Company to Hold Update Conference Call Today, August 16, 2013 at
10:00 a.m. ET
NEW YORK--(BUSINESS WIRE)--
Retrophin, Inc. (OTCQB: RTRX) today announced that it has received
positive survival results from interim preclinical tests for the
Company's compound, RE-024, for the treatment of the ultra-orphan
disease Pantothenate Kinase-Associated Neurodegeneration (PKAN). RE-024
is a replacement therapy for phosphopantothenate, the substrate that is
missing in patients with PKAN. Tests were conducted on mice that were
administered a PANK inhibitor to induce a PKAN-like phenotype.
The interim results of this two-week study assessed the effect of RE-024
in mice dosed with RE-024 and Hopantenate (HoPan), a pantothenate kinase
inhibitor that simulates PKAN by inhibiting PANK, compared to mice dosed
with HoPan alone. Data from the study showed that 92.5% (37/40) of the
mice dosed with HoPan and RE-024 survived the experiment, while no
(0/14) mice dosed with HoPan alone were alive at the end of the study
period (p < 0.0001).
PKAN is a rare and life-threatening neurological disorder caused by a
mutation in the PANK2 gene, which prevents patients from being able to
properly metabolize vitamin B5 (pantothenate) into phosphopantothenate.
The disruption of this metabolic pathway ultimately leads to decreased
levels of Coenzyme A (CoA) and iron accumulation in the brain. As a
result, patients present with dystonia (sustained muscle contraction
leading to abnormal posture), rigidity, dysphagia (problems swallowing),
weakness, pigmentary retinopathy (visual impairment), tremors, as well
as a number of other symptoms. Onset of PKAN typically occurs prior to
the age of 10 and has an estimated prevalence of 5,000-10,000 patients
worldwide. Many patients die within 10 years of being diagnosed.
"We are proud of what we have accomplished with this program in a short
period of time," said Martin Shkreli, Founder, President and Chief
Executive Officer of Retrophin. "When our team read about the plight of
PKAN patients, we moved quickly to develop a series of
phosphopantothenate analogs that we believed would rescue the phenotype
of patients suffering from this horrific, catastrophic disease. Today I
am so proud of our team for its dedication to these patients. We expect
that we will be able to start a human study in first-quarter 2014."
Suzanne Jackowski, PhD, a member of the faculty at St. Jude Children's
Research Hospital and a leading expert in Coenzyme A metabolism,
commented, "I am encouraged by the results of this most recent
experiment with RE-024. The positive preclinical survival data generated
by this study serve to further confirm our own proof-of-concept
research, conducted earlier this year, pointing to RE-024 as a potential
promising new approach to treating this debilitating and
Company to Host Conference Call
Management will host a corporate update conference call and audio
webcast on Friday, August 16, 2013 at 10:00 a.m. ET. To participate in
the conference call, dial 866-953-6857 (U.S.) or 617-399-3481
(International), confirmation code 95270920 shortly before 10:00 a.m.
ET. The audio webcast can be accessed at: www.retrophin.com,
in the Investor Relations section. A replay of the call will be
available from 12:00 p.m. ET on Friday August 16, 2013, through 11:59
p.m. ET on Friday, August 23. The replay number is 888-286-8010 (U.S.)
or 617-801-6888 (International), confirmation code 74457089.
Retrophin is a pharmaceutical company focused on the discovery and
development of drugs for the treatment of debilitating and often
life-threatening diseases for which there are currently no viable
patient options. The Company is currently focused on several
catastrophic diseases affecting children, including Focal Segmental
Glomerulosclerosis (FSGS), Pantothenate Kinase-Associated
Neurodegeneration (PKAN), Duchenne Muscular Dystrophy and others.
Retrophin's lead compound, RE-021, is scheduled to begin enrollment in a
potentially pivotal Phase 2 clinical trial for FSGS during 2013. For
additional information, please visit www.retrophin.com.
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. 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.
Forward-looking statements in the press release should be evaluated
together with the many uncertainties that affect the Company's business.
The Company undertakes no obligation to publicly update any
forward-looking statement, whether as a result of new information,
future events, or otherwise.
Marc Panoff, 917-261-3684
Paula Schwartz, 917-322-2216
Source: Retrophin, Inc.
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