Revolutionizing Growth Hormone Deficiency Treatment

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madman

Super Moderator

LUM 201 (ibutamoren) is a focus of recent research for treating idiopathic pediatric growth hormone deficiency. This rare endocrine disorder, characterized by insufficient growth hormone secretion from the anterior pituitary gland, can manifest at birth, as a result of a medical condition or brain injury, or be idiopathic. It leads to growth retardation, short stature, and various associated health issues such as metabolic abnormalities, impaired cardiovascular function, fatigue, delayed puberty, osteoporosis, and reduced muscle strength. The research aims to explore the efficacy of LUM 201 in addressing these challenges.




Key points:
  • LUM-201 (ibutamoren) is an investigational small molecule that stimulates Growth Hormone (GH) secretion.
  • Studies presented at ENDO 2023 demonstrated the effectiveness of LUM-201 in treating idiopathic pediatric growth hormone deficiency.
  • Two Phase 2 trials analyzed the annualized height velocity (AHV) for two different doses (1.6 mg/kg/day and 3.2 mg/kg/day).
  • AHV results showed 8.1±1.9 cm/year for the lower dose and 8.0±1.5 cm/year for the higher dose initially, and 7.8±1.7 cm/year and 7.4±1.2 cm/year after 12 months of treatment.
  • LUM-201 was well tolerated with no safety concerns.
  • A predictive enrichment marker (PEM) identified 1.6 mg/kg/day as the optimal dose for efficacy and durability.
 
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