Source: Hemispherx Biopharama, Inc. 10/22/18
Hemispherx Biopharma, Inc. (NYSE American: HEB)
announces that Thomas K. Equels, President and Chief Executive Officer,
has issued a Letter to Stockholders providing a business update. The
full text of the Letter, which has also been posted to the Company's
website, is as follows.
To Our Stockholders,
The
primary focus of today’s Letter is to update you on the great strides
we have made in our oncology programs with Ampligen. In recent months
Hemispherx has made significant advancements with our Ampligen oncology
program and Ampligen’s potential use as an immuno-oncology agent for the
treatment of multiple types of cancer. This has been a priority for our
Company, and me personally, since I became CEO in 2016. We have
remained focused on fulfilling our promises to advance these important
clinical goals. I am pleased to announce that we are supporting six
separate clinical studies in eight solid tumors at major research
institutions.
In parallel, we continue to pursue Ampligen for
the treatment of severe myalgic encephalomyelitis/chronic fatigue
syndrome (ME/CFS). Ampligen is approved for this indication in Argentina
(the first and only meaningful therapy approved anywhere in the world
for ME/CFS) and is available through an U.S. Food and Drug
Administration (FDA) authorized expanded access program (EAP), AMP-511, a
program where the FDA has approved the reimbursement rate of $200 per
vial for the direct costs and where we are expanding participation at
clinics in Nevada and North Carolina.
Ampligen and Oncology
In
collaboration with major cancer research centers in the U.S. and around
the world, we have been making tremendous progress and strongly believe
in the future of our cancer-fighting efforts. We believe the true value
of Ampligen is not yet well-understood by the pharma industry, many
clinicians or investors. Therefore, I believe it is important that you,
our stockholders, understand not only what we are doing in this field,
but also
why we are doing it. It is also important that
you have the opportunity to see what these top experts in
immuno-oncology have to say about their recent successes with Ampligen
and its importance as a promising agent for advances in oncology.
Progress in oncology provides a clear opportunity for commercial success
for Hemispherx.
We are working closely with and have
initiated the steps to proceed with important clinical studies at the
University of Pittsburgh Medical Center (UPMC), the University of
Nebraska Medical Center (UNMC) and Roswell Park Comprehensive Cancer
Center (Roswell Park). We expect that the Institutions will conduct and
complete these clinical trials on an expedited basis. The goal of these
various studies will be to conclusively test the hypothesis that the
combination of Ampligen with checkpoint blockade therapies will improve
clinical tumor responses, time to progression and survival in humans.
Checkpoint inhibitors have provided dramatic responses for a number of
advanced end stage cancers by enabling the patient’s immune system to
attack the cancer. Although a minority of patients respond dramatically
to the checkpoint inhibitors, we believe that use of combinational
immune therapy to enhance immune killing of tumor cells provides a
rational mechanism for expanding the clinical response rate for
checkpoint inhibitor therapy.
In early October, Hemispherx
signed a Clinical Trial Agreement with Roswell Park in Buffalo, N.Y. to
conduct clinical studies of Ampligen in combination with checkpoint
inhibitors in three solid tumors, namely urothelial carcinoma (bladder
and associated structures), renal cell carcinoma and melanoma.
Additional clinical studies of Ampligen plus checkpoint blockade
therapies in five other solid tumors — triple-negative breast,
colorectal, ovarian, non-small cell lung and pancreatic carcinomas — are
at various stages of development, from early protocol drafts to having
secured FDA authorization to proceed with human subjects. These five
other clinical trials will be conducted at Roswell Park, UPMC or UNMC
beginning in the near future.
In summary, Hemispherx is
currently collaborating on the start of clinical trials with three
U.S.-based world-class cancer research centers to study Ampligen in
combination with checkpoint inhibitors that are already approved by the
FDA. Checkpoint inhibitors are powerful immuno-therapy drugs that block
proteins that restrain the body’s immune system from fighting cancer,
and they are used in a number of advanced solid tumor malignancies. The
object of these combination clinical trials is to measure improvements
in therapeutic outcomes. The need stems from the fact that checkpoint
blockade therapies work poorly with so-called “cold” tumors. Clinical
proof-of-concept findings using an Ampligen cocktail in colorectal
carcinoma demonstrated a more favorable ratio of killer T-cells to
regulatory T-cells in the tumor microenvironment. Killer T-cells attack
cancer cells, and their presence in the tumor microenvironment can be
inhibited by regulatory (suppressor) T-cells in the tumor
microenvironment. The suppressor cells thereby reduce a patient’s immune
response to cancer. Increases in killer T-cells, without a
corresponding increase in suppressor T-cells, therefore, is an indicator
of the body’s increased ability to mount a potentially effective immune
response, supporting strong pre-clinical evidence of Ampligen’s
activity in converting “cold” tumors into “hot” tumors.
In a
head-to-head study in explant culture models, Ampligen activated the
TLR3 pathway and promoted an accumulation of killer T-cells, but unlike
two other TLR3 agonists, it did so without causing regulatory T-cell
(Treg) attraction. These findings, published in the journal Cancer
Research (
http://cancerres.aacrjournals.org/content/early/2018/05/31/0008-5472.CAN-17-3985),
are important because they show that Ampligen selectively reprograms
the tumor microenvironment by inducing the beneficial aspects of tumor
inflammation (attracting killer T-cells), without amplifying
immune-suppressive elements such as regulatory, ‘suppressor’, T-cells.
The anti-tumor activity of Ampligen has been recognized by oncology
investigators in a multiplicity of peer reviewed scientific
publications. The ability to reprogram the tumor microenvironment
favoring enhanced anti-cancer activity of the checkpoint inhibitors has
provided the rationale for initiating clinical proof of concept studies
in a variety of advanced (lethal) cancers.
"Ampligen has the
potential to be clinically significant because a robust killer T-cell
population in the tumor microenvironment without attracting Treg cells
is important to help optimize checkpoint blockade induced tumor
shrinkage," said Robert P. Edwards, MD, chair of gynecologic services at
Magee-Women’s Hospital of the University of Pittsburgh School of
Medicine (UPMC), Professor of Obstetrics, Gynecology & Reproductive
Services at UPMC, a world-class expert in ovarian cancer and a pioneer
in Ampligen research.
His views are vigorously reiterated by
another world renowned expert and Ampligen pioneer, Pawel Kalinski, MD,
PhD, Rustum Family Professor for Molecular Therapeutics and
Translational Research, Vice Chair for Translational Research and
Director of Cancer Vaccine and Dendritic Cell Therapies at Roswell Park
Comprehensive Cancer Center, who states, “There is significant and
pressing need for new therapy options that can extend the benefits of
checkpoint inhibition to the patients who currently do not respond to
these treatments. We’re excited about initiating our planned basket
trial incorporating Ampligen to see whether these combination approaches
can successfully convert ‘cold’ tumors into ‘hot’ ones that will
respond to these therapies. Our preclinical findings indicate that this
is a worthwhile strategy to pursue.”
One of the solid tumors
that we are targeting at UNMC in our combination therapy clinical
studies with Ampligen is pancreatic cancer, which is a prime example of
the unmet medical needs we are striving to meet. We also have an ongoing
Dutch-approved standalone Ampligen EAP in pancreatic cancer at Erasmus
M.C. that is more than a year old. Statistically, only 6-7% of patients
diagnosed with pancreatic cancer are alive five years after diagnosis,
and more than 100,000 people will die this year in the U.S. and Europe
from this dreaded malignancy. Today, a diagnosis of pancreatic cancer is
a virtual death sentence. Hemispherx, working with Erasmus and UNMC,
intends to change that. We have every hope that the upcoming clinical
trial at UNMC will pave the way for progress in pancreatic cancer. We at
Hemispherx strongly believe that this work is important, will make a
difference and ultimately will save lives. As one of the world’s top
pancreatic cancer researchers, Michael A. (Tony) Hollingsworth, PhD,
head of pancreatic cancer research at UNMC’s Buffett Cancer Center
states, “We are working hard to discover better treatments for
pancreatic cancer. We believe Ampligen is an agent that holds tremendous
promise — not only for pancreatic cancer, but also for a variety of
other cancers.”
I hope that if, as stockholders, you
understand this program, you may better appreciate why we are moving so
aggressively on this clinical path. I believe success in oncology will
lead to lasting commercial success for our company. Over the past two
years we have aggressively and successfully pursued meaningful
relationships with top cancer research centers in the U.S. We then, at
these same institutions, aggressively and successfully pursued
pre-clinical animal studies to show our hypothesis has great merit.
Initial proof of concept clinical work was similarly successful. Now we
are commencing at these same top institutions, led by top experts in
their fields, clinical studies in a variety of solid tumors which we
have every expectation will establish Ampligen as an important agent in
immuno-oncology.
Fighting Cancer and Beyond
We
have two pipeline drugs that show tremendous progress in fighting
debilitating conditions for countless people with unmet medical needs,
Ampligen, and our FDA approved product, Alferon® N Injection. We have
our own manufacturing site and a proven ability to produce our pipeline
products. We have an active EAP in the Netherlands in pancreatic cancer,
and are in midst of expanding our EAP for ME/CFS in the U.S. We have
the first ever commercial approval in severe ME/CFS from Argentina.
What’s more, over the past two years, we have successfully collaborated
with Erasmus M.C., the Hillman Cancer Center at UPMC, the Fred &
Pamela Buffett Cancer Center at UNMC and Roswell Park. These are all
highly respected research centers in the field of cancer treatment. We
strongly believe that successes in our six independent immuno-oncology
clinical studies targeting eight types of solid tumors will create
significant long-term value for our stockholders.
In addition to the above detailed clinical work:
We can manufacture our product
- Two
commercial sized lots totaling over 16,000 vials have been filled,
finished and released for use. We announced in August that the new
Ampligen batch manufactured earlier this year was released for patient
use in the pancreatic cancer EAP in the Netherlands. This was the
initial shipment (500 vials) of a previously announced 2,100 vial
standing stock order issued by myTomorrows, our EAP provider in Europe.
We are making a difference in the U.S. and Globally
- We are working to expand the U.S.-based EAP, known as AMP-511, an FDA-authorized open-label program for severe ME/CFS.
- We
announced in September that our regulatory inspection and clearance for
distribution of our second commercial scale lot of Ampligen, slated for
multiple uses including product launch in Argentina for the treatment
of ME/CFS, our ME/CFS EAP in the U.S. and Europe and the pancreatic
cancer EAP in the Netherlands.
Hemispherx is a small
company, but holds great promise. Our pledge to stockholders is that we
will continue to pursue all available opportunities to advance our
pipeline, with the goals of offering therapeutics that will truly make a
difference in the lives of patients and, at the same time, build
stockholder value.
We will continue to keep you apprised of
our progress in these efforts as we go forward. Thank you for your
continued support of Hemispherx Biopharma.
Sincerely,
Thomas K. Equels
President and Chief Executive Officer
October 22, 2018