Catabasis Pharmaceuticals, founded in 2008 and based in

Cambridge, Massachusetts Cambridge ( ) is a city in Middlesex County, Massachusetts, United States. As part of the Boston metropolitan area, the cities population of the 2020 U.S. census was 118,403, making it the fourth most populous city in the state, behind Boston ...

, is a clinical-stage

biopharmaceutical A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. Different from totally synthesized pharmaceuticals, t ...

company. Their lead program is edasalonexent, an NF-kB inhibitor in development for the treatment of

Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy that primarily affects boys. Muscle weakness usually begins around the age of four, and worsens quickly. Muscle loss typically occurs first in the thighs and pelvis follow ...

. In Catabasis’s Phase 2 MoveDMD clinical trial and open-label extension, edasalonexent was shown to preserve muscle function as assessed by the North Star Ambulatory Assessment and Timed Function Tests compared with the rate of change during an off-treatment control period. It also showed a significant decrease in CRP, muscle enzymes including CK, and decrease in heart rate to age-normative values.

References

{{Reflist Biopharmaceutical companies Companies listed on the Nasdaq Pharmaceutical companies established in 2008 Health care companies based in Massachusetts Companies based in Cambridge, Massachusetts