BenevolentAl Therapeutics Pipeline and Triage
Current Status: Programme in candidate seeking with
candidate selection due to complete Q4 2021
Efficacy'
Demonstrated with
humanised ALS cellular
models using motor
neurons and motor
neuron astrocyte
co-cultures
Multi-donor screens in
these cell types currently
ongoing, to provide
evidence of effects in
sporadic and defined
genetic ALS subtypes
(including TDP-43
mutants)
In vivo efficacy models
initiated
Safety²
In-vitro toxicity and
safety assays showing
better or equivalent safety
to clinical comparators
Panel of cellular toxicity
screen including
✓ cardiomyocytes,
hepatocytes, kidney and
HUVEC cells complete
In-vivo rat tox study (7
days) complete
DMPK
Demonstrable target
engagement in the
mouse CNS
PK/PD using target
engagement biomarkers
in mouse CNS
Comprehensive human
dose predictions
Dose projections
commensurate with BID
dosing
Cyp inhibition/
induction/TDI
IP/Chemistry
Composition of matter
patent applications filed
.
At the end of candidate seeking (Q4 2021), we will have completed our efficacy, safety/in-vivo toxicity and DMPK studies for BAI-5002
• Planning for preclinical and future clinical studies now ongoing
Internal Company drug programme data using SITraN assay. 2 Combined internal Company drug programme
data and CRO specific safety assays
Benevolent 50
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