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 is needed most, announces the
presentation of data signaling increased survival in inoperable
glioblastoma multiforme (GBM) patients enrolled in the 19-patient
lead-in portion of its open-label, dose-escalation Phase 3 study
with Trans Sodium Crocetinate (TSC) plus standard of care (SOC).
Chief Scientific Officer John Gainer, Ph.D. presented these
findings today at the inaugural Glioblastoma Drug Development
Summit in Boston, sponsored by Hanson Wade.
Dr. Gainer’s presentation included earlier research
demonstrating that TSC safely reduces tumor hypoxia by a novel
mechanism of action, making the tumor up to 3 times more
susceptible to radiation and chemotherapy. In Phase 2 testing of
TSC in newly diagnosed inoperable GBM patients, a nearly 4-fold
increase in 2-year survival was seen compared with historical
controls.
The current Phase 3 INTACT (INvestigating Tsc Against Cancerous
Tumors) study – an open-label, randomized, controlled trial – was
designed to examine this finding in a fully powered safety and
efficacy registration study. The study was further designed
to test a new TSC dosing regimen under which, in addition to the 18
injections of TSC administered during the radiation therapy phase,
patients receive 18 additional injections of TSC during the
post-radiation chemotherapy phase, at doses up to 6-times higher
than those received during radiation. The addition of this
high-dose regimen was based on research performed by Dr. Gainer
over the last three years in his laboratory at Diffusion’s
headquarters.
In the randomized portion of the INTACT trial, subjects will be
randomized at baseline to SOC for first-line treatment of GBM plus
TSC, or to SOC alone. The SOC for GBM is temozolomide plus
radiation therapy for 6 weeks, followed by 28 days of rest, then by
6 cycles of post-radiation temozolomide treatment.
In the 19-patient open-label, dose-escalation, lead-in portion
of the INTACT trial, safety was demonstrated in all patients
receiving TSC at all dosing levels. Further, the Company
believes that, while the number of high dose patients (7) is small,
the addition of the 18 higher-dose treatments of TSC may be having
a positive effect on survival. In his talk, Dr. Gainer
presented a Kaplan-Meier survival plot highlighting the difference
in survival seen with the addition of high-dose TSC treatment
compared with both SOC and the earlier Phase 2 study. The current
data show an 86% “Fraction Surviving,” with 38% for the completed
Phase 2 TSC study and 13% for SOC.
The ability of patients to perform daily activities as measured
by Karnofsky performance scores also increased from baseline
following completion of high-dose treatment with TSC, with scores
showing a differential of 15% improvement over the Phase 2 results
at 18 weeks, which is the time at which a decline rather than an
improvement normally occurs.
Dr. Gainer’s slide presentation is available at
http://investors.diffusionpharma.com/Presentations and includes
graphics of these data.
“Even when taking into account the small number of patients
enrolled, the difference in results with the new TSC dosing regimen
compared with both the former TSC regimen and the standard of care
is notable,” said David Kalergis, Diffusion Pharmaceuticals CEO.
“We are gratified by the continued clean safety profile of
TSC. We also believe that observed survival differences of
this magnitude send a meaningful signal about TSC, further
de-risking the INTACT Phase 3 study during the randomized
phase.”
Mr. Kalergis continued, “Patients diagnosed with inoperable GBM
have a dire prognosis and very limited treatment options.
Given TSC’s proven safety profile and observed efficacy signals, we
need to make all efforts to provide a new treatment option to these
patients.”
The Company is seeking a partner to continue development of TSC
in the inoperable GBM indication, and has begun patient enrollment
in its Phase 2 on-ambulance trial with TSC for the treatment of
stroke.
About Hanson Wade
Hanson Wade's goal is to accelerate progress within
organizations and across industries. Its primary method for
achieving this is by creating exclusive business conferences that
gather together the world's smartest thinkers and doers. The
inaugural Glioblastoma Drug Development Summit is designed with two
critical and ambitious objectives: to help overcome the major
biological challenges limiting effective glioblastoma treatment;
and to evaluate novel therapies and innovative trial designs to
prevent more tragic Phase 2 failures.
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 is needed most, offering new
hope for the treatment of life-threatening medical conditions.
Diffusion’s lead drug 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 and
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 signals of enhanced survival and patient
performance. Diffusion’s on-ambulance PHAST-TSC trial for
acute stroke has begun patient enrollment. In addition, preclinical
data supports the potential for 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 disease.
Further, 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, 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 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
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)
220-0718dkalergis@diffusionpharma.com
LHA Investor RelationsKim Sutton Golodetz(212)
838-3777kgolodetz@lhai.com
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