Nkarta, Inc. · NASDAQ:NKTX
Nkarta — Investor Presentation (April 2021)
30 slides from Nkarta's investor presentation, April 2021
Slide 1: Next Generation Natural Killer Cells
NEXT GENERATION Natural Killer Cells Engineered to Beat Cancer APRIL 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: Evolving body of clinical data validates NK approach
Evolving body of clinical data validates NK approach NKG2D and non-engineered NK cells CD19 MD Anderson study with CD19 CAR-NK cells New England Journal of Medicine, Feb 2020 ~30 clinical studies No reported CRS, Well tolerated and no GvHD GvHD or (non-transplant) neurotoxicity CRs in advanced B cel
Slide 7: 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 r/r AML expansion pivotal trials (NKG2D heme) NKX101 IND Ph1 dose finding: (NKG2D solid tumor) Amend Solid Tumors NKX019 Ph1 dose finding: Potential IND B c
Slide 8: Platform
Platform nkarta THERAPEUTICS,
Slide 9: 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 10: 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 11: 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 12: 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 13: Our cryopreserved products are highly cytotoxic
Our cryopreserved products are highly cytotoxic NON- ENGINEE CONTRO RED NK NKX019 NKX019 6x109 L (PBS) 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
Slide 14: Manufacturing
Manufacturing 14
Slide 15: 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 16: 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 17: 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 to support clinical Design and engineering process initiated development Facility expected to supply pivotal trials of
Slide 18: Pipeline
Pipeline al <e lake} THERAPEUTICS,
Slide 19: 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- CAR engineered NK cells OX40 selected based on superiority vs. other co
Slide 20: NKG2D: ligands in multiple tumors, responses in AML
NKG2D: ligands in multiple tumors, responses in AML NKG2D ligand expression is documented Clinical responses observed in R/R AML with in multiple tumor types non-engineered allo-NKs validate NKG2D TUMOR TYPE REFERENCE STUDY RESPONSES* AML, ALL, CML, CLL Hilpert, J Immunol 2012 Bachanova, Crit Rev On
Slide 21: 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 Day 12 NKG2D TARGETS ARE OVER-EXPRESSED in AML blasts Day 18 Post tumor CLINICAL ACTIVITY with non-engineered NKs Day 24 UNMET NEED: Day 30 • AML US in
Slide 22: NKX101 Trial Design
NKX101 Single-arm two-part multi-center Phase 1 study Trial Design evaluating safety and efficacy of NKX101 • Modified 3+3 design in r/r AML and higher-risk MDS patients • 3 dose levels Lymphodepletion NKX101 dosing cycle Efficacy assessment • Multiple doses per cycle Cyclophosphamide (300 Total exp
Slide 23: 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 24: NKX019: CD19 targeted CAR-NK
NKX019: OX40 CD19 targeted CAR- costimulatory domain Proprietary CD19 binder NK CD3ζ signaling moiety LARGE OPPORTUNITY AFTER CD19 CAR-T APPROVALS • Gr3+ CRS: 13–49%; Gr3+ neurotoxicity: 18–31% CAR • Limited number of specialized sites can treat • 9–34% of patients in pivotal trials didn’t receive c
Slide 25: 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 26: Corporate
Corporate nkarta THERAPEUTICS,
Slide 27: 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 r/r AML expansion pivotal trials (NKG2D heme) NKX101 IND Ph1 dose finding: (NKG2D solid tumor) Amend Solid Tumors NKX019 Ph1 dose finding: Potential IND B c
Slide 28: Pioneering the next revolution in cell therapy
Pioneering the next revolution OX40 costimulatory domain in cell therapy Targeting receptor CD3ζ signaling moiety Efficient and robust next generation NK cell platform for blood cancers and solid tumors CAR Allogeneic and off-the-shelf products Industrialized manufacturing Developed for outpatient a
Slide 29: 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 30: Thank you!
al <elare Thank you!