Amyotrophic lateral sclerosis (ALS) is a relatively rare, but invariably fatal form of motor neuron disease. The hallmark of the disorder is the progressive degeneration of motor neurons that control the muscle movement, resulting in muscular atrophy (weakness and deterioration of the muscle).
The multimodal activities of AX200 (stop of neuronal cell death, enhancement of neurogenesis and arteriogenesis etc.) make it a promising candidate to combat ALS.
The SYGNIS research team has demonstrated in pre-clinical trials that AX200 counteracts the neuronal cell death and thereby slowing down the progression of the disease. In an in vivo disease model, protection of the motor neurons led to a reduced degeneration of muscles whilst maintaining muscular strength for a longer period of time as well as leading to prolonged survival.
SYGNIS has established pre-clinical proof of concept for AX200 in ALS. Furthermore, the company has obtained Orphan Drug Designation for ALS by the European Commision. Now, SYGNIS is seeking partners to advance AX200 into clinical development in this devastating orphan indication.