Treating Self-Abuse in Autism and Mental Retardation

Serenix制药有限责任公司（SPL）将开发治疗自闭症、图雷特综合症和智力迟钝患者的冲动、暴力和自残行为的药物。这些药物被称为“宁静剂”，在不干扰正常行为的情况下阻止暴力行为。没有其他产品可用于这些适应症。SPL需要来自礼来公司的一系列抗利尿激素受体拮抗剂来阻断参与攻击的抗利尿激素受体亚型。在I期STTR的支持下，SPL对原始先导系列进行了化学修饰，产生了几种具有高结合亲和力和阻断目标人类受体生物效应的候选分子。II期研究的总体目标是为两个有希望的候选药物提交临床新药（IND）申请。为了实现这一目标，SPL将测试两种候选分子：i)口服吸收和生物利用度，ii)急性和慢性毒性，以及iii)制备这些分子用于GMP级批量合成和配方。拟议研究的完成将提供满足FDA为IND申请制定的化学和生物学标准所需的数据。建议的商业应用：SPL专注于开发药物，以帮助控制冲动、暴力和自我伤害行为，用于治疗自闭症、图雷特综合征和智力迟钝。据估计，仅在美国就有50多万患者需要这些药物，而全球市场为150万。这些药物也可用于控制与躁狂症相关的暴力，ADHD儿童子集的行为障碍和阿尔茨海默病。目前市场上还没有针对这些适应症的其他产品。

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.