Diffusion Pharmaceuticals Receives Accelerated FDA Response to Proposed TSC COVID-19 Clinical Trial Program
May 26 2020 - 8:00AM
Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN)
(“Diffusion” or “the Company”) announces it has received an
accelerated response from the U.S. Food and Drug Administration
(FDA) to the Company’s Pre-Investigational New Drug (PIND) meeting
request. The PIND was filed on April 27, seeking FDA guidance on
the Company’s proposed clinical development program for the use of
trans sodium crocetinate (TSC) in COVID-19 patients displaying
severe respiratory symptoms and low oxygen levels.
The FDA’s accelerated response to the PIND
meeting request recommended that the first U.S. TSC COVID-19 study
employ a double-blinded, controlled, randomized clinical trial
design to address the wide variability in standard of care due to
the rapidly evolving COVID-19 experience. The FDA also recommended
incorporating, where available, the drug remdesivir, which has been
newly approved for emergency use, into TSC clinical trials as a
component of standard of care for patients hospitalized with severe
disease. The FDA agreed with the safety and oxygenation marker
endpoints proposed by the Company for early trials of TSC in
COVID-19 patients and suggested a range of potential functional
outcomes which might be used as primary endpoints in possible
later-stage trials supporting product approval.
“We are gratified that in their accelerated
response to our PIND for studies of TSC in COVID-19 patients the
FDA has suggested this randomized, blinded clinical trial design
allowing oxygenation endpoints in our initial U.S. trial, rather
than the smaller unblinded safety trial we had expected,” said
David Kalergis, chief executive officer of Diffusion. “This more
robust first U.S. study is fully funded by existing Company
resources, and, with FDA guidance now in hand, we intend to move
forward quickly under current expedited FDA and hospital-specific
policies. Meanwhile, we expect a June 2020 first patient enrollment
for the European arm of our TSC COVID-19 program being conducted at
the Romanian National Institute of Infectious Diseases. We further
expect the European trial to provide first human data regarding
TSC’s oxygenation-enhancing potential in this disease by the end of
the summer.”
Patients with COVID-19 respiratory tract
infections often present with significantly impaired oxygen levels.
Diffusion and its affiliated researchers believe the
oxygen-enhancing mechanism of action of TSC could benefit such
patients. Preclinical data indicate TSC increases oxygen
availability and provides a functional benefit in animal models of
acute lung injury and hemorrhagic shock. Clinical data from over
150 patients receiving TSC for other indications demonstrate that
the drug has an acceptable human safety profile in both healthy and
critically ill patients.
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 U.S. Office
of Naval Research, which was seeking a way to treat multiple organ
failure and its resulting mortality caused by low oxygen levels
from blood loss on the battlefield. Evolutions in research have led
to Diffusion’s focus today on addressing some of medicine’s most
intractable and difficult-to-treat diseases, including multiple
organ failure from respiratory distress, stroke and glioblastoma
multiforme (GBM) brain cancer. In each of these diseases, lack of
available oxygen presents a significant obstacle for medical
providers and is the target for TSC’s novel mechanism.
In 2019, the Company reported favorable safety
data in a 19-patient dose-escalation run-in to its Phase 3 INTACT
program, using TSC to target inoperable GBM. That trial is
currently paused, while the Company prioritizes its resources to
address COVID-19. 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, while not officially
paused, is expected to be minimal until the COVID-19 pandemic
abates. The Company is currently partnering with both U.S. and
European-based institutions in an expedited research program to
develop TSC as a treatment for the low oxygen levels and associated
multiple organ failure in COVID-19 patients.
Preclinical data supports the potential for TSC
as a treatment for other conditions where low oxygen availability
plays an important role, such as myocardial infarction, peripheral
artery disease, and neurodegenerative conditions such as
Alzheimer’s and Parkinson’s disease. In addition to the development
of TSC, 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 FDA will approve the IND submission
for commencement of a trial in the U.S.; 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; there can be no assurance as to when the
program in the U.S. might be able to commence, if at all; the
uncertainty that as of yet the FDA has not 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; whether Diffusion has sufficient
funding to complete the trial described; the uncertainty as to
whether the protocol for the Romanian trial will be ultimately
acceptable to the Romanian healthcare regulatory authorities or
that such regulators 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 Romanian
regulators, there can be no assurance as to when they might provide
such guidance or when the program might be able to commence, if at
all; the uncertainty that as of yet the Romanian regulators have
not 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
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