PRINCETON, N.J., Aug. 28, 2019 /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 received a positive
recommendation from the independent Data Monitoring Committee (DMC)
to continue enrolling into the company's Phase 3 "DOM–INNATE" study
(Dusquetide treatment in Oral Mucositis – by modulating INNATE
immunity) for SGX942 (dusquetide) in the treatment of oral
mucositis in patients with head and neck cancer (HNC).
Following its prospectively defined interim analysis,
including unblinded assessment of the study's primary efficacy
endpoint, the DMC recommended that approximately 70 additional
subjects be randomized into the trial, increasing the study sample
size from 190 to 260 evaluable subjects. The DMC's
recommendation indicates that a beneficial SGX942 effect has been
observed; however, to maintain the rigorous assumption of 90%
statistical power for the primary efficacy endpoint, an increase
was required to take into account any potential variability and/or
distribution changes observed in the Phase 3 study patient
population that may have differed from the initial protocol design
assumptions. No safety concerns were reported by the DMC based
on the interim analysis. The study remains on target to
complete enrollment and provide topline results in the first half
of 2020.
"We are pleased to have received the DMC's recommendation to
continue enrolling to the adjusted target of 260 subjects in order
to maintain our conservative power calculation," stated
Christopher J. Schaber, PhD,
President and Chief Executive Officer of Soligenix. "Since reaching
the 90 subject enrollment threshold required for the conduct of the
interim analysis in April, we currently have over 160 subjects
enrolled in the study. With this new level of clarity from the
DMC's analysis of the interim Phase 3 study data and given our
current rate of patient enrollment, we are confident that we will
remain on target to announce topline results in the first half of
2020. We have invested a significant amount of the Company's
resources over the last several years into the oral mucositis
development program and it is gratifying to have received this
feedback from the DMC. Given our current cash resources, we
anticipate that the available funds are sufficient to cover the
additional study subjects needed. We believe SGX942 has the
potential to be a valuable therapy in the treatment of oral
mucositis, which is an area of high unmet medical need."
"The DMC's recommendation from the interim analysis is very
encouraging and provides for a more precise understanding of the
patient population and treatment effect, as it is based on the
actual data from the ongoing Phase 3 clinical trial," stated
Richard Straube, MD, Senior Vice
President and Chief Medical Officer of Soligenix. "Our
understanding of the historic variability in oral mucositis
patients, especially placebo patients, and the variability that can
occur when going from small Phase 2 to larger Phase 3 clinical
trials, is precisely the reason we included the interim
analysis. It is to ensure that we do not get misdirected by
our initial set of assumptions and stop the trial with a
substantial but non-statistically significant benefit in the SGX942
group."
Dr. Straube continued, "Although we remain blinded to the
potential reasons that informed the DMC's recommendation to
increase the sample size, we do know that the DMC's recommendation
reflects that they saw a prospectively defined promising signal in
the primary endpoint, which will allow us to aggressively pursue
completing the trial in order to demonstrate SGX942's potential to
successfully ameliorate the devastating impact of oral mucositis in
patients with HNC receiving chemoradiation therapy
(CRT). Further, the added subjects will also allow us to more
rigorously assess any ancillary benefits of SGX942 (reduced
infection, increased survival and increased tumor clearance rate)
as well as build a more robust safety database that is important to
support potential marketing authorizations with the US and EU
health authorities. We would like to thank the DMC members for
their assistance, as well as our esteemed medical advisory board
and our dedicated clinical investigators for their ongoing efforts
in the design and conduct of this important clinical
trial."
About the Phase 3 DOM–INNATE Study
Based on the positive and previously published Phase 2 results
(Study IDR-OM-01), the pivotal Phase 3 clinical trial (Study
IDR-OM-02) is a highly powered, double-blind, randomized,
placebo-controlled, multinational trial originally targeted to
enroll approximately 190 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 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 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 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) 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 U.S.
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 U.S. 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 or the Phase 3 clinical trial of SGX301
(synthetic hypericin) for the treatment of cutaneous T-cell
lymphoma. There also can be no assurance as to timing or
success of the preclinical/clinical trials
of RiVax®,
that RiVax® will be approved for
the PRV program or the amount for which a PRV
for RiVax® can be sold. 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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