Jasper Therapeutics, Inc. · NASDAQ:JSPR
Jasper Therapeutics — Investor Presentation (January 2023)
51 slides from Jasper Therapeutics's investor presentation, Jan 24, 2023
Slide 1: Harnessing the Power of Jasper's Differentiated c-Kit Therapeutic Approach
Harnessing the Power of Jasper’s Differentiated c - Kit Therapeutic Approach Nasdaq: JSPR January 2023 Exhibit 99.1
Slide 2: Safe Harbor Statements
2 Briquilimab is an investigative drug and is not approved for any indication Safe Harbor Statements Forward - Looking Statements This investor presentation and any accompanying oral presentation (together, this “Presentation”) contain forward - looking statements . All statements other than stateme
Slide 3: Briquilimab is an anti c-Kit antibody for acute and chronic therapy
3 Briquilimab is an investigative drug and is not approved for any indication Briquilimab is an anti c - Kit antibody for acute and chronic therapy in multiple mast and stem cell diseases Clinical validation in 5 for 5 transplant indications Therapeutic development in chronic disease Efficient devel
Slide 4: KIT plays a central role in regulating mast and stem cell survival
4 Briquilimab is an investigative drug and is not approved for any indication Tyrosine kinase KIT plays a central role in regulating mast and stem cell survival Play a key role in immune system through the release of compounds that induce inflammation (degranulation) Unregulated mast cell activation
Slide 5: Briquilimab blocks SCF binding to c-Kit (CD117)
5 Briquilimab is an investigative drug and is not approved for any indication SCF c - KIT ( CD117) Briquilimab Briquilimab Blocks SCF binding to c - Kit (CD117) to directly inhibit receptor signaling Validated Mechanism of Action Briquilimab is designed to directly block SCF from binding to c - Kit
Slide 6: Briquilimab Therapeutic Development
6 Briquilimab is an investigative drug and is not approved for any indication Briquilimab Therapeutic Development
Slide 7: Briquilimab for Chronic Urticaria
7 Briquilimab for Chronic Urticaria
Slide 8: Briquilimab c-Kit and mast cell activity
8 Briquilimab is an investigative drug and is not approved for any indication Briquilimab c - Kit and mast cell activity Activity Assay Result Binding affinity to human c - Kit Fc dimer Kd < 5 pM Biacore assessment of c - Kit signaling neutralization Ki 70pM Human mast cell c - Kit phosphorylation b
Slide 9: Mast cell depletion in non-human primates following cutaneous wound injury
9 Briquilimab is an investigative drug and is not approved for any indication Source: Amgen Research Report 2006488 Briquilimab mast cell depletion in non - human primates following cutaneous wound injury
Slide 10: Mast cell depletion in lungs of non-human primates
10 Briquilimab is an investigative drug and is not approved for any indication Source: Amgen research report R2006481 Briquilimab mast cell depletion in lungs of non - human primates
Slide 11: Briquilimab can be dosed IV or subcutaneously
11 Briquilimab is an investigative drug and is not approved for any indication Briquilimab can be dosed IV or subcutaneously (SQ) Briquilimab IV serum concentrations Time (days) C o n c e n t r a t i o n o f J S P 1 9 1 i n S e r u m ( n g / m L ) 0 7 14 21 1 10 100 1000 10000 100000 -0001 (0.1 mg/k
Slide 12: Phase I: Briquilimab robustly depletes tryptase and c-Kit positive skin mast cells
12 Briquilimab is an investigative drug and is not approved for any indication Phase I: Briquilimab robustly depletes tryptase and c - Kit positive skin mast cells *Punch Biopsy Wound Model Source: Amgen Phase 1a (SAD = 71 briquilimab patients; MAD = 8 briquilimab patients) Briquilimab Phase 1a (
Slide 13: Barzolvolimab proof-of-concept of c-Kit blockade in CSU
13 Briquilimab is an investigative drug and is not approved for any indication Celldex’s barzolvolimab has demonstrated proof - of - concept of c - Kit signal blockade for Chronic Spontaneous Urticaria patients Source: Celldex EAACI 2022 Presentation (June 30, 2022) Barzolvolimab Phase 1b MAD (IV) S
Slide 14: Potential initial clinical study in Chronic Spontaneous Urticaria
14 Briquilimab is an investigative drug and is not approved for any indication Potential initial clinical study for Briquilimab in Chronic Spontaneous Urticaria Chronic Spontaneous Urticaria (CSU) Patient Population • H1 - antihistamines refractory • Xolair - naïve and/or Xolair - failed Expected En
Slide 15: Differentiation between Briquilimab and Barzolvolimab
15 Briquilimab is an investigative drug and is not approved for any indication Key points of differentiation between Briquilimab and Barzolvolimab (CDX - 0159) Key Similarities Key Differences 1 c - Kit (CD117) monoclonal antibody o Both antibodies are humanized, aglycosylated IgG1 inhibitors of c -
Slide 16: Chronic urticaria is a significant and expanding market
16 Briquilimab is an investigative drug and is not approved for any indication Chronic urticaria represents a significant and expanding market, with estimated 1+ million potential patients Given Xolair’s low penetration, experts believe the antihistamine - failed market is poised for significant gro
Slide 17: Unmet need for Chronic Urticaria patients who fail antihistamines
17 Briquilimab is an investigative drug and is not approved for any indication There is a significant unmet need for Chronic Urticaria patients who fail antihistamines – safer, more effective therapies are needed *Montelukast = Leukotriene receptor antagonist; cyclosporine = immunosuppressant; dapso
Slide 18: Expansion beyond Chronic Urticaria into other mast cell diseases
18 Briquilimab is an investigative drug and is not approved for any indication Briquilimab can expand beyond Chronic Urticaria into other mast cell - mediated and inflammatory diseases Chronic Inducible Urticaria Severe Asthma Other Mast Cell Diseases Mast Cell - Mediated Diseases (Addressed by c -
Slide 19: Briquilimab for Lower to Intermediate Risk MDS
19 Briquilimab for Lower to Intermediate Risk MDS
Slide 20: Depleting cancerous stem cells in lower to intermediate risk MDS
20 Briquilimab is an investigative drug and is not approved for any indication Briquilimab’s ability to directly deplete cancerous stem cells may be leveraged as a disease - modifying therapeutic in lower to intermediate risk MDS patients LR - MDS: Lower - Risk Myelodysplastic syndromes MDS HSCs Bri
Slide 21: Depletion may enable healthy HSCs to restore hematopoiesis
21 Briquilimab is an investigative drug and is not approved for any indication Briquilimab depletion may enable healthy HSCs to take over the bone marrow niche and restore functional hematopoiesis [1] Kwon et al, Blood 2019 [2] Amgen Phase I healthy volunteer data (unpublished) Briquilimab depletion
Slide 22: Phase I study in lower-risk myelodysplastic syndrome
22 Briquilimab is an investigative drug and is not approved for any indication A Phase I open - label, dose - escalation, safety and tolerability study of briquilimab as a second line therapy in patients with lower - risk myelodysplastic syndrome HI - E: Hematologic Improvement - Erythroid; Hematolo
Slide 23: Lower to intermediate risk MDS market opportunity
23 Briquilimab is an investigative drug and is not approved for any indication Lower to intermediate risk MDS market opportunity MDS INCIDENCE (G7) 1 ~32,000 Lower to Intermediate Risk MDS (IPSSR ≤3.5)* 2,3 ~22,500 (70%) RBC - Transfusion Dependent 4,5 ~11,250 (50%) Erythropoietin Stimulating Agent
Slide 24: Briquilimab Transplant Development
24 Briquilimab is an investigative drug and is not approved for any indication Briquilimab Transplant Development
Slide 25: Sickle Cell & Beta Thalassemia
25 Sickle Cell & Beta Thalassemia
Slide 26: Briquilimab in sickle cell disease transplant
26 Briquilimab is an investigative drug and is not approved for any indication » Sickle cell disease (SCD) is a group of inherited red blood cell / hemoglobin disorders; patients with SCD have misshapen red blood cells, which prevents normal cell function / behavior » Patients typically develop chro
Slide 27: SCID
tjJasp per THERAPEUTICS INC. SCID
Slide 28: Briquilimab conditioning for SCID
28 Briquilimab is an investigative drug and is not approved for any indication Briquilimab conditioning for severe combined immunodeficiency (SCID) T - B - : A form of severe combined immunodeficiency (SCID) is the T cell - negative (T - ), B cell - negative (B - ) SCID phenotype Jasper SCID Strateg
Slide 29: SCID Phase I results: safety and tolerability
29 Briquilimab is an investigative drug and is not approved for any indication Briquilimab SCID phase I results: Safety and tolerability to date Clinical Safety • No briquilimab - related serious adverse events (SAEs) • No myelosuppression • No significant infusion reactions Source: Agarwal R, et al
Slide 30: Potential near-term BLA and PRV opportunity in SCID
30 Briquilimab is an investigative drug and is not approved for any indication Potential near - term biologics license application (BLA) and PRV opportunity for briquilimab in SCID SCID re - transplant patients are ultra orphan, high unmet need population Focus on current clinical data with briquili
Slide 31: Fanconi Anemia
tjJasp per THERAPEUTICS INC Fanconi Anemia
Slide 32: Briquilimab-based conditioning in Fanconi anemia
32 Briquilimab is an investigative drug and is not approved for any indication Fludarabine Cyclophosphamide » Fanconi is an inherited DNA repair deficiency that leads to progressive failure of hematopoietic stem and progenitor cells and transfusion dependency » Allogeneic stem cell transplant is the
Slide 33: Potential Fanconi Anemia development path
33 Briquilimab is an investigative drug and is not approved for any indication Potential Fanconi Anemia development path for briquilimab Continue to enroll under current protocol (n up to 12) Discuss path to BLA with FDA with 6 - month data in first three patients o Example: Rocket Pharmaceuticals h
Slide 34: Briquilimab development plan
34 Briquilimab is an investigative drug and is not approved for any indication Briquilimab development plan 2023 2024 2025 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q LR - MDS SCID Urticaria Additional LR - MDS Study (n=12 - 16) LR - MDS Expansion (n=40 - 50) Natural History Follow - Up BLA Submission (Rolling) I
Slide 35: Harnessing the Power of Jasper's Differentiated c-Kit Therapeutic Approach
Harnessing the Power of Jasper’s Differentiated c - Kit Therapeutic Approach Nasdaq: JSPR January 2023
Slide 36: Additional Slides
36 Briquilimab is an investigative drug and is not approved for any indication Additional Slides
Slide 37: AML / MDS
THERAPEUTICS INC AML / MDS
Slide 38: Single agent briquilimab depletes diseased stem cells in 5 to 7 days
38 Briquilimab is an investigative drug and is not approved for any indication AML/MDS Study: Single agent briquilimab depletes diseased stem & progenitor cells in bone marrow in 5 to 7 days Stem and progenitor cell depletion following briquilimab 0.6 mg/kg single dose in AML/MDS patients Muffly L e
Slide 39: Donor cell takeover and elimination of detectable cancer in older patients
39 Briquilimab is an investigative drug and is not approved for any indication Briquilimab based conditioning demonstrates donor cell takeover of bone marrow stem cells and elimination of detectable cancer in older patients (Median donor chimerism) Clearance of detectable cancer Donor cell takeover
Slide 40: No briquilimab-related SAEs and outpatient transplant proof of concept
40 Briquilimab is an investigative drug and is not approved for any indication No briquilimab - related SAEs, no 100 - day transplant related mortality and proof of concept for outpatient stem cell transplant Clinical Safety • No briquilimab - related SAEs • No significant briquilimab infusion react
Slide 41: 0.6 mg/kg IV Briquilimab pharmacokinetics
41 Briquilimab is an investigative drug and is not approved for any indication 0.6 mg/kg IV Briquilimab Pharmacokinetics: consistent and predictable clearance 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 100 1000 10000 Time (days) Concentration of JSP191 in Serum (ng/mL) 3000 300 30000 Muffly L et al. Tand
Slide 42: Preclinical
tjJasp per THERAPEUTICS INC. Preclinical
Slide 43: 6-month non-human primate toxicology study
43 Briquilimab is an investigative drug and is not approved for any indication Briquilimab 6 - month non - human primate (NHP) toxicology study • 4 groups (control, 1, 30, 300 mg/kg weekly subcutaneous), n=8 to 12 per group, 50% female • Dosing for 26 weeks followed by 32 - week treatment - free pha
Slide 44: Reticulocytes and Hemoglobin Levels – NHP toxicology study
44 Briquilimab is an investigative drug and is not approved for any indication Reticulocytes and Hemoglobin Levels – 6mo NHP toxicology study Source: Amgen Report 109076
Slide 45: White Blood Cells – NHP toxicology study
45 Briquilimab is an investigative drug and is not approved for any indication White Blood Cells – 6mo NHP toxicology study 0 5 10 15 20 25 Pre 15Pre 22 Dose 15 Dose 29 Dose 57 Dose 85 Dose 113 Dose 141 Dose 169 Dose 183 Post 29Post 57Post 85 Post 113 Post 141 Pose 169 Post 197 Post 225 WBC E3/uL Ma
Slide 46: Phase I Healthy Volunteers
46 Phase I Healthy Volunteers
Slide 47: Phase I SAD Study: adverse events of interest
47 Briquilimab is an investigative drug and is not approved for any indication * All mild (Grade 1) AEs except for two Grade 2 events Briquilimab (JSP191/AMG191) Phase I SAD Study: Adverse events of interest
Slide 48: Phase I – Human SAD
48 Briquilimab is an investigative drug and is not approved for any indication Phase I – Human SAD
Slide 49: Phase I – Human SAD Study Day
49 Briquilimab is an investigative drug and is not approved for any indication Phase I – Human SAD Study Day
Slide 50: MAD – Grade adverse event
50 Briquilimab is an investigative drug and is not approved for any indication MAD – Grade 3 adverse event One subject reported an allergic reaction 36 hours after 2nd dose in cohort 1 (14 mg q2w) •Urticaria, facial angioedema, no respiratory symptoms, no clinically relevant changes in vital signs •
Slide 51: Healthy volunteer immunogenicity summary
51 Briquilimab is an investigative drug and is not approved for any indication Phase I: Healthy volunteer immunogenicity summary • Pre - existing ADAs: 2/77 (Briquilimab (AMG191) treated subjects) • Developing ADAs: 13/77 • Neutralizing ADAs: 8/77 • Post - dose ADAs seen in subjects given 20mg SC or
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