Catabasis' Duchenne Drug Obtains Orphan Drug Designation

James Radke

Plenty of interesting news about Duchenne muscular dystrophy today. First, Biomarin announced they are planning to buy Prosensa for $840 Million, and now, Catabasis Pharmaceuticals has announced their drug candidate, CAT-1004, has been granted Orphan Drug Designation for the treatment of Duchenne muscular dystrophy by the U.S. Food and Drug Administration (FDA).

CAT-1004 inhibits NF-ĸB, a key mediator of cellular injury that may contribute to the underlying pathology of Duchenne muscular dystrophy. CAT-1004 is designed to reduce muscle inflammation and subsequent degeneration, and increase regenerating muscle cells. In a phase I study, CAT-1004 was well-tolerated and demonstrated a significant reduction of activated NF-ĸB.

Michael Jirousek, PhD, co-founder and chief scientific officer of Catabasis said:

“Orphan drug designation for CAT-1004 underscores the importance of bringing new and disease-modifying therapies to treat DMD to the market, and we believe CAT-1004 is a novel approach to address this fatal disorder.”

“We look forward to progressing CAT-1004 into Phase 2 clinical development in 2015.”

In addition to CAT-1004 for Duchenne muscular dystrophy, Catabasis is using its SMART linker technology to develop oral, small molecules for a number of rare diseases, including CAT-2004 for patients with chylomicronemia.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with Duchenne muscular dystrophy lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.

There are an estimated 35,000 patients with Duchenne in the United States and Europe but the population has many subsets based on mutations of the dystrophin gene. CAT-1004 could theoretically work on all subsets of patients with Duchenne muscular dystrophy.
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