A First-in-Class Antibody Therapeutic for Treatment of Acromegaly

项目摘要：肢端肥大症是一种由良性脑垂体瘤引起的内分泌紊乱。 美国大约有25,000名成年人。如果不治疗，患者从确诊起的平均预期寿命为10年。它 以骨骼过度生长、软组织肥大和永久性毁容为特征，伴随 由于肿瘤分泌过量的生长激素而导致的代谢功能障碍。这种疾病与 死亡率增加2到3倍，严重的健康并发症和合并症。这些措施包括 心血管疾病，约占肢端肥大症死亡的50%，呼吸系统疾病， 它们是第二大致死原因，以及代谢性疾病，如2型糖尿病和受损 脂代谢导致更大的心血管风险和死亡率。广泛的共识是， 有效的治疗是通过严格的激素控制来实现的，这体现在对合并症的有益影响上。 和死亡率。当手术不能完全切除肿瘤或不是一种选择时，药物治疗是 长期控制荷尔蒙所需的。然而，只有一半的患者通过现有的 治疗。显然，医学上需要一种实现长期激素控制的疗法，这种需求尚未得到满足。 在肢端肥大症患者群体中保持一致。Elixera的目标是开发一种新的疗法，作为第一个- 对肢端肥大症的一流治疗，满足了这一未得到满足的医疗需求。第一阶段拨款的目的是确定 并描述新的产品线索。第二阶段拨款的目的是让一位治疗候选人做好准备 为FDA生产的IND应用程序，将允许对肢端肥大症患者进行临床试验测试 美国。

Project Summary: Acromegaly is an endocrine disorder caused by a benign pituitary tumor that affects approximately 25,000 adults in the US. Left untreated, the average life expectancy is 10 years from diagnosis. It is characterized by excess skeletal growth, soft-tissue enlargement, and permanent disfigurement, accompanied by metabolic dysfunction resulting from excess growth hormone secreted by the tumor. The disease is associated with 2- to 3-fold increased mortality and serious health complications and comorbidities. These include cardiovascular disease, which is responsible for approximately 50% of acromegalic deaths, respiratory disorders, which represent the second leading cause of death, and metabolic diseases such as type 2 diabetes and impaired lipid metabolism that contribute to greater cardiovascular risk and mortality. The broad consensus is that effective treatment is achieved by strict hormone control, demonstrated by the beneficial effects on comorbidities and mortality. When surgery either fails to completely remove the tumor or is not an option, medical therapy is required for long-term hormone control. However, control is achieved in only half of patients by the existing therapies. There is a clear unmet medical need for a therapy that achieves long-term hormone control consistently across the acromegaly patient population. Elixera’s goal is to develop a novel therapeutic as a first- in-class treatment of acromegaly that fulfills this unmet medical need. The aim of the Phase 1 grant to identify and characterize new product leads. The aim of the Phase 2 grant is to have a therapeutic candidate ready for manufacture for an IND application with the FDA that will permit clinical trial testing in acromegaly patients in the US.