2024-06-01

CASE STUDY: Discovering Best-in-Class LRRK2 Therapies for Parkinson’s Disease

Case study
3

LRRK2 (G2019S) Inhibitors for Parkinson’s disease. Promising target for Parkinson’s disease (> $5B market). Still no FDA-approved drug targeting LRRK2.

Target LRRK2 (G2019S)
Chemical space Enamine REAL 39B
Models we used B1
Hit rate (<10 μM) 4.6-15.6%
Best hit IC50 0.19 μM

Challenge

Following Alzheimer’s disease, Parkinson’s disease is the second most common neurodegenerative disorder in the US. LRRK2 is one of the most promising drug targets for Parkinson’s disease. There are currently some drug candidates in clinical trials, but no FDA-approved drug. The goal: to pioneer the first approved therapy in this large and critical market.

Solution & Impact

BIOPTIC B1 model quickly identified a chemically novel 0.86 uM LRRK2 inhibitor from a vast chemical space of 39B molecules. In the second iteration we improved potency 4.4 times achieving 0.19uM. This discovery brings us closer to delivering a best-in-class solution for an underserved patient population.

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