Catalyst Pharmaceutical Partners Reports Positive Results From Phase I(a) Clinical Study of CPP-115
CPP-115 Safe and Well Tolerated Across All Dose Levels
CORAL GABLES, Fla., May 22, 2012 (GLOBE NEWSWIRE) -- Catalyst
Pharmaceutical Partners, Inc. (Nasdaq:CPRX) today announced
positive results from a Phase I(a) study to evaluate the safety,
tolerability and pharmacokinetic (PK) profile of CPP-115, a novel
small molecule drug candidate for the potential treatment of
epilepsy (initially infantile spasms), cocaine addiction and other
select CNS indications.
The study results demonstrated that CPP-115 was well tolerated at
all six doses administered. This Phase I(a) study is also the first
step of an infantile spasms clinical development plan that was
discussed with the U.S. Food and Drug Administration's (FDA)
Division of Neurology Products during a pre-IND meeting in October
2011.
"The positive results of our CPP-115 first-in-man study mark an
important initial milestone toward our goal of becoming a leading
CNS specialty pharmaceutical company," said Patrick J. McEnany,
Chief Executive Officer of Catalyst. "The results of the study are
promising and are consistent with our preclinical findings. We
believe that
CPP-115 has the potential to address important unmet needs in both
epilepsy and addiction, highlighted by its orphan designations in
the United States (infantile spasms) and European Union (West
Syndrome), as well as its Fast Track status for cocaine addiction
in the United States."
Highlights from the CPP-115 Phase I(a) study include:
- Study Design
-- Double-blind, placebo-controlled, single ascending dose of
CPP-115
solution administered orally to healthy volunteers
-- 55 subjects in 7 cohorts of 8 each (1 had 7); 6 subjects
randomized to
CPP-115 and 2 subjects randomized to placebo
-- Dosing ranged from 5 mg to 500 mg
- Primary Outcome Measure
-- Safety of single oral doses, defined as number of subjects
with
clinically significant changes in vital signs,
electrocardiogram
abnormalities based on changes in cardiac rhythm, serious or
severe
adverse events, and/or clinically significant changes in
clinical
laboratory evaluations
- Secondary Outcome Measures
-- Comparative PK profiles of single ascending oral doses, with
profile
comparisons based on plasma concentrations, including: i) Cmax -
maximum
observed plasma concentration; ii) Tmax - time to maximum
observed
plasma concentration; iii) AUC (0 - infinity) - area under
the
concentration-time curve from time zero extrapolated to infinite
time;
and iv) AUC (0 - last) - area under the concentration-time curve
from
time zero to the last measurable concentration
- Key Findings
-- No serious or severe adverse events
-- No cardiovascular or respiratory events
-- Rapidly absorbed (time to peak blood concentration about 30
minutes)
-- Elimination half-life of 4-6 hours
-- Cmax increases in a dose proportional manner over the range of
doses
studied, 5-500 mg; whereas there is a greater than proportional
increase
in AUCs in the dose range
-- Top dose studied of 500 mg is greater than 10 times the
predicted
effective doses from animal models of 15-30 mg/day
- Future Development Plans (subject to funding)
-- Preclinical studies in juvenile animals of sufficient duration
to span
Phase II study designs
-- Double-blind, placebo-controlled multiple ascending dose Phase
I(b)
study to evaluate safety and PK, and to determine the effective
dose
using GABA magnetic resonance spectroscopy
-- Phase II studies to evaluate the use of CPP-115 in the treatment
of
infantile spasms (West Syndrome) and cocaine addiction
- Advisors for CPP-115 Development to Treat Infantile Spasms
-- Dr. Jack Pellock and Dr. Donald Shields, who are world-renowned
experts
in the clinical development and treatment of infantile spasms; both
of
them attended Catalyst's pre-IND meeting and are medical
consultants to
Catalyst on the development of CPP-115 for the treatment of
infantile
spasms
"I am pleased that Catalyst has successfully completed the first
study necessary to move CPP-115 forward into a pilot trial in
babies diagnosed with infantile spasms, a serious disease that is
in need of safer, more efficacious treatments," stated Dr. Jack
Pellock, Professor and Chairman, Division of Child Neurology,
Virginia Commonwealth University. "I look forward to working with
Catalyst to advance CPP-115 expeditiously into a Phase II trial to
determine its safety and efficacy in such patients."
About CPP-115
CPP-115 is a novel GABA aminotransferase inhibitor and
vigabatrin analogue that, based on preclinical studies to-date, is
greater than
100 times more potent than vigabatrin and may have reduced side
effects (e.g., visual field defects, or VFDs and sedation) from
those associated with vigabatrin. Catalyst licensed CPP-115 from
Northwestern University where it was invented by Dr. Richard B.
Silverman, the John Evans Professor of Chemistry, and a team of
scientists. Dr. Silverman holds more than 40 patents and is the
inventor of Pfizer's drug, pregabalin (Lyrica(R)). CPP-115's
development progress was recently presented at the 2012 Epilepsy
Pipeline Update Conference in San Francisco, CA. CPP-115 has been
granted Orphan Drug Designation for the treatment of infantile
spasms by the FDA, and has been granted Orphan Medicinal Product
Designation for the treatment of West Syndrome by the European
Commission. It has also been granted Fast Track status by the FDA
for the treatment of cocaine addiction.
About West Syndrome / Infantile Spasms
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 development-stage
specialty pharmaceutical company focused on the development and
commercialization of prescription drugs targeting diseases and
disorders of the central nervous system, including addiction and
epilepsy. Catalyst has two products in development, CPP-109 and
CPP-115, and is currently evaluating its lead product and
first-in-class GABA aminotransferase inhibitor candidate, CPP-109,
for the treatment of cocaine addiction.
CPP-109 has been granted "Fast Track" status by the FDA for the
treatment of cocaine addiction. Catalyst also plans to evaluate
CPP-109 for the treatment of other addictions. Catalyst believes
that it controls all current intellectual property for drugs that
have a mechanism of action related to the inhibition of GABA
aminotransferase.
For more information about Catalyst, go to
www.catalystpharma.com.
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 be determined to
be effective for the treatment of addiction, infantile spasms or
other CNS indications, whether CPP-115 will have reduced side
effects compared to CPP-109, whether the funding required for
future studies of CPP-115 will be obtained, whether
CPP-115 will ever be approved for commercialization, and those
other factors described in the Company's filings with the U.S.
Securities and Exchange Commission (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.
CONTACT: Patrick J. McEnany
Catalyst Pharmaceutical Partners
Chief Executive Officer
(305) 529-2522
pmcenany@catalystpharma.com
Melody Carey
Rx Communications Group
Co-President
(917) 322-2571
mcarey@rxir.com
Posted: May 2012
