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0129 ALS pipeline
BioCentury & BioRender

Product Development

ALS looks up as clinical pipeline explodes

Three late-stage readouts this year and multiple mechanisms in the clinic could lift the lid on ALS treatments

With half a dozen programs in late-stage development and multiple mechanisms in the clinic, the lid on ALS treatments might soon lift.

Jan 30, 2021 | 4:08 AM GMT

Propelled by new insights into disease biology, the clinical pipeline for ALS has grown to at least 43 programs attacking the disease from a dozen different directions. With six of those programs in Phase III testing, the largely empty arsenal for the fatal neurodegenerative disease could soon start to fill. 

Undeterred by the long string of failures in Alzheimer’s disease, drug developers and investors are sustaining their rise in interest in neurodegenerative diseases, fueled in part by inroads made into disease pathology via human genetic studies.

Within the field, the large clinical pipeline for amyotrophic lateral sclerosis stands out among rare, age-related neurodegenerative diseases.

The number of industry programs for some of the most well-studied among them, plotted below, indicates that ALS has about two and a half times the activity of Huntington disease and outstrips other indications more than fivefold.


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