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

NIH RePORTER · NIH · R43 · $284,100 · view on reporter.nih.gov ↗

Abstract

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.

Key facts

NIH application ID
10082375
Project number
1R43DK126579-01
Recipient
ELIXERA, INC.
Principal Investigator
James Zanghi
Activity code
R43
Funding institute
NIH
Fiscal year
2020
Award amount
$284,100
Award type
1
Project period
2020-08-01 → 2022-07-31