- First IND application cleared by FDA for AXA1665
- Initiation of AXA1665 Phase 2 clinical trial in OHE expected in
Q2 2021
- Initiation of AXA1125 Phase 2b clinical trial in NASH expected
in Q2 2021
- Company to host conference call at 8:30 a.m. ET today
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 fourth quarter and full
year ended December 31, 2020 and provided a business update.
“The year 2020 was a key inflection point for Axcella as we
completed multiple clinical studies with positive results and
shifted our full attention to our upcoming later-stage clinical
trials,” said Bill Hinshaw, President and Chief Executive Officer
of Axcella. “We were pleased to advance our preparations in the
fourth quarter with a successful Type B pre-IND meeting with the
U.S. Food and Drug Administration (FDA) regarding our nonalcoholic
steatohepatitis (NASH) candidate, AXA1125, and began 2021 with our
first investigational new drug (IND) clearance for AXA1665, our
candidate for the reduction in risk of recurrent overt hepatic
encephalopathy (OHE). Axcella’s strong execution has positioned us
for an exciting year ahead as we seek to swiftly enroll our
upcoming trials, expand our pipeline of EMM compositions and
further our mission to improve the lives of patients with complex
diseases.”
Recent Accomplishments and Next
Steps
AXA1665 for OHE
- Cleared IND Application: In January 2021, Axcella’s IND
application was cleared by the FDA for AXA1665. This first IND
clearance marks a key milestone and validates the company’s
clinical approach with its EMM compositions.
- Planned Phase 2 Clinical Trial Initiation: Axcella plans
to initiate a 24-week Phase 2 clinical trial of AXA1665 in the
second quarter of 2021. This will be a randomized, double-blind,
placebo-controlled, multi-center trial evaluating the efficacy and
safety of AXA1665 in patients who have experienced at least one
prior OHE event and have neurocognitive dysfunction at screening.
Approximately 150 patients on lactulose ± rifaximin (stratified by
rifaximin use) will be enrolled and randomized 1:1 to receive
either 53.8 grams per day of AXA1665 or a calorie-matched placebo
in three divided doses for 24 weeks, with a four-week safety
follow-up period. The trial will be conducted globally with a
primary endpoint assessing the proportion of subjects with a ≥2
point increase in the psychometric hepatic encephalopathy score
(PHES) after the 24-week treatment period. Secondary endpoints will
include the proportion of patients experiencing an OHE breakthrough
event; time to first OHE breakthrough event, including time to
hospitalization; changes in physical function; and patient-reported
outcomes. Other endpoints include measures of circulating ammonia,
amino acids, and inflammation-related markers.
AXA1125 for NASH
- Advanced IND Preparations: Axcella participated in a
Type B pre-IND meeting with the FDA regarding AXA1125 in the fourth
quarter of 2020. This engagement provided the insight required for
the company to advance its Phase 2b clinical trial design and
finalize its preparation of an IND submission.
- Planned Phase 2b Clinical Trial: Following IND clearance
by the FDA, Axcella plans to proceed directly into a 48-week Phase
2b serial biopsy clinical trial of AXA1125 enrolling adult patients
with NASH, with a primary endpoint based on liver histology. This
trial is expected to be initiated in the second quarter of
2021.
- Presented Data at The Liver Meeting 2020: Two posters
regarding AXA1125 were presented in November 2020 at The Liver
Meeting® 2020, the Annual Meeting of the American Association for
the Study of Liver Diseases (AASLD). Both presentations featured
data from AXA1125-003, a placebo-controlled, randomized clinical
study that enrolled 102 subjects with presumed nonalcoholic
steatohepatitis (NASH) to assess the impact of AXA1125 and AXA1957
on safety, tolerability and effects on structures and functions of
the liver for 16 weeks.
- Presented Data at NASH-TAG 2021: Last week, multiple
presentations regarding AXA1125 were included in NASH-TAG 2021, an
event bringing clinicians and researchers together to focus on the
diagnosis and therapy of NASH and liver fibrosis. Among them was a
poster presentation providing novel insights regarding AXA1125’s
multi-targeted mechanism of action and a AXA1125-003 clinical study
data presentation that was recognized with a Distinguished Abstract
and Poster Award.
Management Team
- Added President of R&D: Axcella announced the
appointment of Alison D. Schecter, M.D., as the company’s President
of Research and Development. In this role, Dr. Schecter oversees
all of the company’s research, product candidate design, clinical
and regulatory efforts. She brings more than 20 years of research,
clinical and regulatory experience to Axcella at companies such as
Selecta Biosciences, Sanofi-Genzyme, Baxalta, Novartis and Johnson
& Johnson.
Financial Results
Cash Position: As of December 31, 2020, cash, cash
equivalents, and marketable securities totaled $107.3 million,
compared to $92.1 million at 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. Axcella expects that
its cash balance will be sufficient to meet the company’s operating
needs into the third quarter of 2022.
R&D Expenses: Research and development expenses were
$10.6 million and $10.8 million for the quarters ended December 31,
2020 and 2019, respectively. For the years ended December 31, 2020
and 2019, research and development expenses were $37.0 million and
$41.7 million, respectively. The year-over-year decrease is
primarily due to the completion of the company’s AXA1665-002 and
AXA1125-003 clinical studies.
G&A Expenses: General and administrative expenses
were $3.9 million and $4.6 million for the quarters ended December
31, 2020 and 2019, respectively. For the years ended December 31,
2020 and 2019, general and administrative expenses were $16.8
million and $15.8 million, respectively. The year-over-year
increase is primarily due to greater costs associated with becoming
a public company.
Net Loss: Net loss for the quarter ended December 31,
2020 was $15.2 million, or $0.40 per basic and diluted share. This
compares with a net loss of $15.7 million, or $0.68 per basic and
diluted share, for the quarter ended December 31, 2019. Net loss
for the year ended December 31, 2020 was $56.5 million, or $1.78
per basic and diluted share. This compares with a net loss of $59.0
million, or $3.55 per basic and diluted share, for the year ended
December 31, 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 (844) 808-7139 (U.S. toll free) or (412) 902-0127
(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 90 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 trials 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. 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 AXA1665, AXA1125 and potential future EMM
compositions, the potential for our product candidates to improve
the quality of life for patients with complex diseases, the status
and timing of the company’s planned Phase 2 clinical trial of
AXA1665 and planned Phased 2b clinical trial of AXA1125, the timing
and outcome of IND application submissions and the company’s
expected cash runway into the third quarter of 2022. 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 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 planned clinical trials of AXA1665 and AXA1125,
other potential impacts of COVID-19 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 initiation plans and timing, clinical trial design
and target indications for AXA1665 and AXA1125, the clinical
development and safety profile of AXA1665 and AXA1125 and their
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 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)
December 31,
2020
December 31,
2019
Assets:
Cash and cash equivalents
$
71,590
$
92,053
Marketable securities
35,739
—
Other assets
2,263
2,306
Total assets
$
109,592
$
94,359
Liabilities and stockholders' equity:
Liabilities
$
34,211
$
34,135
Stockholders' equity
75,381
60,224
Total liabilities and stockholders'
equity
$
109,592
$
94,359
Axcella Health Inc.
Unaudited Condensed Consolidated Statements of Operations
(in thousands, except share and per share data)
Three Months Ended December
31,
Year Ended December
31,
2020
2019
2020
2019
Operating expenses:
Research and development
$
10,598
$
10,758
$
37,039
$
41,658
General and administrative
3,869
4,582
16,797
15,781
Total operating expenses
14,467
15,340
53,836
57,439
Loss from operations
(14,467
)
(15,340
)
(53,836
)
(57,439
)
Other income (expense):
Interest income (expense) and other income
(expense), net
(722
)
(373
)
(2,691
)
(1,598
)
Total other income (expense), net
(722
)
(373
)
(2,691
)
(1,598
)
Net loss
$
(15,189
)
$
(15,713
)
$
(56,527
)
$
(59,037
)
Net loss per share, basic and diluted
$
(0.40
)
$
(0.68
)
$
(1.78
)
$
(3.55
)
Weighted average common shares
outstanding, basic and diluted
37,536,350
23,137,014
31,747,676
16,624,941
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210317005106/en/
Company Contact Jason Fredette
jfredette@axcellahealth.com (857) 320-2236
Axcella Health (NASDAQ:AXLA)
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