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AEZS-130 (Macimorelin) 
Ghrelin Agonist for the Treatment of Amyotrophic Lateral Sclerosis
(ALS; Lou Gehrig's disease)


MOA of Macimorelin in ALS

  • Macimorelin potently stimulates GH release and leads to increased body weight gain after repeated dosing in healthy mice

  • Positive effects on body weight and on the GH/IGF-1 axes observed in mouse ALS models

  • Neuroprotective activity demonstrated in lower motorneuron cultures derived from induced pluripotent stem cells from ALS patients

  • Enhanced innervation of motorneurons in mouse ALS models

Treatment of Amyotrophic Lateral Sclerosis (ALS; Lou Gehrig’s Disease)

  • Fatal progressive and degenerative neurological disease affects the motor neurons that control voluntary muscle movement

  • Over time, motor neurons degenerate and die, leading to muscle weakness, loss of muscle control and eventually paralysis

  • Affects ~31,000 people in the US

  • Significant unmet medical need with no treatment for halting progression

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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