Lumos Pharma is a clinical-stage biopharmaceutical company committed to identifying, developing and commercializing life-changing therapies for patients and families living with rare diseases.  The Company’s pipeline features development programs affecting both pediatric and adult rare diseases populations: LUM-201 in diseases associated with growth hormone deficiencies and LUM- 001 for Creatine Transporter Deficiency (CTD/SLC6A8).


  • Also known as ibutamoren mesylate, LUM-201 is a potent, orally administered small molecule that promotes the secretion (secretagogue) of Growth Hormone (GH) from the pituitary gland.
  • LUM-201 has demonstrated to increase the release of growth hormone and its downstream effector molecules in humans in a pulsatile manner which mimics natural secretion of GH.
  • It has been studied in more than 1000 patients, both adult and pediatric, and was generally well-tolerated.
  • Clinical development of this orally administered product to treat children with pediatric growth hormone deficiency (PGHD) is scheduled for 2019.

Learn more about PGHD


  • LUM-001 is a repurposed small molecule that is a biochemical analog of creatine and has shown promising therapeutic potential for the treatment of Creatine Transporter Deficiency in preclinical studies.
  • It is a small molecule with the chemical characteristics necessary to cross membranes.
  • It has been studied in a previously filed Investigational New Drug (IND) application for use in a solid tumor indication, however, Lumos Pharma is required to repeat some preclinical studies with an oral formulation, as the previous IND studied LUM-001 in an intravenous formulation.
  • Currently, Lumos is sponsoring an observational study of males with Creatine Transporter Deficiency males, called the Vigilan Study. https://clinicaltrials.gov/ct2/show/NCT0293168

 Learn more about CTD