Blueprint
Exhibit 99.1
CorMedix Inc. Granted Orphan Drug Designation for Taurolidine for
Treatment of Neuroblastoma
Berkeley Heights, NJ – February 26, 2018 – CorMedix Inc. (NYSE American: CRMD),
a biopharmaceutical company focused on developing and
commercializing therapeutic products for the prevention and
treatment of infectious and other diseases, today announced that it
received notification on Friday, February 23, 2018 that the U.S.
Food and Drug Administration (FDA) granted orphan drug designation
to taurolidine for the treatment of neuroblastoma. Taurolidine is
currently in preclinical development for neuroblastoma and is a key
component in the Company’s lead product, Neutrolin®, a
novel anti-infective solution for the reduction and prevention of
catheter-related infections and thrombosis in patients requiring
central venous cathers in end stage renal
disease.
“We are pleased to expand on our potential indications for
taurolidine and to receive orphan drug designation for treatment of
neuroblastoma,” said Khoso Baluch, Chief Executive Officer of
CorMedix. “We believe taurolidine could have potential
activity in this rare pediatric disease area, based on ongoing
laboratory studies. Current treatments offer poor outcomes and
based on its potential synergy with other drugs approved in this
area, we believe taurolidine could become an important therapy for
neuroblastoma. Our goal in this area is to partner with an
appropriate cancer-focused company to advance taurolidine into
clinical development and ultimately obtain marketing
approval.”
Orphan drug designation is granted by the FDA to novel drugs or
biologics that treat rare diseases or conditions affecting fewer
than 200,000 patients in the U.S. The designation provides CorMedix
with a potential seven-year period of U.S. marketing exclusivity
upon approval of the drug for commercial distribution, and
eligibility for grant funding, clinical trial design assistance, as
well as tax credits for research costs, and the waiver of
Prescription Drug User Fee Act (PDUFA) filing fees.
Neuroblastoma is a severe form of cancer that originates in certain
types of nerve tissues and most often begins in the adrenal glands,
which are the small glands on top of the kidneys. It can develop in
the chest, stomach, neck, pelvis, or bones. Children ages five
or younger are most commonly affected and symptoms include fatigue,
loss of appetite, and fever. The tumor can cause a large bump in
the neck region and bulging of the eyes and dark circles around the
eyes. In infants it can cause difficulty breathing. A genetic
pattern of inheritance for neuroblastoma can be passed from parent
to child and resistant forms of the disease are prevalent and can
recur.
The Pediatric Oncology Experimental Therapeutics
Investigators’ Consortium (POETIC) has been contracted by
CorMedix to carry out experiments on cell lines and in animal
models. Dr. Antony Pffafle, Chief Scientific Officer of CorMedix,
spearheads the collaboration with POETIC on preclinical development
of taurolidine-based therapies for cancer. Initial candidate
therapies are being developed and formulated for delivery to
tumors, including potential combination therapies involving
taurolidine plus another chemotherapy. The anti-cancer
activity of these candidates was evaluated successfully in various
cancer cell lines, and animal studies are
ongoing.
About POETIC
The Pediatric Oncology Experimental Therapeutics Investigators'
Consortium (POETIC) was founded in February 2003 by Dr. Lia Gore at
the University of Colorado Cancer Center and Dr. Tanya Trippett at
Memorial Sloan-Kettering Cancer Center. POETIC is composed of ten
large academic medical centers in North America with a major
emphasis on comprehensive cancer care and research that provide the
collaborative and research strength needed to complete intensive
phase I and II studies.
POETIC’s participating centers are:
Alberta Children's Hospital (Aru Narenderan, MD, Ph.D)
Arkansas Children's Hospital, Little Rock (Kathleen A. Neville, MD,
MS)
Arnold Palmer Hospital for Children/MD Anderson Cancer Center
Orlando (Amy Smith, MD)
Dana-Farber/Children's Hospital Cancer Center (Susan Chi,
MD)
MD Anderson Cancer Center (Cynthia Herzog, MD)
Lucile Salter Packard Children's Hospital, Stanford (Norman James
Lacayo, MD)
Memorial Sloan-Kettering Cancer Center (Tanya Trippett,
MD)
Penn State Hershey Children's Hospital (Lisa M. McGregor, M.D.,
Ph.D.)
Phoenix Children's Hospital (Jessica Boklan, MD)
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (Dr.
Patrick Brown)
University of Texas Health Science Center (Steve Weitman, M.D.,
Ph.D.)
About CorMedix
CorMedix Inc. is a biopharmaceutical company focused on developing
and commercializing therapeutic products for the prevention and
treatment of infectious and inflammatory diseases. The Company is
focused on developing its lead product Neutrolin®,
a novel, non-antibiotic antimicrobial solution designed to prevent
costly and dangerous bloodstream infections associated with the use
of central venous catheters, currently in a Phase 3 clinical trial
enrolling patients undergoing chronic hemodialysis. Such infections
cost the U.S. healthcare system approximately $6 billion annually
and contribute significantly to increased morbidity and mortality.
Neutrolin has FDA Fast Track status and is designated as a
Qualified Infectious Disease Product, which provides the potential
for priority review of a marketing application by FDA and
allows for 5 additional years of QIDP market exclusivity in the
event of U.S. approval. Neutrolin is already marketed as a CE
Marked product in Europe and other territories. In parallel,
CorMedix is leveraging its taurolidine technology to develop a
pipeline of antimicrobial medical devices, with active programs in
surgical sutures and meshes, and topical hydrogels. The
company is also working with top-tier researchers to develop
taurolidine-based therapies for rare pediatric cancers. For
more information, visit: www.cormedix.com.
Forward-Looking Statements
This press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995,
including with respect to possible uses of taurolidine, that are
subject to risks and uncertainties. All statements, other than
statements of historical facts, regarding management’s
expectations, beliefs, goals, plans or CorMedix’s prospects,
future financial position, financing plans, future revenues and
projected costs should be considered forward-looking. Readers are
cautioned that actual results may differ materially from
projections or estimates due to a variety of important
factors, including: preclinical
results are not indicative of success in clinical trials and might
not be replicated in any subsequent studies or trials; the possible
inability to capture sufficient CRBSI events in the ongoing Phase 3
clinical trial for Neutrolin®;
the cost, timing and results of the ongoing and planned Phase 3
trials for Neutrolin® in
the U.S. and the resources needed to commence and complete those
trials; the risks and uncertainties associated with
CorMedix’s ability to manage its limited cash resources and
the impact on current, planned or future research, including the
ongoing LOCK-IT 100 trial and research for additional uses for
taurolidine; obtaining additional financing to support
CorMedix’s research and development and clinical activities
and operations; and the ability
to retain and hire necessary personnel to staff our operations
appropriately. These and other risks are described in greater
detail in CorMedix’s filings with the SEC, copies of which
are available free of charge at the SEC’s website at
www.sec.gov or upon request from CorMedix. CorMedix may not
actually achieve the goals or plans described in its
forward-looking statements, and investors should not place undue
reliance on these statements. CorMedix assumes no obligation and
does not intend to update these forward-looking statements, except
as required by law.
Investor
Contacts:
Dan
Ferry
Managing
Director
LifeSci
Advisors