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.

“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”

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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.”

2020 Milestones

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.

About Axcella

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.