Diffusion Pharmaceuticals Receives FDA Guidance for International Phase 1b/2b COVID-19 Clinical Program with TSC
July 27 2020 - 8:00AM
Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN)
(“Diffusion” or “Company”), a biotechnology company developing
novel therapeutics for the treatment of unmet medical needs, today
announced receipt of guidance from the U.S. Food and Drug
Administration (FDA) on the Company’s recently-filed
Investigational New Drug (IND) application for trans sodium
crocetinate (TSC) in COVID-19 patients. This guidance suggests
certain study design changes with regard to endpoints and
statistical considerations that Diffusion believes will enhance the
prospect of regulatory approvals upon program completion, should
the clinical results be favorable.
Because these changes will be harmonized
throughout the overall TSC global development program, they are
being submitted for clearance by the Romanian National Agency for
Medicines and Medical Devices (NAMMD). NAMMD has regulatory
oversight over the Company’s first planned clinical trial, an
open-label Phase 1b lead-in trial in 24 hospitalized COVID-19
patients at the Romanian National Institute of Infectious Diseases
(NIID).
The Company previously announced expectations
that the first patient would be enrolled in the Phase 1b study by
the end of July. Considering the FDA’s suggested protocol
modifications and an additional NAMMD review cycle, the Company now
expects the first patient will be enrolled by the end of August,
with first data available early in the fourth quarter of 2020.
“Getting the clinical trial protocol right is
imperative as it will support regulatory reviews and
determinations,” said David Kalergis, chief executive officer of
Diffusion. “We are grateful for the FDA’s guidance.
Incorporating their views into the Romanian arm of the program will
delay the program start by just a few weeks, yet should have no
material impact on overall program timelines.”
About TSC and COVID-19
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 more
than 150 patients receiving TSC for other indications demonstrate
that the drug has an acceptable 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. 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.
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 responsibilities of
the Company’s participating emergency medical services providers,
enrollment in this trial, while not officially paused, is expected
to be minimal until the COVID-19 pandemic abates.
Preclinical data support 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; the uncertainty as to whether the
protocol for the Romanian trial will be ultimately acceptable to
the Romanian healthcare regulatory authorities and local ethics
committees 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; whether Diffusion can enroll and complete the trials and
provide data on the timelines indicated; whether Diffusion can
efficiently transition from the Phase 1b to Phase 2b portion of the
Romanian trials and to the 2b U.S. trial; whether the data from the
Romanian trials can be combined with data generated in any U.S.
trials; whether Diffusion has sufficient funding to complete the
trials described; 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 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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