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Axcella's Multi-Targeted Approach To Treat Complex Diseases Holds Transformational Potential

Axcella's Multi-Targeted Approach To Treat Complex Diseases Holds Transformational Potential

Axcella Health (NASDAQ: AXLA) was one of the sponsors for the Benzinga Biotech Global Small Cap Conference that took place on March 24-25, 2021. The information contained in this article in no way represents investment advice or opinion on the part of Benzinga or its writers and is intended for informational purposes only.

Precision medicine — providing the right treatment, for the right patient, at the right time — is saving lives. 

To battle diseases that are tough to treat, biotech companies typically use a small molecule or a biological drug to target one pathway within the body. Though, traditional single-target mechanisms may only address an individual metabolic or disease node.

There is another class of important and promising candidates that are overlooked: multi-target drugs powered by endogenous metabolic modulators (EMMs).

Axcella Health, a Massachusetts-based clinical-stage biotechnology company, is pioneering a multi-targeted approach using EMM compositions to treat complex diseases. 

What Are EMMs?

Endogenous metabolic modulators, or EMMs, are a critical set of naturally occurring molecules that have a fundamental role in biology. EMMs include a broad set of molecular families, including Axcella’s current focus: amino acids and their derivatives.

Amino acids serve as master regulators and signaling agents for metabolic pathways throughout the body while also having a well-established track record for clinical safety and tolerability. 

They can help provide the ability for cells and organs to:

  • Select fuel sources.
  • Create biomolecules such as nucleic acids, proteins, lipids and carbohydrates.
  • Sense the availability of nutrients.
  • Eliminate waste products.
  • Produce immune responses.
  • Activate signaling pathways.

Unique Approach

Leveraging these master regulators and signaling agents, Axcella has compiled a portfolio of multi-targeted product candidates that hold transformational potential, working with the body’s systems to tackle complex diseases such as nonalcoholic steatohepatitis (NASH) and overt hepatic encephalopathy (OHE).

Photo: Axcella Health

Drug Candidate To Treat NASH

AXA1125 is Axcella’s drug candidate to treat NASH. Nonalcoholic steatohepatitis (NASH) is a severe form of nonalcoholic fatty liver disease that is caused by the accumulation of liver fat, which can lead to complications such as cirrhosis and liver cancer.

Between 3% to 12% of adults in the U.S. (up to 40 million) suffer from NASH. While there are currently no FDA-approved drugs to combat this chronic and deadly disease, virtually all analysts expect NASH to become a multi-billion-dollar market over the next decade.

Designed just four years ago, AXA1125 is now set to enter a Phase 2b clinical trial. Axcella believes the trial’s initiation will demonstrate distinct advantages of the company’s differentiated approach. For instance, while it normally would take seven or more years to reach Phase 2 development with a drug candidate, Axcella is accomplishing this in roughly half of the time. The company also is armed with more data entering this phase of development than many companies.

Drug Candidate To Target The Reduction In Risk Of Recurrent OHE

AXA1665 is Axcella’s product candidate for the prevention of recurring OHE. OHE is a manifestation of cirrhosis in which patients are severely and cognitively impaired to where they are unable to care for themselves and may ultimately become comatose. 

It is estimated that about one-third of cirrhotic patients experience at least one OHE event during the course of their disease, leading to roughly $1 billion being spent annually on OHE treatments in the U.S. Additionally, many of these patients experience repeated events even while on today’s approved medicines, namely lactulose and rifaximin. Axcella believes this is because these agents focus on only one of the disease drivers, namely elevated ammonia, and because tolerability is a challenge and thus compliance is limited. 

AXA1665 holds the potential to improve the standard of care by addressing this disease more comprehensively and safely. The drug candidate is currently entering a Phase 2 clinical trial.


As of December 31, 2020, cash, cash equivalents and marketable securities totaled $107.3 million, compared to $92.1 million on December 31, 2019. The increase is primarily the result of net proceeds from the company’s follow-on stock offering that was completed in May 2020.

Axcella expects that its cash balance will be sufficient to meet the company’s operating needs into the third quarter of 2022.

To learn more about Axcella Health, you can visit its website here.

Photo by National Cancer Institute on Unsplash


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