Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN)
(“Diffusion” or “the Company”), a cutting-edge biotechnology
company developing new treatments for life-threatening medical
conditions by improving the body’s ability to deliver oxygen to the
areas where it is needed most, today announced the pre-IND
submission to the U.S. Food and Drug Administration (FDA) of a
planned clinical program using trans sodium crocetinate (TSC) in
COVID-19 patients displaying severe respiratory symptoms and low
oxygen levels. Under federal regulations, the FDA has up to 60 days
to hold an advisory meeting with the Company, but for
COVID-19-related submissions, the FDA has announced its
intention to significantly shorten this period under
its Coronavirus Treatment Acceleration Program. Clinical
trial start-up preparations are continuing as the Company awaits
the FDA’s response.
To aid in timely trial enrollment, Diffusion is
conducting expedited discussions with institutions
located in areas of severe COVID-19 incidence, both in the
U.S. and in Eastern Europe, to determine their possible
participation.
Acute Respiratory Distress Syndrome (ARDS)
develops in nearly all patients hospitalized with COVID-19,
triggered by lack of sufficient oxygen to vital organs as a
consequence of damage to the lungs. In severe cases, patients need
to receive life support through mechanical ventilation. In general,
ARDS carries a high mortality rate (up to 40% in those on a
breathing machine, and likely higher in COVID-19-related ARDS).
Diffusion believes that through successful implementation of its
TSC/COVID-19 clinical program, TSC’s oxygen-enhancing mechanism of
action could provide an important new treatment option for this
life-threatening unmet medical need. Further, the Company believes
that TSC’s novel mechanism of action would be compatible with many
of the COVID-19 treatment modalities currently used or under
development.
Although the number, design and projected
enrollment of the clinical trials are subject to change,
Diffusion’s pre-IND submission for TSC envisions three studies to
be conducted in rapid succession. The first is an open-label study
that will examine the basic safety of the TSC dosing paradigm in up
to 12 COVID-19 patients who have been admitted to Intensive Care
Units (ICUs). In addition to the primary endpoint of safety, blood
gas and pulse oximetry data also will be collected to observe TSC’s
possible effect on increasing oxygenation in these oxygen-starved
patients.
The second trial is planned as a larger,
double-blinded, randomized, placebo-controlled study to confirm
safety in the ICU patient population and determine the statistical
significance of any effect on the patient’s oxygenation status from
TSC, again using blood gas analysis and pulse oximetry. The number
of patients enrolled will be determined by statistical
considerations. Data also will be collected for other endpoints
including mortality.
Assuming results from the second study warrant
program continuation, the third study, also double-blinded,
randomized and placebo controlled, would enroll a statistically
determined number of hospitalized COVID-19 patients who have not
yet been admitted to an ICU, and therefore may not have arterial
blood lines in place. While pulse oximetry readouts and other
parameters will be monitored for possible improvements in patient
status, the primary goal of this third study, intended to be a
registration trial, will be to show that TSC lowers the probability
of COVID-19 patient admittance to the ICU. Diffusion believes that
achieving this goal would provide an important new tool for
healthcare professionals as they fight to prevent the cascade of
negative effects from oxygen deficiency in hospitalized COVID-19
patients, which too often lead to disease progression and
mortality. Meeting this goal could also provide significant cost
savings to the health care system, lessening the use of critical
resources associated with ICU admittance and fostering TSC’s more
widespread use.
Diffusion’s COVID-19 program is a cooperative
research effort with the University of Virginia Health System (UVA)
and the Integrated Translational Research Institute of Virginia
(iTHRIV.) iTHRIV is a National Institutes of Health (NIH)-funded
Clinical and Translational Awards (CTSA) program. Dr. Andrew
Southerland, Associate Professor of Neurology and Public Health
Sciences at UVA, serves as lead Principal Investigator, working
with co-investigator Dr. Alex Kadl, Assistant Professor of Medicine
and Pharmacology in the UVA Division of Pulmonary & Critical
Care Medicine.
“Despite 50 years of research,” said Dr. Kadl,
“there are no pharmaceutical therapies for ARDS. We further know
that maintaining adequate blood oxygenation with needed mechanical
ventilation can cause further harm and damage to the lungs.
Improving oxygenation with TSC may prevent severe hypoxia and the
need for mechanical ventilation, giving the lungs the time needed
to recover from COVID-19 infection.”
“We believe TSC’s novel oxygen-enhancing
mechanism could have a direct effect on the systemic hypoxemia in
COVID-19 patients, altering the downward spiral of disease
progression and decreasing the necessity for ARDs-related
admissions to the ICU,” said David Kalergis, chief executive
officer of Diffusion. “We are committed to working with hospitals
and regulatory authorities both in the U.S. and Eastern Europe to
forge the fastest possible pathway to TSC’s approval, consistent
with good clinical practices.”
About Diffusion Pharmaceuticals
Inc.
Diffusion Pharmaceuticals Inc. is an innovative
biotechnology company developing new treatments that improve the
body’s ability to deliver oxygen to the areas where it is needed
most, offering new hope for the treatment of life-threatening
medical conditions. Diffusion’s lead drug trans sodium crocetinate
(TSC) was originally developed in conjunction with the Office of
Naval Research, which was seeking a way to treat multiple organ
failure and its resulting mortality caused by the systemic
hypoxemia from blood loss on the battlefield. Evolutions in
research have led to Diffusion’s focus today: Fueling Life by
taking on some of medicine’s most intractable and
difficult-to-treat diseases, including multiple organ failure,
stroke and glioblastoma multiforme (GBM) brain cancer. In each of
these diseases, hypoxia – oxygen deprivation of essential tissue in
the body – has proved to be a significant obstacle for medical
providers and is the target for TSC’s novel mechanism.
In July 2019 the Company reported favorable
safety data in a 19-patient dose-escalation run-in study to its
Phase 3 INTACT program, using TSC to target inoperable GBM. Further
findings from the dose-escalation run-in study, released in
December 2019, also showed possible signals of enhanced survival
and patient performance. Diffusion’s in-ambulance PHAST-TSC trial
for acute stroke began enrolling patients last year. Given the
heightened responsibilities of the Company’s emergency medical
services providers, enrollment in this trial is expected to be
minimal until the COVID-19 pandemic abates. The Company is also
currently partnering with the University of Virginia and iTHRIV in
a research program to develop its novel small molecule TSC as a
treatment for Acute Respiratory Distress Syndrome (ARDS) from
COVID-19, specifically targeting the associated multiple organ
failure.
Preclinical data supports the potential for TSC
as a treatment for other conditions where hypoxia plays a major
role, such as myocardial infarction, peripheral artery disease, and
neurodegenerative conditions such as Alzheimer’s and Parkinson’s
disease. In addition, RES-529, the Company’s PI3K/AKT/mTOR pathway
inhibitor that dissociates the mTORC1 and mTORC2 complexes, is in
preclinical testing for GBM.
Diffusion is headquartered in Charlottesville,
Virginia – a hub of advancement in the life science and
biopharmaceutical industries – and is led by CEO David Kalergis, a
30-year industry veteran and company co-founder.
Forward-Looking Statements
To the extent any statements made in this news
release deal with information that is not historical, these are
forward-looking statements under the Private Securities Litigation
Reform Act of 1995. Such statements include, but are not limited
to, statements about the company's plans, objectives, expectations
and intentions with respect to future operations and products, the
potential of the company's technology and product candidates, and
other statements that are not historical in nature, particularly
those that utilize terminology such as "would," "will," "plans,"
"possibility," "potential," "future," "expects," "anticipates,"
"believes," "intends," "continue," "expects," other words of
similar meaning, derivations of such words and the use of future
dates. Forward-looking statements by their nature address matters
that are, to different degrees, uncertain. Uncertainties and risks
may cause the Diffusion’s actual results to be materially different
than those expressed in or implied by such forward-looking
statements. Particular uncertainties and risks include: the
uncertainty as to whether the protocol described above, which is a
pre-IND submission, will be ultimately acceptable to the FDA for an
IND submission or that the FDA will not require significant changes
that might take significant time to implement, if at all, or that
any such required changes will be financially feasible; moreover,
if this or a revised protocol is acceptable to the FDA for an IND
submission, there can be no assurance as to when the FDA might
provide such guidance or when the program might be able to
commence, if at all; the uncertainty that as of yet the FDA has no
approved a trial evaluating TSC for the treatment of ARDS, or if
approved, such a trial possibly entailing significant additional
time, effort and expense, particularly in light of the difficulty
of doing business during the COVID-19 pandemic; Diffusion’s ability
to maintain its Nasdaq listing, market conditions, the difficulty
of developing pharmaceutical products, obtaining regulatory and
other approvals and achieving market acceptance; general business
and economic conditions; the sufficiency of the company’s cash, the
company's need for and ability to obtain additional financing or
partnering arrangements; and the various risk factors (many of
which are beyond Diffusion’s control) as described under the
heading “Risk Factors” in Diffusion’s filings with the United
States Securities and Exchange Commission. All forward-looking
statements in this news release speak only as of the date of this
news release and are based on management's current beliefs and
expectations. Diffusion undertakes no obligation to update or
revise any forward-looking statement, whether as a result of new
information, future events or otherwise.
Contacts:David Kalergis, CEODiffusion
Pharmaceuticals Inc.(434)
825-1834dkalergis@diffusionpharma.comorLHA Investor RelationsKim
Sutton Golodetz(212) 838-3777kgolodetz@lhai.com
Diffusion Pharmaceuticals (NASDAQ:DFFN)
Historical Stock Chart
From Mar 2024 to Apr 2024
Diffusion Pharmaceuticals (NASDAQ:DFFN)
Historical Stock Chart
From Apr 2023 to Apr 2024