Metabolic Dysregulation Is At The Heart Of Many Diseases
Multifactorial diseases, where numerous pathologies and cellular networks are involved, require safe, multifunctional treatments. AXA candidates are intended to elicit powerful, multifunctional effects through the cell and integrate the multiple cellular functions of metabolic modulators, such as amino acids. Each AXA candidate is a rationally designed combination of metabolic modulators that harnesses this diverse activity and is intended to yield emergent synergies and reprogram dysregulated pathways in complex diseases and conditions with high unmet needs. Our robust pipeline includes programs in liver, muscle, CNS and other target indications.
Amino acid biology impacts multiple cellular network systems implicated in disease including:
Immune response and inflammation
- Mitochondrial activation and TCA cycle
- Protein synthesis
The potential benefits of AXA candidates include:
- Multifunctionality - simultaneously targeting multiple networks of dysregulated pathways
- Potential to treat a broad range of complex diseases
- Signal seeking and confirming studies to understand the AXA impact on human biology*, integrated with preclinical development, inform candidate optimization
- Designed and studied using our platform in a highly capital efficient manner
- Opportunity to be used as stand-alone or as a foundational combination agent in multi-drug therapeutic regimens
*Axcella conducts pre-IND, IRB-approved studies to evaluate the safety and tolerability of its AXA products in human subjects, or the product’s effects on the normal structure or function of the body. Therapeutic claims would need to be established under IND.