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

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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.