Rocket Pharmaceuticals, Inc. · NASDAQ:RCKT
Rocket Pharmaceuticals — Investor Presentation (September 2022)
49 slides from Rocket Pharmaceuticals's investor presentation, Sep 9, 2022
Slide 1: Seeking Gene Therapy Cures
INVESTOR DECK INVESTOR DECK INVESTOR DECK SEEKING GENE THERAPY CURES
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 physical beings.” — GAURAV SHAH, MD | CEO
Slide 4: Vision: Seeking Gene Therapy Cures
v Vision: Seeking Gene Therapy Cures To develop first-in-class and best-in-class curative gene therapies for patients with devastating diseases Generosity Trust Elevate Values Mission Curiosity ABOUT ROCKET PHARMACEUTICALS
Slide 5: Multi-platform, first-, best- and only-in-class approach
ABOUT ROCKET PHARMACEUTICALS Multi-platform, first-, best- and only-in-class approach to treating complex and life-threatening childhood disorders ???? Generating Value-based Gene Therapies Late-stage Science and Innovation Strong Capabilities and Financials Collaboration and Expertise Promising top
Slide 6: Expert Leadership With Proven Track Record
Expert Leadership With Proven Track Record ABOUT ROCKET PHARMACEUTICALS Raj Prabhakar, MBA Chief Business Officer, SVP ~20 years cell, gene and biotech business development Jonathan Schwartz, M.D. Chief Medical Officer, SVP Led multiple biologics approvals Kinnari Patel, Pharm.D., MBA President and
Slide 7: Four programs with clinical proof of concept
On-target MOA; clear endpoints Sizeable market to maximize patient impact First-, best- and only-in-class Criteria used to select programs Four programs with compelling clinical proof of concept Fast Track, Orphan Drug (US), Rare Pediatric Designation RMAT, ATMP, Fast Track, Rare Pediatric, Orphan D
Slide 8: Developing First-, Best- and Only-in-Class Therapies for Rare Diseases
2023 Developing First-, Best- and Only-in-Class Therapies for Rare Diseases With Extensive Unmet Needs Right technology for the target Clean MOAs: correct proteins are made in correct cells for disorders caused by single gene mutations Well-defined, achievable endpoints In-house AAV cGMP manufacturi
Slide 9: Strategically Building a Leading Gene Therapy Company
Strategically Building a Leading Gene Therapy Company ASGCT, American Society of Gene & Cell Therapy; ASH, American Society of Hematology; CHCO, Children’s Hospital of Colorado; CHOP, Children’s Hospital of Philadelphia; CIEMAT, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas;
Slide 10: Strong, Strategic Approach to Gene Therapy Manufacturing
Strong, Strategic Approach to Gene Therapy Manufacturing facility in Cranbury, NJ ~100,000 ft2 In-house capabilities manufacturing with capabilities to support commercial products and scaling Streamlined manufacturing capabilities to allow for cost-effective commercialization AAV, adeno-associated v
Slide 11: World-class Scientific Experts and Partners
World-class Scientific Experts and Partners Data on file. Rocket Pharmaceuticals. 2022. ABOUT ROCKET PHARMACEUTICALS Stanford Medicine
Slide 12: Unmet Needs and Market
UNMET NEEDS AND MARKET “We never went through the bone marrow transplant route and only had to deal with cancer and the complications associated with chemotherapy and radiation therapies. We lost two children…to this awful condition. May future research yield positive outcomes.” — FATHER OF TWO CHIL
Slide 13: Rare Diseases Are Associated With a Reduced Lifespan
Rare Diseases Are Associated With a Reduced Lifespan1 UNMET NEEDS AND MARKET FDA, Food and Drug Administration.1. Global Genes. Accessed April 2022. https://globalgenes.org/rare-disease-facts/ people globally are affected by a rare disease1 400 million of rare diseases have monogenic origins1 80% of
Slide 14: Market for Rare Disease Treatment Is Rising
Market for Rare Disease Treatment Is Rising Rare disease treatment market is projected to grow from $161.4 billion in 2020 to $547.5 billion by 20302 CAGR of 13.1% projected by 20302 Rare disease treatment market by region, 2015-2026 (USD million)1 2015 2026 2025 2024 2023 2022 2021 2020 2019 2018 2
Slide 15: Costs Associated With Rare Diseases Have Increased Exponentially
Feelings of depression Feelings of anxiety/stress Less interaction with friends/family Isolation from friends/family Worry about how their health will change in the future Lack of information on rare disease caused worry Felt they had no one to turn to in the medical system for information/support T
Slide 16: Pioneering Gene Therapy Clinical Programs
PIONEERING GENE THERAPY CLINICAL PROGRAMS “During the kids’ entire childhood they had multiple infections – ‘you name it they had it’ – and were admitted to the hospital several times due to these infections. Since treatment, the kids are back in day care and have scraped their knees – but unlike th
Slide 17: Rocket Offers Multi-platform Gene Therapy Expertise
Rocket Offers Multi-platform Gene Therapy Expertise IN VIVO platform EX VIVO platform RP-A501: Danon Disease RP-L102: Fanconi Anemia RP-L201: Leukocyte Adhesion Deficiency-I RP-L301: Pyruvate Kinase Deficiency CLINICAL PROGRAMS AAV, Adeno-Associated Virus; LV, Lentiviral Vector. Data on file. Rocket
Slide 18: In Vivo Platform: Adeno-associated Virus (AAV)
In Vivo Platform: Adeno-associated Virus (AAV) GOAL Express an adequate quantity of normal protein to normalize cardiomyocyte structure and function IDEAL FOR AAV platform ideal for disorders that affect the heart, liver, eye or central nervous system DANON DISEASE Multi-system disorder with severe
Slide 19: RP-A501 for Danon Disease: LAMP2B Gene Mutation
Standard of care: Heart transplant Limitations: Considerable morbidity and mortality Not curative Available to ~20% of patients Therapeutic challenges LAMP2 mutation Autosomal dominant, monogenic X-linked disease Disease etiology RP-A501 for Danon Disease: LAMP2B Gene Mutation Impaired autophagy Pro
Slide 20: First AAV Program in History to Address Monogenic Cardiomyopathy
First AAV Program in History to Address Monogenic Cardiomyopathy Clinical study N=7 (Phase 1) Primary endpoints: Safety Cardiomyocyte transduction, LAMP2B protein expression, histologic normalization Clinical stabilization or improvement Selected secondary endpoints: Sustained stabilization or impro
Slide 21: Reduction in Heart Wall Thickness Indicates Cardiac Remodeling
40 20 0 -20 -40 -60 Baseline M1 M2 M3 M6 M12 M15 M18 M24 % change from baseline septum (end-diastole) Months post gene therapy Stabilization Improvement Danon Disease Natural History: septal wall thickens by 0.92 ± 0.15 mm/year in untreated Danon males* Danon Disease Natural History: LV posterior wa
Slide 22: RP-A501: Danon Disease Cardiac Remodeling Images
RP-A501: Danon Disease All echocardiographic parameters are from local laboratory assessment by a single reader. LVPW IVS Representative images from patient 1005 Month 15 Baseline Reduction in Heart Wall Thickness Indicates Cardiac Remodeling
Slide 23: Improved Cardiac Function Across Dose Levels
Cohort Patient ID Age at enrollment (years) Variable Baseline Most recent follow-up Time of follow-up (months) Adult – Low dose 1001* 17.5 NYHA class II II 24 BNP (pg/mL) 70 30 6MWT (meters) 443 467 1002 20.4 NYHA class II I 18 BNP (pg/mL) 942 200 6MWT (meters) 405 410 1005 18.3 NYHA class II I 15 B
Slide 24: Improved Protein Expression Across Dose Levels
Cohort Patient ID LAMP2B protein expression (by IHC)* Month 12 LAMP2B protein expression (by Western Blot) Month 5-18 Adult – Low dose 1001† 2.5% (previously <15%)a 17.9%d 1002 67.8% 21.2%e 1005 92.4%b 61.1%f Adult – High dose 1006 100% 18.2%d Adult – High dose 1007 100%c RV: 45.1%g LV: 44.0%g Impro
Slide 25: Robust LAMP2 Cardiac Protein Expression
* Representative images from patient 1005: endomyocardial biopsy Baseline* Month 9* Normal Control Immunohistochemistry Electron Microscopy RP-A501: Danon Disease Robust LAMP2 Cardiac Protein Expression by Immunohistochemistry Vacuole Reduction and Restored Myofibrillar Structure by Electron Microsc
Slide 26: Manageable Safety Profile
Rituximab + Corticosteroids + Sirolimus Minimal complement activation and ↓ potential for TMA Early steroid taper and no exacerbation of Danon disease-associated skeletal myopathy Enhanced risk management plan: Safety results Infusion well tolerated with no drug-related SAEs Mitigation of complement
Slide 27: Development Plan: Moving toward pivotal Phase 2 study
Development Plan Moving toward pivotal Phase 2 study PLANNED GLOBAL REGISTRATIONAL PHASE 2 STUDY Pediatric cohort update in late Q3 2022 Expanded natural history study End of Phase 1 Regulatory meeting with FDA Initiate Phase 2 Global Pivotal Study Activities Initiate female study PLANNED Phase 1 tr
Slide 28: Ex Vivo Platform: Lentiviral Vector (LV)
Ex Vivo Platform: Lentiviral Vector (LV) EX VIVO (outside the body) LV gene therapy IDEAL FOR Modifying HSCs to address hematologic and immune disorders GOAL Promote sufficient quantities of a healthy therapeutic protein to be manufactured by patients’ own blood cells HSCs transduced with a lentivir
Slide 29: RP-L102 for Fanconi Anemia Complementation Group A
FA-A is an autosomal recessive disease caused by FANCA gene mutations FA proteins enable DNA repair FA-A accounts for 60-70% of FA cases Disease etiology RP-L102 for Fanconi Anemia Complementation Group A (FA-A) BMF, bone marrow failure; FA, Fanconi Anemia; FA-A, Fanconi Anemia, group A; FANC, Fanco
Slide 30: Clinical Studies Overview
Clinical Studies Overview Autologous HSCs transduced with LV carrying FANCA transgeneConditioning is not required because gene-corrected HSCs display proliferative advantage over time Description Clinical studies EU FANCOLEN I study (N=9) completed US Phase 1 study (N=2) completed US Phase 2 study o
Slide 31: Progressive Increase in Peripheral Blood and Bone Marrow VCNs
Progressive Increase in Peripheral Blood and Bone Marrow VCNs Progressive increases in gene markings in peripheral blood and bone marrow cells in 6 patients FA, Fanconi Anemia; VCN, vector copy number.Data on file. Rocket Pharmaceuticals. 2022. Data Cut-off: April 4, 2022 Peripheral blood VCN Bone m
Slide 32: Strong Evidence for Phenotypic Reversal
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, increased BM CFC MMC resistance ranging from 51% to 94% observed at 18 to 24 months post–RP-L102 administration MMC resistance of >20% achieve
Slide 33: Blood Count Stabilization and Sustained Phenotypic Reversal
Blood Count Stabilization and Sustained Phenotypic Reversal Increased MMC resistance in BM CFCs associated with hematologic stabilization at ≥1 year post RP-L102 BM CFC, bone marrow colony forming cell; FA, Fanconi Anemia; MMC, mitomycin-C. Data on file. Rocket Pharmaceuticals. 2022. Data Cut-off: A
Slide 34: Development Plan: Moving toward BLA/MAA filing
Development Plan Moving toward BLA/MAA filing Additional life-cycle management activities: Expansion to FANC C & G Exploration of non-genotoxic conditioning and HSC expansion Rejection of null hypothesis with minimum of 5 patients with increased MMC resistance >10% at 2 timepoints between 12 and 36
Slide 35: RP-L201 for LAD-I: ITGB2 Gene Mutation
Patients suffer from recurrent infections; fatal in majority Severe LAD-I: Death prior to age 2 in 60% to 75% of patients, infrequent survival >5 y in absence of alloHSCT Moderate LAD-I: Death prior to age 40 in >50% of patients, extensive morbidity with recurrent infections and inflammatory lesions
Slide 36: Clinical Study Overview
Clinical Study Overview Autologous HSCs transduced with LV carrying ITGB2 transgene Description Clinical study Treatment completed Phase 1/2 (N=9) Primary endpoints: Safety (Phase 1) Survival and safety (Phase 2) Safety Well tolerated; no drug product-related SAEs No graft rejection, no GvHD Initial
Slide 37: Sustained CD18 Expression in Peripheral Blood PMNs
Sustained CD18 Expression in Peripheral Blood PMNs At 3 to 24 months after infusion, 9/9 patients sustained stable CD18 expression (median: 56%) with no therapy-related serious adverse events *Dim/weak CD18 expression reported at baseline for Subject L201-003-1004 in ~63% of cells in conjunction wit
Slide 38: Sustained VCN in PBMCs
Sustained VCN in PBMCs At 3 to 24 months after infusion, 9/9 patients sustained stable CD18 expression (median: 56%) with no therapy-related serious adverse events LAD-I, Leukocyte Adhesion Deficiency-I; PB, peripheral blood; PBMCs, peripheral blood mononuclear cells; VCN, vector copy number. Data o
Slide 39: Significant Reduction in Hospitalizations and 100% Overall Survival
Significant Reduction in Hospitalizations and 100% Overall Survival Survival without allogeneic HSCT Primary outcomes ≥2 years of age AND ≥1-year post–RP-L201 infusion 100% overall survival Kaplan–Meier estimate Significant reduction in incidence of hospitalizations Post engraftment EOS, end of stud
Slide 40: Development Plan: Moving toward BLA/MAA filing
Development Plan Moving toward BLA/MAA filing Life-cycle management Potential label expansion to include moderate LAD-I population Potential study initiation in 2023 ATMP, advanced therapy medicinal product; BLA, Biologics License Application; H1, first half of the year; LAD-I, leukocyte adhesion de
Slide 41: RP-L301 for PKD: PKLR Gene Mutation
Lifelong chronic hemolysis Other clinical manifestations: Anemia Jaundice Iron overload Clinical manifestations Standard of care: Chronic blood transfusions and splenectomy Limitations: Iron overload Extensive end-organ damage Splenectomy confers lifelong infection and thrombotic risk Therapeutic ch
Slide 42: Clinical Study Overview
Clinical Study Overview Autologous HSCs transduced with LV containing human PKLR transgene Description Clinical study N = 4-5 (Phase 1) Primary endpoints: Safety Toxicity Selected secondary endpoints: Genetic correction Transfusion independence Reduction in anemia Reduction of hemolysis Safety Appea
Slide 43: Hemoglobin Normalization and Transfusion Independence
Hemoglobin normalized (from ~7.4 to 13.0 g/dL) sustained at 18 months post infusion No transfusion requirements following engraftment PATIENT 1001 PATIENT 1002 Dotted lines indicate average Hb for each patient prior to gene therapy Hemoglobin normalized (from ~7.0 to 14.8 g/dL) sustained at ~18 mont
Slide 44: Development Plan: Moving toward pivotal Phase 2 study
Development Plan Moving toward pivotal Phase 2 study Key endpoints selected Hemoglobin increase ↓ 50% transfusions or transfusion independence Well-delineated natural history in recent PKD NHS publications Complete Phase 1 pediatric cohort dosing (N=2-3) End of Phase 1 regulatory meeting with FDA Ap
Slide 45: Future Directions
FUTURE DIRECTIONS pharma
Slide 46: Rocket Pharmaceuticals: Elevating Gene Therapy to New Heights
Recognized as a premier gene therapy company Specialized against monogenic diseases Pioneer in the development of both ex vivo LV and in vivo AAV therapies AAV9-based gene therapy for Danon disease, a major value driver based on size of indication and lack of other therapies LV-based programs to pro
Slide 47: Anticipated Milestones
Anticipated Milestones and Wave 2 Q2 2022 LAD-I: Top-line data will be achieved by June FA: Top-line data achieved early In-house AAV cGMP manufacturing Q3 2022 Danon disease: Pediatric cohort data Q4 2022 Danon disease: Initiate Phase 2 pivotal study activities FA, PKD: Additional data BLA/MAA fili
Slide 48: Future Therapies: Wave 2 (AAV)
Future Therapies: Wave 2 (AAV) We continue to build our pipeline based on our core R&D strategy; identifying the “most productive” indications for the most efficient development path. MOA, mechanism of action; R&D, research & development Data on file. Rocket Pharmaceuticals. 2022. On-target MOA; cle
Slide 49: Thank You!
THANK YOU!
Rocket Pharmaceuticals — Investor Presentation (September 2022) on 6ix