FDA grants fast-track designation to novel ALS therapeutic

Key takeaways:

AMX0114 was developed to target calpain-2, a driver of axonal neurodegeneration.

A pending clinical trial will evaluate safety and efficacy of the novel therapeutic.

The FDA has granted fast-track designation to an investigational antisense oligonucleotide that targets a specific driver of axonal degeneration in those with ALS, according to the manufacturer.

In a press release, Amylyx Pharmaceuticals said that AMX0114 was developed to address calpain-2, a calcium-activated protease, which contributes to disease progression in ALS.

The FDA granted fast track designation to an antisense oligonucleotide that targets a driver of axonal degeneration in those with ALS. I mage : Adobe Stock

“The FDA’s fast-track designation is a meaningful acknowledgment of both the urge