PRINCETON, N.J., Dec. 22, 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 preliminary top-line results for its
pivotal Phase 3 DOM-INNATE (Dusquetide treatment in Oral Mucositis
– by modulating INNATE Immunity) trial evaluating SGX942
(dusquetide) in the treatment of severe oral mucositis (SOM) in
patients with head and neck cancer (HNC) receiving
chemoradiation. The study enrolled 268 patients randomized
1:1 to receive either SGX942 or placebo. The primary endpoint
of median duration of SOM did not achieve the pre-specified
criterion for statistical significance (p≤0.05); although
biological activity was observed with a 56% reduction in the median
duration of SOM from 18 days in the placebo group to 8 days in the
SGX942 treatment group. Despite this clinically meaningful
improvement, the variability in the distribution of the data
yielded a p-value that was not statistically significant.
Other secondary endpoints supported the biological activity of
dusquetide, including a statistically significant 50% reduction in
the duration of SOM in the per-protocol population, which decreased
from 18 days in the placebo group to 9 days in the SGX942 treatment
group (p=0.049), consistent with the findings in the Phase 2
trial. Similarly, incidence of SOM also followed this
biological trend as seen in the Phase 2 study, decreasing by 16% in
the SGX942 treatment group relative to the placebo group in the
per-protocol population. The per-protocol population was
defined as the population receiving a minimum of 55 Gy radiation
and at least 10 doses of study drug (placebo or SGX942) throughout
the intended treatment period, with no major protocol deviations
(e.g. breaks in study drug administration longer than 8 days
between successive doses).
"We are obviously very disappointed with the unanticipated
outcome of the study," stated Christopher
J. Schaber, PhD, President and Chief Executive Officer of
Soligenix. "Despite the fact that SGX942 demonstrated
clinically meaningful reductions in oral mucositis consistent with
the Phase 2 study, the Phase 3 trial did not achieve the
statistically significant benefit we expected. Over the coming
weeks, we will be analyzing the data to better determine why the
study did not meet expectations. If there is any clarity
gained from further analysis of the dataset, especially with
respect to specific subsets of patients that may benefit from
SGX942 therapy, we will certainly communicate our findings and
explore follow-up discussions with the FDA and the
EMA."
Dr. Schaber continued, "With approximately $20 million of cash and our non-dilutive
government funding, we will evaluate strategic options as we
continue to execute on the multiple development programs across our
rare disease pipeline. Most importantly, this will include
the preparation of a New Drug Application for SGX301 in the
treatment of cutaneous T-cell lymphoma, which demonstrated
statistical significance in its pivotal Phase 3 clinical trial
earlier this year, as well as continuing activities towards SGX301
U.S. commercialization where we expect peak annual sales to exceed
$75 million."
The Company will host a webcast and conference call today at
8:30 AM EST to review the top-line
findings.
Conference Call, December 22,
2020 at 8:30 AM Eastern Time
The Company will share information on its Phase 3 top-line
results for its SGX942 program in oral mucositis. A question
and answer (Q&A) session with management will follow the
presentations. If you would like to ask a question during the
Q&A, please submit your request via
email to ir@soligenix.com at least 15 minutes prior
to the scheduled start of the call.
U.S. toll free: 1-866-652-5200
International: 1-412-317-6060
Please request to be entered into the Soligenix call.
A transcript of the presentation will be archived for 30 days
following the event.
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 U.S. 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 U.S., 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
enrolled 268 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 were
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 SOM, 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. SOM is defined as a WHO Grade of
≥3. A positive interim analysis was conducted in August 2019, resulting in the recommended
addition of 35 subjects / group to the study to maintain 90%
power. Subjects are being 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.
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 SOM 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 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 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,
SGX943, our therapeutic candidate for antibiotic resistant and
emerging infectious disease, and our research programs to identify
and develop novel vaccine candidates targeting viral infection
including Ebola, Marburg and SARS-CoV-2 (the cause of COVID-19).
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, such as
experienced with the COVID-19 outbreak. 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. Despite the statistically significant result
achieved in the SGX301 Phase 3 clinical trial for the treatment of
cutaneous T-cell lymphoma, there can be no assurance that a
marketing authorization from the FDA or EMA will be
successful. 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®. Also, no assurance can be
provided that the Company will receive or continue to receive
non-dilutive government funding from grants and contracts that have
been or may be awarded or for which the Company will apply in the
future. 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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