Amylyx Pharmaceuticals Gains FDA Fast Track for ALS Drug AMX0114

Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) is one of the best biotech stocks to invest in now. On June 3, Amylyx Pharmaceuticals announced that the US FDA granted Fast Track designation to AMX0114. AMX0114 is an investigational antisense oligonucleotide/ASO that is specifically designed to target calpain-2. This protein is believed to be a significant contributor to axonal degeneration, which is a key driver in the progression of amyotrophic lateral sclerosis/ALS.

ALS is a rapidly progressive and fatal neurodegenerative disorder with limited therapeutic options. The Fast Track designation is a crucial step for Amylyx in its mission to develop treatments for ALS. The FDA’s decision acknowledges both the severity of ALS and the encouraging preclinical data supporting AMX0114’s potential.

Amylyx Pharmaceuticals Gains FDA Fast Track for ALS Drug AMX0114

A medical scientist in a lab coat gazing at a microscopic view of a drug in development.

Calpain-2 is a calcium-activated protease. Research has indicated that overactive calpain-2 activity might accelerate disease progression in ALS and other neurodegenerative conditions by causing the degeneration of axons, which are vital for transmitting signals between neurons and muscles. Preclinical studies of AMX0114 have demonstrated improved neuronal survival and reduced levels of extracellular neurofilament light (NfL), which is a biomarker for neuronal damage across various disease models. AMX0114 was also well-tolerated in preclinical safety studies.

Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) is a clinical-stage pharmaceutical company that discovers and develops treatments for neurodegenerative diseases and endocrine conditions in the US.

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Disclosure: None. This article is originally published at Insider Monkey.