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Abstract

Grant Number: 5R42HD037290-03
PI Name: SIMON, NEAL G.
PI Email: ngs0@lehigh.edu
PI Title: CHAIR
Project Title: Treating Self-Abuse in Autism and Mental Retardation

Abstract: Serenix Pharmaceuticals LLC (SPL) will develop drugs to treat impulsivity, violence and self-injurious behavior in patients with autism, Tourette's syndrome, and mental retardation. These drugs, termed serenics, stop violent behavior without disrupting normal behaviors. No other products are available for these indications. SPL required a series of vasopressin receptor antagonists from Eli Lilly Company that block the vasopression receptor subtype involved in aggression. With Phase I STTR support, SPL chemically modified the original lead series, producing several candidate molecules that exhibit high binding affinity and block biological effects at the target human receptor. The overall objective of Phase II research is to file Investigational New Drug (IND) applications are two promising candidates. To reach this objective, SPL will test two candidates for i) oral absorption and bioavailability, ii) acute and chronic toxicity, and iii) prepare these molecules for GMP grade bulk synthesis and formulation. The completion of the proposed research will provide the data required to meet the chemical and biological criteria established by the FDA for IND applications. PROPOSED COMMERCIAL APPLICATIONS: SPL is focusing on the development of drugs to help in the control of impulsivity, violence, and self-injurious behavior for the management of autism, Tourette'S syndrome, and mental retardation. It is estimated that more than 500,000 patients in the United States alone would have a need for these drugs, with a worldwide market of 1.5 million. These drugs also may be used in the control of violence associated with mania, conduct disorder in a subset of children with ADHD, and Alzheimer's disease. There are no other products on the market for these indications.

Thesaurus Terms:
Tourette's syndrome, autism, chemical structure function, drug design /synthesis /production, mental disorder chemotherapy, mental retardation, self destructive behavior, vasopressin
aggression, drug screening /evaluation, gastrointestinal drug absorption, impulsive behavior, pharmacokinetics, violence
cell line, guinea pig, laboratory mouse, laboratory rat

Institution: SERENIX PHARMACEUTICALS, INC.
111 RESEARCH DR, B217
BETHLEHEM, PA 18015
Fiscal Year: 2003
Department:
Project Start: 01-JUL-2002
Project End: 30-JUN-2004
ICD: NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT
IRG: ZRG1


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