We have built a first of its kind integrated knowledge platform that has proven capable of rapidly designing our novel and proprietary AXA Candidates. In our Non-IND, IRB Approved Clinical Studies*, we have rapidly generated a rich dataset in healthy subjects and subjects with certain disease conditions. We believe that generating human data at this early stage of development enables us to make high-insight, capital efficient development decisions and potentially increases the probability of success for these candidates.

Our AXA Development Platform allows us to efficiently design and test AXA Candidates that simultaneously target multiple biologies and metabolic pathways by integrating:

  • Advanced analytics of metabolism regulation and dysregulation;

  • Correlative reasoning algorithms to interrogate data in our proprietary database, AxcellaDB;

  • Proprietary human primary cell systems to directly test the multiple biologies that drive any particular disease or metabolic dysregulation;

  • Predictive combinatorial drug metabolism and pharmacokinetics, or DMPK, analytics to inform dose exposure relationships; and,

  • What we believe to be the world’s leading EMM safety database.

The data and learnings generated from our AXA Candidate design process further inform the design pathway, increasing the AXA Development Platform’s efficiency. Our platform has produced a rich pipeline of AXA candidates with programs in liver, muscle, and blood among other target areas.

*Axcella conducts Non-IND, IRB-approved Clinical studies to evaluate the safety and tolerability of its AXA candidates in human subjects, or effects on the normal structure or function of the body. Studies intended at the time of initiation to support drug development will be conducted under an IND.