Lumos Pharma is a clinical-stage biopharmaceutical company focused on developing therapeutics for rare diseases, particularly Pediatric Growth Hormone Deficiency (PGHD). Their lead product, LUM-201, is an investigational small molecule that is administered orally once daily. LUM-201 functions by promoting the secretion of Growth Hormone (GH) from the pituitary gland, effectively mimicking the natural pulsatile pattern of GH release. This innovative approach aims to provide an alternative to the current treatment options, which primarily involve painful daily or weekly injections of recombinant human growth hormone. The company has conducted extensive studies involving over 1,300 patients, demonstrating that LUM-201 is generally well-tolerated, with increased appetite being the most frequently reported treatment-related adverse event.

In addition to its therapeutic development, Lumos Pharma employs a Predictive Enrichment Marker (PEM) strategy to identify children who are more likely to respond positively to LUM-201 treatment. This strategy utilizes clinical markers, including baseline IGF-1 levels and GH response to a single oral dose of LUM-201, to categorize patients as PEM positive or negative. By focusing on this predictive approach, Lumos Pharma aims to enhance the efficacy of its clinical trials and ensure that the investigational therapy is directed towards those who would benefit the most. Overall, Lumos Pharma's commitment to addressing the challenges of PGHD through innovative oral therapies and strategic patient selection underscores its mission to transform the lives of patients and families affected by rare diseases.

United States

Lumos Pharma is a clinical-stage biopharmaceutical company whose mission is to develop new therapies for people with rare diseases, prioritizing its focus where the medical need is high, and the pathophysiology is clear.

Endocrine System Diseases

Genetic Diseases

Rare diseases

Lumos Pharma

Pediatric growth hormone deficiency

LUM-201, also known as ibutamoren, is an orally-administered small molecule that promotes the secretion of GH from the pituitary gland. LUM-201 has been observed to increase the amplitude of endogenous pulsatile GH secretion, which mimics the natural pattern of GH secretion.

Lumos Pharma

Company Details