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Rare Disease Therapy Company Raises $60 Million in IPO

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26 June 2015. Catabasis Pharmaceuticals Inc., a biotechnology company creating treatments for rare diseases and cholesterol-related disorders, is raising $60 million in its initial public stock offering, trading on the Nasdaq exchange under the symbol CATB. The Cambridge, Massachusetts enterprise yesterday priced its 5 million shares at $12.00, and as of 4:00 pm today is trading at $13.41.

The company’s technology is based on research by Steven Shoelson at Harvard Medical School and Joslin Diabetes Center. Shoelson, a co-founder of the company in 2008, conducts research on the key role of inflammation linking obesity to insulin resistance, type 2 diabetes, and cardiovascular disease. He serves as a scientific adviser to Catabasis.

The Catabasis platform is called Safely Metabolized And Rationally Targeted or SMART linker technology that targets multiple points along particular disease pathways. Linkers, says the company, are small sub-molecular components that connect therapeutic molecules, but remain inactive in the bloodstream until they reach the target cells at various points along the pathway.

Once at their targets, the drug molecules are broken off into their active components where they can have an immediate therapeutic impact.  The company says this technology makes it possible to produce drugs that address their targets more precisely, with greater safety and fewer adverse effects.

Catabasis’s pipeline includes therapies for rare diseases including amyotrophic lateral sclerosis or ALS, Friedreich’s ataxia, an inherited disease causing damage to the nervous system and movement problems, and Duchenne muscular dystrophy, a genetic disorder resulting in muscle degeneration and weakness, primarily in the shoulders, arms, hips, and thighs of boys. Earlier this month, the company began recruiting participants for an early- and intermediate-stage clinical trial of its treatment for Duchenne muscular dystrophy code-named CAT-1004. A single therapy for ALS and Friedreich’s ataxia, code-named CAT-4001, is still in preclinical development.

Therapies for lipid disorders, particularly those related to cholesterol, are further along in the Catabasis pipeline. Its first candidate, code-named CAT-2003, addresses a pathway synthesizing fatty acids and in an intermediate-stage trial as a treatment for hypertriglyceridemia, a condition where triglyceride levels become elevated as a result of diabetes or obesity.

A second-generation therapy, targeting the same pathway, is being developed to treat hypercholesterolemia, an inherited form of high cholesterol, in people who do not respond to other types of treatments. That candidate, code-named CAT-2054, is in an early-stage clinical trial.

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Hat tip: Fortune/Term Sheet

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