Harnessing the Power of
Jasper's Differentiated
c-Kit Therapeutic Approach
Nasdaq: JSPR
January 2023
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Briquilimab is an investigative drug | 2 |
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
Sickle cell disease
(SCD)
Severe combined immunodeficiency (SCID)_
Fanconi anemia (FA)
Acute myeloid leukemia
(AML)
Myelodysplastic syndrome (MDS)
Therapeutic development
in chronic disease
Chronic Spontaneous
Urticaria (CSU)
(Expected Start Q3 2023)
Low to Int. Risk MDS
(Expected Start
March/ April 2023)
Expansion
opportunities: chronic
inducible urticaria,
allergic asthma, prurigo nodularis, eosinophilic esophagitis
Efficient development with
near-term catalysts
Initial data expected:
Late 2023: Lower to Int.
Risk MDS
Mid 2024: CSU
SCID BLA submission
targeted for 2024
Multiple transplant studies currently funded and run by partners
Briquilimab is an investigative drug | 3 |
and is not approved for any indication | |
Tyrosine kinase KIT plays a central role in regulating mast and stem cell survival
Mast Cell
c-Kit
Blocking c-Kit signaling depletes
mast cells
Mast Cells
Play a key role in immune system through the release of compounds that induce inflammation (degranulation)
Unregulated mast cell activation may cause episodes of hives and inflammation (i.e. Chronic Urticaria)
Briquilimab blocks c-Kit signaling and may lead to lasting depletion of mast cells in Chronic Urticaria and other mast cell diseases
Stem Cells
Hemopoietic stem cells are the pluripotent cells that drive ongoing production of red blood cells, platelets and immune cells
Many blood cancers and genetic diseases can be cured by depleting diseased stem cells and replacing with donor or gene modified stem cells
Briquilimab can be added to existing transplant regimens to block c-Kit signaling on stem cells and potentially deliver more effective transplants
Briquilimab is an investigative drug | 4 |
and is not approved for any indication | |
Briquilimab is optimally designed to directly block c-Kit signaling
Briquilimab
Blocks SCF binding to c-Kit (CD117) to
directly inhibit receptor signaling
Briquilimab
SCF
c-KIT (CD117)
Validated
Mechanism
of Action
Favorable
Drug
Properties
Encouraging
Clinical
Profile
Briquilimab is designed to directly block SCF from binding to c-Kit (CD117) with high affinity and avidity
- Aglycoslyated IgG1 antibody directly inhibits stem cell factor from binding to the c-Kit receptor on mast and stem cells
- Inhibition of SCF signaling leads to depletion of mast cells in the skin and hematopoietic stem cells in the bone marrow
- Wide therapeutic potential across a range of mast and stem cell- mediated diseases
- Kd < 5pM affinity to human c-Kit with IC50 ~ 70pM
- Human mast cell survival bioassay IC50 ~12.5nM
- No Fc mediated ADCC or complement mediated cytotoxicity which reduces risk of adverse effects related to express CD117
- Human clinical data as IV or Sub-Q delivery
- Predictable clearance from ages 3yrs to 79yrs, no need to monitor PK
- Demonstrated single agent lasting depletion of mast cells
- Demonstrated efficacy in 5 clinical transplant studies
- No briquilimab-related SAEs reported
Briquilimab is an investigative drug | 5 |
and is not approved for any indication | |
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Jasper Therapeutics Inc. published this content on 24 January 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 24 January 2023 21:25:28 UTC.