Feb 9 2010
Psyadon Pharmaceuticals, Inc. today announced that the United States
Food and Drug Administration (FDA) has accepted its Investigational New
Drug Application (IND) to study ecopipam in patients with Lesch-Nyhan
Disease (LND). The first center at which the drug will be evaluated is
Emory University under the direction of Dr. Hyder A. Jinnah, MD, PhD.
The study is designed to evaluate the safety and tolerability of
different doses of ecopipam in adults (group 1), adolescents (group 2)
and children (group 3) with LND.
“the potential to significantly ameliorate the behavioral
symptoms seen in these patients.”
Dr. Jinnah is a world-renowned expert in the molecular biology and
clinical pathology of LND having published numerous peer-reviewed
articles. He believes that dopamine D1-receptor antagonists like
ecopipam have “the potential to significantly ameliorate the behavioral
symptoms seen in these patients.”
“We are very excited to be starting our clinical trial with ecopipam in
patients with Lesch-Nyhan Disease. There are currently no treatments
available for these patients, and we are hopeful that this drug will
provide some benefit,” said Richard E. Chipkin, PhD, President and CEO
of Psyadon Pharmaceuticals.
Ecopipam is a selective antagonist of the D1-family of dopamine
receptors. Psyadon Pharmaceuticals licensed worldwide rights to ecopipam
from Schering-Plough Corporation, an affiliate of Merck & Co., Inc. in
2008. Although an optimal clinical indication has not yet been
identified, ecopipam has already been studied in over 2,000 patients.
LND is a rare genetic disorder that is predominantly seen in males. The
patients have a well-studied mutation of a specific enzyme which causes
a characteristic metabolic, motor, and behavioral syndrome. The onset is
disease is usually between ages 1-3 years, and patients suffer life-long
debilitation. It has been estimated that in the United States there are
between 500 and 1000 patients.
ORPHAN DRUG DESIGNATION
Psyadon Pharmaceuticals has received orphan drug designation for the use
of ecopipam in LND in both the United States and the European Union.
This provides market exclusivity and other benefits in these territories.
Source:
Psyadon Pharmaceuticals, Inc.