On September 29, 2022, the Company reported interim results from
the ongoing global Phase 2b randomized, double-blind, placebo-controlled, dose ranging EMMPACT study to evaluate the safety, tolerability,
and efficacy of AXA1125 for the treatment of NASH. These interim results report findings regarding the effects of AXA1125 administration
on selected outcome measures after 12 and 24 weeks of treatment.
This interim analysis was preplanned to be conducted when enrollment
reached 30% of the target of 270 subjects with biopsy confirmed stage 2 or 3 NASH across all trial arms. Data from this ongoing blinded
study included 82 subjects at week 12 and 58 subjects at week 24; approximately half of the subjects have type 2 diabetes mellitus (T2DM).
In addition to effects on hepatic fat and alanine aminotransferase (ALT), previously reported in 2 other studies, this study also included
vibration controlled transient elastography (FibroScan), a widely accepted and accessible non-invasive test (NIT) that assesses both liver
fat and stiffness. Specifically, the study examines liver stiffness, changes of which have been correlated with improvements in liver
fibrosis and outcomes in clinical studies. Study participants were randomized 1:1:1 to receive either a placebo or 22.6g or 33.9g of AXA1125
twice daily.
At 24-weeks there were statistically significant improvements in the
liver stiffness measurement (LSM) compared to placebo in the high dose arm for all subjects. Absolute changes in LSM were 0.13, -2.01,
and -4.07 kilopascals (kPa) in the placebo, low dose and high dose arms, respectively (p= 0.0992 and 0.0096 for the low and high dose,
respectively, compared to placebo). These results were supported by statistically significant improvements in other NITs of liver fibrosis:
ELF and FIB-4. Statistically significant improvements in ALT were seen at both weeks 12 and 24 in all subjects (placebo-adjusted difference
of -28.61% (p=0.0183) and -36.3% (p=0.0017) for the low and high doses, respectively). All subjects experienced significantly greater
changes from baseline in MRI-PDFF at 12-weeks compared to the change from baseline in the placebo group (placebo adjusted difference of
-18.98% (p=0.0082) and -21.24% (p=0.0014) for the low and high doses, respectively). Numerical trends of improvement relative to placebo
in PDFF were seen at week 24 but these were not statistically significant in the small number of subjects. Overall, these positive results
confirm AXA1125’s multi-targeted impact, a differentiated approach to directly and simultaneously targeting multiple pathways that
are dysregulated in NASH. Consistent with previous results, AXA1125 was found to be very safe and well-tolerated in this study. Both dose
levels are active and will be continued. Consistent with prior clinical trials, T2DM showed results comparable to non-diabetics.
Cautionary Note Regarding Forward-Looking Statements
This Form 8-K contains forward-looking statements within the meaning
of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the potential for AXA1125 to serve
as a first-line treatment option. 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 Form 8-K 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 Form 8-K, 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 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 clinical trials of AXA1125, other potential impacts on the Company’s business and financial results, including with respect
to its 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 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
SEC filings, including Axcella’s Annual Report on Form 10-K, Quarterly Reports 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 Form 8-K 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.