Full Press Release Details
Catalyst Pharmaceutical Partners Announces Publication of Preclinical Proof-of-principle
of CPP-115 Efficacy in Suppressing Infantile Spasms in Epilepsia
Phase I Multiple-Ascending Dose Study to be Initiated in 2014
CORAL GABLES, Fla., Nov. 18, 2013 Catalyst Pharmaceutical Partners, Inc. (Nasdaq: CPRX), a specialty pharmaceutical company focused on
developing safe and effective approved medicines targeting orphan neuromuscular and neurological diseases, today announced that the journal Epilepsia has accepted for publication an original research paper that demonstrates proof of concept
of CPP-115 suppressing infantile spasms (IS) in a pre-clinical study. CPP-115 is Catalyst s next-generation GABA aminotransferase inhibitor being developed for the treatment of IS. Catalyst also announced plans to initiate a Phase I
multiple-dose ascending study in the first half of 2014 to evaluate the safety and tolerability of multiple ascending oral doses of CPP-115.
author (Dr. Aristea Galanopoulou) and principal investigator of the preclinical rodent study described in this publication stated, This publication provides the first proof-of-principle preclinical evidence in support of CPP-115 as a candidate
treatment for IS. Dr. Galanopoulou continued Because vigabatrin is particularly effective in tuberous sclerosis related IS, these patients are likely an ideal target for this analogue that seems to be more potent and less
Epilepsia, the official journal of the International League Against Epilepsy, has published Dr. Galanopoulou s paper,
CPP-115, a vigabatrin analogue, decreases spasms in the multiple-hit rat model of infantile spasms online as an early view of its upcoming publication. The research paper describes the following study conclusions:
There is a significant unmet medical need in this area, as parents of children who have infantile spasms
have a very difficult choice when it comes to treatment options, as they must choose between drug-related risks and adequate treatment, said Patrick J. McEnany, Chairman and CEO of Catalyst. Thanks to Dr. Galanopoulou and her team
at Albert Einstein College of Medicine, we are able to take next steps in the development of CPP-115 shown in this study to be a better and safer treatment option for Infantile Spasms than current options. This study demonstrates that CPP-115 likely
has more than a hundred-fold increase in potency and a better safety profile in the expected therapeutic dose range compared to vigabatrin, as well as increased overall efficacy in this model.
About the Upcoming Phase I Study
study for CPP-115 will be a randomized, double-blind, placebo-controlled, safety, tolerability and pharmacokinetic study of multiple ascending oral doses of CPP-115 in healthy volunteers. The primary objective will be to evaluate the safety and
tolerability of multiple ascending oral doses of CPP-115. Secondary objectives will be to determine the pharmacokinetic profile of CPP-115 and to determine the effects of CPP-115 on brain GABA levels as measured by Magnetic Resonance
Spectroscopy (MRS) following administration of multiple oral daily doses.
(SABRIL ), CPP-115 irreversibly inhibits GABA-aminotransferase, raising brain GABA concentrations. Therefore, the anticipated MRS findings of increased brain GABA are a well characterized
biomarker, which can be correlated to the potential efficacy of CPP-115 to treat infantile spasms and epilepsy, as well as other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette
About the Published Paper
research paper was authored by Stephen W. Briggs, Wenzhu Mowrey, Charles B. Hall, and Aristea Galanopoulou (corresponding author), of the Saul R. Korey Department of Neurology, Albert Einstein College of Medicine of Yeshiva University, Laboratory of
Developmental Epilepsy and Dominick P. Purpura Department of Neuroscience, Albert Einstein College of Medicine of Yeshiva University, Bronx, New York. The abstract can be accessed at http://onlinelibrary.wiley.com/doi/10.1111/epi.12424/abstract.
About Albert Einstein College of Medicine
The Albert Einstein College of Medicine of Yeshiva University is a premier, research-intensive medical school dedicated to innovative biomedical investigation
and to the development of ethical and compassionate physicians and scientists. Inspired by the words of its namesake, the school welcomes students, faculty and staff from diverse backgrounds who strive to enhance human health in the community and
About West Syndrome / Infantile Spasms
West Syndrome is a rare disease having a prevalence in the U.S. of under 200,000 patients. Its estimated prevalence is 2.5 to 6 cases per 10,000 live births or
between 2150 and 5160 patients.
An infantile spasm is a type of seizure seen in an epilepsy syndrome of infancy and childhood known as West Syndrome. The onset of infantile spasms is usually in the first year of life, typically
between 4-8 months. Spasms often occur in clusters of up to 100 at a time, and infants may have dozens of clusters and several hundred spasms per day. Infantile spasms usually stop by age five, but may be replaced by other seizure types. Many
underlying disorders, such as birth injury, metabolic disorders and genetic disorders can give rise to spasms, making it important to identify them (symptomatic IS). In some children, no cause can be found (cryptogenic IS). Mental retardation occurs
in 70-90% of persons with infantile spasms, usually involving severe to profound retardation. Early control of seizures is critical for reducing developmental delays and levels of mental retardation, but ~5% of infants with this condition eventually
die from complications caused by the seizures.
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc. is a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs
targeting rare (orphan) neuromuscular and neurological diseases, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette Syndrome. Catalyst s lead candidate, Firdapse for the treatment of LEMS, is currently
undergoing testing in a global, multi-center, pivotal Phase 3 trial and recently received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA). In 2012, Catalyst licensed Firdapse from BioMarin and
Catalyst assumed management of the Phase 3 pivotal trial, initiated by BioMarin. Firdapse is the first and only European approved drug for symptomatic treatment in adults with LEMS.
Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115) to treat infantile spasms, and epilepsy, as well as
other neurological conditions associated with reduced GABAergic signaling, like post-traumatic stress disorder and Tourette Syndrome. CPP-115 has been granted U.S. orphan drug designation for the treatment of infantile spasms by the FDA and has been
granted EU orphan medicinal product designation for the treatment of West Syndrome by the European Commission.
Forward-Looking Statements
This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause the
Company s actual results in future periods to differ materially from forecasted results. A number of factors, including whether CPP-115 will prove to be an effective treatment for infantile spasms in humans, whether CPP-115 will ultimately
prove to have significantly reduced risks of visual field defects compared to vigabatrin, whether CPP-115 will ever be approved for commercialization, and those other factors described in the Company s filings with the SEC, could adversely
affect the Company. Copies of the Company s filings with the SEC are available from the SEC, may be found on the Company s website or may be obtained upon request from the Company. The Company does not undertake any obligation to update
the information contained herein, which speaks only as of this date.