Axcella Announces AXA1125’s IND Clearance for the Treatment of NASH
- Multi-targeted oral candidate with the potential to be a first-line treatment option for adult and pediatric patients with NASH
- Phase 2b clinical trial in adults expected to begin in Q2
“This IND follows close on the heels of AXA1665’s IND clearance earlier this year, ushering in an exciting new era for Axcella as we seek to tackle a variety of complex diseases and address important unmet needs for patients utilizing multi-targeted EMM compositions,” said
Axcella expects to initiate its Phase 2b clinical trial in the second quarter of 2021. This randomized, double-blind, placebo-controlled, multi-center trial will evaluate the efficacy, safety and tolerability of AXA1125 in adult patients with biopsy-confirmed F2/F3 NASH. Approximately 270 patients will be enrolled and randomized 1:1:1 to receive either 45.2 or 67.8 grams per day of AXA1125 or a matched placebo in two divided doses for 48 weeks, with a four-week safety follow-up period. Patients will be stratified based on the presence or absence of type 2 diabetes.
The trial will be conducted globally across more than 70 clinical sites with a primary endpoint assessing the proportion of patients with a biopsy-confirmed ≥2 point improvement in NAFLD Activity Score (NAS) after the 48-week treatment period. Secondary endpoints will include the proportion of patients achieving biopsy-confirmed resolution of NASH without worsening of fibrosis and the proportion of patients achieving a ≥1 stage improvement in fibrosis without worsening of NASH. A range of non-invasive biomarkers, including MRI-PDFF and Fibroscan, will be utilized for additional endpoints and an interim analysis in the trial.
“AXA1125 leverages a mechanism that engages multiple pathophysiologic pathways involved in the development of NASH using a modality with well-precedented safety, which is particularly important given the many comorbidities associated with this chronic disease,” said
The upcoming Phase 2b clinical trial follows two earlier non-IND clinical studies in which reductions were seen with AXA1125 in key measures of hepatic fat, insulin resistance, inflammation and fibrosis with a safe and well tolerated profile. Presentations containing those findings can be found by visiting https://axcellahealth.com/publications/.
About AXA1125 and Nonalcoholic Steatohepatitis (NASH)
NASH is the most severe form of non-alcoholic fatty liver disease (NAFLD). This chronic, complex disease is associated with significant morbidity and mortality globally, and it is estimated to impact up to 40 million Americans, including up to 10% of American children. Despite its severity and increasing prevalence, there are currently no approved therapies for NASH in
AXA1125, Axcella’s NASH product candidate, is a composition of six amino acids and derivatives that is designed to target multiple metabolic pathways involved in NASH’s progression from metabolism to inflammation to fibrosis. In prior clinical studies, this oral product candidate has been safe and well tolerated and has demonstrated clinically meaningful reductions in a range of well-accepted, non-invasive NASH biomarkers, with the most pronounced activity noted in subjects with type 2 diabetes. AXA1125 is now entering Phase 2b development in adults with NASH. Axcella also plans to investigate AXA1125 in pediatric NASH.
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 company’s clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under
Internet Posting of Information
Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company’s website in the “Investors and News” section. Accordingly, investors should monitor this portion of the company’s website, in addition to following its press releases,
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 AXA1125, the design, status and timing of the company’s planned Phase 2b clinical trial of AXA1125, 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 AXA1125, 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 AXA1125, the clinical development and safety profile of AXA1125 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