Through its newsletter PharmaGPS Bytes, BioXcel, headquartered in Branford, regularly publishes analyses of pipelines of neuro-degenerative diseases. Their current analysis concerns ALS.

ALS (amyotrophic lateral sclerosis) is a complex neurodegenerative disease characterized by the progressive degeneration of motor neuron circuitry, which includes neurons and cells that reside in the cortex, spinal cord, and the brainstem.

According to the BioXcel analysis, clinical success has been dismal in ALS owing to critical challenges such as poor understanding of ALS etiology, lack of translation of efficacy from preclinical to human studies, and lack of candidate drugs that would aid in disease modification.