Axcella Announces Positive Top-Line Data from AXA1125-003 Clinical Study Showing Multifactorial Activity in Adult Subjects with NAFLD
-
Clinically relevant reductions in liver fat content, insulin resistance and fibroinflammation markers observed with AXA1125 along with favorable tolerability, supporting its potential to be a first-line
NASH therapy - Greater activity in key markers seen among subjects with type 2 diabetes receiving AXA1125
-
Company plans to engage with FDA regarding IND submission for AXA1125, proposed Phase 2b clinical trial in adult
NASH and pediatric development program -
Conference call to be held today at
8:30 a.m. ET
AXA1125-003 is a placebo-controlled, randomized, multi-arm clinical study assessing the impact of AXA1125 and AXA1957 on safety, tolerability and effects on structures and functions of the liver, as measured by a comprehensive panel of imaging and soluble biomarkers related to metabolism, inflammation and fibrosis. Both of these distinct product candidates are proprietary compositions of amino acids and derivatives that have been designed to support liver health. In this non-IND study, 102 adult non-alcoholic fatty liver disease (NAFLD) subjects with presumed nonalcoholic steatohepatitis (
Results from the study showed that AXA1125 and AXA1957 were generally well-tolerated, with sustained reductions noted for both product candidates versus placebo in key biomarkers of metabolism, inflammation and fibrosis over 16 weeks. Overall, as compared to placebo, AXA1125 demonstrated larger and more consistent reductions in clinically relevant biomarkers than AXA1957. Among subjects receiving AXA1125, 39% achieved a ≥30% relative reduction in liver fat content (MRI-PDFF), 39% achieved a ≥17 U/L reduction in alanine aminotransaminase (ALT), and 35% achieved a ≥80 mSec reduction in corrected T1 (cT1). Among the 11 subjects with type 2 diabetes receiving AXA1125, a greater proportion achieved each of these thresholds. Emerging evidence suggests that these thresholds of activity increase the likelihood of histopathological improvement in
“These data indicate that AXA1125 holds the potential to be a first-line therapy in
Select Measures of Relevance at 16 Weeks
Measure |
Placebo |
AXA1125 |
AXA1957 Low |
|
Subjects dosed |
15 |
29 |
26 |
32 |
Mean relative reduction in liver fat content (MRI-PDFF) |
-6% |
-23% |
-20% |
-8% |
Subjects with ≥30% relative reduction in liver fat content |
8% |
39% |
23% |
19% |
Mean relative reduction in ALT |
-7% |
-22% |
-19% |
-21% |
Subjects with ≥17 U/L reduction in ALT |
25% |
39% |
32% |
37% |
Subjects with ≥80 mSec reduction in cT1 |
17% |
35% |
23% |
23% |
Mean absolute change in proC3 (ng/mL) |
-0.7 |
-3.4 |
-3.1 |
-4.1 |
* Mean values and percentages above only include subjects for whom data was available at the week 16 timepoint.
AXA1125 and AXA1957 were both generally well tolerated in the study. The adverse events (AEs) experienced in ≥10% of subjects were gastrointestinal (diarrhea, nausea, reduced appetite) and upper respiratory infection. Gastrointestinal AEs were generally mild and transient, self-resolving in two to three weeks on average. Two serious adverse events were reported, both of which were determined to be unrelated to study product administration.
“The findings from this clinical study further validate the strength of our EMM platform and its ability to identify product candidates with the potential to address complex diseases in a multi-targeted manner,” said
Conference Call Information
Axcella will host a conference call today at
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 Clinical Studies
Each of the company’s clinical studies to date, including AXA1125-003, 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 such portions of the company’s website, in addition to following its press releases,
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 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 (
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 characteristics, competitive position and development potential of the company’s EMM product candidates, including AXA1125, the design, status and timing of the company’s ongoing clinical studies and planned IND-enabled clinical trials, including with respect to the company’s planned adult and pediatric clinical trials for AXA1125, the subject and timing of the company’s interactions with the FDA, including with respect to an IND application, Phase 2b clinical trial in adults and pediatric development plans for AXA1125, and the potential of the company’s product candidates to impact health and/or disease, including AXA1125’s potential in
View source version on businesswire.com: https://www.businesswire.com/news/home/20200506005139/en/
Company/Investor Contact
jfredette@axcellahealth.com
(857) 320-2236
Media Contact
Azeem Zeekrya
HDMZ
azeem.zeekrya@hdmz.com
(312) 506-5244
Source: Axcella