ALS new drug research and development both suffered setbacks: Biogen/Ionis terminated the phase I trial of Amylyx's drug listing, which was questioned by FDA

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Amyotrophic lateral sclerosis (ALS), also known as ALS, motor neuron disease, Charcot disease, Lou Gehrig's disease, etc.

On March 28, Biogen and its partner Ionis Pharmaceuticals released the first-line results of the Phase I clinical trial of BIIB078

BIIB078 is an investigational antisense oligonucleotide drug for the treatment of C9orf72-related ALS

According to the results disclosed by the company, BIIB078 was well tolerated and its adverse events (AEs) were mostly mild to moderate in severity

The relevant person in charge of Ionis said that the scientific community believes that there are many mechanisms by which the C9orf72 gene causes disease, and in this test, it is believed that the C9orf72-related ALS disease mechanism is caused by the related toxicity of RNA and corresponding dipeptide repeat sequences

Meanwhile, Amylyx Pharmaceuticals is also holding back progress on ALS drugs

Amylyx stock fell sharply, down more than 40%, within half an hour of the FDA's announcement on the news

Still, despite concerns about the data, the FDA may still eventually consider approving the ALS drug due to its high demand

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