Nkarta, Inc. · NASDAQ:NKTX
Nkarta — Investor Presentation (November 2020)
28 slides from Nkarta's investor presentation, November 2020
Slide 1: Allogeneic Natural Killer Cells Engineered to Beat Cancer
Allogeneic Natural Killer Cells Engineered to Beat Cancer November 2020
Slide 2: Forward looking statements
Forward looking statements This presentation contains forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995, regarding future events and the future results of the company that are based on current expectations, estimates, forecasts, and projections ab
Slide 3: Highlights: Nkarta engineered CAR-NKs
Highlights: Nkarta engineered CAR-NKs Natural Killer cells are the cornerstone of innate immune surveillance » Allogeneic and off-the-shelf OX40 Targeting CD3ζ » Attractive cost of manufacturing CAR » Proprietary expansion, persistence, tumor targeting and cryopreservation technologies » Potential f
Slide 4: Nkarta proprietary technologies
Nkarta proprietary technologies Expansion Persistence Targeting Cryopreservation Co-culture with proprietary Expression of proprietary Engineered for expression Maintains NK cell viability K562 stimulatory cell line to membrane bound IL-15 to of optimized CARs and potency achieve high cell doses enh
Slide 5: Extensive clinical experience validates NK approach
Extensive clinical experience validates NK approach NKG2D CD19 Patients have been treated with non-engineered NK MD Anderson study with CD19 CAR-NK cells cells across ~30 studies - Published in New England Journal of Medicine, Feb 2020 ü 7 / 11 CRs in patients with advanced B cell Well tolerated mal
Slide 6: Pipeline
Pipeline 2021 2022 2023 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1H 2H r/r AML Potential Ph1 dose finding: Heme NKX101 expansion pivotal trials (NKG2D) IND Ph1 dose finding: Amend. Solid tumors Ph1 dose finding: Potential NKX019 (CD19) IND Dose expansion B cell malignancies pivotal trials Program 3 IND Ph1 dose find
Slide 7: Management Team and Board
MANAGEMENT TEAM BOARD Paul Hastings Ali Behbahani, MD President & CEO Chairman Tiba Aynechi, PhD Ralph Brandenberger, PhD Director VP, Technical Operations Fouad Azzam, PhD Director Alicia Hager, JD, PhD Chief Legal Officer Mike Dybbs, PhD Director Nadir Mahmood, PhD Simeon George, MD Chief Financia
Slide 8: Platform
al. <elare! THERAPEUTICS Platform
Slide 9: Nkarta CAR-NKs: engineered to enhance activity
Nkarta CAR-NKs: engineered to enhance activity APPROVED ALLO CAR-T CAR-T NK THERAPIES THERAPIES CELLS CAR-NK CELLS OPPORTUNITY FOR IMPROVED SOLID TUMOR ACTIVITY P PERSISTENCE PP P P LOW GVHD RISK P TBD P P LOW RISK OF CRS OR NEUROTOXICITY P P ALLOGENEIC, OFF-THE-SHELF MANUFACTURING P P COST OF MANUF
Slide 10: Superior persistence from membrane bound IL-15
Superior persistence from membrane bound IL-15 In vitro persistence In vivo persistence and expansion in NSG mice 400 Non-engineered NK cells 20 Non-engineered Unmodified NKNKcells cells NK cells expressing mbIL-15 NK NKcells expressing cells mbIL-15 expressing mbIL-15 (NKX101) NK cells expressing s
Slide 11: Persistence and targeting to maximize activity
Persistence and targeting to maximize activity NKG2D-CAR Control GFP-expressing NKG2D-CAR + mbIL-15 1.2×10 10 Control (PBS) NK cells NK cells NK cells GFP-expressing NKs Day 7 NKG2D-CAR NKs 1×10 10 NKG2D-CAR + mbIL-15 NKs Day 14 8×10 9 BLI (photon/sec) Day 21 Post tumor 6×10 9 Day 28 4×10 9 Day 35 2
Slide 12: Allogeneic, commercially-enabling manufacturing
Allogeneic, commercially-enabling manufacturing 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 driven Harvesti
Slide 13: Proprietary expansion to enable large scale manufacturing
Proprietary expansion to enable large scale manufacturing 10,000 » Extensive optimization enables truly off- 3,364 the-shelf products 1,000 » In-house cGMP manufacturing suite Fold Expansion 100 construction recently completed 35 » Projected cost of commercial 10 manufacturing at peak: ~$2,000 / dos
Slide 14: Cryopreserved NKX101 retains in vitro cytotoxicity
Cryopreserved NKX101 retains in vitro cytotoxicity 7 Control 6 Non-engineered NKs Fresh NKX101 Frozen / thawed NKX101 (run 1) Tumor cells (Normalized to T=0) 5 Frozen / thawed NKX101 (run 2) Frozen / thawed NKX101 (run 3) 4 3 2 1 0 0 12 24 36 48 60 72 Time (hours) Cryopreserved NKX101 retains cytoto
Slide 15: In-house clinical GMP facility: construction complete
In-house clinical GMP facility: construction complete 15
Slide 16: Pipeline
al. <elare! THERAPEUTICS Pipeline
Slide 17: NKX101: CAR-NK targeting NKG2D ligands
NKX101: CAR-NK targeting NKG2D ligands » NKG2D receptor is primary driver of NK cell activation and tumor killing NKG2D OX40 CD3ζ » >10x increase in NKG2D expression vs. CAR non-engineered NK cells » OX40 selected based on superiority vs. other costimulatory domains mbIL-15 » Targets of NKG2D are se
Slide 18: NKG2D: ligands enriched in tumors, demonstrated responses
NKG2D: ligands enriched in tumors, demonstrated responses 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
Slide 19: NKX101: Acute myeloid leukemia (AML)
NKX101: Acute myeloid leukemia (AML) » AML US incidence: ~21K / yr GFP- expressing NK NKX101 NKX101 NKX101 • 5-year survival rate ~28% cells (5 x 106 cells) (1 x 107 cells) (2 x 107 cells) » NKG2D targets are over-expressed in Day 6 AML blasts Day 12 » Clinical activity with non-engineered NKs Day 1
Slide 20: NKX101: Heme dose finding and expansion
NKX101: Heme dose finding and expansion Dose finding (3+3 design, 3 cohorts, 108 / 3x108 / 109 CAR-NK cells / dose) Pre-treatment Cycle 1 Cycle 2 (~28 days) 5 days 28 days 5 days 28 days Leuka- Haplomatched donor pheresis NKX101 Mfg. Manufacturing Treatment beyond Cycle 1 with FDA concurrence & Rele
Slide 21: NKX101: Solid tumors
NKX101: Solid tumors » Liver & bile cancer US incidence: ~42K / yr NKX101 activity in NSG mice • 5-year survival rate ~18% PBS control GFP-expressing NKs NKX101 » NKG2D targets over-expressed Day 7 on HCC and CRC cells » NK cells are important in liver Day 14 immunity and tumor surveillance Post tum
Slide 22: NKX019: CD19 targeted CAR-NK; IND 1Q21
NKX019: CD19 targeted CAR-NK; IND 1Q21 » Large opportunity after CAR-T approvals: • Gr3+ CRS: 13–49%; Gr3+ neurotoxicity: 18–31% • Limited number of specialized sites can treat CD19 binder • 9–34% of patients in pivotal trials didn’t receive cells CD3ζ OX40 (primarily due to mfg. challenges) CAR » R
Slide 23: NKX019: Activity in lymphoma model
NKX019: Activity in lymphoma model Non- 6E+09 6x109 Control engineered NKX019 NKX019 (PBS) NK cells (fresh) Control (PBS) (cryo) 5E+09 5x109 Non-engineered NK cells Day 2 NKX019 (fresh) 4E+09 4x109 NKX019 (cryo) Day 7 3E+09 3x109 Day 12 2E+09 2x109 Day 23 1E+09 1x109 Day 30 00 1 6 12 23 30 56 Day 56
Slide 24: Corporate
al. <elare! THERAPEUTICS Corporate
Slide 25: Intellectual property
Intellectual property PLATFORM NKX101 NKX019 NK cell expansion NKG2D target CD19 » Multiple issued patents and pending » Issued US patents and multiple pending » Provisional applications applications US/OUS/PCT applications » Cells expressing tumor-targeting » Compositions and methods » Claims to va
Slide 26: Anticipated Milestones
Anticipated Milestones 2021 2022 2023 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1H 2H r/r AML Potential Ph1 dose finding: Heme NKX101 expansion pivotal trials (NKG2D) IND Ph1 dose finding: Amend. Solid tumors Ph1 dose finding: Potential NKX019 (CD19) IND Dose expansion B cell malignancies pivotal trials Program 3 IND
Slide 27: Highlights: Nkarta engineered CAR-NKs
Highlights: Nkarta engineered CAR-NKs Natural Killer cells are the cornerstone of innate immune surveillance » Allogeneic and off-the-shelf OX40 Targeting CD3ζ » Attractive cost of manufacturing CAR » Proprietary expansion, persistence, tumor targeting and cryopreservation technologies » Potential f
Slide 28: Thank you!
al. <elare! THERAPEUTICS Thank you!