Pneumoreductive Surfactant Therapy for Emphysema

描述（由申请人提供）：Aeris Therapeutics正在开发一种用于晚期肺气肿患者的新型药物治疗，称为肺减容表面活性剂（PRS）。PRS是通过安全地改变天然肺表面活性剂的表面张力特性来增加肺回缩的吸入药物。与现有的慢性阻塞性肺疾病药物疗法不同，PRS是唯一专门设计用于通过改善肺回缩和减少过度充气来治疗肺气肿的药物疗法。在I期SBIR资助期间（2004年9月至2005年3月），Aeris确定了两种PRS配方，可在体外有利地改变肺表面活性物质功能，并在体内肺气肿模型中有效增加肺回缩。这些制剂中最好的已被选择用于产品开发。剂量-反应和剂量递送研究表明，使用常规雾化器系统可以有效地将PRS递送至肺部。重复给药研究证实了PRS给药的安全性，长达两周。已确定生产PRS的原材料的GMP来源，并制定了生产方案、临床计划和监管策略，以推进本产品的临床试验。Aeris正在请求第二阶段SBIR支持，以推进这一令人兴奋的新治疗产品的开发。

DESCRIPTION (provided by applicant): Aeris therapeutics is developing a novel medical treatment for patients with advanced emphysema known as Pneumoreductive Surfactant (PRS). PRSs are inhaled drugs that increase lung recoil by safely altering the surface tension properties of native lung surfactant. Unlike existing medical therapies for chronic obstructive pulmonary disease, PRS is the only medical therapy designed specifically to treat emphysema by improving lung recoil and decreasing hyperinflation. During Phase I SBIR funding (9/2004 to 3/2005), Aeris identified two PRS formulations that favorably alter lung surfactant function in vitro, and effectively increase lung recoil in emphysema models in vivo. The best of these preparations has been selected for product development. Dose-response and dose delivery studies demonstrate that PRSs can be effectively delivered to the lung using conventional nebulizer systems. Repeat dosing studies confirm the safety of PRS administration, out to two weeks. GMP sources of raw materials for manufacturing PRS have been identified, and a manufacturing protocol, clinical plan, and regulatory strategy have been developed for advancing this product into clinical testing. Aeris is requesting Phase II SBIR support to advance the development of this exciting new therapeutic product.