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Safety, Activity Assessed in New Duchenne Drug

Muscle tissue cross section

Cross-section of muscle tissue from a person with Duchenne muscular dystrophy shows extensive replacement of dark colored muscle fiber with light-colored adipose or fat cells. (Centers for Disease Control and Prevention)

25 January 2016. An early-stage clinical shows an experimental drug for Duchenne muscular dystrophy is safe for patients and produces the desired chemical activity in the body. Results of the trial, conducted by Catabasis Pharmaceuticals were released today. The Cambridge, Massachusetts company says it is now extending the study to test the drug against a placebo.

Duchenne muscular dystrophy is a rare genetic disorder resulting in progressive muscle degeneration and weakness, primarily in the shoulders, arms, hips, and thighs. The disease affects mainly boys starting at age 3 to 5, and caused by a defective gene that fails to produce the protein dystrophin for strengthening muscle fiber and protecting muscles from injury. While life expectancy can vary, people with Duchenne muscular dystrophy do not often survive past their 20s or 30s, with death caused by respiratory or cardiac failure.

Catabasis’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.

The clinical trial tested Catabasis’s therapy for Duchenne muscular dystrophy code-named CAT-1004, among 17 boys age 4 to 7 with the disorder at three sites in the U.S. CAT-1004 is a small molecule that aims to inhibit NF-kB proteins, which when activated, play a role in controlling a number of cellular processes and organ functions, including those associated with muscle wasting. The study tested three dosage levels of CAT-1004, looking primarily for signs of adverse reactions and tolerability, but also evidence of NF-kB protein suppression in plasma samples taken at several points after administering the drug.

Catabasis says individuals taking CAT-1004 experience no serious adverse effects, with gastrointestinal problems, mainly diarrhea the most frequent adverse effect. The therapy, the company adds, was well tolerated at all three dosage levels, with no participants dropping out of the study. In addition, plasma exposure levels among the participant are, on average, equivalent to adults with inhibited NF-kB proteins.

The company expects to extend the study with as many of the same group of participants as possible, testing CAT-1004 against a placebo over 12 weeks. The study team collected data from MRI images and tests of strength and physical function at the beginning of the trial, which will be used to measure any progress among individuals receiving the treatments. The extended trial is expected to begin early in 2016.

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