- 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
- Presented AXA1125 late-breaker at EASL Digital International
Liver Congress
- Company to hold conference call today at 8:30 a.m. ET
Axcella (Nasdaq: AXLA), a clinical-stage biotechnology company
pioneering a new approach to treat complex diseases and improve
health using endogenous metabolic modulator (EMM) compositions,
today announced financial results for the third quarter ended
September 30, 2020 and provided a business update.
“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 Bill Hinshaw,
President and Chief Executive Officer of Axcella. “We are pleased
to report that we recently completed a successful Type B pre-IND
meeting with the U.S. Food and Drug Administration (FDA) that
enabled us to affirm our plans to submit an Investigational New
Drug (IND) application and initiate a Phase 2b biopsy clinical
trial of AXA1125, our product candidate for nonalcoholic
steatohepatitis (NASH). We also presented positive top-line data in
the third quarter for AXA1665, our product candidate for the
reduction in risk of overt hepatic encephalopathy (OHE) recurrence.
Having already completed our pre-IND FDA engagement for AXA1665,
preparations for its IND submission and Phase 2 clinical trial are
now underway.”
“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,” Mr. Hinshaw continued. “With
key catalysts for AXA1125 and AXA1665 as well as our decision
regarding enrollment expansion for our AXA4010-001 clinical study
on the near-term horizon, our excitement continues to build.”
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.
- 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 by Stephen A. Harrison, M.D.,
Medical Director of Pinnacle Clinical Research in San Antonio, TX
and visiting professor of Hepatology at the University of Oxford,
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
virtually November 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
- 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 September 30, 2020, cash, cash
equivalents and marketable securities totaled $117.3 million,
compared to $92.1 million as of December 31, 2019. The increase is
primarily the result of net proceeds from the company’s follow-on
stock offering that was completed in May 2020.
R&D Expenses: Research and development expenses for
the quarter and nine months ended September 30, 2020 were $7.5
million and $26.4 million, respectively. Research and development
expenses for the same periods ended September 30, 2019 were $12.2
million and $29.1 million. The decrease for the three and nine
months ended September 30, 2020 is primarily due to the completion
of the company’s AXA1125-003 and AXA1665-002 clinical studies.
G&A Expenses: General and administrative expenses for
the quarter and nine months ended September 30, 2020 were $4.2
million and $12.9 million, respectively. General and administrative
expenses for the same periods ended September 30, 2019 were $4.8
million and $13.0 million.
Net Loss: Net loss for the quarter and nine months ended
September 30, 2020 was $12.4 million, or $0.34 per basic and
diluted share, and $41.3 million, or $1.39 per basic and diluted
share, respectively. This compares with a net loss of $17.3
million, or $0.75 per basic and diluted share, and $43.3 million,
or $3.01 per basic and diluted share, for the quarter and nine
months ended September 30, 2019. Included in the net loss for the
quarter and the nine months ended September 30, 2020 was $1.4
million and $4.9 million, respectively, of non-cash expense related
to stock-based compensation, as compared to $1.7 million and $4.3
million, respectively, for the same periods in 2019.
Conference Call Reminder
Axcella will host a conference call today at 8:30 a.m. ET to
discuss the company’s financial results and other recent business
updates. The conference call webcast will be accessible in the
Investors & News section on the company’s website at
www.axcellahealth.com. To access the call via telephone, please
dial (866) 652-5200 (U.S. toll free) or (412) 317-6060
(international) five minutes prior to the start time. For those
unable to listen in live, a webcast archive will be available on
the company’s website for 30 days following the call.
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 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, any subsequent clinical 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 this portion
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 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 (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 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 NASH
and AXA1665’s potential in OHE, and the importance of any
intellectual rights granted to the company. 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, including
planned IND submissions for AXA1125 and AXA1665, 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 ongoing AXA4010-001 clinical study and potential
delays in disclosure of the same, other potential impacts of
COVID-19 on the company’s our business and financial results,
including with respect to the company’s 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 the
company’s IND submission and clinical trial initiation plans and
timing, clinical trial design and target indications 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 in
clinical studies or IND-enabled clinical trials, and other risks
identified in the company’s SEC filings, including Axcella’s Annual
Report on Form 10-K, Quarterly Report 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 press release 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.
Axcella Health Inc.
Unaudited Condensed
Consolidated Balance Sheets
(in thousands)
September 30,
December 31,
2020
2019
Assets:
Cash and cash equivalents
$
114,063
$
92,053
Marketable securities
3,192
—
Other assets
2,898
2,306
Total assets
$
120,153
$
94,359
Liabilities and stockholders' equity:
Liabilities
$
31,391
$
34,135
Stockholders' equity
88,762
60,224
Total liabilities and stockholders'
equity
$
120,153
$
94,359
Axcella Health Inc.
Unaudited Condensed
Consolidated Statements of Operations
(in thousands, except share
and per share data)
Three Months Ended
September 30,
Nine Months Ended
September 30,
2020
2019
2020
2019
Operating expenses:
Research and development
$
7,541
$
12,157
$
26,441
$
29,063
General and administrative
4,184
4,840
12,928
13,036
Total operating expenses
11,725
16,997
39,369
42,099
Loss from operations
(11,725)
(16,997)
(39,369)
(42,099)
Other income (expense), net
(712)
(307)
(1,969)
(1,225)
Net loss
$
(12,437)
$
(17,304)
$
(41,338)
$
(43,324)
Net loss per share, basic and diluted
$
(0.34)
$
(0.75)
$
(1.39)
$
(3.01)
Weighted average common shares
outstanding, basic and diluted
36,942,475
23,083,367
29,804,034
14,430,397
View source
version on businesswire.com: https://www.businesswire.com/news/home/20201112005213/en/
Company Contact Jason Fredette
jfredette@axcellahealth.com (857) 320-2236
Axcella Health (NASDAQ:AXLA)
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