Axcella Reports First Quarter Financial Results and Provides Business Update
- Reported positive top-line data from AXA1125-003, with clinically relevant reductions in liver fat content, insulin resistance and fibroinflammation observed in subjects with NAFLD receiving AXA1125
- Completed enrollment of AXA1665-002, with data readout expected in Q3 2020
- Announced AXA2678 patent issuance and oral presentation at ICSFR
- Launched new corporate website at www.axcellahealth.com
“These past few months have been a particularly exciting period for Axcella, culminating in our recent report of positive top-line data for AXA1125, our NASH product candidate, that we believe further validates our platform,” said
Recent Highlights
Liver Programs
- Reported positive top-line data from AXA1125-003, a clinical study assessing the impact of AXA1125 and AXA1957 on safety, tolerability and physiology in subjects with non-alcoholic fatty liver disease (NAFLD). 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 both placebo and AXA1957, AXA1125 demonstrated larger and more consistent reductions in clinically relevant biomarkers.
- Completed enrollment of AXA1665-002, an ongoing clinical study assessing the impact of AXA1665 on safety, tolerability and physiology in adult subjects with mild and moderate hepatic insufficiency. AXA1665 has been generally safe and well tolerated in this study to date. The company expects to report data from this study in the third quarter of 2020.
Muscle Program
-
Discussed Axcella’s AXA2678 muscle product candidate during an oral presentation at the 2020
International Conference on Frailty and Sarcopenia Research (ICFSR) inMarch 2020 . -
Announced the issuance of
U.S. Patent 10,596,136, which broadly covers the use of AXA2678 and related EMM compositions for the reduction of fat infiltration in muscle.
Corporate
- Launched a new corporate website at www.axcellahealth.com that includes additional detail about the company’s platform, product candidates and culture.
Anticipated 2020 Milestones
Liver Programs
- Q3 2020: Report data from AXA1665-002 in adult subjects with mild and moderate hepatic insufficiency.
-
Q4 2020: Submit an Investigational New Drug (IND) application to the
U.S. Food and Drug Administration (FDA) and initiate a potential Phase 2b/3 registrational clinical trial of AXA1665 for the reduction in risk of OHE recurrence. - 2H 2020: Engage with the FDA to discuss the company’s planned IND application for AXA1125, proposed Phase 2b clinical trial in adults and pediatric development program.
Blood Program
- Q4 2020: Report top-line data from Cohort 1 of AXA4010-001, a clinical study on safety, tolerability and blood physiology in subjects with sickle cell disease.
Financial Results
R&D Expenses: Research and development expenses were
G&A Expenses: General and administrative expenses were
Net Loss: Net loss for the quarter ended
Cash Position: Cash and cash equivalents 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 ongoing clinical studies are being conducted as non-investigational new drug (IND) application 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 (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. 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 characteristics, competitive position and development potential of the company’s EMM product candidates, including for AXA1125 and AXA1665, the design, status and timing of the company’s ongoing clinical studies and planned IND-enabled clinical trials, the company’s anticipated program milestones, including the timing of AXA1665-002 and AXA4010-001 data readouts, the subject and timing of the company’s planned interactions with the FDA on the AXA1665 and AXA1125 programs including potential timing of IND application submissions, and the potential of the company’s product candidates to impact health and/or disease, including AXA1125’s potential in NASH and AXA1665 potential in OHE. 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 and planned interactions and submissions to FDA or other regulatory authorities 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 we are able to collect in our ongoing AXA1665-002 and AXA4010-001 clinical studies and potential delays in disclosure of the same, other potential impacts of COVID-19 on our business and financial results, including with respect to our ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts and/or FDA feedback support our IND submission and clinical trial initiation plans and timing, clinical trial design and target indication for AXA1125 and AXA1665, 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 FDA or other comparable regulatory authorities, and for which, if any, indications, competition from other biotechnology companies, past results from clinical studies not being representative of future results, and other risks identified in the company’s
|
||||||||
Unaudited Condensed Consolidated Balance Sheets |
||||||||
(in thousands) |
||||||||
|
|
|
|
|
||||
|
|
|
|
|
||||
|
|
2020 |
|
2019 |
||||
Assets: |
|
|
|
|
||||
Cash and cash equivalents |
|
$ |
75,522 |
|
|
$ |
66,769 |
|
Other assets |
|
2,060 |
|
|
$ |
4,149 |
|
|
Total assets |
|
$ |
77,582 |
|
|
$ |
70,918 |
|
Liabilities and stockholders' equity (deficit): |
|
|
|
|
||||
Liabilities |
|
$ |
30,768 |
|
|
$ |
33,253 |
|
Preferred stock |
|
— |
|
|
$ |
197,888 |
|
|
Stockholders' equity (deficit) |
|
46,814 |
|
|
$ |
(160,223 |
) |
|
Total liabilities and stockholders' equity |
|
$ |
77,582 |
|
|
$ |
70,918 |
|
|
||||||||
Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss |
||||||||
(in thousands, except share and per share data) |
||||||||
|
|
|||||||
|
Three Months Ended |
|||||||
|
||||||||
|
2020 |
|
2019 |
|||||
Operating expenses: |
|
|
|
|||||
Research and development |
$ |
10,335 |
|
|
$ |
7,563 |
|
|
General and administrative |
4,125 |
|
|
3,468 |
|
|||
Total operating expenses |
14,460 |
|
|
11,031 |
|
|||
Loss from operations |
(14,460) |
|
|
(11,031) |
|
|||
Other income (expense), net |
(549) |
|
|
(542) |
|
|||
Net loss and comprehensive loss |
$ |
(15,009) |
|
|
$ |
(11,573) |
|
|
Net loss per share, basic and diluted |
$ |
(0.65) |
|
|
$ |
(2.43) |
|
|
Weighted average common shares outstanding, basic and diluted |
23,188,816 |
|
|
4,775,828 |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20200511005106/en/
Company/Investor Contact
jfredette@axcellahealth.com
(857) 320-2236
Media Contact
Azeem Zeekrya
azeem.zeekrya@hdmz.com
(312) 506-5244
Source: