PRINCETON, N.J., March 22, 2018 /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 European Commission, acting on the
positive recommendation from the European Medicines Agency (EMA)
Committee for Orphan Medicinal Products, has granted orphan drug
designation to the Company's recombinant modified ricin toxin
A-chain subunit (the active pharmaceutical ingredient in
RiVax®) for the prevention of ricin poisoning.
RiVax® has previously been granted orphan drug
designation from the US Food and Drug Administration (FDA).
The European Commission grants orphan designations for medicines
that treat a life-threatening or chronically debilitating condition
affecting no more than five in 10,000 persons in the European Union
(EU) and where no satisfactory treatment is available. In
addition to a 10-year period of marketing exclusivity in the EU
after product approval, orphan drug designation provides incentives
for companies seeking protocol assistance from the EMA during the
product development phase, and direct access to the centralized
authorization procedure which allows access to all 28 EU member
states' markets.
"We are extremely pleased to have received European orphan drug
designation for the RiVax® program," stated Christopher J. Schaber, PhD, President and Chief
Executive Officer of Soligenix, Inc. "This EU orphan designation,
combined with our US orphan designation, positions this biodefense
program for a potentially accelerated global regulatory product
development pathway to address this unmet need.
RiVax® has shown up to 100% protection against
aerosolized ricin exposure in non-human primates and safety in
humans and we look forward to further developing our thermostable
formulation for human use."
About Ricin Toxin
Ricin toxin is a lethal plant-derived toxin and potential
biological weapon because of its stability and high potency, and
the fact it is readily extracted from by-products of castor oil
production. Ricin comes in many forms including powder, mist
or pellet. Ricin can also be dissolved in water and other
liquids. The US Centers for Disease Control and Prevention
estimates that the lethal dose in humans is about the size of a
grain of salt. Ricin toxin illness causes tissue necrosis and
general organ failure leading to death within several days of
exposure. Ricin is especially toxic when inhaled. Ricin
works by entering cells of the body and preventing the cells from
making the proteins it needs. Without the proteins, cells
die, which is eventually harmful to the entire body.
There are currently no effective treatments for ricin
poisoning. The successful development of an effective vaccine
against ricin toxin may act as a deterrent against the actual use
of ricin as a biological weapon and could be used to vaccinate
military personnel and civilian emergency responders at high risk
of potential exposure in the event of a biological attack.
About RiVax®
RiVax® is Soligenix's proprietary heat stable
recombinant subunit vaccine developed to protect against exposure
to ricin toxin. With RiVax®, Soligenix is a world
leader in the area of ricin toxin vaccine research.
RiVax® contains a genetically altered version of
a ricin toxin A-chain containing two mutations that inactivate the
toxicity of the ricin molecule. A Phase 1A clinical trial was
conducted with a formulation of RiVax® that did not
contain an adjuvant. This trial revealed dose dependent
seroconversion as well as lack of toxicity of the molecule when
administered intramuscularly to human volunteers. The
adjuvant-free formulation of RiVax® induced toxin
neutralizing antibodies that lasted up to 127 days after the third
vaccination in several individuals.
To increase the longevity and magnitude of toxin neutralizing
antibodies, RiVax® was subsequently formulated with
an adjuvant of aluminum salts (known colloquially as Alum) for a
Phase 1B clinical trial. Alum
is an adjuvant that is used in many human vaccines, including most
vaccines used in infants. The results of the Phase
1B study indicated that
Alum-adjuvanted RiVax® was safe and well tolerated,
and induced greater ricin neutralizing antibody levels in humans
than adjuvant-free RiVax®. In preclinical animal
studies, the Alum formulation of RiVax® also
induced higher titers and longer-lasting antibodies than the
adjuvant-free vaccine. Vaccination with the thermostabilized
Alum-adjuvanted RiVax® formulation in a large
animal model provided 100% protection (p<0.0001) against acute
exposure to aerosolized ricin, the most lethal route of exposure
for ricin. The protected animals also had no signs of gross
lung damage, a serious and enduring ramification with long-term
consequences for survivors of ricin exposure. These results
are described in a publication available here.
Heat stabilization of RiVax® is achieved with
the Company's proprietary ThermoVax® technology,
designed to eliminate the cold-chain production, distribution and
storage logistics required for most vaccines. The technology
utilizes precise lyophilization of protein immunogens with
conventional aluminum adjuvants in combination with secondary
adjuvants for rapid onset of protective immunity with the fewest
number of vaccinations. By employing
ThermoVax® during the final formulation of
RiVax®, the vaccine has demonstrated enhanced stability
and the ability to withstand temperatures at least as high as 40
degrees Celsius (104 degrees Fahrenheit) for up to one year.
The development of RiVax® has been sponsored
through a series of grants from both National Institute of Allergy
and Infectious Diseases (NIAID) and the FDA and ongoing development
is sponsored by NIAID contract # HHSN272201400039C.
RiVax® potentially would be added to the Strategic
National Stockpile and dispensed in the event of a terrorist
attack. RiVax® has received orphan drug
designation from the FDA and EMA, conferring 7 and 10 years
marketing exclusivity, respectively.
As a new chemical entity, an FDA approved
RiVax® vaccine has the potential to qualify for a
biodefense Priority Review Voucher (PRV), which allows the holder
accelerated review of a drug application. Approved under the
21st Century Health Cures Act in late 2016, the
biodefense PRV is awarded upon approval as a medical countermeasure
when the active ingredient(s) have not been otherwise approved for
use in any context. PRVs are transferable and can be sold,
with sales in recent years ranging between $125 million to $350
million. When redeemed, PRVs entitle the user to an
accelerated review period of six months, saving a median of seven
months' review time as calculated in 2009. However, the FDA
must be advised 90 days in advance of the use of the PRV and the
use of a PRV is associated with an additional user fee
($2.7 million in 2017).
About ThermoVax®
The ThermoVax® technology is designed to eliminate
the cold chain production, distribution and storage logistics
required for most vaccines. The technology utilizes precise
lyophilization of protein immunogens with conventional aluminum
adjuvants in combination with secondary adjuvants for rapid onset
of protective immunity with the fewest number of vaccinations. Cold
chain requirements add considerable cost to the production and
storage of current conventional vaccines. Elimination of the cold
chain would also enhance the utility of these vaccines for emerging
markets and for other applications requiring but lacking reliable
cold chain capabilities. For vaccines that are intended for
long-term stockpiling, such as for use in biodefense or in pandemic
situations, the utilization of ThermoVax® has the
potential to facilitate easier storage and distribution of
Strategic National Stockpile vaccines in emergency situations. The
underlying ThermoVax® technology has been developed by
Drs. John Carpenter and Theodore Randolph at the University of Colorado.
By employing ThermoVax® during the final formulation
of RiVax®, the vaccine has demonstrated enhanced
stability and the ability to withstand temperatures at least as
high as 40 degrees Celsius (104 degrees Fahrenheit) for up to one
year. Similar stabilization at temperatures as high as 50 degrees
Celsius for up to three months (maximum timepoint tested) have also
been demonstrated with other antigens (e.g., human papillomavirus,
Ebola and anthrax).
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 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 Vaccines/BioDefense 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) 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 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. 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. Further, there can be no assurance
that RiVax® will qualify for a biodefense PRV or that
the prior sales of PRVs will be indicative of any potential sales
price for a PRV for RiVax®. 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.