CAMBRIDGE, Mass.--(BUSINESS WIRE)--Axcella (Nasdaq: AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today provided an update from its ongoing clinical study in adult subjects with non-alcoholic fatty liver disease (NAFLD) and announced its anticipated milestones for 2020.
Interim Analysis of Ongoing Clinical Study of AXA1125/AXA1957 (AXA1125-003)
Axcella has completed the enrollment of 102 adult subjects with NAFLD in an ongoing randomized, dose-ranging study in which subjects receive either AXA1125, one of two AXA1957 doses or placebo for 16 weeks. This study, which is being conducted at 18 U.S. medical centers, is assessing the impact of AXA1125/AXA1957 on safety, tolerability and physiology, as measured by a comprehensive panel of imaging and soluble biomarkers related to metabolism, inflammation and fibrosis.
An interim analysis has been conducted that includes data from approximately half of the study population through the full 16 weeks of administration. The analysis shows that AXA1125 and both doses of AXA1957 have been safe and well tolerated to date. Additionally, both AXA1125 and AXA1957 demonstrated clinically relevant responses on the three biological nodes fundamental to liver health and disease: metabolism (MRI-PDFF and HOMA-IR), inflammation (ALT, CK-18, cT1) and fibrogenesis (proC3). The onset of response in some biomarkers was seen as early as the eight-week, post-baseline assessment with continued improvement through 16 weeks.
“These interim non-invasive data indicate that AXA1125 and AXA1957 are having a positive impact on multiple dysregulated biological pathways related to health and disease that are common in NAFLD/NASH patients,” said Stephen A. Harrison, M.D., the principal investigator (PI) of the study, medical director of Pinnacle Clinical Research in San Antonio, TX, and visiting professor of Hepatology at the University of Oxford, UK. “These are particularly encouraging early findings, providing hope that a multifactorial effect can be generated from novel compositions of endogenous metabolic modulators.”
These findings will be included in the company’s presentation at the J.P. Morgan Healthcare Conference next week. Details regarding this presentation will be provided in a separate press release.
“We are pleased with the swift pace of enrollment and the data generated to date in our sizable clinical study of AXA1125 and AXA1957. The interim analysis increases our confidence in the potential for these candidates to become foundational therapeutics for NASH patients,” said Bill Hinshaw, President and CEO of Axcella. “Our excitement continues to build as we begin a major year for the company, with five planned clinical readouts and the initiation of our first planned Phase 2b/3 clinical trial in 2020.”
Liver Product Candidates
- AXA1125 and AXA1957 are being investigated in the aforementioned ongoing clinical study in adult NAFLD subjects. The company expects to report top-line data from this study in mid-2020.
- AXA1957 is also being investigated in a placebo-controlled ongoing clinical study enrolling approximately 30 adolescent subjects with NAFLD (AXA1957-002). This study is assessing the impact of AXA1957 on safety, tolerability and physiology. Top-line data from this study are expected to be reported in the second half of 2020.
- AXA1665 is being investigated in a 12-week, placebo-controlled ongoing clinical study that is enrolling approximately 60 subjects with mild and moderate hepatic insufficiency (AXA1665-002). This study is assessing the impact of AXA1665 on safety, tolerability and physiology. AXA1665 has been safe and well tolerated in clinical studies conducted to date. The company expects to report top-line data from the study in mid-2020. It also plans to submit an IND and initiate a Ph2b/3 clinical trial in the second half of 2020 to study AXA1665’s ability to reduce the risk of recurrence of overt hepatic encephalopathy (OHE).
Blood Product Candidate
- AXA4010, Axcella’s first hematology product candidate, is being investigated in an ongoing clinical study to assess safety, tolerability and impact on red blood cell physiology in approximately 24 subjects with sickle cell disease ages 12 and older in a staged, sequential design of three separate cohorts of eight subjects, each for up to 12 weeks (AXA4010-001). Axcella expects to report top-line data from this study in the second half of 2020.
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 ongoing clinical studies are being conducted as non-investigational new drug (IND) application clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate’s ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125/1957, subsequent studies will be conducted under an IND.
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 such portions of the company’s website, in addition to following its press releases, SEC filings and public conference calls and webcasts.
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 that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella’s pipeline includes lead therapeutic candidates for overt hepatic encephalopathy (OHE) and non-alcoholic steatohepatitis (NASH). Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.
Forward Looking Statements
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 development potential of the company’s product candidates, including AXA1665, AXA1125 and AXA1957, potential expansion into new therapeutic fields, the ability of endogenous metabolic modulators to impact dysregulated metabolism and health and the timing of the company’s clinical studies and trials and the timing of receipt of data from the same. 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 breadth and potential uses of the company’s pipeline of product candidates, the potential of AXA1125 and AXA1957 to become foundational therapies, interim and topline data readouts and timing of the same, the planned timing of an IND filing for AXA1665, the strength of the AXA Development Platform, the efficiency of the company’s discovery and development approach, the clinical development and safety profile of the company’s product candidates and their health or therapeutic potential, whether and when, if at all, the company’s product candidates will receive approval from the U.S. Food and Drug Administration, or other comparable regulatory authorities, and for which, if any, indications, competition from other biotechnology companies, the company’s liquidity, its ability to successfully develop product candidates through current and future milestones on the anticipated timeline, if at all, past results from non-IND clinical studies not being representative of future results, and other risks identified in the company’s SEC filings, including Axcella’s Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.