10-K
1
azrx10k_dec312020.htm
ANNUAL REPORT
azrx10k_dec312020
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 20549
FORM 10-K
For the fiscal year ended December 31, 2020
or
Commission file No. 001-37853
AZURRX BIOPHARMA, INC.
(Exact name of registrant as specified in its charter)
1615 South Congress Avenue, Suite 103
Delray Beach, Florida 33445
(Address of principal executive offices)
(646) 699-7855
(Issuer’s telephone number)
Securities registered pursuant
to Section 12(b) of the Act:
Title of Each Class Trading Symbol Name of Each Exchange on Which Registered
Common stock, par value $0.0001 per share AZRX Nasdaq Capital Market
Securities registered under Section 12(g) of the Exchange Act:
None
Indicate by check mark if the registrant is a well-known seasoned
issuer, as defined in Rule 405 of the Securities Act. Yes [ ]
No [X]
Indicate by check mark if the registrant is not required to file
reports pursuant to Section 13 or Section 15(d) of the Act. Yes [
] No [X]
Indicate by check mark whether the registrant (1) has filed all
reports required to be filed by Section 13 or 15(d) of the
Securities Exchange Act of 1934 during the preceding 12 months (or
for such shorter period that the registrant was required to file
such reports), and (2) has been subject to such filing requirements
for the past 90 days. Yes [X]No
[ ]
Indicate by check mark whether the registrant has submitted
electronically every Interactive Data File required to be submitted
pursuant to Rule 405 of Regulation S-T (§232.405 of this
chapter) during the preceding 12 months (or for such shorter period
that the registrant was required to submit such files). Yes [X] No
[ ]
Indicate by check mark whether the registrant is a large
accelerated filer, an accelerated filer, a non-accelerated filer,
smaller reporting company, or an emerging growth company. See the
definitions of “large accelerated filer,”
“accelerated filer,” “smaller reporting
company,” and “emerging growth company” in Rule
12b-2 of the Exchange Act.
Large accelerated filer [ ] Accelerated filer [ ]
Non-accelerated filer [X] Smaller reporting company [X]
Emerging growth company [X]
If an emerging growth company, indicate by check mark if the
registrant has elected not to use the extended transition period
for complying with any new or revised financial accounting
standards provided pursuant to Section 13(a) of the Exchange
Act.[
]
Indicate by check mark whether the registrant has filed a report on
and attestation to its management’s assessment of the
effectiveness of its internal control over financial reporting
under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b))
by the registered public accounting firm that prepared or issued
its audit report. [ ]
Indicate by check mark whether the registrant is a shell company
(as defined in Rule 12b-2 of the Exchange Act). Yes [ ] No
[X]
The aggregate market value of common stock held by non-affiliates
of the registrant, based on the closing price of a share of the
registrant’s common stock on June 30, 2020, which is the last
business day of the registrant’s most recently completed
second fiscal quarter, as reported by the Nasdaq Capital Market on
such date, was approximately $25.2 million.
There were 74,439,377 shares of the registrant’s common
stock, par value $0.0001 per share (the “Common
Stock”), outstanding as
of March 29, 2021.
AZURRX BIOPHARMA, INC.
ANNUAL REPORT ON FORM 10-K
YEAR ENDED DECEMBER 31, 2020
TABLEOF CONTENTS
Page
PART I
Item 1. Description of Business 1
Item 1A. Risk Factors 29
Item 1B. Unresolved Staff Comments 57
Item 2. Properties 57
Item 3. Legal Proceedings 57
Item 4. Mine Safety Disclosures 57
PART II
Item 6. Selected Financial Data 58
Item 7A. Quantitative and Qualitative Disclosures About Market Risk 66
Item 8. Financial Statements and Supplementary Data 66
Item 9A. Controls and Procedures 66
Item 9B. Other Information 67
PART III
Item 10. Directors, Executive Officers and Corporate Governance 68
Item 11. Executive Compensation 73
Item 14. Principal Accountant Fees and Services 87
PART IV
Item 15. Exhibits, Financial Statement Schedules 88
Signatures 91
Index to Consolidated Financial Statements F-1
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CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Annual Report on Form 10-K
(“Annual
Report”) contains
forward-looking statements that involve substantial risks and
uncertainties. All statements contained in this Annual Report other
than statements of historical facts, including statements regarding
our strategy, future operations, future financial position, future
revenue, projected costs, prospects, plans, objectives of
management and expected market growth, are forward-looking
statements. These statements involve known and unknown risks,
uncertainties and other important factors that may cause our actual
results, performance or achievements to be materially different
from any future results, performance or achievements expressed or
implied by the forward-looking statements.
The
words “anticipate”, “believe”,
“estimate”, “expect”, “intend”,
“may”, “plan”, “predict”,
“project”, “target”,
“potential”, “will”, “would”,
“could”, “should”, “continue”
and similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words. These forward-looking statements include,
among other things, statements about:
●
statements regarding the impact of the COVID-19
pandemic and its effects on our operations, access to capital,
research and development and clinical trials and potential
disruption in the operations and business of third-party vendors,
contract research organizations (“CROs”), contract development and manufacturing
organizations (“CDMOs”), other service providers, and
collaborators with whom we conduct business;
●
the
availability of capital to satisfy our working capital
requirements;
●
our
current and future capital requirements and our ability to raise
additional funds to satisfy our capital
needs;
●
the
accuracy of our estimates regarding expense, future revenue and
capital requirements;
●
our ability to
continue operating as a going concern;
●
our
plans to develop and commercialize our drug candidates, including
MS1819, and niclosamide;
●
our
ability to initiate and complete our clinical trials and to advance
our principal drug candidates into additional clinical trials,
including pivotal clinical trials, and successfully complete such
clinical trials;
●
regulatory
developments in the U.S. and foreign countries;
●
the
performance of our third-party vendor(s), CROs, CDMOs and other
third-party non-clinical and clinical development collaborators and
regulatory service providers;
●
our
ability to obtain and maintain intellectual property protection for
our core assets;
●
the
size of the potential markets for our drug candidates and our
ability to serve those markets;
●
the
rate and degree of market acceptance of our drug candidates for any
indication once approved;
●
the
success of competing products and drug candidates in development by
others that are or become available for the indications that we are
pursuing;
●
the
loss of key scientific, clinical and nonclinical development,
regulatory, and/or management personnel, internally or from one of
our third-party collaborators; and
●
other risks and uncertainties, including those
listed under Part I, Item 1A., “Risk
Factors” of this Annual
Report.
Factors that may cause actual results to differ materially from
current expectations include, among other things, those set forth
in Part I, Item 1A, “Risk
Factors,” herein and for
the reasons described elsewhere in this Annual Report on Form 10-K.
Any forward-looking statement in this Annual Report on Form 10-K
reflects our current view with respect to future events and is
subject to these and other risks, uncertainties and assumptions
relating to our operations, results of operations,industry and future growth. Given
these uncertainties, you should not rely on these forward-looking
statements as predictions of future events. Although we believe
that the expectations reflected in the forward-looking statements
are reasonable, we cannot guarantee future results, levels of
activity, performance or achievements. Except as required by law,
we assume no obligation to update or revise these forward-looking
statements for any reason, even if new information becomes
available in the future.
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This Annual Report on Form 10-K also contains estimates,
projections and other information concerning our industry, our
business and the markets for certain drugs and consumer products,
including data regarding the estimated size of those markets, their
projected growth rates and the incidence of certain medical
conditions. Information that is based on estimates, forecasts,
projections or similar methodologies is inherently subject to
uncertainties and actual events or circumstances may differ
materially from events and circumstances reflected in this
information. Unless otherwise expressly stated, we obtained these
industry, business, market and other data from reports, research
surveys, studies and similar data prepared by third parties,
industry, medical and general publications, government data and
similar sources and we have not independently verified the data
from third party sources. In some cases, we do not expressly refer
to the sources from which these data are derived.
In this Annual Report on Form 10-K, unless otherwise stated or as
the context otherwise requires, references to
“AzurRx,”
the “Company,”
“we,”
“us,”
“our”
and similar referencesare to
AzurRx BioPharma, Inc. and its subsidiaries on a consolidated
basis. References to “AzurRx
BioPharma” refer to
AzurRx BioPharma, Inc. on an unconsolidated basis. References to
“AzurRx SAS” refer to AzurRx SAS, AzurRx
BioPharma’s wholly-owned subsidiary through which we conduct
our European operations.
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PART
I
ITEM 1.BUSINESS
Overview
We are engaged in the research and development of targeted,
non-systemic therapies for the treatment of patients with
gastrointestinal (“GI”) diseases. Non-systemic therapies are
non-absorbable drugs that act locally, i.e. in the intestinal
lumen, skin or mucosa, without reaching an individual’s
systemic circulation.
We are currently focused on developing our pipeline of
gut-restricted GI clinical drug candidates. Our lead drug candidate
is MS1819, a recombinant lipase for the treatment of exocrine
pancreatic insufficiency (“EPI”)
in patients withcystic
fibrosis (“CF”) and chronic pancreatitis
(“CP”), currently in two
Phase 2 CF clinical trials. In 2021, we plan to launch two
clinical programs using proprietary formulations of niclosamide, a
pro-inflammatory pathway inhibitor; FW-1022, for Severe Acute
Respiratory Syndrome Coronavirus 2 (“SARS-CoV-2,” or“COVID-19”)
gastrointestinal infections, and FW-420, for Grade 1 Immune
Checkpoint Inhibitor-Associated Colitis (“ICI-AC”)
and diarrhea in oncology patients. Each drug candidate is described
below.
MS1819
MS1819, a recombinant lipase enzyme for the treatment of EPI
associated with CF and CP, is supplied as an oral non-systemic
biologic capsule. MS1819 is derived from
the Yarrowia
lipolytica yeast lipase
and breaks up fat molecules in the digestive tract of EPI patients
so that they can be absorbed as nutrients. Unlike the standard of
care, the MS1819 lipase does not contain any animal
products.
EPI is a condition characterized by deficiency of
exocrine pancreatic enzymes, primarily lipase, resulting
in a patient’s inability to digest food properly, or
maldigestion. The deficiency of these enzymes can be responsible
for greasy diarrhea, fecal urge, abdominal pain and weight loss. We
believe that there are two principal therapeutic indications for
EPI compensation by MS1819: (i) children and adults affected by CF,
and (ii) adult patients with CP. There are more than 30,000
patients with EPI caused by CF according to the Cystic Fibrosis
Foundation and there are approximately 90,000 patients in the U.S.
with EPI caused by CP according to the National Pancreas
Foundation. Patients are currently treated with porcine pancreatic
enzyme replacement (“PERT”) pills.
We have determined to initially pursue the indication for adults in
CF.
Completed Phase 2 OPTION Bridging Dose Monotherapy
Study
In
October 2018, the U.S. Food and Drug Administration
(“FDA”) cleared
our Investigational New Drug (“IND”)application for MS1819 in patients
with EPI due to CF. In connection with the FDA’s clearance of
the IND, we initiated a multi-center Phase 2 bridging dose safety study in the fourth
quarter of 2018 in the U.S. and Europe (the “OPTION Bridging Dose Study”). We
targeted enrollment of 30 to 35 patients and dosed the first
patients in February 2019. In June 2019, we reached its enrollment
target for the study and in September 2019, we announced positive
results from the OPTION Bridging Dose Study.
Results
showed that the primary efficacy endpoint of coefficient of fat absorption (“CFA”) was comparable to the CFA in a
prior Phase 2a study in patients with CP, while using the same
dosage of MS1819. The dosage used in the OPTION Bridging Dose Study
was 2.2 grams per day, which was determined in agreement with the
FDA as a bridging dose from the highest safe dose used in the Phase
2a CP dose escalation study. Although the OPTION Bridging Dose
Study was not powered for statistical significance, we believe the
data demonstrated meaningful efficacy results, with approximately
50% of the patients showing CFA
high enough to reach non-inferiority with standard PERTs.
Additionally, the coefficient of nitrogen absorption
(“CNA”) was
comparable between the MS1819 and PERT arms, 93% vs. 97%,
respectively, in the OPTION Bridging Dose Study. This important
finding confirms that protease supplementation is not likely to be
required with MS1819 treatment. A total of 32 patients, ages 18 or
older, completed the OPTION Bridging Dose Study.
Ongoing Phase 2b OPTION 2 Monotherapy Trial
In October 2019, the Cystic Fibrosis Foundation Data Safety
Monitoring Board (the “CFF DSMB”) completed its review of our final results
of the OPTION Bridging Dose Study. It found no safety concerns for MS1819, and
supported our plan to proceed to a higher 4.4 gram dose of MS1819
with enteric (delayed release) capsules in multi-center dose escalation Phase 2b
clinical trial (the “OPTION 2
Trial”). In December
2019, we submitted the clinical trial protocol for the OPTION 2
Trial to the existing IND at
the FDA. The clinical trial
protocol was reviewed by the FDA with no comments. In April 2020,
we received approval to conduct the OPTION 2 Trial in Therapeutics
Development Network clinical sites in the U.S. as well as
Institutional Review Board (“IRB”) approval to commence the OPTION 2
Trial.
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The OPTION 2 Trial was designed to investigate the safety,
tolerability and efficacy of MS1819 (2.2 gram and 4.4 gram doses in
enteric capsules) in a head-to-head manner versus the current
standard of care, PERT pills. The OPTION 2 Trial was an open-label,
crossover study, conducted in 15 sites in the U.S. and
Europe. Enrollment included a total of 30 CF patients 18
years or older. MS1819 was administered in enteric capsules to
provide gastric protection and test for optimal delivery of enzyme
to the duodenum. Patients were initially randomized into two
cohorts: to either the MS1819 arm, where they received a 2.2 gram
daily oral dose of MS1819 for three weeks; or to the PERT arm,
where they received their pre-study dose of PERT pills for three
weeks. After three weeks, stools were collected for analysis of
CFA. Patients were then crossed over for another three weeks
of the alternative treatment. After three weeks of cross-over
therapy, stools were again collected for analysis of CFA. A
parallel group of patients were randomized and studied in the same
fashion, using a 4.4 gram daily dose of MS1819. All patients
were followed for an additional two weeks after completing both
crossover treatments for post study safety
observation. Patients were assessed using descriptive methods
for efficacy, comparing CFA between MS1819 and PERT arms, and for
safety.
In
November 2020, we announced that we would submit protocol amendment
request to the FDA for the OPTION 2 Trial to add a study arm
utilizing immediate release MS1819 capsules. In January 2021, we
announced that we had initiated the additional study arm. This
extension phase tested patients 18 years or older, who had already
completed the cross-over phase, with immediate release capsules at
higher 4.4 and 6.6 gram doses relative to the 2.2 gram capsules
previously used in the OPTION Bridging Dose Study. The purpose of
the additional study arm was to allow us to compare data from the
existing crossover arm using enteric (delayed release) capsules
with data from the new extension arm, to help us identify the
optimal dose and delivery method for MS1819.
In
March 2021, we announced topline data results from the OPTION 2
Trial. The data demonstrated MS1819 to be safe and well-tolerated.
In addition, we believe the data from the OPTION 2 Trial also
demonstrates meaningful drug activity, as was also the case with
our OPTION Bridging Dose Study and a prior Phase 2a study in
patients with CP, and also with the interim data in our ongoing
Combination Trial (as defined and discussed in further detail
below). However, patients in the OPTION 2 Trial did not
consistently meet the primary efficacy endpoint. Some patients were
able to achieve CFA at levels beyond what is required to
demonstrate non-inferiority with PERT therapies, but the majority
did not. As such, we did not meet our primary endpoint for the
trial.
We
believe that the underlying cause of the MS1819’s uneven
efficacy performance in the OPTION 2 Trial lies with the enteric
capsule formulation. While we believe the enteric coating protects
the capsule from breaking down in the stomach acid, the trial data
suggests it may dissolve too slowly in the small intestine to
release the lipase enzyme in time to aid with proper digestion and
nutrient absorption.
As a
result, we have announced plans to develop a new formulation for
MS1819, employing a capsule filled with acid-resistant granules, or
microbeads, similar to what is used in CREON®, ZENPEP®
and other PERT therapies. These beads will be placed into immediate
release capsules that are intended to dissolve in the stomach,
dispersing the beads, which should then pass through to the small
intestine and break down, releasing the lipase enzyme so that it
thoroughly mixes with food as it is being digested.
We are
accelerating discussions with contract manufacturers to develop
this new formulation. We believe we have sufficient capital on hand
to fund this development and initiate a further Phase 2 study to
evaluate the new formulation’s efficacy, without
substantially delaying our development efforts in other
areas.
Ongoing Phase 2 Combination Therapy Trial
In
addition to the CF monotherapy studies, we launched a Phase 2
multi-center clinical trial (the “Combination Trial”) in Europe
(Hungary and Turkey) to investigate the safety, tolerability and
efficacy of escalating doses of MS1819, in combination with PERT,
in order to increase CFA levels and relieve abdominal symptoms in
CF patients who suffer from severe EPI but continue to experience
clinical symptoms of fat malabsorption despite taking the maximum
daily dose of PERTs.
Ideally,
a stable daily dose of PERT will enable CF patients to eat a normal
to high-fat diet and minimize unpleasant gastrointestinal symptoms.
In practice, however, approximately 25-30% of CF patients do not
achieve normal absorption of fat with PERTs. Achieving an optimal
nutritional status, including normal fat absorption levels, in CF
patients is important for maintaining better pulmonary function,
physical performance and prolonging survival. Furthermore, a
decline of body mass index around the age of 18 years predicts a
substantial drop in lung function. We believe a combination therapy
of PERT and MS1819 has the potential to: (i) correct macronutrient
and micronutrient maldigestion; (ii) eliminate abdominal symptoms
attributable to maldigestion; and (iii) sustain optimal nutritional
status on a normal diet in CF patients with severe
EPI.
The
Combination Trial enrolled 18 patients, 12 years of age or older,
with severe EPI, with CFA levels less than 80%. Patients enrolled
in the study receive escalating doses of 700mg, 1200mg, and 2240mg
of MS1819 once daily for 15 days per dosing level, in addition to
their standard PERT dose. Baseline CFA is established by measuring
CFA levels while on standard of care therapy only, before beginning
combination therapy. The primary efficacy endpoint of the trial is
improvement in CFA; secondary endpoints of the study are
improvements in the stool weight, stool consistency, number of
bowel movements, the incidence of steatorrhea, and increase of body
weight.
We
dosed the first patients in the Combination Trial in Hungary in
October 2019.
We announced positive interim data on the first five patients in
the Combination Trial in August 2020. The primary efficacy endpoint
was met, with CFAs greater than 80% for all patients across all
visits. For secondary efficacy endpoints, we observed that stool
weight decreased, the number of stools per day decreased,
steatorrhea improved, and body weight increased. Additionally, no
serious adverse events were reported. In October 2020, we
opened a total of five clinical sites in Turkey and dosed the first
patients in November 2020.
In
March 2021, we announced that we completed enrollment of 18
patients and expect to report top-line data in the second quarter
of 2021.
Niclosamide
Niclosamide,
a pro-inflammatory pathway
inhibitor, is a prescription small molecule drug that has
been safely used on millions of patients. Niclosamide is listed as
an essential medicine by the World Health Organization
(WHO). In the U.S., niclosamide was approved by the
FDA in 1982 for the treatment of intestinal tapeworm infections.
Niclosamide’s activity as an antihelminthic result from
direct action in the intestinal lumen where it disrupts parasite
oxidative metabolism, killing parasites. Niclosamide has been
commercially available worldwide for more than 50 years as 500mg
tablets intended for use in pediatric and adult populations, at a
dose rate of 2g per adult or child over six years of age. No safety
issues have ever been identified. In addition to its antihelminthic
activity, niclosamide has novel anti-inflammatory and
anti-viral properties.
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We
believe
niclosamide, and more specifically the patented and proprietary
micronized niclosamide formulation developed by First Wave, has the
potential to be an ideal therapeutic to treat multiple GI
indications due to the following favorable properties: (i) it has a
reduced particle size (D(90) between 5 and 9 μM) as compared
to regular non-micronized niclosamide (approximately D(90) ≥
60μM) with greater surface
to solvent ratio, (ii) low oral bio-availability with minimal
systemic absorption / exposure, (iii) improved dissolution with
broader distribution allowing for higher local GI concentrations
(up to approximately 200 times based on preclinical study results),
and (iv) it exhibits anti-inflammatory effects while avoiding
steroid-related complications and adverse
events.
FW-1022: COVID-19 GI infections
We are developing FW-1022, a small molecule medicinal product
containing micronized niclosamide in an oral immediate release
tablet formulation as treatment for SARS-CoV-2 intestinal infection
in patients presenting with gastrointestinal symptoms of COVID-19
disease. The formulation to be used has been milled (micronized) to
allow superior dissolution in the gut fluids. This in turn allows
local niclosamide concentrations to reach anti-viral levels. Thus,
FW-1022 has the potential to benefit COVID patients by decreasing
viral load in the GI tract, treating infection symptoms and
preventing transmission of the virus through fecal spread. Evidence
of niclosamide’s antiviral properties is sufficient to expect
a clinical pharmacodynamic response against viral replication and
clinical benefit, justifying the proposed clinical study in
COVID-19 patients and favorable benefit-risk
assessment.
An
IND for FW-1022 micronized niclosamide
for COVID-19 GI infections was cleared by the FDA in September
2020. We are currently preparing to initiate a two-part, two-arm, placebo-controlled Phase 2
clinical trial examining the safety and efficacy of niclosamide in
patients with COVID-19 GI infections.
FW-420: Immune Checkpoint Inhibitor Colitis (ICI-AC)
Immune checkpoint inhibitors (“ICIs”) are monoclonal antibodies that target
down-regulators of the anti-cancer immune response and have
revolutionized the treatment of a variety of malignancies. The
global market for ICIs in in 2019 was estimated to be over $22
billion and growing rapidly. Approximately 44% of patients
with advanced cancer tumors, or about 260,000 patients, are
eligible to receive ICIs
However, many immune-related adverse events, especially diarrhea
and colitis, limit the use of ICIs. While Grade 1 colitis (less
than four stools per day) is treated symptomatically as
outpatients, about 30% or more of patients with Grade 1 colitis
progress to Grade 2 or worse. Those patients must be taken off of
the ICI being used for their cancer, risking relapse. Also, they
receive aggressive immunosuppressive therapies, further aggravating
their underlying cancer.
The incidence of immune-mediated colitis
(“IMC”)
ranges from 1% to 25% depending on the type of ICI and whether they
are without interruption used in combination. Approximately 30% of
ICI patients develop diarrhea, which can progress to colitis. The
onset of diarrhea in ICI-AC patients occurs within six to seven
weeks and progressively worsens, and the progression to colitis is
rapid and unpredictable. For example, in patients taking ipilimumab
(Yervoy), between 25% and 30% of patients developed diarrhea and
approximately 8% to 12% developed colitis. Moreover, the trend is
towards the use of combination ICI therapies (e.g. Yervoy and
Opdivo) which may lead to a concomitant increase in both diarrhea
and colitis.
Administration of corticosteroids, or treatment with certain
immunosuppressive biologics, while withholding ICI therapy are
recommended for Grade 2 or more severe colitis. The impact of
this colitis complication and treatment may reduce the goal of
progression free cancer survival. An oral, non-absorbed treatment,
such as niclosamide, for Grade 1 colitis (diarrhea) may prevent
progression to Grade 2 disease. There currently is no approved
treatment for Grade 1 colitis.
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FW-420 is a niclosamide based small molecule anti-inflammatory
inhibitor therapy which we intend to use for the treatment of
immune checkpoint inhibitor-associated colitis and diarrhea in
metastatic cancer patients. FW-420 will be supplied in two
formulations, as an oral immediate-release tablet and as a topical
rectal enema foam. The standard care for treating inflammatory
bowel diseases (IBD) such as ulcerative colitis and Crohn’s
Disease is corticosteroids and 5-ASAs, which can cause problems
when used for check point inhibitor patients due to their
immunosuppressant effects. FW-420 has the potential to safely
treat Grade 1 ICI colitis and diarrhea and prevent its progression
to more serious and potentially fatal later stages. The overall
goal of early niclosamide treatment is to enable oncology patients
to remain without interruption on, or spend minimal time off of,
their ICI treatment programs.
The primary objective of our planned Phase 1b/2a trial of
niclosamide is to monitor patients receiving ICI treatment, and to
intervene with niclosamide treatment at the first signs of
diarrhea. The primary objective is to compare niclosamide oral,
versus niclosamide oral plus rectal, versus standard of care, and
to prevent Grade 1 colitis progressing to Grade 2 or worse. We
anticipate the trial will need about 100 patients to provide a
signal of efficacy.
We plan to initiate the Phase 1b/2a clinical trial in the first