PPMD To Collaborate With Catabasis Pharmaceuticals, Inc. On CAT-1004 Development

Parent Project Muscular Dystrophy (PPMD) announced today a collaboration with Catabasis Pharmaceuticals, Inc. (Catabasis) to assist in a Phase 2 clinical trial of its CAT-1004 product candidate for the treatment of patients with Duchenne. As part of this collaboration, PPMD will provide funding to support participant travel. The trial is expected to begin in the first half of 2015.   


CAT-1004 is designed to block a key inflammatory pathway, mediated by a molecule called “NF-ĸB,” that is thought to contribute to the muscle wasting and loss that occurs in Duchenne. In mice that lack dystrophin, CAT-1004 has been shown to reduce inflammation and increase muscle regeneration, and the drug was well-tolerated and demonstrated a significant reduction of activated NF-ĸB in a study of healthy volunteers. Late last month, the U.S. Food and Drug Administration (FDA) granted CAT-1004 Orphan Drug Designation for the treatment of Duchenne. 

“As more and more potential therapies make it to clinical trial, it is critical that we ease the burden of patients willing to participate in these important next steps – especially in a rare disease like Duchenne, where the patient population is limited. Subsidizing travel expenses is one way PPMD can at least help ease the financial burden, especially in a therapy we believe in like CAT-1004. The team at Catabasis is committed to helping the Duchenne community so it is our obligation to our children and their future to help Catabasis help us.”

-- Pat Furlong, PPMD Founding President and CEO

“We are very pleased to be collaborating with PPMD for the execution of our Phase 2 trial of CAT-1004 in DMD. PPMD’s willingness to help with patient participation in our innovative trial is a great example of PPMD’s commitment to finding a cure for this devastating disease.”

-- Jill Milne, PhD, Catabasis co-founder and CEO


To learn more about other projects PPMD is currently funding, visit our website

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