Nkarta, Inc. · NASDAQ:NKTX
Nkarta — Investor Presentation (October 2021)
32 slides from Nkarta's investor presentation, October 2021
Slide 1: Natural Killer Cells Engineered to Beat Cancer
NEXT GENERATION Natural Killer Cells Engineered to Beat Cancer October 2021 1
Slide 2: Forward looking statements
Forward looking statements This presentation contains forward-looking statements, within the any of these forward-looking statements. Should one or more of these meaning of the Private Securities Litigation Reform Act of 1995, regarding risks or uncertainties materialize, or should underlying assump
Slide 3: Pioneering the next revolution in cell therapy
Pioneering the next revolution in cell therapy OX40 costimulatory domain Efficient, robust, next generation Targeting receptor NK cell platform built for CD3ζ signaling moiety Blood cancers and solid tumors CAR Allogeneic and off-the-shelf Industrialized manufacturing Outpatient administration membr
Slide 4: They're called Natural Killer cells for a reason
They’re called Natural Killer cells for a reason Because Therefore Innate power of Highly active, cytotoxic NK cells to identify and cells as foundation and kill transformed cells starting material Low risk of GvHD Naturally allogeneic Potential for routine Low risk of CRS and administration and neu
Slide 5: Next gen platform enlists natural, healthy human NK cells
Which allows for: Donor selection for desired cell Potential for universal donors Next gen features and master cell platform banks enlists natural, Process starts with healthy human highly active, Efficient cytotoxic, NK cells manufacturing NK immune enables rapid, cells for large-scale optimal Mult
Slide 6: Global Collaboration to Develop Gene Edited Cell Therapies
Global Collaboration to Develop Gene Edited Cell Therapies GENOME ENGINEERING CAPABILITY Best-in-class, clinically validated CRISPR gene editing Ability to deploy up to 5 CRISPR/Cas9 gene edits in unlimited number of Nkarta product candidates Cell therapy leaders EXPERIENCED CLINICAL DEVELOPMENT PAR
Slide 7: Staying Ahead of the Curve: A Platform That Incorporates Multiple Next Generation Enhancements
ü Armored cells with membrane-bound IL-15 for persistence ü Multiplexed CRISPR/Cas9 genome engineering Staying Ahead of the Curve: ü Enhanced expansion, persistence and TME resistance via CISH deletion A Platform That Incorporates ü Cytokine activation using IL-12, -15 and -18 to enhance Multiple Ne
Slide 8: Evolving body of clinical data validates NK approach
Evolving body of clinical data validates NK approach NKG2D and non-engineered NK cells – AML CD19 – advanced B cell malignancies Multiple clinical studies show tolerability ~30 clinical studies and activity of engineered NK cells Well tolerated and no GvHD (non-transplant) MD Anderson study with CD1
Slide 9: Platform-driven pipeline with multiple upcoming milestones
Platform-driven pipeline with multiple upcoming milestones 2021 2022 2023 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 H1 H2 NKX101 Potential Ph1 dose finding: Heme Dose expansion (NKG2D heme) pivotal trials NKX101 IND Ph1 dose finding: (NKG2D solid tumor) Amend Solid Tumors NKX019 Ph1 dose finding: Potential Dose expan
Slide 10: Platform
Platform nkarta THERAPEUTICS,
Slide 11: Harnessing the power and efficiency of healthy adult NK cells
Harnessing the power and efficiency of healthy adult NK cells for the next revolution in cell therapy CELL SOURCE FEATURE AUTOLOGOUS IPSC DONOR Robust and scalable Complex NK differentiation and MANUFACTURING Highly difficult to scale 2-week process starting expansion over 4-8 weeks with real NK cel
Slide 12: Proprietary technologies in place for a best-in-class NK cell platform
Expansion Persistence Donor NK cells are co- NK cells are engineered cultured with for expression of Proprietary proprietary K562 stimulatory cell line to proprietary membrane bound IL-15 to enhance technologies achieve high cell numbers time in circulation in place for a best-in-class NK cell platf
Slide 13: Superior NK cell persistence from membrane bound IL-15
Superior NK cell persistence from membrane bound IL-15 IN VITRO PERSISTENCE IN VIVO PERSISTENCE AND EXPANSION IN NSG MICE (per 10,000 live leukocytes) 100 500 6 NK non-engineered NT-NK 5x10 NK non-engineered 5x106 NT-NK Percent remaining 6 80 CAR NK + NKX019 5MmbIL15 400 CAR NK + NKX019 5x10 mbIL15
Slide 14: Persistence and targeting to maximize anti-tumor activity
Persistence and targeting to maximize anti-tumor activity NKG2D-CAR Control GFP-expressing NKG2D-CAR + mbIL-15 (PBS) NK cells NK cells NK cells 1.2×10 10 Control Day 7 GFP-expressing NKs NKG2D-CAR NKs 1×10 10 NKG2D-CAR + mbIL-15 NKs Day 14 BLI (photon/sec) 8×10 9 Post tumor Day 21 6×10 9 Day 28 4×10
Slide 15: Our cryopreserved products are highly cytotoxic
Our cryopreserved products are highly cytotoxic NON- CONTROL ENGINEERED NKX019 NKX019 6x109 (PBS) NK CELLS (FRESH) (CRYO) DAY 2 5x109 4x109 DAY 7 3x109 DAY 12 2x109 DAY 23 1x109 DAY 30 0 DAY 56 Nalm-6 lymphoma model. 107 cells administered one day post tumor. Graphical data above are an average of m
Slide 16: Manufacturing
Manufacturing 16
Slide 17: A powerful and efficient process for off-the-shelf products
A powerful and efficient process for off-the-shelf products 1 Collect & Isolate 2 Activate 3 Engineer 4 Expand 5 Cryopreserve 6 Thaw & Administer NK cell Proprietary K562 stimulatory g-retro-virus cell line NK cells collected from NK cell expansion using Expanded NK cells are Continued expansion Har
Slide 18: Proprietary expansion enables industrial-scale manufacturing
Proprietary expansion enables industrial-scale manufacturing Robust, rapid expansion produces 10,000 3,364 doses in a single manufacturing run, 1,000 with potential of of doses Fold Expansion per run 100 35 Projected cost of commercial 10 manufacturing at peak: 1 per dose* 0 6 15 Days of Expansion D
Slide 19: In-house manufacturing to control process and production
Clinical GMP Facility South San Francisco, CA In-house manufacturing to control process and production CLINICAL GMP FACILITY FUTURE COMMERCIAL-SCALE FACILITY Multi-product facility 88,000 sq ft facility in South San Francisco Combined manufacturing hub and company Support early clinical trials and r
Slide 20: Pipeline
Pipeline nkarta THERAPEUTICS,
Slide 21: NKX101: CAR-NK targeting NKG2D ligands
NKX101: CAR-NK targeting NKG2D ligands OX40 costimulatory domain CD3ζ signaling moiety NKG2D activating receptor NKG2D receptor is primary driver of NK cell activation and tumor killing >10x increase in NKG2D expression vs. non-engineered CAR NK cells OX40 selected based on superiority vs. other cos
Slide 22: NKG2D: ligands in multiple tumors, responses in AML
NKG2D: ligands in multiple tumors, responses in AML NKG2D ligand expression in multiple tumor types Clinical responses observed in r/r AML • Non-engineered allogeneic NK cells • Heterogeneous patient population • Single center academic studies • 19% true CR rate (aggregate) TUMOR TYPE REFERENCE STUD
Slide 23: NKX101: Rationale in acute myeloid leukemia (AML)
NKX101: Rationale in GFP-expressing NKX101 NKX101 NKX101 acute myeloid NK cells (5 x 106 cells) (1 x 107 cells) (2 x 107 cells) leukemia (AML) Day 6 NKG2D TARGETS Day 12 ARE OVER-EXPRESSED in AML blasts Day 18 Post tumor CLINICAL ACTIVITY with non-engineered NKs Day 24 UNMET NEED: • AML US incidence
Slide 24: NKX101 Trial Design
NKX101 Single-arm two-part multi-center Phase 1 study Trial Design evaluating safety and efficacy of NKX101 in r/r AML and higher-risk MDS patients • Multi-cycle • Multi-dosing per cycle Lymphodepletion NKX101 multi-dosing cycle Efficacy assessment Cyclophosphamide (300 Two dosing regimens: 2 or 3 d
Slide 25: NKX101 demonstrates anti-tumor activity in solid tumors
NKX101 demonstrates anti-tumor activity in solid tumors NKX101 activity in NSG mice LIVER & BILE CANCER US PBS CONTROL GFP-EXPRESSING NKS NKX101 INCIDENCE: ~42K / YR 5-year survival rate ~18% DAY 7 NKG2D TARGETS OVER-EXPRESSED DAY 14 on HCC and CRC cells Post tumor NK CELLS ARE IMPORTANT IN LIVER DA
Slide 26: NKX019: CD19 targeted CAR-NK
NKX019: OX40 CD19 targeted CAR-NK costimulatory domain Proprietary CD19 binder CD3ζ signaling moiety UNMET NEED REMAINS DUE to SAFETY, SPEED, ACCESS of APPROVED CD19 CAR-T THERAPIES • Gr3+ CRS: 13 to 49%; Gr3+ neurotoxicity: 18 to 31% • Limited number of specialized sites can treat CAR • 9 to 34% of
Slide 27: NKX019 kills tumors with high or low levels of CD19 expression
NKX019 kills tumors with high or low levels of CD19 expression High CD19 Expressing Cells Low CD19 Expressing Cells NKX019 NKX019 CD19 CAR-T CD19 CAR-T Effector : Target Effector : Target NKX019 can achieve high levels of cytotoxicity even when tumor cells express low levels of CD19 antigen, whereas
Slide 28: NKX019 Trial Design
NKX019 Single-arm multi-center Phase 1 study evaluating Trial Design safety and efficacy of NKX019 • Multi-cycle in patients with r/r B cell malignancies • Multi-dosing per cycle - 3 doses per cycle Lymphodepletion NKX019 dosing cycle Efficacy assessment Cyclophosphamide (300 Total exposure per cycl
Slide 29: Corporate
Corporate nkarta THERAPEUTICS,
Slide 30: Platform-driven pipeline with multiple upcoming milestones
Platform-driven pipeline with multiple upcoming milestones 2021 2022 2023 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 H1 H2 NKX101 Potential Ph1 dose finding: Heme Dose expansion (NKG2D heme) pivotal trials NKX101 IND Ph1 dose finding: (NKG2D solid tumor) Amend Solid Tumors NKX019 Ph1 dose finding: Potential Dose expan
Slide 31: Our Vision and Mission
Our Vision and Mission OUR VISION OUR MISSION To be the leading company delivering We strive to discover, develop and deliver novel innovative, accessible cell therapies for cancer off-the-shelf NK cell therapy product candidates patients, their caregivers and families that have a profound impact on
Slide 32: Thank you!
al <elare Thank you!