GERMANTOWN, Md. -- (BUSINESS WIRE) -- Psyadon Pharmaceuticals, Inc. announced today that the first patients have been enrolled in its Phase 3 study of ecopipam for the treatment of self-injurious behaviors in patients with Lesch-Nyhan Disease.
“We consider this a significant milestone in the development of ecopipam and the history of the company,” said Dr. Richard Chipkin, President and CEO of Psyadon. “The self-injurious behaviors seen in these patients are devastating and in some cases sufficiently severe to require full-time restraint in specially designed wheelchairs.”
The study’s Principal Investigator, Dr. H. Jinnah (Emory University) said “This international study is the largest and most rigorously controlled therapeutic intervention ever attempted in patients with Lesch-Nyhan Disease. If positive, it will provide relief for hundreds of sufferers who have almost no good options.”
ABOUT THE STUDY
Protocol PSY102 is a multicenter, double-blind, randomized three-way crossover study to assess the efficacy and safety of ecopipam in subjects with Lesch-Nyhan Disease having moderate-to-severe self-injurious behaviors. Eligible subjects are instructed to take ecopipam (50 or 100 mg/day depending on body weight) or placebo before bedtime over an 18-week treatment period (comprised of three six-week treatment periods with either ecopipam or placebo). Self-injurious behavior frequency and severity will be rated using the Behavior Problems Inventory (BPI), a well-validated, standard rating scale typically used when testing new pharmaceuticals.
The study will recruit patients from all over the world. The following sites are already open and enrolling patients: Emory University in Atlanta, US (Dr. Jinnah), University of California at San Diego, US (Dr. Nyhan), and La Paz Hospital in Madrid, Spain (Dr. Torres). A center at Radboud University/Nijmegen Medical Center in the Netherlands (Dr. Visser) is expected to open for patient enrollment shortly. Additional study sites are being evaluated in France, Great Britain, and Argentina.
Site selection, patient recruitment, monitoring and data management support for the ecopipam study is being provided by SGS Clinical Research Services, Gaithersburg, Maryland, USA.
Ecopipam is a first-in-class molecule that selectively blocks the actions of the neurotransmitter dopamine at its receptor. Dopamine receptors can be broadly classified into two families based on their structures (“D1 receptors” and “D2 receptors”). Ecopipam blocks dopamine only at D1 receptors. In contrast, all currently marketed dopamine antagonists and agonists act at D2 receptors.
Based on studies in a large number of patients and normal volunteers, ecopipam is generally well-tolerated. To evaluate whether patients with Lesch-Nyhan Disease had a peculiar sensitivity to the drug, the safety and tolerability of the doses used in this Phase 3 study were first tested in an open label study in a small group of these patients. The results showed that it was well-tolerated and that its side effect profile was similar to that seen in other patient populations.
ABOUT PSYADON PHARMACEUTICALS
Psyadon Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the discovery and development of drugs for serious neurological and psychiatric disorders. The company focuses its efforts on drugs for orphan indications because of the high medical need for effective treatments in these areas. Psyadon has received orphan designation from both the US and the EU regulatory authorities for the use of ecopipam to treat Lesch-Nyhan Disease.