AzurRx BioPharma, Inc. (NASDAQ: AZRX), (“AzurRx” or the “Company”),
a clinical stage biopharmaceutical company specializing in the
development of targeted, non-systemic therapies
for gastrointestinal (GI) diseases, today issued a letter to
its shareholders from James Sapirstein, President, Chief Executive
Officer and Chairman, highlighting the company’s corporate and
clinical achievements during the first quarter of 2021 and recent
weeks.
The full text of the letter, which has also been
posted to the Company's website, is as follows:
Dear AzurRx Shareholders,
It was just a few months ago that AzurRx began
2021 with news of a transformative in-licensing deal with First
Wave Bio that added a new asset, proprietary formulations of
micronized niclosamide, with two new therapeutic indications, for
COVID-19 GI infections and immune checkpoint inhibitor-associated
colitis (ICI-AC), to our clinical-stage pipeline.
I am pleased to report that we are on track to
launch both niclosamide programs in the clinic. In April 2021, we
announced the initiation of RESERVOIR, our Phase 2 (Proof of
Principle) clinical trial of niclosamide for the treatment of
COVID-19-related gastrointestinal infections, and that we engaged
PPD, Inc., a leading clinical research organization (CRO) to manage
the Phase 1b/2a ICI-AC trial that we plan to initiate in Q2
2021.
Additionally, we continued the development of
MS1819 as a potential treatment for exocrine pancreatic
insufficiency (EPI) in patients with cystic fibrosis (CF). We
reported topline data from our Phase 2b OPTION 2 monotherapy data
at the end of Q1 2021 and expect to report topline data from our
Phase 2 combination therapy trial using MS1819 in combination with
the current standard of care, pancreatic enzyme replacement therapy
(PERT), in Q2 2021. We continue to believe that MS1819 has the
potential to supplant PERT as the gold standard treatment for EPI
in patients with CF and chronic pancreatitis (CP).
These events are just the tip of the iceberg.
AzurRx remains focused on its mission to develop valuable therapies
with significant market opportunities into the GI space, and, as it
does so, generate value for shareholders.
AzurRx – Focused on Targeted,
Non-Systemic GI Therapeutics
Our most advanced clinical asset, MS1819, is a
recombinant lipase enzyme designed to treat EPI in patients with CF
and CP. EPI is a common complication of CF and CP caused by an
enzyme deficiency that leaves patients unable to properly digest
food and absorb nutrients. Our goal is to provide a safe and
effective therapy to control EPI and improve upon PERT, which is
primarily derived from pig pancreases and requires patients to take
upwards of 40 capsules per day to control symptoms. The target
market for EPI is substantial, at more than $1.4 billion in the
U.S. and over $2 billion globally in 2019. We continue to believe
that MS1819 has strong potential with the ability to attract
partnership opportunities and licensing interest from Big
Pharma.
With micronized niclosamide, we are exploring
two separate indications -- COVID-19-related GI infections and
immune checkpoint inhibitor-associated colitis in advanced stage
cancer patients. We believe both indications are synergistic and a
good fit for AzurRx, which also have high unmet need and sizable
market opportunities.
Niclosamide – Launching Two Clinical
Trials in 1H 2021
FW-1022: Phase 2 COVID-GI Infection
TrialIn early April, we initiated our Phase 2 RESERVOIR
trial, a two-part, two-arm, placebo-controlled study examining the
safety and efficacy of FW-1022, an oral micronized niclosamide
tablet, for COVID-19-related GI infections.
COVID-19 continues to impact hundreds of
thousands of people worldwide every day in addition to the millions
who have already been infected. For many, the after-effects of
COVID-19 can be as bad as the disease itself, and this includes a
growing number who experience “long haul” GI complications due to
what many believe is the ability of the SARS-CoV-2 virus to hide in
reservoirs within the GI tract. Gastrointestinal infection symptoms
(severe diarrhea, vomiting and abdominal pain) have been reported
in ~18% of COVID-19 cases, with viral RNA positive stool samples
being reported in ~48% of all COVID-19 patients in a recent
study.1 Furthermore, approximately 10%2 of patients who were
infected with COVID have persistent symptoms months after their
initial diagnosis. Approximately 86% of these COVID “long haulers”
are reported to have GI infection symptoms, with 60% having
diarrhea months after their initial infection.3
It is important to understand that despite all
the progress made with vaccines that they are not going to end the
COVID pandemic – the disease is becoming endemic. Vaccines have
given us hope, but the emergence of new and more transmissible
SARS-CoV-2 variants which keep cropping up all over the world,
suggest that COVID-19 may remain with us for several years to come.
Moreover, we will not be able to vaccinate everyone we need to.
There remains a strong need to find therapeutics that can help most
of the world’s population deal with the effects of COVID-19
infections and we believe that our niclosamide therapeutic has a
strong and lasting role to play in treating patients.
There is a growing body of evidence supporting
the potential of niclosamide as a COVID-19 therapy. Niclosamide was
identified by the Institut Pasteur Korea as a potent inhibitor of
SARS-CoV-2, the virus causing COVID-19, with potency 40X greater
than remdesivir.4 A recent study published in the journal
Nature found that niclosamide could prevent long-term lung damage
in COVID-19 patients.5 We believe that our micronized oral
niclosamide therapy has the potential to target SARS-CoV-2 directly
in the gut and to become an important addition to the numerous
therapeutics that may unfortunately be required by many who
contract COVID-19. There currently is no targeted treatment for
COVID GI infections and we are optimistic that we will be the first
therapy to market in this indication.
Patient enrollment is underway for the RESERVOIR
trial, with topline data anticipated in the first quarter of
2022.
FW-420: Phase 1b/2a Immune Checkpoint
Colitis TrialMeanwhile, we are preparing to launch a
second clinical trial soon. This is a Phase 1b/2a study evaluating
oral micronized niclosamide tablets, known as FW-420, for Grade 1
Immune Checkpoint Inhibitor-Associated Colitis (ICI-AC). Immune
checkpoint inhibitors have been a major advance in cancer
therapeutics, but the drugs can induce a serious inflammation of
the bowels, which if left unchecked, can be life-threatening and
force patients to halt treatment. There currently is no approved
treatment for Grade 1 colitis. Our goal is to develop FW-420 as a
therapeutic that halts ICI-AC from progressing and enables patients
to continue their immunotherapy treatment regimen
uninterrupted.
We recently announced that we engaged PPD, a leading CRO, to
manage the clinical trial and anticipate initiation of the study in
Q2 2021.
We believe that both niclosamide programs can be
completed relatively quickly and have the potential for accelerated
regulatory approval, either through the FDA’s 505(b)(2) approval
pathway (COVID-19-related GI infections) or a potential
breakthrough designation (ICI-AC). The result could greatly reduce
development costs and timelines for FW-1022 and FW-420.
MS1819 – A Potential Advance in the
Treatment of EPI
Phase 2b Monotherapy
TrialRecently, we announced topline results from our Phase
2b OPTION 2 clinical trial investigating MS1819 as a monotherapy in
CF patients with EPI. As discussed on our March 31 conference call,
results were mixed, although we did not consistently meet the
primary efficacy endpoint of a coefficient of fat absorption (CFA)
≥80%. MS1819 demonstrated itself to be safe and well-tolerated and
data from OPTION 2, and our other Phase 2 clinical trials, have
clearly demonstrated drug activity. 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, and
as such, we did not meet our trial goal.
We believe the underlying cause of the drug’s
uneven efficacy performance in OPTION 2 lies with the formulation.
In response, we are moving aggressively to develop a new
formulation for MS1819 that utilizes a capsule filled with
acid-resistant granules, or microbeads. Such a capsule is intended
to dissolve in the stomach, disperse the beads, and then pass
through to the small intestine where the beads would break down and
release the lipase enzyme so that it thoroughly mixes with food and
deliver the lipase enzyme in the duodenum.
We are now in a position to develop a microbead
formulation similar to that used by the leading PERT brands in the
market. Due to prior cost constraints, we were unable to develop a
microbead formulation and conducted the OPTION 2 clinical trial
using a powder formulation with different delivery mechanisms -
both immediate release and delayed-release enteric capsules. This
was a development pathway that certain PERT competitors initially
pursued as well, because they too were unable to achieve a CFA ≥80%
using powder immediate-release formulations. Once they reformulated
their products using enteric-coated microbeads they were able to
achieve CFA levels over 80%.
Furthermore, in addition to the acid-resistant
microbead formulation, we have also been investing in several
manufacturing processes designed to optimize production yields and
incorporate more drug product into capsules - with the goals of
reducing the cost of goods, improving competitive pricing, and
further decreasing the number of daily capsules needed by patients.
We continue to believe that we will be able to reduce pill burden
and help improve patient compliance through process
improvements.
Phase 2 Combination Therapy
ProgramIn addition to the MS1819 monotherapy program, we
continue to advance a Phase 2 clinical trial evaluating MS1819 in
combination with PERT. Patient enrollment in this trial is
complete, and we expect to report top line data in Q2 2021. As
previously reported in August 2020, early data was encouraging, and
we believe that the combination regimen has potential to help the
approximately 25% to 30% of refractory CF patients with severe EPI
who are unable to achieve adequate nutrition using PERT alone.
MS1819 -- A Clear Path
Forward
We have already initiated discussions with
contract manufacturers to develop the optimized microbead
formulation of MS1819. We expect to have a product ready to advance
into clinical studies by Q3 2021. This process will of course
require more time and resources. However, to our benefit, we have
sufficient capital on hand to fund this development.
We believe the cost-benefit profile with MS1819
is clearly in our favor. The drug has a proven mechanism of action
and compared to PERTs, it offers numerous therapeutic, safety, and
compliance advantages, while remaining relatively easy to
manufacture. Based on these factors, we believe that if this
optimized microbead formulation proves successful in the clinic and
receives regulatory approval, MS1819 could eventually eclipse pig
PERT as the standard of care, as similarly seen in the early 1980’s
with the advent of synthetic “human” insulin which completely
replaced the cow and pig derived insulin that had been used for
treating diabetes.
Finance
During the first quarter of 2021, we raised
aggregate gross proceeds of approximately $18.0 million from the
sale of preferred stock and common stock in public offerings and
private placement transactions, plus the receipt of aggregate cash
proceeds of approximately $4.6 million from the exercise of
warrants.
Outlook Ahead
These are exciting times for AzurRx and we
anticipate several catalysts on the horizon. I look forward to
working with my management team and fellow board members to execute
a business and clinical strategy that has the potential to
transform AzurRx and generate substantial returns for our
shareholders.
We will continue to pursue our efforts to drive
long-term shareholder value by delivering safe and effective GI
therapies for patients who need them the most, raising our global
visibility and broadening our shareholder base. We look forward to
continuing what has been a productive 2021, and we thank you for
your continued support.
Sincerely,James SapirsteinChairman, President
and CEOAzurRx BioPharma, Inc.
About AzurRx BioPharma,
Inc.AzurRx BioPharma, Inc. (NASDAQ: AZRX) is a clinical
stage biopharmaceutical company specializing in the development of
targeted, non-systemic therapies for gastrointestinal (GI)
diseases. The Company has a pipeline of three gut-restricted GI
assets. The lead therapeutic candidate is MS1819, a recombinant
lipase for the treatment of exocrine pancreatic insufficiency (EPI)
in patients with cystic fibrosis and chronic pancreatitis. AzurRx
is also advancing two clinical programs using proprietary
formulations of niclosamide, a pro-inflammatory pathway inhibitor;
FW-1022, for COVID-19 GI infections and FW-420, for Grade 1 Immune
Checkpoint Inhibitor-Associated Colitis and diarrhea in oncology
patients. The Company is headquartered in Delray Beach, Florida
with clinical operations in Hayward, California. For more
information visit www.azurrx.com.
Forward-Looking StatementThis
press release may contain certain statements relating to future
results which are forward-looking statements. It is possible that
the Company’s actual results and financial condition may differ,
possibly materially, from the anticipated results and financial
condition indicated in these forward-looking statements, depending
on factors including whether results obtained in preclinical and
nonclinical studies and clinical trials will be indicative of
results obtained in future clinical trials; whether preliminary or
interim results from a clinical trial will be indicative of the
final results of the trial; the size of the potential markets for
the Company’s drug candidates and its ability to service those
markets; and the Company’s current and future capital requirements
and its ability to raise additional funds to satisfy its capital
needs. Additional information concerning the Company and its
business, including a discussion of factors that could materially
affect the Company’s financial results are contained in the
Company’s Annual Report on Form 10-K for the year ended December
31, 2020 under the heading “Risk Factors,” as well as the
Company’s subsequent filings with the Securities and Exchange
Commission. All forward-looking statements included in this press
release are made only as of the date of this press release, and we
do not undertake any obligation to publicly update or correct any
forward-looking statements to reflect events or circumstances that
subsequently occur or of which we hereafter become aware.
For more information:
AzurRx BioPharma, Inc.1615 South Congress AvenueSuite 103Delray
Beach, Florida 33445Phone: (646) 699-7855info@azurrx.com
Media contact:
Tiberend Strategic Advisors, Inc.Johanna Bennett/Ingrid
Mezo(212) 375-2665/(646)
604-5150jbennett@tiberend.com/imezo@tiberend.com
References1 Gut Journal: Vol 69, Issue 6:
2020; Gut Journal: Vol 69, Issue 6: 2020; JAMA Network:
Vol 3, Issue 6: 2020; Lancet Gastroenterol Hepatol: Vol 5,
Issue 5: 2020; Cheung Gastroenterology: Vol. 159, Issue 1: 2020.2
Rubin, R. “As their numbers grow, COVID-19 “Long Haulers” Stump
Experts”. https://jamanetwork.com/journals/jama/fullarticle/2771111
September 23, 2020.3 Davis, et al. “Characterizing Long Covid in an
International Cohort: 7 Months of Symptoms and their Impact”.
https://www.medrxiv.org/content/10.1101/2020.12.24.20248802v2.full.pdf4
Jeon S, Ko M, Lee J, Choi I, Byun SY, Park S, Shum D, Kim S. 2020.
Identification of antiviral drug candidates against SARS-CoV-2 from
FDA-approved drugs. Antimicrob Agents Chemother
64:e00819-20. https://doi.org/10.1128/AAC.00819-20.5 Braga,
L., Ali, H., Secco, I. et al. Drugs that inhibit TMEM16 proteins
block SARS-CoV-2 Spike-induced syncytia. Nature (2021).
https://doi.org/10.1038/s41586-021-03491-6
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