Retrophin, Inc. (NASDAQ:RTRX) and Asklepion Pharmaceuticals, LLC, a
privately held rare disease pharmaceutical development company, today
announced the signing of a definitive agreement under which Retrophin
has acquired the exclusive right to purchase from Asklepion, all
worldwide rights, titles, and ownership of cholic acid for the treatment
of bile acid synthesis defects, if approved by the U.S. Food and Drug
Administration (FDA). Under the terms of the agreement, Retrophin will
pay Asklepion an upfront payment of $5 million and up to $73 million in
milestones based on approval and net product sales, plus tiered
royalties on future net sales of cholic acid. Retrophin has secured a
line of credit from current lenders to cover necessary payments.
“We are very pleased with this agreement, which has the potential to
significantly accelerate Retrophin’s growth in 2015 and beyond,” said
Stephen Aselage, Chief Executive Officer of Retrophin. “Cholic acid is a
natural complement to our current commercial portfolio and upon its
approval, Retrophin would be positioned to become the leading provider
of bile acid treatments for a number of rare diseases.”
Mr. Aselage continued, “The effort by Asklepion and its investigators is
a testament to their dedication and hard work in bringing an important
medication closer to the marketplace. The disorders treated in their
clinical trial are devastating and we are grateful to have an
opportunity to address those patients’ needs.”
Cholic acid is being considered for approval in a late-stage review by
the FDA and is currently approved in Europe for the treatment of inborn
errors in primary bile acid synthesis. It restores endogenous bile acid
pool levels while inhibiting abnormal bile acid synthesis. The clinical
use of cholic acid has been documented in medical literature for several
single enzyme bile acid synthesis defects since the mid-1990s.
“This agreement could represent a significant milestone for both our
company and patients suffering from bile acid disorders,” said Gary
Pasternack, Chief Executive Officer of Asklepion. “If cholic acid is
approved, we are confident that Retrophin’s expertise in this area would
allow for the greatest number of patients to benefit from this treatment.
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®,
Thiola® and Vecamyl®, and its pipeline includes compounds for several
catastrophic diseases, including focal segmental glomerulosclerosis
(FSGS), pantothenate kinase-associated neurodegeneration (PKAN),
schizophrenia, infantile spasms, nephrotic syndrome and others. For
additional information, please visit www.retrophin.com.
About Asklepion
Asklepion is a pharmaceutical company focused on the clinical research
and development through regulatory approval of innovative therapies for
rare pediatric diseases. Asklepion’s pipeline products address other
liver diseases and critical care applications in children.
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 Company faces risk
that cholic acid may not be approved by the FDA, that certain conditions
to the purchase of cholic acid by the Company will not be met and that
the Company will not be able to successfully launch and commercialize
cholic acid in the United States following FDA approval. The Company
faces risk that it will be unable to raise additional funding; risk
relating to the dependence on contractors for 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.
Copyright Business Wire 2015