Cambria Pharmaceuticals Inc.
This article was originally published in Start Up
Executive Summary
Although only a small percentage of ALS cases seem tied to the mutant form of SOD1, the deep knowledge about it provides Cambria Pharmaceuticals with a platform for assaying compounds it intends to develop against ALS and other neurodegenerative diseases.
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A preview of the emerging health care companies profiled in the current, all-profiles issue of Start-Up. This month's profile groups are "Glioblastoma Start-Ups Get Specific," "Glaucoma Start-Ups Reach Critical Mass," and "ALS on the Cusp." Plus these Start-Ups Across Health Care: EarlySense, NeoChord, and Pantec Biosolutions.
Knopp Neurosciences Inc.
Knopp Neurosciences is developing the right-oriented enantiomer of Parkinson's and restless leg syndrome drug pramipexole (Mirapex) for amyotrophic lateral sclerosis. The drug has received orphan designation from the FDA, and is now in Phase IIa studies in 80 ALS patients.
ALS on the Cusp
Amyotrophic lateral sclerosis, the devastating neurodegenerative disease, has largely defied R&D efforts, but emerging knowledge, much of it through nonprofit research organizations, raises hopes for better results and treatments with blockbuster potential. In this issue we profile two emerging drug developers hoping to make an impact: Cambria Pharmaceuticals and Knopp Neurosciences.