LRRK2

LRRK2 (Leucine-rich repeat kinase 2) is a promising drug target in Parkinson's disease. Mutations in LRRK2 are among the most common genetic contributors to this neurodegenerative disorder. By targeting LRRK2, researchers aim to develop treatments that can potentially slow or even halt the progression of Parkinson’s. Furthermore, LRRK2's roles in other diseases, including certain cancers and inflammatory conditions, highlight its broader therapeutic potential. The exploration of LRRK2 underscores a shift towards understanding and targeting the root molecular causes of diseases, promising more precise and long-lasting solutions for patients.

BT-0267

Our drug candidate has demonstrated impressive efficacy against LRRK2, exhibiting nanomolar potencies against both the LRRK2[WT] and the [G2019S] mutation. BT-0267 and its backup molecules have shown remarkable selectivity, achieving a 500-fold preference against other kinases and surpassing 1,000-fold selectivity against other safety panel targets. This high specificity paves the way for a targeted therapeutic approach, significantly reducing the risk of off-target effects.

The compound exhibits an outstanding pharmacokinetic (PK) profile, characterized by high and sustained brain exposure with minimal peripheral distribution. This ensures superior efficacy while mitigating the risk of on-target off-tissue toxicity. The pristine safety profile has been confirmed through GLP-compliant studies and ongoing safety and PK clinical evaluations in healthy volunteers.