BenevolentAl Therapeutics Pipeline and Triage
Amyotrophic Lateral Sclerosis (ALS)
Affects 0.02% US population over age 40 years(1),
~75 thousand patients in 8MM(2), forecast
$1.04bn market by 2029(3)
• ALS is a rare and devastating fatal neurodegenerative disease in
which the motor neurons degenerate or die, and stop sending
messages to the muscles. Fewer than 50% of patients survive 30
months from symptom on-set5
• Efficacy and Safety - Current treatments (riluzole and radicava) are
largely ineffective and only extend patient survival by ~6 months.
Patients are largely treated with oral riluzole, however radicava is an
intense intravenous treatment placing significant burden on patient
quality of life
• New, safe and effective disease-modifying therapies are urgently
needed
10%
Familial
ALS
90%
Sporadic
BEN-9160: CNS-Penetrant c-Abl inhibitor for the
Treatment of Sporadic and Familial ALS Subtypes,
with potential to expand to Parkinson's Disease
Deployment of the Benevolent PlatformTM led to the
discovery of c-Abl - a target with demonstrated capacity
to modulate pathways critical to ALS pathology
Our Molecular Design expertise resulted in a potent,
brain-penetrant small molecule c-Abl inhibitor
BEN-9160 with a pharmacokinetic profile enabling
significant target engagement
BEN-9160 is expected to provide an efficacious oral
treatment for ALS, targeting key disease-relevant
mechanisms
Disease modifying treatment for the benefit of
both the Sporadic and Familial ALS patient
populations
Delay of disease progression with extension of life
significantly better than Standard of Care
Clean safety profile (no relevant drug-drug
interactions, hepatotoxicity, CV liability, CNS effects
on memory or cognition or myelosuppresion)
Source (1) GlobalData: Amyotrophic Lateral Sclerosis: Epidemiology Forecast to 2029; (2) and (3) GlobalData,Amyotrophic Lateral Sclerosis (ALS):
Opportunity Analysis and Forecasts to 2029; (4) Company internal drug programme data; (5)
https://www.sciencedirect.com/science/article/pii/S0140673610611567
https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(10)61156-7/fulltext
Benevolent
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