Axcella is developing a robust pipeline of DAAC candidates targeting multiple indications with high unmet medical need where a multifunctional treatment could produce meaningful results for patients.
We are initially focused on programs in liver, muscle, CNS and other indications.
Axcella is investigating DAAC candidates across a spectrum of liver conditions spanning from non-alcoholic fatty liver (NAFL) at the earliest stage of liver disease, to steatohepatitis (NASH), to cirrhosis at the later stage of liver disease. Axcella also plans to investigate candidates for the potential treatment of NAFLD in pediatric populations, where there is a growing unmet need.
AXA1125 is Axcella’s lead liver candidate in development for NAFLD, inclusive of NASH. A rationally designed DAAC candidate intended to correct multiple core mechanisms of disease, AXA1125 is designed to modulate metabolism, inflammation and fibrosis pathology in an integrated manner. The Axcellerator™ program for AXA1125 has yielded mechanistic and preclinical data to date to support the therapeutic potential of this candidate. AXA1125 has demonstrated consistent improvement on hepatocyte injury and fibrosis in the established animal models for NASH. AXA1125 is currently in ongoing preclinical studies, and IRB-approved human studies*.
AXA1665 is a rationally designed DAAC candidate intended to address added complications in cirrhosis, such as nitrogen imbalance (leading to hyperammonemia), hypercatabolism and increased anabolic resistance (leading to frailty).
Axcella is focusing on muscle atrophy as a springboard to study multiple related indications, including rotator cuff injury and hip fracture-related myopenia.
AXA2678 is a rationally designed DAAC candidate intended to improve muscle protein synthesis, thereby leading to potentially improved muscle mass and function under conditions of atrophy. To achieve this, AXA2678 simultaneously mitigates a diverse set of disease biologies that underlie muscle atrophy including those that contribute to anabolic resistance, reduced protein synthesis, autophagy, junction integrity and energy deficit. AXA2678 is currently in an IRB-approved human study*.
CENTRAL NERVOUS SYSTEM
Axcella’s strategy in diseases affecting the central nervous system (CNS) is to focus on mild traumatic brain injury (mTBI or concussion) and adult intractable epilepsy. Pending sufficiently robust signals, especially in adults with intractable epilepsy, other orphan epilepsies, such as Dravet’s, are anticipated for future study.
AXA3359 is a DAAC candidate designed to address the cascade of consequences resulting from mild trauma to the brain. Specifically, AXA3359 is intended to address deficits in brain signaling, TCA cycle, anaplerosis and mitochondrial function, to modulate the microglial response to pro-inflammatory signals, to promote glutathione synthesis and reactive oxygen species scavenging, and to reduce inflammation.
AXA3116 is a DAAC candidate rationally designed to engage novel biological mechanisms to reduce difficult-to-treat seizures, including imbalanced signaling, imbalanced neurotransmitters, aberrant neurotransmission, energy imbalance and abnormal brain cell activity.
*Studies conducted under food and dietary supplement guidelines