PRINCETON, N.J., March 19, 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 positive preliminary top-line results
for its pivotal Phase 3 FLASH (Fluorescent Light Activated
Synthetic Hypericin) trial evaluating SGX301 (Synthetic Hypericin)
in the treatment of cutaneous T-cell lymphoma (CTCL). The
study enrolled 169 patients randomized 2:1 to receive either SGX301
or placebo, demonstrating statistically significant treatment
response (p=0.04) in the Composite Assessment of Index Lesion Score
(CAILS) primary endpoint assessment at 8 weeks for Cycle 1. In
addition, preliminary assessment of the open-label Cycle 2 results
suggest a significantly more robust response rate after 12 weeks of
SGX301 treatment. These data are expected to be announced in
June 2020.
"This is an important outcome for patients suffering from CTCL.
SGX301 has successfully demonstrated efficacy in this challenging
chronic cancer, with no safety concerns, making it a potentially
preferred first-line option for the treatment of early stage CTCL,
which is the large majority of patients suffering from this
disease," stated Ellen Kim, MD,
Director of the Dermatology Clinic, Perelman Center for Advanced
Medicine and Lead Investigator of the FLASH study. "The treatment
showed a statistically significant improvement after just 6 weeks
of treatment. This successfully proves that the drug has biologic
activity in combating this disease in a relatively short time
window, with preliminary data suggesting that the improvement
continues to increase with extended treatment. In addition to
the efficacy demonstrated, SGX301 was well-tolerated and its
mechanism of action is not associated with DNA damage like other
currently available therapies."
"On behalf of everyone at Soligenix, I would like to extend
my sincere appreciation to the patients, families, investigators,
and advisors involved in the pivotal Phase 3 FLASH study," stated
Christopher J. Schaber, PhD,
President and Chief Executive Officer of Soligenix. "We are
extremely pleased with the results, which demonstrate successful
treatment with SGX301 and reinforces its potential to be an
important new treatment for early stage CTCL. We will now look
to move as quickly as possible to complete a full analysis of the
data for publication, as well as begin preparations for a robust
discussion with the FDA regarding this important dataset."
Dr. Schaber continued, "With approximately $7.6M in cash, not including the anticipated sale
of our New Jersey net operating
loss carryovers and United Kingdom
tax incentive receivable of approximately $1.0M or our non-dilutive government funding, we
will evaluate the potential need for a larger capital raise only
after top-line results from our Phase 3 study in oral
mucositis. This will afford us the opportunity to more
thoroughly assess commercialization and/or partnership of SGX301 in
tandem with preparing for the New Drug Application submission to
FDA. These results support our long-standing belief that SGX301 has
the potential to be a valuable therapy in the treatment of early
stage CTCL, which is an orphan disease and area of unmet medical
need."
Conference Call Thursday, March 19 at 8:30 AM
Eastern Time
The Company will share trial results on Thursday, March 19,
2020 during an investor conference call. Investors may submit
questions electronically at: 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 conference call will be archived for 30 days
following the event.
The Phase 3 FLASH trial enrolled 169 patients (166 evaluable)
with Stage IA, IB or IIA CTCL. The trial consists of three
treatment cycles, each of 8 weeks duration. 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
subjects received SGX301 treatment (0.25% synthetic hypericin) and
50 received placebo treatment of their index lesions. A total
of 16% of the patients receiving SGX301 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). SGX301 treatment
in the first cycle was safe and well tolerated. In the second
open-label treatment cycle (Cycle 2), all patients received SGX301
treatment. Of note, preliminary results from blinded data to
date suggest more than a 35% response rate (inclusive of patients
receiving both 12 weeks and 6 weeks of therapy), indicating the
response increases with continued treatment. Further independent
review of lesion photographs may be conducted to provide for a more
uniform confirmation of response. Results from Cycle 2 are
expected to be announced in June
2020.
In the third (optional) treatment cycle (Cycle 3), all subjects
could receive SGX301 treatment of all their lesions. Of note,
the majority of patients enrolled have elected to continue with
this optional cycle of the study. Moreover, in a subset of
patients evaluated in this cycle, it was demonstrated that SGX301
is not systemically available, consistent with the general safety
of this topical product observed to date. Results from Cycle 3
and the subsequent 6-month follow-up after completion of treatment
will be further announced as the final patients continue to
complete their designated visits.
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 a rash and eventually
forming raised plaques and tumors as the disease
progresses. 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 US, with approximately
3,000 new cases seen annually.
About SGX301
SGX301 is a novel first-in-class photodynamic therapy utilizing
safe visible light for activation. The active ingredient in
SGX301 is synthetic hypericin, a potent photosensitizer that is
topically applied to skin lesions, is taken up by the malignant
T-cells, and then activated by fluorescent light 16 to 24 hours
later. This treatment approach avoids the risk of secondary
malignancies (including melanoma) inherent with the frequently
employed DNA-damaging chemotherapeutic drugs and other photodynamic
therapies 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. SGX301 has received orphan drug and fast
track designations from the US Food and Drug Administration (FDA),
as well as orphan designation from the European Medicines Agency
(EMA).
Based on the positive results demonstrated in the Phase 2 study
of SGX301, the Phase 3 protocol is a highly powered, double-blind,
randomized, placebo-controlled, multicenter trial targeted to
enroll 160 evaluable subjects. The trial consists of three
treatment cycles, each of 8 weeks duration. Treatments are
administered twice weekly for the first 6 weeks and treatment
response will be determined at the end of Week 8. In the first
treatment cycle, 116 subjects received SGX301 and 50 subjects
received placebo treatment of their index lesions. In the second
cycle, all subjects received SGX301 treatment of their index
lesions and in the third cycle all subjects could receive SGX301
treatment of all their lesions. Subjects are followed
for an additional 6 months after the completion of
treatment. The primary efficacy endpoint was assessed on the
percent of patients in each of the two treatment groups (i.e.,
SGX301 and placebo) achieving a Partial or Complete Response
(yes/no) of the treated lesions defined as a ≥ 50% reduction in the
total Composite Assessment of Index Lesion Disease Severity (CAILS)
score for three index lesions at the Cycle 1 evaluation visit (Week
8) compared to the total CAILS score at baseline. Assessment
of the primary endpoint revealed that 16% patients receiving SGX301
responded (i.e., had ≥ 50% reduction in index lesion size) while
only 4% receiving placebo responded (p=0.04). Preliminary results
from blinded data to date suggest more than a 35% response rate
(inclusive of patients receiving both 12 weeks and 6 weeks of
therapy), indicating the response increases with continued
treatment.
Other secondary measures assessed are treatment response
(including duration), degree of improvement, time to relapse and
safety, and will be available as the subsequent cycles and
follow-up visits are completed for all
subjects.
Overall safety of SGX301 is a critical attribute of this
treatment and will continue to be monitored throughout the
additional treatment cycles and the 6-month follow-up
period. SGX301'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. SGX301 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 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 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 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 any of our other clinical/preclinical
trials. 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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