SAN DIEGO--(BUSINESS WIRE)--
Retrophin, Inc. (NASDAQ:RTRX) today announced that the U.S. Food and
Drug Administration (FDA) has reviewed the Company's Investigational New
Drug (IND) application for RE-024, a novel phosphopantothenate
replacement therapy, and has granted clearance to begin clinical
studies. The Company is initiating a Phase 1 trial in healthy adult
subjects to evaluate safety, tolerability, and pharmacokinetics, with
enrollment expected to begin in the coming weeks. RE-024 is being
developed for the treatment of pantothenate kinase-associated
neurodegeneration (PKAN), a rare and lethal autosomal recessive
neurodegenerative disorder believed to affect approximately one to three
persons per million worldwide, with no approved treatment currently
available.
"We are excited to see RE-024 move forward into the clinic and get one
step closer to reaching patients who are suffering from this rare and
debilitating disease," said Dr. Alvin Shih, Executive Vice President and
Head of Research & Development for Retrophin. "With the continued
support of the scientific and patient communities, our research and
development team will continue to rapidly advance RE-024 through
clinical development with the hope of providing these patients with a
much needed treatment option."
The Phase 1 trial is a randomized, double-blind, placebo-controlled
single ascending dose study of orally administered RE-024 in healthy
volunteer subjects. The primary objective of the study is to assess
safety and tolerability of single oral doses of RE-024. As a secondary
objective, pharmacokinetics of RE-024 will be assessed at multiple time
points. Pending study completion, safety and pharmacokinetic data will
be submitted for publication.
About RE-024 & PKAN
RE-024 is a small molecule discovered by Retrophin that is being
developed as a replacement therapy for phosphopantothenate. Preclinical
studies in PANK-deficient animal models and cell lines have demonstrated
the ability of RE-024 to restore Coenzyme A (CoA) levels. PKAN is caused
by a mutation in the PANK2 gene, which encodes a critical protein that
metabolizes vitamin B5 (pantothenate) to phosphopantothenate. The
disruption of this metabolic pathway ultimately leads to decreased
levels of CoA, an important substrate for many functions such as
mitochondrial energy metabolism. Clinical manifestations of PKAN include
developmental delay in children, dystonia sometimes causing intractable
pain, parkinsonism with Parkinson's-like freezing and bradykinesia,
choreoathetosis, status dystonicus, dysarthria, spasticity, rigidity,
and dysphagia often leading to feeding tube placement.
About Retrophin
Retrophin is a pharmaceutical company focused on the development,
acquisition and commercialization of drugs for the treatment of serious,
catastrophic or rare diseases for which there are currently no viable
options for patients. The Company's approved products include Chenodal®,
Cholbam™, and Thiola®, and its pipeline includes compounds for several
catastrophic diseases, including focal segmental glomerulosclerosis
(FSGS), pantothenate kinase-associated neurodegeneration (PKAN),
infantile spasms, nephrotic syndrome and others. For additional
information, please visit www.retrophin.com.
Forward-Looking Statements
This press release contains "forward-looking statements" as that term is
defined in the Private Securities Litigation Reform Act of 1995,
regarding the research, development and commercialization of
pharmaceutical products. Without limiting the foregoing, these
statements are often identified by the words "may", "might", "believes",
"thinks", "anticipates", "plans", "expects", "intends" or similar
expressions. In addition, expressions of our strategies, intentions or
plans are also forward-looking statements. Such forward-looking
statements are based on current expectations and involve inherent risks
and uncertainties, including factors that could delay, divert or change
any of them, and could cause actual outcomes and results to differ
materially from current expectations. No forward-looking statement can
be guaranteed. Among the factors that could cause actual results to
differ materially from those indicated in the forward-looking statements
are risks and uncertainties associated with the Company's business and
finances in general, as well as risks and uncertainties associated with
the Company's pre-clinical and clinical stage pipeline as well as its
sales and marketing strategies. Specifically, the risks and
uncertainties the Company faces with respect to its RE-024 program
include risk that the Company will be unable to initiate and/or complete
Phase 1 clinical trials, risk that RE-024 will not progress to Phase 2
clinical trials for safety, regulatory or other reasons; risk associated
with enrollment of clinical trials for rare diseases; risk that the
company's later stage RE-024 clinical studies will fail to demonstrate
that RE-024 is safe and effective. The Company also faces risk that it
will be unable to raise additional funding required to complete
development of any or all of its product candidates; risk relating to
the Company's dependence on contractors for clinical drug supply and
commercial manufacturing; uncertainties relating to patent protection
and intellectual property rights of third parties; risks and
uncertainties relating to competitive products and technological changes
that may limit demand for the Company's products. You are cautioned not
to place undue reliance on these forward-looking statements as there are
important factors that could cause actual results to differ materially
from those in forward-looking statements, many of which are beyond our
control. The Company undertakes no obligation to publicly update
forward-looking statement, whether as a result of new information,
future events, or otherwise. Investors are referred to the full
discussion of risks and uncertainties as included in the Company's
filings with the Securities and Exchange Commission.
Retrophin, Inc.
Chris Cline, CFA, 646-564-3680
Manager,
Investor Relations
IR@retrophin.com
Source: Retrophin, Inc.
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