Neumora Therapeutics IPO Presentation Deck
NMRA-CK18 for Amyotrophic Lateral Sclerosis
Pharmacology
- Focused on inhibiting the protein casein
kinase-16 (CK18) to reduce levels of the
pathological form of TDP-43 and slow
disease progression in ALS
-
CK16 also regulates tau biology which may
enable indication expansion
Epidemiology
-5,000 people diagnosed in U.S. each year and
~16,000 patients in U.S. live with ALS at a given timeĀ¹
Status
Discovery stage; identified compounds demonstrating
CK10 selectivity, nanomolar potency, and properties
consistent with favorable CNS penetration
Strong IP Protection
Expect exclusivity through 2043+, based on
composition of matter protection plus potential for
patent term extension
1) CDC.gov, 2018.
Strong Biological
Rationale
CK18
phosphorylates
TDP-43, a key driver
of TDP-43-driven
pathology in ALS
ALS PATHOLOGY
Motor
neuron
Cytoplasm
Nucleus
CK18
O-ATP
TDP-43
NMRA-
CK18
Inhibits CK18 to
reduce levels of
pathological TDP-43
Phosphorylation
TDP-43 aggregation can
promote neurodegeneration
Toxic TDP-43
aggregates
Endogenous pathways.
Enhanced in disease
Drug impact
Confidential
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