Michael Thorner, MB, Vice President of Endocrine Science at Lumos Pharma discusses the latest research on LUM 201 (ibutamoren) to treat idiopathic pediatric growth hormone deficiency.
Growth hormone deficiency is a rare endocrine disorder. Insufficient levels of growth hormone secreted from the anterior pituitary gland characterize this disease. The condition may be present at birth congenital, acquired, or idiopathic.
Typically, the main symptom of prolonged growth hormone deficiency is growth retardation or deceleration, as well as short stature. Additionally, the disease is associated with other symptoms such as:
- metabolic abnormalities
- impaired cardiovascular function
- fatigue
- delayed or incomplete puberty
- osteoporosis
- reduced muscle strength
LUM-201, also known as ibutamoren, is an investigational, once-daily, orally administered small molecule. It is designed to promote the secretion of Growth Hormone (GH) from the pituitary gland.
At the ENDO 2023 meeting, researchers presented studies showing the efficacy of LUM-201 in treating patients with idiopathic pediatric growth hormone deficiency.
Study
As noted by Dr. Thorner, Michael Tansey, MD, and colleagues combined data from two Phase 2 trials studying LUM-201 at two different doses (1.6 mg/kg/day or 3.2 mg/kg/day) and analyzed the calculated annualized height velocity (AHV). They observed the calculated AHV was 8.1±1.9 cm/year in the 1.6 mg/kg/day group and 8.0±1.5 cm/year in the 3.2 mg/kg/day group (N=15 in both groups). After 12 months of treatment, the calculated AHV was 7.8±1.7 cm/year in the 1.6 mg/kg/day group and 7.4 ±1.2 cm/year in the 3.2 mg/kg/day group (N=6 in both groups). Patients tolerated LUM-201 well and researchers did not identify any safety concerns.
In the second abstract, Fernando Cassorla, MD et al looked at a predictive enrichment marker (PEM) to identify patients likely to respond to LUM-201 at various doses and it was determined that 1.6 mg/kg/day is the optimum dose for efficacy and durability.
To stay up-to-date on the latest developments in endocrinology, visit checkrare.com/diseases/endocrine-disorders/
