Preclinical and clinical development pipeline

Phase 1a: completed QA 2024

Target Market: Moderate-to-severe Ulcerative colitis

Potential Key Differentiators: 

• Novel therapeutic approach: potential first-in-class peripherally restricted small molecule for the treatment of UC
• Potential for meaningful differentiation from existing immunosuppressive standard-of care treatments, through disease modifying efficacy

IND-ready: from Q4 2023

Target Market: Naive and recurrent GBM regardless of MGMT methylation status

Potential Key Differentiators:

• Potential first-in-class CNS penetrant drug for GBM and metastatic brain tumours
• Potential efficacy in patients resistant to chemotherapeutic SoC agents
• Strong rationale for combination therapy approaches in non-CNS cancers

BEN-28010 is an oral brain penetrant CHK1 inhibitor under development as a potential first-in-class CNS penetrant drug for GBM and metastatic brain tumours with the potential for meaningful differentiation in efficacy in patients resistant to chemotherapeutic standard of care agents and the potential to be used in combination therapy approaches. BenevolentAI has completed all IND-enabling studies.

Amyotrophic Lateral Sclerosis

IND-ready: from Q2 2024

Target Market: Sporadic and familial forms of ALS 

Potential Key Differentiators:

• Potential best-in-class CNS penetrant subtype-selective approach to drive efficacy and minimise side effect profile
• Neuroprotective mechanism of action, with positive effects in SOD1 mouse model

BEN-34172 is an oral, potent and selective brain-penetrant RARɑβ (retinoic acid receptor alpha beta) selective agonist as a potential best-in-class treatment for amyotrophic lateral sclerosis (ALS). BEN-34712 is currently in IND-enabling studies and it is expected to be IND ready by Q2 2024.

Target Market: Parkinson's and related synucleinopathies

Potential Key Differentiators: 

• Potential first-in-class CNS penetrant drug with neuroprotective activity

Target Market: Fibrotic indications including NASH

Potential Key Differentiators: 

• Novel target focused on the underlying mechanisms of fibrotic diseases - broad spectrum therapeutic potential

Further AZ target selection potental across Heart Failure

Further AZ target selection potental across Systenuc Lupus Erythematosus

Initial delivery of 3 novel small molecule drug candidates