PRINCETON, N.J., Sept. 17, 2014 /PRNewswire/ -- Soligenix,
Inc. (OTCQB: SNGX) (Soligenix or the Company), a late-stage
biopharmaceutical company developing products that address unmet
medical needs in the areas of inflammation, oncology and
biodefense, announced today that agreement has been reached with
the US Food and Drug Administration (FDA) on the design of a
pivotal, Phase 3 clinical trial evaluating its product SGX301
(synthetic hypericin) for the treatment of cutaneous T-cell
lymphoma (CTCL).
SGX301 is a novel, first-in-class, photodynamic therapy
utilizing safe visible light for activation. The active
ingredient, synthetic hypericin, is a potent photosensitizer which
is topically applied to skin lesions and activated by visible
fluorescent light. 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 A (UVA)
exposure. Topical hypericin has demonstrated safety in a Phase
1 clinical study in healthy volunteers. In a Phase 2,
double-blind, placebo-controlled clinical study in CTCL patients,
the drug was safe and well tolerated, with 58.3% of the CTCL
patients responding to SGX301 treatment compared to only 8.3%
receiving placebo (p < 0.04).
"I enthusiastically support Soligenix in their efforts to
improve outcomes for patients with CTCL, affecting up to 50,000
patients in the US," stated Alain
Rook, MD, Director, Cutaneous Lymphoma Program, Hospital of
the University of Pennsylvania. "I have had a lengthy
scientific and clinical interest in hypericin and am pleased that
the Soligenix team is advancing this product to a Phase 3 clinical
study. I believe that SGX301 has the potential to be a major step
forward in the treatment of CTCL by providing a safer alternative
therapy over the course of the patients' disease than is currently
available."
Based on the positive results demonstrated in the Phase 2 study
of SGX301, the upcoming Phase 3 protocol will be a highly powered,
double-blind, randomized, placebo-controlled, multicenter trial and
will seek to enroll approximately 120 patients. The trial will
consist of three treatment cycles, each of 8 weeks duration.
Treatments will be 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, approximately 80 patients will receive
SGX301 and 40 will receive placebo treatment of their index
lesions. In the second cycle, all patients will receive SGX301
treatment of their index lesions and in the third (open-label)
cycle all patients will receive SGX301 treatment of all their
lesions. Subjects will be followed for an additional 6 months
after the completion of treatment. The primary clinical efficacy
endpoint is treatment response assessed using the CAILS (Composite
Assessment of Index Lesion Severity) score evaluating the three
worst index lesions at the end of Cycle 1 (Week 8). The trial is
anticipated to begin in the first half of 2015 with primary data
available in the second half of 2016.
"We are pleased to have FDA agreement on our Phase 3 protocol
design in CTCL," stated Christopher J.
Schaber, PhD, President and Chief Executive Officer of
Soligenix. "We are excited to move forward with this pivotal trial
in an effort to address the significant unmet medical need in this
orphan disease."
About CTCL
Cutaneous T-cell lymphoma (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 skin-trafficking
malignant T-cells migrate to the skin, causing various lesions to
appear that may change shape as the disease progresses, typically
beginning as a rash and eventually forming plaques and
tumors. Mycosis fungoides (MF) is the most common form of
CTCL. It generally presents with skin involvement only, manifested
as scaly, erythematous patches. Advanced disease with diffuse
lymph node and visceral organ involvement is usually associated
with a poorer response rate to standard therapies. A
relatively uncommon sub-group of CTCL patients present with
extensive skin involvement and circulating malignant cerebriform
T-cells, referred to as Sezary syndrome. These patients have
substantially graver prognoses than those with MF.
With CTCL mortality is related to stage of disease, 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. Treatment of early-stage disease
generally involves skin-directed therapies. Most MF
treatments are not approved by the FDA. One of the most common
unapproved therapies used for early-stage disease is oral 5 or
8-methoxypsoralen (Psoralen) given with ultraviolet A (UVA) light,
referred to as PUVA. Although having demonstrated a level of
efficacy, psoralen is a mutagenic chemical that interferes with DNA
causing mutations and other malignancies. Moreover, UVA is a
carcinogenic light source that when combined with the psoralen,
results in serious adverse effects including secondary skin
cancers; therefore, the FDA requires a Black Box warning for
PUVA.
CTCL constitutes a rare group of NHLs, occurring in about 4% of
the approximate 500,000 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 20,000 individuals in the US, with approximately
2,800 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 which is topically applied to skin lesions and then
activated by fluorescent light 16 to 24 hours later.
Hypericin is also found in several species of Hypericum
plants, although the drug used in SGX301 is chemically synthesized
by a proprietary manufacturing process and not extracted from
plants. Importantly, hypericin is optimally activated with visible
light thereby avoiding the negative consequences of ultraviolet
light.
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 both settings, it appears that the mode of
action is an induction of cell death in a concentration as well as
a light dose-dependent fashion. These effects appear to
result, in part, from the generation of singlet oxygen during
photoactivation of hypericin.
Hypericin is one of the most efficient known generators of
singlet oxygen, the key intermediate for phototherapy. The
generation of singlet oxygen induces necrosis and apoptosis in
adjacent cells. The use of topical hypericin coupled with
directed visible light results in generation of singlet oxygen only
at the required site. The use of visible light (as opposed to
cancer-causing ultraviolet light) is a major advance in
photodynamic therapy. In a published Phase 2 clinical study in
CTCL, patients experienced a significant response with topical
hypericin treatment as compared to placebo: 58.3% compared to
8.3% (p < 0.04), respectively.
SGX301 has received orphan drug designation from the US FDA. The
US Orphan Drug Act is intended to assist and encourage companies to
develop safe and effective therapies for the treatment of rare
diseases and disorders. In addition to providing a seven year term
of market exclusivity for SGX301 upon final FDA approval, orphan
drug designation also positions Soligenix to be able to leverage a
wide range of financial and regulatory benefits, including
government grants for conducting clinical trials, waiver of
expensive FDA user fees for the potential submission of a New Drug
Application for SGX301, and certain tax credits.
About Soligenix, Inc.
Soligenix is a late-stage biopharmaceutical company developing
products that address unmet medical needs in the areas of
inflammation, oncology and biodefense. Soligenix is developing
proprietary formulations of oral BDP (beclomethasone
17,21-dipropionate) for the prevention/treatment of
gastrointestinal disorders characterized by severe inflammation,
including pediatric Crohn's disease (SGX203) and acute radiation
enteritis (SGX201). Soligenix is also advancing its novel innate
defense regulator (IDR) technology SGX942 for the treatment of oral
mucositis and SGX301, its novel first-in-class photodynamic
technology utilizing synthetic hypericin with safe visible light,
for the treatment of cutaneous T-cell lymphoma.
Through its BioDefense Division, Soligenix is developing
countermeasures pursuant to the Biomedical Advanced Research and
Development Authority (BARDA) Strategic Plan of 2011-2016 for
inclusion in the US government's Strategic National Stockpile.
Soligenix's biodefense products in development are a recombinant
subunit vaccine called RiVax™, which is designed to protect against
the lethal effects of exposure to ricin toxin and VeloThrax™, a
vaccine against anthrax exposure. RiVax™ has demonstrated
statistically significant survival results in a lethal aerosol
exposure non-human primate model and has also been shown to be well
tolerated and immunogenic in two Phase 1 clinical trials in healthy
volunteers. Both RiVax™ and VeloThrax™ are currently the
subject of a $9.4 million National
Institute of Allergy and Infectious Diseases (NIAID) grant
supporting development of Soligenix's new vaccine heat
stabilization technology known as ThermoVax™. Soligenix is
also developing OrbeShield™ for the treatment of gastrointestinal
acute radiation syndrome (GI ARS) under a BARDA contract award
valued up to $26.3 million and a
NIAID contract award valued up to $6.4
million. OrbeShield™ has previously demonstrated
statistically significant preclinical survival results in a canine
model of GI ARS funded by the NIAID. Additionally, Soligenix
has an exclusive worldwide collaboration with Intrexon Corporation
(NYSE: XON) focused on the joint development of a treatment
for Melioidosis, a high priority biothreat and an area of unmet
medical need.
For further information regarding Soligenix, Inc., please visit
the Company's website at www.soligenix.com.
This press release contains 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," "intends,"
"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 vaccines against bioterror
threats conducting preclinical and clinical trials of vaccines,
obtaining regulatory approvals and manufacturing 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. 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.
SOURCE Soligenix, Inc.