Manageable Safety Profile Enhanced immunomodulatory regimen for pediatric cohort was associated with limited side effects: Mitigation of AEs observed in low- and high-dose adult cohorts Limited complement activation Rituximab + Corticosteroids + Sirolimus Low dose pediatric co
Slide 25: Development Plan
RP-A501: Danon Disease Development Plan Moving toward pivotal Phase 2 study CURRENT PLANNED • Phase 1 treatment completed in • Pediatric cohort update in late Q3 2022 males • Expanded natural history study PLANNED GLOBAL • Orphan Drug, Rare Pediatric and • End of Phase 1 Regulatory meeting with FDA
Slide 26: 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 27: 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 28: 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 29: 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 30: 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 31: 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 32: 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 33: RP-L201 for LAD-I: ITGB2 Gene Mutation
PROGRAMRP-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 adhesi
Slide 34: 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 35: 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 (median: L201-003-1001 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 36: Sustained VCN in PBMCs
RP-L201: LAD-I Sustained VCN in PBMCs 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 L201-004-2009 L201-004-2010 L201-003-
Slide 37: 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 38: 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 39: 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 40: 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 41: 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 42: 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 43: Future Directions
FUTURE DIRECTIONS 43
Slide 44: 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 45: Anticipated Milestones and Wave 2
FUTURE DIRECTIONS Anticipated Milestones and Wave 2 2022 2023 2024 • BLA/MAA filing for LAD-I Q2 2022 Q3 2022 Q4 2022 • Label expansion • BLA/MAA filing for • LAD-I: Top-line • Danon disease: • Danon disease: studies for FA data will be Pediatric Initiate Phase 2 Danon disease achieved by June cohor
Slide 46: 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 47: Thank You!
THANK YOU! 47