Catabasis is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics based on its proprietary Safely Metabolized And Rationally Targeted, or SMART, linker drug discovery platform. Catabasis’ SMART linker drug discovery platform enables the Company to engineer product candidates that can simultaneously modulate multiple targets in a disease. The Company’s proprietary product candidates impact pathways that are central to diseases where efficacy may be optimized by a multiple target approach. The Company’s primary focus is on treatments for rare diseases. Catabasis is also developing other product candidates for the treatment of serious lipid disorders. The Company has applied its SMART linker drug discovery platform to build an internal pipeline of product candidates for rare diseases and plan to pursue partnerships to develop additional product candidates.
The Company’s lead product candidate, CAT-1004, an oral small molecule targeting the NF-kB (nuclear factor kappa-light-chain-enhancer of activated B cells) pathway, is being studied to treat boys for Duchenne muscular dystrophy (DMD). In January 2016, the Company reported top-line results from Part A of the Phase I/II MoveDMD trial. Part B data is expected by year-end 2016. If the results from the MoveDMD trial are positive, the Company plans to initiate a six-month Phase III pivotal trial in 2017. The FDA has granted orphan drug, fast track and rare pediatric disease designations to CAT-1004 for the treatment of DMD.