Diffusion Pharmaceuticals Inc. (Nasdaq: DFFN), a
cutting-edge biotechnology company developing new treatments for
life-threatening medical conditions by improving the body’s ability
to bring oxygen to the areas where it’s needed most, announced
today that it has received FDA approval to enroll patients in an
ambulance-based Phase 2 clinical trial testing its lead drug, trans
sodium crocetinate (TSC), for the treatment of acute stroke.
The trial, named PHAST-TSC (Pre-Hospital
Ambulance Stroke Trial-TSC), will involve 23 hospitals across
urban, suburban, and rural areas in Los Angeles and Central
Virginia, working closely with approximately 150 emergency medical
transport groups. Results for the PHAST-TSC trial could be
available in just under two years – a critically fast turnaround
for this potential improvement in the front-line medical treatment
of stroke, which afflicts nearly 800,000 Americans each year at an
estimated cost of over $34 billion to the U.S. economy.
Nearly two million brain cells die each minute
during a stroke. As a result, medical providers seek to provide
treatment for patients as quickly as possible during the hours
immediately after stroke onset. TSC, which will be administered by
paramedics while the stroke victim is still in the ambulance, may
offer new hope for these patients by increasing the amount of
oxygen directed to affected tissue, potentially reducing cell death
and improving patient outcomes. In addition to stroke, TSC has
shown safety and efficacy to date in oncology, with a Phase 3 trial
in glioblastoma (GBM) brain cancer currently enrolling.
The Study Chair for the PHAST-TSC trial is
Jeffrey Saver, M.D., professor of clinical neurology and director
of the stroke unit at the David Geffen School of Medicine at UCLA.
Co-Principal Investigators for the study are Andrew Southerland,
M.D. (UVA) and Nerses Sanossian, M.D. (University of Southern
California).
“Dr. Saver and his team at UCLA have been
pioneers in on-ambulance therapy, and, along with Dr. Johnston and
her team at the University of Virginia, we have the expertise
necessary to test TSC’s effectiveness in patients suffering from
acute stroke,” said John L. Gainer, PhD, Diffusion’s Chief
Science Officer and inventor of the TSC molecule. “While
combining the talents of a pharmaceutical company with those of two
of our country’s leading academic medical centers is, perhaps, a
paradigm shift for creating new clinical trials, it is one which
will hopefully produce substantial benefits for stroke victims and
their families.”
“TSC was originally designed for the US military
to help treat life-threatening injuries on the battlefield – a
place where every minute can be the difference between life and
death. This FDA approval to begin patient enrollment in
PHAST-TSC gives us the opportunity to show that treatment with TSC
could make an impact on a different battlefield, one where stroke
patients are also in a race against the clock,”
said David Kalergis, Chairman and CEO of
Diffusion. “As we return to our company’s roots in
emergency medicine and work to identify possible strategic
partnerships, we will also continue applying TSC to other areas of
medicine, including GBM brain cancer in our ongoing Phase 3
trial.”
“In addition to testing TSC in acute stroke, the
novelty of this study is that patients will receive treatment in
the ambulance prior to hospital arrival,” said Karen C.
Johnston, MD, MSc, Harrison Distinguished Professor of Neurology
and Associate Vice President for Clinical & Translational
Research at UVA. “Dr. Saver and Dr. Southerland are
both leaders in the development of innovative approaches to
treating acute stroke patients in the ambulance.”
Historically, advancements in safe and effective
pre-hospital treatment for patients immediately following a stroke
have eluded researchers. One reason is that ischemic stroke
(resulting from a clot in the brain) and hemorrhagic stroke (caused
by a burst blood vessel in the brain) produce almost identical
symptoms. Treatment is complicated by the fact that differentiating
between these two stroke types requires special imaging equipment
usually found only in-hospital.
There are no existing ischemic stroke
therapeutics which can be administered without first diagnosing the
type of stroke. For hemorrhagic stroke there are currently no
approved therapeutics of any kind, either on-ambulance or
in-hospital.
Despite numerous stroke clinical trials to date
involving “neuroprotectants” – drugs intended to minimize the death
of brain cells – none have shown sufficient efficacy to be
approved. TSC is also a neuroprotectant, but unlike many of the
other drugs which have been studied previously for stroke, TSC is
designed to stabilize brain oxygen levels, starting from the time
the patient first reaches the ambulance.
The one currently FDA-approved stroke drug,
Genentech’s Tissue Plasminogen Activator (tPA) dissolves blood
clots, but can only be used in ischemic stroke and may prove
harmful if given to hemorrhagic stroke patients. Preclinical
studies suggest that patient outcomes may improve synergistically
from the use of TSC and tPA, potentially without increasing risk to
patients. Since TSC can be administered upon ambulance arrival for
any acute stroke, whether ischemic or hemorrhagic, it could become
the immediate first treatment for any acute stroke, with
hospital-administered treatments to follow.
Additional information about the
PHAST-TSC trial
The Phase 2, randomized, double-blind, placebo
controlled PHAST-TSC trial will enroll 160 patients, with 128
coming from up to 20 hospitals in the greater Los Angeles area and
32 patients from 3 central Virginia hospitals. Half the
patients will be randomized to receive TSC and half to
placebo. The primary end point will be mortality or patient
neurological disability as measured by the modified Rankin scale
administered 90 days after stroke occurrence. Because the follow-up
period in the trial is only 90 days post-treatment, complete data
collection is expected within 21 months of first patient
enrollment, offering a timely and cost-effective assessment of a
potential breakthrough therapeutic in one of healthcare’s most
significant unmet medical needs.
The PHAST-TSC Diffusion-sponsored clinical trial
is a cooperative effort by Diffusion’s Chief Science Officer and
inventor of the TSC molecule, Dr. John L. Gainer, PhD and
Diffusion’s Scientific Advisory Board Chair, Dr. Guy Chisolm, PhD,
in close cooperation with the Director of UCLA’s Stroke Center,
Jeffrey Saver, M.D and the Chair of UVA’s Department of Neurology
Karen Johnston, M.D. Co-Principal Investigators for the study are
Andrew Southerland, M.D. (UVA) and Nerses Sanossian, M.D.
(University of Southern California). Other important participants
include the approximately 150 emergency medical transport groups
based in Los Angeles and Central Virginia. Diffusion will be
seeking additional financing or a partnering arrangement to help
fund the trial.
More information about Diffusion’s clinical programs, including
both its INTACT Phase 3 trial in inoperable GBM brain cancer and
its PHAST-TSC acute stroke program, are available on Diffusion’s
Website at www.diffusionpharma.com/clinicalprograms.
About Diffusion Pharmaceuticals
Inc.
Diffusion Pharmaceuticals Inc. is an innovative
biotechnology company developing new treatments that improve the
body’s ability to bring oxygen to the areas where it’s needed most
—offering new hope for the treatment of life-threatening medical
conditions.
Diffusion’s lead drug, Trans Sodium Crocinate
(TSC) was originally developed in conjunction with the Office of
Naval Research, which was seeking a way to treat hemorrhagic shock
caused by massive 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 stroke, GBM
brain cancer, pancreatic cancer, and brain metastases. In each of
these diseases, hypoxia – when essential tissue in your body is
deprived of oxygen – has proved to be a significant obstacle for
medical providers and the target for TSC’s novel mechanism.
In 2018, the Company began enrolling patients in
its Phase 3 INTACT program, using TSC to target inoperable GBM
brain cancer. It’s in-ambulance Phase 2 acute stroe protocol was
granted FDA clearance to proceed in September, 2018.
Additional pre-clinical data supports the potential use of TSC as a
treatment for other conditions where hypoxia plays a major role,
such as myocardial infarction, respiratory diseases such as COPD,
peripheral artery disease, and neurodegenerative conditions such as
Alzheimer’s and Parkinson’s.
In addition, RES-529, the Company’s
PI3K/AKT/mTOR pathway inhibitor that dissociates the mTORC1 and
mTORC2 complexes, is in the preclinical testing phase 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, the anticipated timing of future clinical trials, 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 company's actual results to be materially different
than those expressed in or implied by such forward-looking
statements. Particular uncertainties and risks include: general
business and economic conditions; the company's need for and
ability to obtain additional financing or partnering arrangement;
and the difficulty of developing pharmaceutical products, obtaining
regulatory and other approvals and achieving market acceptance, 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.
Contact:Rob
Corradirob@penpublicaffairs.com410-212-5483
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