Multiple biological pathways go awry to cause disease. Cambridge startup Catabasis is developing a platform to simultaneously modulate multiple targets in a disease pathway, with a focus on diseases like Duchenne muscular dystrophy and ALS
The startup’s in the midst of a $20.4 million fundraise, according to a recent SEC filing. So far, it’s raised about $12.4 million in this round.
The company says its platform can simultaneously modulate a number of biological targets within several related disease pathways. These are small chemicals designed to join two separate bioactives into a single conjugate molecule, Catabasis says. The platform’s called “Safely Metabolized And Rationally Targeted,” shortened to SMART.
The MIT Technology Review wrote up a nice explainer of Catabasis’ technology two years ago:
Catabasis has found a way around the challenge of identifying a single active molecule that can hit multiple pathways effectively: using a chemical linker to bring together two active molecules. The synergistic effect of the linked molecules may arise from the fact that the two compounds both “get to the right place at the right time,” says [cofounder and CSO Mike] Jirousek.
Catabasis has a number of products in its pipeline. Furthest along is its Duchenne muscular dystrophy treatment. It’s received FDA orphan status, and is projected to begin Phase 1/2 clinical trials this year. A Phase 1 trial’s already found that the drug’s well-tolerated, and can reduce inflammation and increase muscle regeneration.
It’s completed a Phase 2a trial for hypertriglyceridemias, projects a Phase 2a trial later this year for hypercholesterolemia and is conducting preclinical work for ALS and Freidreich’s ataxia.
