CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc., a clinical stage drug development company built on a pathway pharmacology technology platform, today announced that CAT-1004 has been granted Orphan Drug Designation for the treatment of Duchenne muscular dystrophy (DMD) by the U.S. Food and Drug Administration (FDA). CAT-1004 is designed to inhibit NF-ĸB, which in preclinical studies, has been shown to reduce inflammation and increase muscle regeneration. In a Phase 1 study, CAT-1004 was well-tolerated and demonstrated a significant reduction of activated NF-ĸB.
“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,” said Michael Jirousek, Ph.D., co-founder and chief scientific officer of Catabasis. “We look forward to progressing CAT-1004 into Phase 2 clinical development in 2015.”
CAT-1004 is a new chemical entity that inhibits activated NF-κB, a key mediator of cellular injury. A growing body of evidence indicates that activated NF-κB contributes to the underlying pathology of DMD. CAT-1004 is designed to reduce muscle inflammation and subsequent degeneration, and increase regenerating muscle cells. Catabasis’ proprietary SMART (Safely Metabolized And Rationally Targeted) Linker technology enables selective intracellular delivery and synergistic activity of CAT-1004, which is a conjugate of salicylate and the omega 3 fatty acid docosahexaenoic acid (DHA). CAT-1004 is expected to enter Phase 2 clinical studies in 1H15.
About Orphan Drug Designation
Orphan Drug Designation is granted by the FDA Orphan Drug Designation program for medicines intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan status provides sponsors with development and commercial incentives for designated compounds and medicines.
Catabasis Pharmaceuticals is leveraging its pathway pharmacology drug development platform to bring important medicines to patients with severe lipid disorders and rare diseases. The Company’s mission is to address difficult-to-treat diseases through the simultaneous modulation of multiple targets in a disease pathway. For more information on our technology and pipeline of drug candidates, please visit www.catabasis.com.