Results of Axcella’s AXA1665-001 Clinical Study Published in Clinical and Translational Gastroenterology
Data support AXA1665’s potential to address hyperammonemia, amino acid dysregulation and physical dysfunction in subjects with cirrhosis
“Excess ammonia, amino acid imbalance and muscle wasting are often underlying contributing factors that impact various complications of cirrhosis, including overt hepatic encephalopathy, which is typically marked by significant cognitive dysfunction,” says Dr.
AXA1665-001 was a domiciled and controlled crossover clinical study that examined two AXA1665 doses (14.7g/day and 44.1g/day), each over a two-week period. Overall, the results showed that both doses of AXA1665 were well tolerated, with no product-related adverse events observed. AXA1665’s effects on hepatic and muscle physiology were measured using clinically relevant biomarkers. Results suggest AXA1665’s potential to mitigate the multifactorial defects of hyperammonemia, dysregulated amino acid metabolism, and physical dysfunction. Key physiological findings in subjects receiving the higher dose of AXA1665 for 15 days as compared to the control group were:
An increase in
Fischer Ratio(branched chain amino acids ÷ aromatic amino acids) of approximately 40%, indicating improved amino acid balance.
- A decreased trend in mean fasted plasma ammonia concentration despite provision of additional nitrogen via the amino acids in AXA1665, suggesting improved nitrogen handling.
- A decrease in the liver frailty index, suggesting improved physical function, accompanied by trends toward a leaner body composition.
“This publication follows our presentations at EASL and AASLD last year and the recent positive data readout for our second clinical study of AXA1665, helping to raise further awareness about Axcella’s pioneering work to develop multi-targeted agents utilizing distinct compositions of EMMs,” explains
About Endogenous Metabolic Modulators (EMMs)
EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.
About Axcella’s Ongoing Clinical Studies
Each of the company’s clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under
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Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company’s product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella’s pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics and development potential of the company’s EMM product candidates and the company’s characterization of the results from its clinical studies and future clinical trials, including for AXA1665, the company’s anticipated program milestones, including the design, status and timing of an IND-enabled clinical trial of AXA1665, the subject and timing of the company’s planned interactions with the FDA on its AXA1665 program, including the timing of IND submissions, and the potential of the company’s product candidates to impact health and/or disease, including AXA1665’s potential in advanced liver disease. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company’s ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its AXA1665-002 clinical study, other potential impacts of COVID-19 on business and financial results, including with respect to the ability to raise additional capital, make planned interactions and submissions to FDA or other regulatory authorities in a timely manner or at all and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts and/or FDA feedback support our IND submission and clinical trial initiation plans and timing, clinical trial design and target indication for AXA1665, whether and when, if at all, the company’s product candidates will receive approval from the FDA or other comparable regulatory authorities, and for which, if any, indications, past results from clinical studies not being representative of future results, and other risks identified in the company’s