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，称为肺还原表面活性剂（PRS）。PRSs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后坐力的吸入药物。与现有慢性阻塞性肺疾病的药物治疗方法不同，PRS是唯一专门通过改善肺反冲和减少恶性通货膨胀来治疗肺气肿的药物治疗方法。在第一阶段SBIR资助期间（2004年9月至2005年3月），Aeris确定了两种PRS配方，它们在体外有利于改变肺表面活性物质的功能，并有效地增加肺气肿模型的肺后坐力。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，使用传统的雾化器系统，PRSs可以有效地递送到肺部。重复给药研究证实了两周内使用PRS的安全性。已确定生产PRS原料的GMP来源，并制定了生产方案、临床计划和监管策略，以推进该产品进入临床试验。Aeris正在申请II期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.