Study Supported by $2.6
Million FDA Orphan Products Development Grant
PRINCETON, N.J., Aug. 10,
2023 /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 patient enrollment has been opened for the
investigator-initiated study (IIS) at the University of Pennsylvania, supported by a
$2.6 million Orphan Products
Development grant award by the U.S. Food and Drug Administration
(FDA). The IIS will evaluate the expanded treatment,
including up to 12 months of treatment, with
HyBryte™ (synthetic hypericin) in patients with early-stage
cutaneous T-cell lymphoma (CTCL). The trial is sponsored by
Ellen Kim, MD, Director, Penn
Cutaneous Lymphoma Program, Vice Chair of Clinical Operations,
Dermatology Department, and Professor of Dermatology at the
Hospital of the University of
Pennsylvania who was a leading enroller in the recently
published positive Phase 3 FLASH (Fluorescent Light Activated
Synthetic Hypericin) study in the treatment of early-stage
CTCL.
"In the Phase 3 FLASH study, HyBryte™ was shown to be
efficacious with a promising safety profile. CTCL patients are
often searching for alternative treatments, with limited options
especially for early-stage disease. HyBryte™ offers a distinct
treatment option which patients found extremely useful and continue
to specifically request. We look forward to demonstrating the
expanded use of HyBryte™ in a "real world" setting and thank both
the FDA and Soligenix for their support of this study," noted Dr.
Kim, principal investigator of the IIS, RW-HPN-MF-01.
"We are pleased the FDA is supporting the HyBryte™ program and
giving patients an opportunity to access the therapy in an
open-label setting," stated Christopher J.
Schaber, President and CEO of Soligenix, Inc. "CTCL is an
incredibly difficult to treat orphan disease and remains an area of
unmet medical need with a very limited number of safe and effective
treatment options. With the demonstration of statistical
significance in the primary endpoint and continued improvement in
treatment response with extended treatment in our published Phase 3
FLASH study, we are continuing discussions with FDA on the design
of a second confirmatory Phase 3 study. This IIS has the potential
to augment the expanding safety database for synthetic hypericin,
as well as provide further real-world evidence into the practical
use of HyBryte™ once commercially available."
The clinical study RW-HPN-MF-01, "Assessment of Treatment with
Visible Light Activated Synthetic Hypericin Ointment in Mycosis
Fungoides Patients" is designed as an open-label, multicenter
clinical trial enrolling approximately 50 patients at select high
enrolling clinical centers that participated in the Phase 3 FLASH
study. Patients have the potential to be treated for up to 12
months with twice a week dosing (visible light activation to follow
ointment application by 24 ± 6 hours). The study also allows
for potential transition to the home-use setting. The primary
endpoint for the study will be evaluating the number of treatment
successes defined as ≥50% reduction in the cumulative CAILS
(Composite Assessment of Index Lesion Severity) score from baseline
to end of the treatment.
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 approximately 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 recently published 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 very limited 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. In addition,
the FDA awarded an Orphan Products Development grant to support the
evaluation of HyBryte™ for expanded treatment in patients with
early-stage CTCL, including in the home use setting. The
grant, totaling $2.6 million over 4
years, was awarded to the University of
Pennsylvania that was a leading enroller in the Phase 3
FLASH study.
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 sodium) 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
expansion of synthetic hypericin (SGX302) into psoriasis, 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).
Our Public Health Solutions business segment includes active
development programs for RiVax®, our ricin toxin vaccine
candidate, as well as 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, and include the
expected amount and use of proceeds from the offering and the
expected closing date of the offering. 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. The
Company cannot offer assurances that the FDA will agree to the
proposed design of a second, Phase 3 pivotal study evaluating
HyBryte™ (hypericin sodium) in the treatment of early stage
cutaneous T-cell lymphoma; that, if accepted, the Company will
conduct the trial; or that, if the Company conducts the trial, the
trial will be successful. Notwithstanding the result in the
HyBryte™ (SGX301) Phase 3 clinical trial for the treatment of
cutaneous T-cell lymphoma and the Phase 2a clinical trial of SGX302
for the treatment of psoriasis, there can be no assurance as to the
timing or success of the clinical trials of SGX302 for the
treatment of psoriasis. 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 (the
"SEC"), including, but not limited to, the Company's preliminary
prospectus (Registration No. 333-271049) filed with the SEC on
May 4, 2023, and 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.