The Sygnis research team has demonstrated in pre-clinical trials that AX200 counteracts the neuronal cell death and thereby decelerates the development of the disease. In the laboratory, protection of the motor neurons led to a reduced degeneration of muscles whilst maintaining muscular strength for a longer period of time.
Alfred Bach, CEO of Sygnis Pharma, said: “We are delighted to have obtained orphan drug status for AX200, our lead development compound, in amyotrophic lateral sclerosis. We intend to begin clinical trials within the next 12 months.”