PIPELINE

 

DAAC CANDIDATES

 

LIVER

Axcella is advancing DAAC candidates across a spectrum of liver diseases spanning from non-alcoholic fatty liver (NAFLD) at the earliest stage, steatohepatitis (NASH), to cirrhosis (laterstage of liver disease) in both adults and pediatric populations.

+ AXA1125

AXA1125 is Axcella’s lead liver candidate in development for NAFLD, inclusive of NASH. A rationally designed DAAC to correct multiple core mechanisms of disease, AXA1125 modulates metabolism, inflammation and fibrosis pathology in an integrated manner with a favorable safety profile in pre-IND human studies. The Axcellerator™ program for AXA1125 has yielded strong 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 a pre-IND, IRB-approved human study.

+ AXA1665

AXA1665 is a rationally designed DAAC to address added complications in cirrhosis, such as nitrogen imbalance (leading to hyperammonemia), hypercatabolism and increased anabolic resistance (leading to frailty). AXA1665 is being prepared for early clinical PK-PD studies.

 

MUSCLE DISEASE

Axcella is focusing on muscle atrophy as a springboard to multiple related indications, including rotator cuff injury and hip fracture-related myopenia.

+ AXA2678

AXA2678 is a rationally designed DAAC intended to safely and effectively  improve muscle protein synthesis, thereby leading to 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  a pre-IND 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) as a lead indication, followed by adult intractable focal epilepsy.  Pending sufficiently robust signals, especially in adults with focal intractable epilepsy, other orphan epilepsies, such as Dravet’s, are anticipated.

+ AXA3555

AXA3555 is a DAAC candidate designed to address the cascade of consequences resulting from mild trauma to the brain. Specifically, AXA3555 is intended to address deficits in brain signaling, TCA cycle, anaplerosis and motrochondrial function, to modulate the microglial response to pro-inflammatory signals, to [promote] gluthathione synthesis and reactive oxygen speces scavaging, and to reduce inflammation. AXA3555 is currently being prepared for an initial IRB-approved clinical trial in subjects experiencing mild concussions.

+ AXA3116

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, irregular brain development, ion channel dysfunction and abnormal brain cell activity. AXA3116 is currently enrolling adult subjects with intractable focal epilepsy in an IRB-approved clinical study .