PRINCETON, N.J., June 24, 2020 /PRNewswire/ -- Soligenix, Inc.
(Nasdaq: SNGX) (Soligenix or the Company), a late-stage
biopharmaceutical company focused on developing and commercializing
products to treat rare diseases where there is an unmet medical
need, announced today it has completed patient enrollment in its
Phase 3 DOM-INNATE ("Dusquetide treatment in Oral Mucositis – by
modulating INNATE Immunity") study for SGX942 (dusquetide) in
the treatment of oral mucositis (OM) in head and neck cancer (HNC)
patients. The study successfully enrolled 268 subjects,
following positive interim analysis, which included a prospectively
defined, unblinded assessment of the study's primary efficacy
endpoint by an independent Data Monitoring Committee (DMC).
With enrollment completed, top-line results are expected in the
fourth quarter of 2020.
SGX942 is a novel, first-in-class, Innate Defense Regulator
(IDR) which both modulates inflammation and enhances anti-infective
and tissue-healing pathways of the innate immune system.
Study enrollment was temporarily extended as Soligenix assessed the
potential impact of COVID-19 on the study (e.g., patient treatment
compliance and completion of necessary assessments). With
extra efforts by participating patients, physicians and clinical
staff, the Company can now successfully report that the negative
impact of the pandemic on the overall study was much less than
initially anticipated. The study remains on-track to provide
top-line results before the end of 2020.
"We are pleased to have completed enrollment and look forward to
the top-line results in the fourth quarter, particularly in light
of the DMC recommendation at the interim analysis which observed a
beneficial drug effect," stated Christopher
J. Schaber, PhD, President and Chief Executive Officer of
Soligenix. "We continue to positively position this
fast-tracked program for approval. With approximately
$8 million in cash as of the end of
the first quarter, not including our non-dilutive government
funding, along with the at-the-market sales issuance agreement with
B. Riley FBR, Inc. to judiciously supplement our cash runway as
needed, we anticipate having sufficient capital to achieve multiple
inflection points across our rare disease pipeline, including
top-line results in the DOM-INNATE study. As there is no FDA
approved drug for the treatment of oral mucositis in head and neck
cancer or other solid tumor settings, we believe SGX942 has the
potential to be the first approved therapy to address this unmet
medical need and dramatically improve the lives of patients
undergoing chemoradiation therapy (CRT)."
"SGX942 has the potential to have a significant impact on the
lives of patients undergoing CRT for squamous cell carcinoma of the
oral cavity and oropharynx," stated Richard
Straube, MD, Senior Vice President and Chief Medical Officer
of Soligenix. "We would like to thank the DMC members, our
esteemed medical advisory board and our dedicated clinical
investigators for their efforts in the design and conduct of this
important clinical trial, as well as all the subjects that are
participating in the trial. Our focus now is to complete the
treatments for all subjects in both the US and Europe and to lock the study database,
facilitating top-line results in the fourth quarter of
2020."
Based on the positive results demonstrated in the Phase 2 study
of SGX942, the Phase 3 trial is a highly powered, double-blind,
randomized, placebo-controlled, multicenter and multinational
trial. The primary endpoint for the study is the median
duration of severe oral mucositis, assessed by oral examination at
each treatment visit and then through six weeks following
completion of CRT. Oral mucositis is evaluated using the WHO
(World Health Organization) Grading system. Other secondary
measures, including incidence of severe oral mucositis, incidence
and duration of ulcerative oral mucositis, and incidence of
infection will also be assessed at topline or during the 12-month
follow-up.
A prospectively defined interim analysis was conducted in
August 2019 by an independent DMC and
was used to verify the underlying assumptions defining the required
sample size of the study to maintain its rigorous 90% statistical
power. The DMC identified a beneficial SGX942 effect and
accordingly adjusted the study sample size to approximately
260. The DMC did not identify any safety concerns. The
interim recommendation is described in the August 2019 press release here.
About Oral Mucositis
Mucositis is the clinical term for damage done to the mucosa by
anticancer therapies. It can occur in any mucosal region, but
is most commonly associated with the mouth, followed by the small
intestine. It is estimated, based upon review of historic
published studies and reports and an interpolation of data on the
incidence of mucositis, that mucositis affects approximately
500,000 people in the US per year and occurs in 40% of patients
receiving chemotherapy. Mucositis can be severely debilitating
and can lead to infection, sepsis, the need for parenteral
nutrition and narcotic analgesia. The gastrointestinal damage
causes severe diarrhea. These symptoms can limit the doses and
duration of cancer treatment, leading to sub-optimal treatment
outcomes.
The mechanisms of mucositis have been extensively studied and
have been recently linked to the interaction of chemotherapy and/or
radiation therapy with the innate defense system. Bacterial
infection of the ulcerative lesions is now regarded as a secondary
consequence of dysregulated local inflammation triggered by
therapy-induced cell death, rather than as the primary cause of the
lesions.
It is estimated, based upon review of historic published studies
and reports and an interpolation of data on the incidence of oral
mucositis, that oral mucositis in HNC is a subpopulation of
approximately 90,000 patients in the US, with a comparable number
in Europe. Oral mucositis almost always occurs in patients
with HNC treated with CRT and is severe, causing inability to eat
and/or drink, in >80% of patients. It is common (40-100%
incidence) in patients undergoing high dose chemotherapy and
hematopoietic cell transplantation, where the incidence and
severity of oral mucositis depends greatly on the nature of the
conditioning regimen used for myeloablation.
In the pediatric population, head and neck cancer is a rarer
occurrence and is caused by different underlying pathologies. The
major types of HNC in children are lymphoma, sarcomas (including
rhabdomyosarcomas), and neuroblastoma rather than squamous cell
carcinoma, the major type of adult HNC cancers. Hematopoietic
stem cell transplantation (HSCT), especially
allogeneic transplantation with higher risk of oral mucositis, is
more frequently used in the pediatric population than in adults
when treating a number of primary tumor types, as seen
in leukemia and lymphoma. Both treatment of HNC and
HSCT are associated with high risk of oral mucositis in the
pediatric population.
Oral mucositis remains an area of unmet medical need where there
are currently no approved drug therapies in the context of any
solid tissue tumors.
About the Phase 3 DOM-INNATE Study
This multinational, placebo-controlled, randomized study is
targeted to enroll approximately 260 subjects with squamous cell
carcinoma of the oral cavity and oropharynx, scheduled to receive a
minimum total cumulative radiation dose of 55 Gy fractionated as
2.0-2.2 Gy per day with concomitant cisplatin chemotherapy given as
a dose of 80-100 mg/m2 every third week. Subjects are
randomized to receive either 1.5 mg/kg SGX942 or placebo given
twice a week during and for two weeks following completion of CRT.
The primary endpoint for the study is the median duration of severe
oral mucositis, assessed by oral examination at each treatment
visit and then through six weeks following completion of CRT. Oral
mucositis is evaluated using the WHO (World Health Organization)
Grading system. Severe oral mucositis is defined as a WHO Grade of
≥3. Subjects are to be followed for an additional 12 months after
the completion of treatment. Soligenix has been working with
leading oncology centers internationally, a number of which
participated in the Phase 2 study.
About Dusquetide
Dusquetide (the active ingredient in SGX942) is an innate
defense regulator (IDR), a new class of short, synthetic
peptides. It has a novel mechanism of action whereby it
modulates the body's reaction to both injury and infection towards
an anti-inflammatory, anti-infective and tissue healing response.
IDRs have no direct antibiotic activity but, by modulating the
host's innate immune system responses, increase survival after
infections caused by a broad range of bacterial Gram-negative and
Gram-positive pathogens. It also accelerates resolution of
tissue damage following exposure to a variety of agents including
bacterial pathogens, trauma and chemo- and/or radiation
therapy. Preclinical efficacy and safety has been demonstrated
in numerous animal disease models including mucositis, colitis,
macrophage activation syndrome (MAS) as well as bacterial
infections, including melioidosis.
SGX942 has demonstrated safety in a Phase 1 clinical study in 84
healthy human volunteers. Positive efficacy results were
demonstrated in an exploratory Phase 2 clinical study in 111
patients with oral mucositis due to CRT for HNC. Soligenix is
working with leading oncology centers in the US
and Europe to advance SGX942 in oral mucositis with the
conduct of a pivotal Phase 3 clinical trial referred to as the
"DOM–INNATE" study (Dusquetide treatment in Oral Mucositis – by
modulating INNATE immunity).
SGX942 has received Fast Track Designation from the FDA for the
treatment of oral mucositis as a result of radiation and/or
chemotherapy treatment in HNC patients, as well as Promising
Innovative Medicine designation in the United Kingdom by
the Medicines and Healthcare Products Regulatory Agency for the
treatment of severe oral mucositis in HNC patients receiving
CRT. In addition, products containing the same active
ingredient, dusquetide, have been granted Fast Track Designation as
an adjunctive therapy with other antibacterial drugs, for the
treatment of melioidosis and Orphan Drug Designations in the
treatment of MAS and the treatment of acute radiation
syndrome.
Soligenix has a strong intellectual property position in the IDR
technology platform, including composition of matter for dusquetide
and related analogs. Dusquetide was developed pursuant to
discoveries made by Professors B. Brett Finlay, PhD and Robert Hancock, PhD
of the University of British
Columbia, Canada. Soligenix has received partial funding
from NIH for its oral mucositis clinical studies. The Phase 2
study was supported with a Phase I SBIR grant (#R43DE024032) award,
with the Phase 3 study being supported by a Phase II SBIR grant
(#R44DE024032) award.
In addition, a high level review of the IDR technology platform
is available here.
About Soligenix, Inc.
Soligenix is a late-stage biopharmaceutical company focused on
developing and commercializing products to treat rare diseases
where there is an unmet medical need. Our Specialized
BioTherapeutics business segment is developing SGX301 as a novel
photodynamic therapy utilizing safe visible light for the treatment
of cutaneous T-cell lymphoma, our first-in-class innate defense
regulator (IDR) technology, dusquetide (SGX942) for the treatment
of oral mucositis in head and neck cancer, and proprietary
formulations of oral beclomethasone 17,21-dipropionate (BDP) for
the prevention/treatment of gastrointestinal (GI) disorders
characterized by severe inflammation including pediatric Crohn's
disease (SGX203) and acute radiation enteritis (SGX201).
Our Public Health Solutions business segment includes active
development programs for RiVax®, our ricin toxin vaccine
candidate, OrbeShield®, our GI acute radiation syndrome
therapeutic candidate and SGX943, our therapeutic candidate for
antibiotic resistant and emerging infectious disease. The
development of our vaccine programs incorporates the use of our
proprietary heat stabilization platform technology, known as
ThermoVax®. To date, this business segment has
been supported with government grant and contract funding from the
National Institute of Allergy and Infectious Diseases (NIAID), the
Defense Threat Reduction Agents (DTRA) and the Biomedical Advanced
Research and Development Authority (BARDA).
For further information regarding Soligenix, Inc., please visit
the Company's website at www.soligenix.com.
This press release may contain forward-looking statements that
reflect Soligenix, Inc.'s current expectations about its future
results, performance, prospects and opportunities, including but
not limited to, potential market sizes, patient populations and
clinical trial enrollment. Statements that are not historical
facts, such as "anticipates," "estimates," "believes," "hopes,"
"intends," "plans," "expects," "goal," "may," "suggest," "will,"
"potential," or similar expressions, are forward-looking
statements. These statements are subject to a number of
risks, uncertainties and other factors that could cause actual
events or results in future periods to differ materially from what
is expressed in, or implied by, these statements. Soligenix
cannot assure you that it will be able to successfully develop,
achieve regulatory approval for or commercialize products based on
its technologies, particularly in light of the significant
uncertainty inherent in developing therapeutics and vaccines
against bioterror threats, conducting preclinical and clinical
trials of therapeutics and vaccines, obtaining regulatory approvals
and manufacturing therapeutics and vaccines, that product
development and commercialization efforts will not be reduced or
discontinued due to difficulties or delays in clinical trials or
due to lack of progress or positive results from research and
development efforts, that it will be able to successfully obtain
any further funding to support product development and
commercialization efforts, including grants and awards, maintain
its existing grants which are subject to performance requirements,
enter into any biodefense procurement contracts with the US
Government or other countries, that it will be able to compete with
larger and better financed competitors in the biotechnology
industry, that changes in health care practice, third party
reimbursement limitations and Federal and/or state health care
reform initiatives will not negatively affect its business, or that
the US Congress may not pass any legislation that would provide
additional funding for the Project BioShield program. In addition,
there can be no assurance as to timing or success of the Phase 3
clinical trial of SGX942 (dusquetide) as a treatment for oral
mucositis in patients with head and neck cancer receiving
chemoradiation therapy (including the outcome of the interim
analysis) or the Phase 3 clinical trial of SGX301 (synthetic
hypericin) for the treatment of cutaneous T-cell lymphoma.
Further, there can be no assurance that RiVax® will
qualify for a biodefense Priority Review Voucher (PRV) or that the
prior sales of PRVs will be indicative of any potential sales price
for a PRV for RiVax®. These and other risk factors are
described from time to time in filings with the Securities and
Exchange Commission, including, but not limited to, Soligenix's
reports on Forms 10-Q and 10-K. Unless required by law,
Soligenix assumes no obligation to update or revise any
forward-looking statements as a result of new information or future
events.
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SOURCE Soligenix, Inc.