April 4, 2012

Mass High Tech

Catabasis gets $120K from MDA to test compounds

Clinical-stage biopharmaceutical company Catabasis Pharmaceuticals Inc. of Cambridge has been awarded $120,000 from the Muscular Dystrophy Association as part of a strategic partnership to test two compounds in a mouse model of Duchenne muscular dystrophy (DMD), one of nine types  of the disorder.

The award was made through the MDA Venture Philanthropy drug development arm of the association’s translational research program.

The two test compounds are called CAT-1004 and CAT-1040, and they are for the research mouse model of DMD, which is a degenerative muscle disease affecting boys almost exclusively. It involves progressive degeneration of voluntary and cardiac muscles, with resulting weakness and heart abnormalities.

Catabasis plans to determine in a new four-month study whether either or both CAT-1004 and CAT-1040 reduce inflammation in muscle tissue and improve muscle function. If the results are favorable, the company plans to begin clinical trials in people with DMD.

The company recently completed a Phase 1 safety, tolerability, and pharmacokinetics study of CAT-1004 in healthy human volunteers. The drug candidate was found to be safe and well-tolerated, according to the company. Catabasis also said that the pharmacokinetics results from the study show chemical proof of concept that the approach works in humans and that CAT-1004 offers broad utility in treating inflammatory diseases.

Catabasis has an open investigational new drug application for the compound with the U.S. Food and Drug Administration that allows it to conduct U.S. clinical trials of the investigational drug.

“MDA is pleased to support further testing of CAT-1004 to assess the compound’s ability to counter muscle-damaging inflammation and support muscle regeneration,” Jane Larkindale, MDA director of translational research, said in a statement. “We’re excited to help determine if this compound, or if the related compound CAT-1040, will be helpful in DMD — and, perhaps, for other diseases in MDA’s program.”

Michael Jirousek, co-founder and chief scientific officer of Catabasis, said the short-term preclinical findings in DMD models are encouraging.

“There is a significant need for new treatment options with improved safety and efficacy for people with DMD. If the preclinical findings prove positive, we will initiate a clinical trial in patients with the disease, which could potentially lead to a new treatment and approach to slow the rate of muscle degeneration in young boys living with DMD.”

Last December, Catabasis added $8 million to a Series A round of venture financing, bringing the firm’s Series A round to $47.6 million.