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

用于治疗肢端肥大症的一流抗体疗法

基本信息

批准号：
10082375
负责人：
James Zanghi
金额：
$ 28.41万
依托单位：
ELIXERA, INC.
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-08-01 至 2022-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10082375
关键词：
Acromegaly Address Adult Affect Affinity Alloys Antibodies Antibody Therapy Benign Binding Biological Assay Cardiovascular Diseases Cause of Death Cell Line Cells Cessation of life Clinical Clinical Trials Cloning Consensus Data Data Analyses Diagnosis Disease Dose Endocrine System Diseases Endocrine system Epitopes Genetic Engineering Goals Grant Growth Growth Hormone Receptor Head Health Hormone secretion Hormones Human Hybridomas Impairment In Vitro Insulin-Like Growth Factor I Lead Left Libraries Life Expectancy Medical Metabolic Diseases Metabolic dysfunction Modeling Monoclonal Antibodies Non-Insulin-Dependent Diabetes Mellitus Operative Surgical Procedures Patients Phase Pituitary Gland Pituitary Neoplasms Population Group Production Pump Research Respiration Disorders Rodent Model Serum Somatotrophin increased Somatotropin Specificity Technology Testing Therapeutic Therapeutic antibodies Toxicity Tests Transgenic Mice Transgenic Organisms Treatment Efficacy Validation base cardiovascular risk factor cell bank comorbidity drug candidate effective therapy growth hormone deficiency human monoclonal antibodies improved in vitro activity in vivo in vivo Model innovation lead candidate lead optimization lead series lipid metabolism manufacturability mortality neonatal Fc receptor neutralizing antibody neutralizing monoclonal antibodies novel therapeutics patient population pegvisomant pre-clinical research clinical testing screening side effect skeletal soft tissue somatostatin analog therapeutic candidate tumor

项目摘要

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.

项目摘要：肢端肥大是由良性垂体肿瘤引起的内分泌疾病在美国约有25,000名成年人。未经治疗，平均预期寿命距离诊断为10年。它以过剩的骨骼生长，软组织扩张和永久毁容为特征通过由肿瘤分泌的超过生长马的代谢功能障碍。该疾病与死亡率增加了2至3倍，严重的健康并发症和合并症。这些包括心血管疾病，大约50％代表死亡的第二大原因，以及诸如2型糖尿病等代谢疾病和受损脂质代谢有助于更大的心血管风险和死亡率。广泛的共识是严格的骑马控制可以实现有效的治疗，这是对合并症的有益影响证明的和死亡率。当手术要么无法完全切除肿瘤或无法选择时，药物治疗是长期控制的必需。但是，现有患者仅在一半的患者中获得控制疗法。明显的未满足的医疗需求可以实现长期骑马控制的治疗始终如一地遍及肢端肥大的患者人群。 Elixera的目标是开发一种新颖的疗法作为首先满足这种未满足的医疗需求的肢端肥大的课堂治疗。第一阶段赠款的目的是确定并描述新产品线索。第2阶段赠款的目的是为治疗候选人做好准备使用FDA进行IND应用的制造，该应用将允许在肢端肥大症患者中进行临床试验测试美国。