- Top-line data from AXA1665-002 show dose dependent improvements
in markers of cognitive function and amino acid metabolism; both
AXA1665 doses safe and well tolerated for 12 Weeks; plan to
initiate Phase 2 clinical trial under IND in 1H 2021
- Reported positive top-line data for AXA1125, Axcella’s NASH
product candidate, with clinically relevant reductions in liver fat
content, insulin resistance and fibroinflammation; plan to initiate
Phase 2b clinical trial under IND in 1H 2021
- Completed follow-on equity offering, raising approximately $60
million in gross proceeds
- Company to hold conference call today at 8:30 a.m. ET
Axcella (Nasdaq: AXLA), a clinical-stage biotechnology company
focused on leveraging endogenous metabolic modulators (EMMs) to
pioneer a new approach for treating complex diseases and improving
health, today reported positive top-line 12-week data from
AXA1665-002, a placebo-controlled clinical study of AXA1665, and
financial results for the second quarter ended June 30, 2020.
“The second quarter of 2020 was a time of significant
accomplishment for Axcella,” said Bill Hinshaw, President and Chief
Executive Officer of Axcella. “This period was highlighted by
positive top-line data from our AXA1125-003 clinical study, which
served as another strong validator for our novel EMM platform.
Preparations are now well underway for our engagement with the U.S.
Food and Drug Administration (FDA) regarding our adult and
pediatric NASH programs as we seek to finalize the design of our
proposed Phase 2b clinical trial of AXA1125 and initiate enrollment
in the first half of 2021. We are appreciative of the support from
the investors that enabled us to bolster our balance sheet via a
follow-on equity offering in May 2020.”
“Today, we are pleased to share our AXA1665-002 top-line data
that once again demonstrate safety, tolerability and activity
across multiple biologies and, for the first time, show positive
changes in neurocognitive measures. After additional data analyses
and consultation with external medical experts, we plan to submit
an IND and initiate a Phase 2 clinical trial of AXA1665 in patients
with advanced liver disease. Ultimately, our goal is to provide a
much-needed new treatment option for the many patients who have
experienced an overt hepatic encephalopathy (OHE) event. We extend
our sincere thanks to all of the subjects and investigators
involved in AXA1665-002 for their participation in and commitment
to this study, particularly in light of the ongoing global
pandemic,” Mr. Hinshaw concluded.
AXA1665-002 Top-Line Data
AXA1665-002 was a placebo-controlled, randomized clinical study
that was designed to investigate the safety, tolerability and
physiological impact of AXA1665, a proprietary composition of eight
amino acids, in 60 subjects with mild (Child Pugh A) and moderate
(Child Pugh B) hepatic insufficiency. Subjects in the study were
randomized in a 2:2:1 ratio to receive either 29.4 g or 53.9 g of
AXA1665 or a matched placebo in three divided doses per day for 12
weeks with a four-week follow up. In addition to safety and
tolerability, the study evaluated plasma amino acid and ammonia
levels as well as markers of neurocognition, muscle structure and
function.
“We believe the data from the AXA1665-002 study show AXA1665’s
potential to address multiple fundamental dysregulations associated
with cirrhosis and hepatic encephalopathy,” said Manu Chakravarthy,
M.D., Ph.D., Chief Medical Officer of Axcella. “This study
replicated findings on amino acid metabolism from our previous
short-term study, AXA1665-001, and we were pleased to see those
effects sustained through 12 weeks. We also noted dose dependent,
directionally consistent changes across all three psychometric
tests that were utilized, which help to bolster our confidence in
AXA1665’s potential to reduce OHE events. We look forward to
initiating a Phase 2 clinical trial to investigate this hypothesis
and further evaluate this candidate’s impact on measures of
physical function and related patient reported outcomes in patients
with advanced liver disease.”
Key results from AXA1665-002 include:
- Safety/Tolerability: Both doses of AXA1665 were safe and
well-tolerated. Rates of adverse events (AEs) were low, mostly
unrelated to study product and generally mild or moderate. There
were four serious adverse events reported in the study and two
deaths (one due to complications of COVID-19; one due to a
myocardial infarction during the study run-in period prior to
dosing), none of which were determined to be related to
AXA1665.
- Neurocognitive Function: Positive, dose dependent trends
were observed in the AXA1665 arms across all three psychometric
tests: Stroop EncephalApp, critical flicker frequency, and
psychometric hepatic encephalopathy score (PHES). In PHES, a highly
specific assessment to diagnose minimal hepatic encephalopathy
(MHE), a statistically significant (p <0.05) improvement of a
clinically relevant magnitude was observed in the AXA1665 high dose
arm vs. placebo. Additionally, the proportion of subjects achieving
a clinically relevant threshold of PHES improvement was higher in
the AXA1665 arms relative to placebo.
- Amino Acid Metabolism: A dose dependent and
statistically significant (p <0.05) percentage increase from
baseline in Fischer Ratio (FR; a measure of branched chain amino
acids ÷ aromatic amino acids) was seen in the AXA1665 arms relative
to placebo (low dose: 21%, high dose: 44%), which was sustained
over 12 weeks. Observed changes in FR were accompanied by
concomitant decreases in circulating aromatic amino acids (Phe and
Tyr), which may suggest their incorporation into protein synthesis
and an improved metabolic state. Published studies suggest that
aromatic amino acids may contribute to impaired neurotransmission
and have correlated lower FR with poor clinical outcomes in
patients with cirrhosis and end-stage liver disease.
- Ammonia Handling: Despite the increased nitrogen load
delivered via the amino acids in AXA1665, fasted plasma ammonia
levels remained stable in the active arms over the 12-week dosing
duration. In a subset of subjects with evidence of MHE at baseline
as assessed by PHES, a mean reduction from baseline of
approximately 7% in fasted plasma ammonia levels was observed in
subjects receiving both doses of AXA1665 at week 12.
- Muscle Structure and Function: Key measures of muscle
structure (e.g. lean mass) and function (e.g. gait speed, liver
frailty index, or LFI) remained essentially stable in all groups
from baseline to week 12. This observation may reflect the mild
hepatic insufficiency and lack of overt sarcopenia in nearly all
enrolled subjects at baseline. A higher proportion of subjects in
the AXA1665 arms achieved a ≥0.3 absolute reduction in LFI (i.e.
less frailty) versus placebo. Previous studies suggest that a ≥0.3
reduction in the LFI score may correlate with an improved ability
to conduct activities of daily living in subjects with end-stage
liver disease.
“Overt hepatic encephalopathy is a complex disease that involves
dysregulation across multiple organ systems, including altered
amino acids, elevated ammonia levels, dysregulated muscle
metabolism, and cognitive dysfunction,” said Dr. Arun Sanyal,
Professor of Medicine, Physiology and Molecular Pathology at
Virginia Commonwealth University School of Medicine, and an
investigator in AXA1665-002. “While previous approaches have
focused on reducing ammonia load from the bowel alone, future
approaches should incorporate the evolving knowledge of the role of
other organs, such as muscle and the brain, in the development of
encephalopathy. I am encouraged by the multifactorial activity seen
with AXA1665 in the 002 study and look forward to its continued
investigation in a subsequent Phase 2 trial to evaluate AXA1665’s
therapeutic potential to prevent OHE recurrence in advanced liver
disease patients who are in dire need of new treatment
options.”
Additional data will be discussed during the company’s
conference call at 8:30 a.m. ET today and will be included in a
presentation that will be posted to “Investors & News” section
of Axcella’s website prior to the call. Details about how to access
this conference call are included below.
Other Recent Developments
- AXA1125 Top-Line Data: 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, with a greater magnitude noted
among subjects with type 2 diabetes.
- AXA1665 Patents: Announced the issuance of two key
patents: U.S. Patent 10,682,325 and U.S. Patent 10,660,870. These
are the first patents related to Axcella’s family of applications
for AXA1665, the company’s product candidate for the reduction in
risk of overt hepatic encephalopathy recurrence, covering both its
composition and methods of use. These patents follow the issuance
of composition and methods of use patents for Axcella’s other lead
product candidate, AXA1125, in 2019.
- Follow-On Stock Offering: Axcella closed an underwritten
public offering of an aggregate of 12,650,000 shares of its common
stock, including the full exercise of the underwriters’ option to
purchase additional shares. The gross proceeds of the offering,
before deducting underwriting discounts and commissions and other
estimated offering expenses payable by Axcella, were approximately
$60.1 million.
- Addition to Board of Directors: The company also today
announced that Chief Development Officer Shreeram Aradhye, M.D.,
will be stepping down from his full-time role to accept another
opportunity and will be appointed to Axcella’s Board of Directors,
effective on September 1, 2020. Dr. Aradhye has more than 20 years
of pharmaceutical industry experience in clinical development and
medical affairs, having previously served as Chief Medical Officer
and Global Head, Medical Affairs for Novartis Pharmaceuticals. In
previous roles at Novartis and Sandoz, he provided functional
leadership for clinical development and medical affairs teams
working on novel and biosimilar medicines across multiple
indications, including multiple sclerosis, Alzheimer’s Disease,
neuropathic pain, muscle disease and migraine.
- Management Update: Chief Medical Officer Manu
Chakravarthy, M.D., Ph.D., has been promoted to Executive Vice
President and will assume Dr. Aradhye’s day-to-day
responsibilities. Additionally, Andrew Suchoff joined Axcella as
Chief People Officer in June 2020. He is driving initiatives aimed
at the company’s culture, talent, learning and development,
employee engagement and compensation. Mr. Suchoff brings more than
20 years of human resources leadership experience to Axcella,
having most recently served as Global Head of People Operations and
Talent Development at Stallergenes Greer, a healthcare company with
more than 1,200 employees and operations throughout the world.
Upcoming Planned Milestones
Liver Programs
- Q3 2020: Present AXA1125-003 data (late-breaker poster
presentation) at The Digital International Liver Congress 2020
(EASL)
- 2H 2020: Engage with the FDA regarding the company’s planned
IND application for AXA1125, proposed Phase 2b clinical trial in
adults and pediatric development program
- 1H 2021: Initiate a Phase 2b clinical trial of AXA1125 in adult
NASH under an IND
- 1H 2021: Initiate a Phase 2 clinical trial of AXA1665 in
patients with advanced liver disease (i.e. in cirrhotic subjects
with at least one prior episode of OHE) under an IND
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 $8.6 million and $9.3 million for the
quarters ended June 30, 2020 and 2019, respectively. The change was
primarily related to the completion of the company’s AXA1125-003
clinical study.
G&A Expenses: General and
administrative expenses were $4.6 million and $4.7 million for the
quarters ended June 30, 2020 and 2019, respectively.
Net Loss: Net loss for the quarter
ended June 30, 2020 was $13.9 million, or $0.48 per basic and
diluted share. This compares with a net loss of $14.4 million, or
$0.95 per basic and diluted share, for the quarter ended June 30,
2019.
Cash Position: Cash and cash
equivalents at June 30, 2020 were $121.3 million, which compares
with $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.
Conference Call Reminder
Axcella will host a conference call today at 8:30 a.m. ET to
discuss the top-line data from AXA1665-002 and other recent
business updates. The conference call webcast and accompanying
slides will be made available shortly before the start of the call
on the company’s website at www.axcellahealth.com in the Investors
& News section. 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 &
News” section. Accordingly, investors should monitor such portions
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 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 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 studies
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, 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, 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 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 AXA4010-001 clinical study 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 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)
June 30,
December 31,
2020
2019
Assets:
Cash and cash equivalents
$
121,326
$
92,053
Other assets
3,222
$
2,306
Total assets
$
124,548
$
94,359
Liabilities and stockholders' equity
(deficit):
Liabilities
$
31,585
$
34,135
Stockholders' equity (deficit)
92,963
$
60,224
Total liabilities and stockholders'
equity
$
124,548
$
94,359
Axcella Health Inc.
Unaudited Condensed
Consolidated Statements of Operations and Comprehensive
Loss
(in thousands, except share
and per share data)
Three Months Ended June
30,
Six Months Ended June
30,
2020
2019
2020
2019
Operating expenses:
Research and development
$
8,565
$
9,343
$
10,335
$
7,563
General and administrative
4,619
4,728
4,125
3,468
Total operating expenses
13,184
14,071
14,460
11,031
Loss from operations
(13,184
)
(14,071
)
(14,460
)
(11,031
)
Other income (expense), net
(708
)
(376
)
(549
)
(542
)
Net loss and comprehensive loss
$
(13,892
)
$
(14,447
)
$
(15,009
)
$
(11,573
)
Net loss per share, basic and diluted
$
(0.48
)
$
(0.95
)
$
(0.65
)
$
(2.43
)
Weighted average common shares
outstanding, basic and diluted
29,202,367
15,230,815
23,188,816
4,775,828
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200805005224/en/
Company/Investor Contact Jason Fredette
jfredette@axcellahealth.com (857) 320-2236
Media Contact Azeem Zeekrya azeem.zeekrya@hdmz.com (312)
506-5244
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