Axcella Reports Third Quarter 2020 Financial Results and Provides Business Update
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Plan to advance AXA1125 directly into a Phase 2b biopsy clinical trial in
NASH under IND following successful Type B pre-IND meeting with FDA - Plan to advance AXA1665 directly into a Phase 2 clinical trial in OHE under IND following positive top-line data from AXA1665-002
- Published peer-reviewed manuscripts highlighting therapeutic benefit of EMMs and results from AXA1665-001 clinical study
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Presented AXA1125 late-breaker at
EASL Digital International Liver Congress -
Company to hold conference call today at
8:30 a.m. ET
“Recent accomplishments serve as further validation of the strength of Axcella’s execution amid the COVID-19 pandemic as well as its clinical and regulatory approach,” said
“Just three years ago, Axcella finalized the designs of these differentiated, multi-targeted product candidates, and we now are prepared to enter later-stage clinical trials. Better still, we have compiled far more human data than would normally be expected at this stage of development, which we believe increases the probability of success in our next phase of development as we seek to make a difference for patients,”
Recent Accomplishments and Next Steps
AXA1125
- Completed Successful Type B Meeting: Axcella recently participated in a Type B pre-IND meeting with the FDA regarding AXA1125, focusing on key elements of the company’s development strategy. This engagement enables Axcella to proceed with its IND submission and Phase 2b clinical trial design as planned.
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Planned Phase 2b Clinical Trial: Axcella plans to submit an IND for AXA1125 and proceed directly into a 48-week Phase 2b serial biopsy clinical trial enrolling adult patients with
NASH , with a primary endpoint based on liver histology. This trial is expected to be initiated in the first half of 2021. -
Presented Late-Breaker at EASL for AXA1125: A late-breaker poster containing key findings from Axcella’s AXA1125-003 clinical study was presented at the
EASL Digital International Liver Congress byStephen A. Harrison , M.D., Medical Director ofPinnacle Clinical Research inSan Antonio, TX and visiting professor of Hepatology at the University ofOxford, UK . -
Planned Presentations at AASLD: Posters regarding AXA1125-003 will be presented at The Liver Meeting® 2020, the Annual Meeting of the
American Association for the Study of Liver Diseases (AASLD), which is taking place virtuallyNovember 13-16, 2020 .
AXA1665
- Reported Positive Top-Line Data: Axcella reported top-line data from AXA1665-002, a 12-week clinical study assessing the impact of AXA1665 on safety, tolerability and physiology in subjects with mild and moderate hepatic insufficiency. Results from the study showed that AXA1665 was generally well-tolerated, with dose dependent changes noted across measures of amino acid metabolism and neurocognition. These included statistically significant (p <0.05) improvements in the Fischer Ratio and the psychometric hepatic encephalopathy score (PHES) in the AXA1665 high dose arm vs. placebo. Additionally, clinically relevant trends were seen in certain measures of nitrogen/ammonia handling and physical function in the AXA1665 arms versus placebo.
- Planned Phase 2 Clinical Trial Initiation: Axcella plans to submit an IND for AXA1665 and proceed directly into a 24-week Phase 2 clinical trial enrolling subjects with liver cirrhosis who have experienced at least one prior OHE event. This trial is expected to be initiated in the first half of 2021.
- Published Findings from Initial Investigation of AXA1665: Clinical and Translational Gastroenterology published a peer-reviewed report detailing results from Axcella’s AXA1665-001 clinical study, the initial clinical investigation of AXA1665’s effect on safety, tolerability, and clinically relevant biomarkers related to hepatic and muscle metabolism and function.
AXA4010
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Planned Enrollment Decision for AXA4010-001: Following Axcella’s receipt of Cohort 1 data in
December 2020 from the ongoing AXA4010-001 clinical study, the company expects to communicate its decision about whether to enroll additional subjects with sickle cell disease in the study by early 2021.
EMM Platform
- Published Manuscript Elucidating the Therapeutic Potential of EMMs: iScience published a peer-reviewed manuscript entitled “Endogenous Metabolic Modulators: Emerging Therapeutic Potential of Amino Acids” that detailed clinical precedents for EMMs as therapeutics and discussed the potential to develop EMM compositions that simultaneously target multiple biological pathways to address unmet needs in a range of complex diseases.
Financial Results
Cash Position: As of
R&D Expenses: Research and development expenses for the quarter and nine months ended
G&A Expenses: General and administrative expenses for the quarter and nine months ended
Net Loss: Net loss for the quarter and nine months ended
Conference Call Reminder
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 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 this portion of the company’s website, in addition to following its press releases,
About Axcella
Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. 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 product candidates and the company’s characterization of the results from its clinical studies and future clinical trials, including for AXA1125 and AXA1665, the design, status and timing of the company’s ongoing clinical study and planned IND-enabled clinical trials, the company’s anticipated program milestones, including the timing of data readout from Cohort 1 of AXA4010-001, the subject and timing of the company’s planned interactions with the FDA on the AXA1665 and AXA1125 programs, including the potential timing of IND application submissions for its product candidates, including AXA1125 and AXA1665, the potential of the company’s product candidates to impact health and/or disease, including AXA1125’s potential in
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Unaudited Condensed Consolidated Balance Sheets |
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(in thousands) |
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2020 |
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2019 |
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Assets: |
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Cash and cash equivalents |
|
$ |
114,063 |
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$ |
92,053 |
|
Marketable securities |
|
3,192 |
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— |
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Other assets |
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2,898 |
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2,306 |
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Total assets |
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$ |
120,153 |
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$ |
94,359 |
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Liabilities and stockholders' equity: |
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Liabilities |
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$ |
31,391 |
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$ |
34,135 |
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Stockholders' equity |
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88,762 |
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|
60,224 |
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Total liabilities and stockholders' equity |
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$ |
120,153 |
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$ |
94,359 |
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Unaudited Condensed Consolidated Statements of Operations |
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(in thousands, except share and per share data) |
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Three Months Ended
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Nine Months Ended
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2020 |
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2019 |
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2020 |
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2019 |
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Operating expenses: |
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Research and development |
$ |
7,541 |
|
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$ |
12,157 |
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$ |
26,441 |
|
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$ |
29,063 |
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General and administrative |
4,184 |
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4,840 |
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12,928 |
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13,036 |
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Total operating expenses |
11,725 |
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16,997 |
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39,369 |
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42,099 |
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Loss from operations |
(11,725) |
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(16,997) |
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(39,369) |
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(42,099) |
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Other income (expense), net |
(712) |
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(307) |
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(1,969) |
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(1,225) |
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Net loss |
$ |
(12,437) |
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$ |
(17,304) |
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$ |
(41,338) |
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$ |
(43,324) |
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Net loss per share, basic and diluted |
$ |
(0.34) |
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$ |
(0.75) |
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$ |
(1.39) |
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$ |
(3.01) |
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Weighted average common shares outstanding, basic and diluted |
36,942,475 |
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23,083,367 |
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29,804,034 |
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14,430,397 |
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View source version on businesswire.com: https://www.businesswire.com/news/home/20201112005213/en/
Company Contact
jfredette@axcellahealth.com
(857) 320-2236
Source: Axcella