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Pipeline        AIM Biologicals         AEZS-150        AEZS-130 (Macimorelin)        Macimorelin Dx         Publications        

AEZS-130 (Macimorelin) 
Potential Therapeutic for Amyotrophic
Lateral Sclerosis (ALS, Lou Gehrig's disease)

  • Stimulates appetite and lowers metabolic rate to promote weight gain 
  • Regulates the release of the neuroprotective and anabolic hormones growth hormone (GH) and insulin-like growth factor-1 (IGF-1) 
  • Acts via the GHSR1a, which is expressed throughout the body, including the cerebral cortex, spinal cord and muscle (i.e. tissues directly impacted in ALS) 
  • Majority of motor neuron disease (MND) patients have a moderate to marked GH deficiency1 
  • Treatment with ghrelin or the ghrelin mimetic GHRP3 was shown to slow weight loss, improve muscle strength, and extend survival in the SOD1G93A mouse model of ALS2 

1: Steyn et al., Endocrinology. 2012 Aug;153(8):3735-46. Impairments to the GH-IGF-I Axis in hSOD1G93A Mice Give Insight into Possible Mechanisms of GH Dysregulation in Patients with Amyotrophic Lateral Sclerosis 
2: Ngo et al., J Neuroendocrinol. 2021 Jan 29;33(7):e12938. Ghrelin as a treatment for amyotrophic lateral sclerosis. 

Macimorelin is a Ghrelin Mimetic and Modulates Various Disease-Relevant Processes 

Macimorelin, a ghrelin receptor agonist, is a small molecule that stimulates the secretion of growth hormone from the pituitary gland. Through this stimulation, it is believed that macimorelin may slow the progression of certain neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease).

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