- Published findings demonstrate that HyBryte™
treatment statistically significantly reduced CTCL lesion size
- HyBryte™ has potential to address a critical gap in
treatment of early-stage CTCL
PRINCETON, N.J., July 20,
2022 /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
that the results of its successful Phase 3 FLASH (Fluorescent Light
Activated Synthetic Hypericin) study evaluating HyBryte™
(synthetic hypericin) for the treatment of cutaneous T-cell
lymphoma (CTCL) has been published in the Journal of the
American Medical Association (JAMA) Dermatology.
"The peer reviewed publication of these data in JAMA
Dermatology is a testament and further validation to the
importance of the findings for the scientific and CTCL disease
communities," stated Ellen Kim, MD,
Professor of Dermatology and Medical Director, Dermatology Clinic,
Perelman Center for Advanced Medicine at the Hospital of the
University of Pennsylvania, and the
Lead Principal Investigator for the Phase 3 FLASH study. "With its
chronic course and major impact on patient quality of life, CTCL is
an orphan disease in urgent need of additional treatment options
that are well-tolerated and safe over the long haul. The results
from this Phase 3 study, which is the largest double-blind,
randomized, placebo-controlled trial in CTCL to date, represents an
important leap forward in the development of potential therapies to
meet this unmet medical need."
The published findings demonstrate that HyBryte™
treatment statistically significantly reduced lesion size, with the
treatment response further improving over successive 6-week
treatment cycles. The primary endpoint evaluated the CAILS
(Composite Assessment of Index Lesion Severity) score of three
treated index lesions and success was defined as ≥50% reduction in
CAILS score relative to baseline. Lesion response continuously
improved with treatment duration. After the first 6-week treatment
window, 16% of patients had a response (p=0.04 versus patients with
6 weeks of placebo treatment; primary endpoint). This response rate
continued to significantly increase to 49% through 18 weeks of
treatment (p<0.0001 versus patients with 6-week hypericin or
placebo treatment). Throughout the study, HyBryte™ was
safe and well-tolerated. Importantly, HyBryte™ was
observed to perform similarly against both patch and thicker plaque
lesions characteristic of CTCL.
"In treating CTCL, which is a chronic cancer with no cure,
long-term safety is of paramount concern. Most current treatment
options for CTCL are associated with significant safety concerns,
including black-box warnings. HyBryte™ treatment has
demonstrated strong and rapid efficacy with a very benign safety
profile," stated Dr. Richard
Straube, MD, Senior Vice President and Chief Medical Officer
of Soligenix. "This is of significant benefit to patients living
with this difficult disease. The substantial increase in efficacy
with longer treatment and the similar performance against both
patch and plaque lesions are particularly encouraging. As one of
the largest studies in CTCL, this study and this publication
establishes a new benchmark in CTCL treatment."
About JAMA Dermatology
JAMA Dermatology is an international peer-reviewed
journal published online weekly and in print/ online issue 12 times
a year. It is one of the highest ranked journals in dermatology,
with an acceptance rate of 9%. The journal, which has been in
continuous publication since 1882, publishes studies in the areas
of medical, surgical, pediatric, geriatric dermatology, oncologic
and aesthetic dermatology. It prioritizes clinical and laboratory
studies that reveal new information pertinent to the interests and
needs of the medical dermatologist, dermatologic surgeon, and all
those concerned with state-of-the-art care of cutaneous disease.
The journal believes that knowledge derived from well-designed
clinical trials and studies of cost-effectiveness are especially
important for improving the practice of dermatology. JAMA
Dermatology is a member of the JAMA Network family of
journals, which includes JAMA, 11 JAMA Network
specialty journals, and JAMA Network Open.
About
HyBryte™
HyBryte™ (research name SGX301) is a novel,
first-in-class, photodynamic therapy utilizing safe, visible light
for activation. The active ingredient in HyBryte™ is
synthetic hypericin, a potent photosensitizer that is topically
applied to skin lesions that is taken up by the malignant T-cells,
and then activated by visible light 16 to 24 hours later. The use
of visible light in the red-yellow spectrum has the advantage of
penetrating more deeply into the skin (much more so than
ultraviolet light) and therefore potentially treating deeper skin
disease and thicker plaques and lesions. This treatment approach
avoids the risk of secondary malignancies (including melanoma)
inherent with the frequently employed DNA-damaging drugs and other
phototherapy that are dependent on ultraviolet exposure. Combined
with photoactivation, hypericin has demonstrated significant
anti-proliferative effects on activated normal human lymphoid cells
and inhibited growth of malignant T-cells isolated from CTCL
patients. In a published Phase 2 clinical study in CTCL, patients
experienced a statistically significant (p=0.04) improvement with
topical hypericin treatment whereas the placebo was ineffective.
HyBryte™ has received orphan drug and fast track
designations from the FDA, as well as orphan designation from the
European Medicines Agency (EMA).
The Phase 3 FLASH trial enrolled a total of 169 patients (166
evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of
three treatment cycles. Treatments were administered twice weekly
for the first 6 weeks and treatment response was determined at the
end of the 8th week of each cycle. In the first double-blind
treatment cycle, 116 patients received HyBryte™
treatment (0.25% synthetic hypericin) and 50 received placebo
treatment of their index lesions. A total of 16% of the patients
receiving HyBryte™ achieved at least a 50% reduction in
their lesions (graded using a standard measurement of dermatologic
lesions, the CAILS score) compared to only 4% of patients in the
placebo group at 8 weeks (p=0.04) during the first treatment cycle
(primary endpoint). HyBryte™ treatment in the first
cycle was safe and well tolerated.
In the second open-label treatment cycle (Cycle 2), all patients
received HyBryte™ treatment of their index lesions.
Evaluation of 155 patients in this cycle (110 receiving 12 weeks of
HyBryte™ treatment and 45 receiving 6 weeks of placebo
treatment followed by 6 weeks of HyBryte™ treatment),
demonstrated that the response rate among the 12-week treatment
group was 40% (p<0.0001 vs the placebo treatment rate in Cycle
1). Comparison of the 12-week and 6-week treatment groups also
revealed a statistically significant improvement (p<0.0001)
between the two groups, indicating that continued treatment results
in better outcomes. HyBryte™ continued to be safe and
well tolerated. Additional analyses also indicated that
HyBryte™ is equally effective in treating both plaque
(response 42%, p<0.0001 relative to placebo treatment in Cycle
1) and patch (response 37%, p=0.0009 relative to placebo treatment
in Cycle 1) lesions of CTCL, a particularly relevant finding given
the historical difficulty in treating plaque lesions in
particular.
The third (optional) treatment cycle (Cycle 3) was focused on
safety and all patients could elect to receive HyBryte™
treatment of all their lesions. Of note, 66% of patients elected to
continue with this optional compassionate use / safety cycle of the
study. Of the subset of patients that received HyBryte™
throughout all 3 cycles of treatment, 49% of them demonstrated a
positive treatment response (p<0.0001 vs patients receiving
placebo in Cycle 1). Moreover, in a subset of patients evaluated in
this cycle, it was demonstrated that HyBryte™ is not
systemically available, consistent with the general safety of this
topical product observed to date. At the end of Cycle 3,
HyBryte™ continued to be well tolerated despite extended
and increased use of the product to treat multiple lesions.
Overall safety of HyBryte™ is a critical attribute of
this treatment and was monitored throughout the three treatment
cycles (Cycles 1, 2 and 3) and the 6-month follow-up period.
HyBryte's™ mechanism of action is not associated with
DNA damage, making it a safer alternative than currently available
therapies, all of which are associated with significant and
sometimes fatal, side effects. Predominantly these include the risk
of melanoma and other malignancies, as well as the risk of
significant skin damage and premature skin aging. Currently
available treatments are only approved in the context of previous
treatment failure with other modalities and there is no approved
front-line therapy available. Within this landscape, treatment of
CTCL is strongly motivated by the safety risk of each product.
HyBryte™ potentially represents the safest available
efficacious treatment for CTCL. With no systemic absorption, a
compound that is not mutagenic and a light source that is not
carcinogenic, there is no evidence to date of any potential safety
issues.
The Phase 3 CTCL clinical study was partially funded by the
National Cancer Institute via a Phase II SBIR grant
(#1R44CA210848-01A1) awarded to Soligenix, Inc.
About Cutaneous T-Cell Lymphoma
(CTCL)
CTCL is a class of non-Hodgkin's lymphoma (NHL), a type of
cancer of the white blood cells that are an integral part of the
immune system. Unlike most NHLs which generally involve
B-cell lymphocytes (involved in producing antibodies), CTCL is
caused by an expansion of malignant T-cell lymphocytes (involved in
cell-mediated immunity) normally programmed to migrate to the skin.
These malignant cells migrate to the skin where they form various
lesions, typically beginning as patches and may progress to raised
plaques and tumors. Mortality is related to the stage of CTCL, with
median survival generally ranging from about 12 years in the early
stages to only 2.5 years when the disease has advanced. There is
currently no cure for CTCL. Typically, CTCL lesions are treated and
regress but usually return either in the same part of the body or
in new areas.
CTCL constitutes a rare group of NHLs, occurring in about 4% of
the approximate 700,000 individuals living with the disease. It is
estimated, based upon review of historic published studies and
reports and an interpolation of data on the incidence of CTCL that
it affects over 25,000 individuals in the U.S., with approximately
3,000 new cases seen annually.
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 and moving toward
potential commercialization of HyBryte™ (SGX301 or
synthetic hypericin) as a novel photodynamic therapy utilizing safe
visible light for the treatment of cutaneous T-cell lymphoma
(CTCL). With a successful Phase 3 study completed, regulatory
approval is being sought and commercialization activities for this
product candidate are being advanced initially in the U.S.
Development programs in this business segment also include our
first-in-class innate defense regulator (IDR) technology,
dusquetide (SGX942) for the treatment of inflammatory diseases,
including 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, and our vaccine programs
targeting filoviruses (such as Marburg and Ebola) and
CiVax™, our vaccine candidate for the prevention of
COVID-19 (caused by SARS-CoV-2). 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 Agency
(DTRA) and the Biomedical Advanced Research and Development
Authority (BARDA).
For further information regarding Soligenix, Inc., please visit
the Company's website at https://www.soligenix.com and
follow us on LinkedIn and Twitter at @Soligenix_Inc.
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 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 the
timing or success of any of its clinical/preclinical trials.
Despite the statistically significant result achieved in the
HyBryte™ (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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SOURCE Soligenix, Inc.