Development of a long-acting growth hormone antagonist to address the unmet need for treatment in acromegaly

肢端肥大症是一种生长激素分泌过多的疾病，如果不治疗，患者会遭受毁容、高血压、糖尿病、关节炎和心力衰竭。肢端肥大症患者的死亡率是普通人群的两倍。一线治疗是手术，但在50%的患者中，肿瘤不能手术或不能完全切除，这些患者需要药物治疗。目前的药物治疗并不令人满意，因为它要么只能控制一部分患者的疾病，要么涉及每天注射一种不符合成本效益的药物。我们开发了一种新技术，Profuse，用于制造一种长效药物，可以阻断生长激素的作用，预计将具有成本效益，并在>95%的患者中控制疾病。DPFS申请是一项为期3年的资助，用于提供准备用于临床概念验证的Profuse药物。这是将Profuse药物推向市场所需的第一阶段。接下来的阶段是在患者中进行临床试验（1/2a期概念验证试验），然后是上市许可的2期和3期试验。

Acromegaly is a disease of excess growth hormone secretion and if untreated patients suffer from disfigurement, hypertension, diabetes, arthritis and heart failure. Acromegalic patients have twice the mortality of the general population. First line therapy is surgery but in 50% of patients the tumours are inoperable or cannot be fully removed and these patients need drug therapy. Current drug therapy is not satisfactory as it either only controls the disease in a proportion of patients or involves daily injections with a drug that is not cost effective. We have developed a new technology, Profuse, for making a long-acting drug that blocks growth hormone actions and is expected to be cost effective and control the disease in >95% of patients. The DPFS application is for a 3 year grant to deliver a Profuse drug ready for clinical proof-of-concept. It is the first phase required to bring the Profuse drug to market. The next stages are a clinical trial (Phase 1/2a proof of concept trial) in patients, followed by Phase 2 & 3 trials for market authorisation.