Encouraging Preclinical Results in Membrane Sealant Drug for DMD

Phrixus Pharmaceuticals in Ann Arbor, Mich., reported July 19, 2012, that its experimental drug Carmeseal demonstrated a beneficial effect on the diaphragm (the primary breathing muscle) in mice with a disease resembling Duchenne muscular dystrophy (DMD).

Also known as poloxamer 188, or P188, Carmeseal is designed to act like a molecular bandage, repairing tears in muscle-cell membranes, and originally was targeted at heart muscle degeneration. New study results show that the synthetic compound may be useful not only for cardiac problems but for the life-threatening respiratory problems associated with DMD.  

Low doses effective in mice

The effects of Carmeseal in DMD mice were evaluated at a number of different dosing regimens ranging from daily to weekly dosing, and at doses from 3 to 300 milligrams per kilogram of body weight per day, which translates into a daily dose of 105 milligrams for a child weighing about 75 pounds. The treatment was administered by subcutaneous (under the skin) injection.

Study results indicate that Carmeseal has a maximal effect on tidal volume, an important measure of respiratory performance that assesses the amount of air inhaled and exhaled with each breath under resting conditions.

The results "open a new, convenient route of administration for Carmeseal, similar to the subcutaneous administration of insulin, a route that has been found acceptable for millions of individuals in diabetes,"said Phrixus President and CEO Thomas A. Collet.

In June 2012, the National Institutes of Health (NIH) awarded a $623,000 grant to Phrixus for preclinical studies to test subcutaneous delivery of Carmeseal in DMD.

Results from an earlier MDA-supported study, announced May 16, 2011, showed that daily abdominal injections of P188 benefited heart function in DMD mice. And a study published in March 2012, which also received MDA support, showed that dogs with a DMD-like disease that were given intravenous infusions of P188 showed much less heart damage than dogs receiving saline infusions without P188.

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