Axcella Announces Initiation of EMMPOWER℠ Phase 2 Clinical Trial of AXA1665
- Initial clinical sites activated and patient screening underway
- Potential to improve the standard of care for cirrhotic patients by addressing multiple key drivers of overt hepatic encephalopathy (OHE)
“OHE is a condition that has devastating consequences for patients and families and places a substantial economic burden on the overall healthcare system,” said
Axcella has branded the Phase 2 trial EMMPOWER based on the potential for AXA1665, an EMM composition, to help patients, physicians and other caregivers overcome significant challenges associated with cirrhosis and OHE. This randomized, double-blind, placebo-controlled, multi-center investigation will evaluate the efficacy and safety of AXA1665 in patients who have experienced at least one prior OHE event and have neurocognitive dysfunction at screening. Approximately 150 patients on lactulose ± rifaximin (stratified by rifaximin use) will be randomized 1:1 to receive either 53.8 grams per day of AXA1665 or a calorie-matched placebo in three divided doses for 24 weeks, with a four-week safety follow-up period.
“The initiation of EMMPOWER is an important milestone, ushering in an exciting new era of clinical development for Axcella as we seek to tackle a variety of complex diseases utilizing proprietary, multi-targeted EMM compositions,” said
The EMMPOWER trial will be conducted globally at more than 70 clinical sites with a primary endpoint assessing the proportion of patients with a ≥2 point increase in the psychometric hepatic encephalopathy score (PHES) after the 24-week treatment period. The trial’s key secondary endpoints will focus on the proportion of patients experiencing an OHE breakthrough event and time to first OHE breakthrough event, including time to hospitalization. Other secondary endpoints include changes in physical function, including the liver frailty index, and patient-reported outcomes. Measures of circulating ammonia, amino acid profile, and inflammation-related markers will also be included as endpoints.
“By taking on the core drivers of OHE more comprehensively, we not only see the potential for AXA1665 to improve today’s standard of care, but also to eventually become a first-line treatment option,” said
Additional trial information can be found on https://clinicaltrials.gov/ via the identifier NCT04816916.
About AXA1665 and Overt Hepatic Encephalopathy (OHE)
Hepatic encephalopathy (HE), one of the most common complications of cirrhosis, is a condition involving amino acid imbalance, ammonia toxicity and muscle wasting, all of which contribute to diminished brain function. OHE refers to the presence of neurological abnormalities that are clinically apparent and do not require specialized psychometric testing. OHE is well established as a significant cause of morbidity and mortality in the cirrhotic population and is an area that continues to have unmet medical needs.
AXA1665, Axcella’s product candidate for reduction in risk of recurrent OHE, is a composition of eight amino acids and derivatives that is designed to target multiple metabolic pathways intersecting key organ systems, including the liver, muscle and gut. In prior clinical studies, this oral product candidate has been safe, well tolerated and has demonstrated the potential to improve ammonia handling, physical function, amino acid balance and neurocognition with a safe and well tolerated profile. AXA1665 is now in Phase 2 development.
About Endogenous Metabolic Modulators (EMMs)
EMMs are a broad family of naturally occurring 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 underlying causes of various complex diseases and improve health.
About Axcella’s Clinical Studies
Each of the clinical investigations that the company has completed to date have been conducted as non-investigational new drug application (IND) clinical studies under
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Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. 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. 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, competitive position, and development potential of AXA1665, the potential for AXA1665 to improve upon the standard of care for OHE patients and reduce OHE events, and the company’s ability to address other complex diseases utilizing EMM compositions. 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 planned clinical trial of AXA1665, other potential impacts of COVID-19 on the company’s our business and financial results, including with respect to the company’s ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company’s clinical trial initiation plans and timing, clinical trial design and target indications for AXA1665, the clinical development and safety profile of AXA1665 and its therapeutic potential, whether and when, if at all, the company’s product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company’s target indications, and other risks identified in the company’s