Axcella is developing a robust pipeline of AXA 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 conducting human signal seeking and confirming studies* to understand the impact of various AXA candidates on human liver biology with the goal of taking candidates into the clinic to target non-alcoholic fatty liver (NAFLD) at the earliest stage of liver disease, to steatohepatitis (NASH), to cirrhosis at the later stage of liver disease.

AXA1125 is Axcella’s lead liver candidate targeted for NAFLD, inclusive of NASH. Intended to correct multiple core disease mechanisms, AXA1125 is designed to modulate metabolism, inflammation and fibrosis pathology in an integrated manner. The 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.

AXA1665 is designed to address complications in cirrhosis, such as nitrogen imbalance (leading to hyperammonemia), hypercatabolism and increased anabolic resistance (leading to frailty).



Axcella is focusing on studying human muscle atrophy biology* as a springboard to study multiple related indications, including knee and hip arthroplasty and hip fracture-related atrophy.

AXA2678 was designed to improve muscle mass and function under conditions of disuse atrophy. To achieve this, AXA2678 simultaneously mitigates a diverse set of biologies that underlie muscle atrophy including those that contribute to anabolic resistance, reduced protein synthesis, autophagy, junction integrity and energy deficit.



Axcella’s strategy in 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 AXA 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 mitrochondrial function, to modulate the microglial response to pro-inflammatory signals, to promote gluthathione synthesis and reactive oxygen species scavenging, and to reduce inflammation.


*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.