PHRIXUS PHARMACEUTICALS, INC. REPORTS POSITIVE PRECLINICAL RESULTS IN
RESPIRATORY DISEASE IN DUCHENNE MUSCULAR DYSTROPHY AFTER
SUBCUTANEOUS DELIVERY OF CARMESEAL™ AT VERY LOW DOSES
Results extend the utility of Carmeseal™ from cardiac disease to respiratory disease, the main
cause of mortality in boys with DMD
ANN ARBOR, Mich. (July 19 2012)
Phrixus Pharmaceuticals, Inc., a specialty pharmaceutical
company focused on innovative therapies for Duchenne muscular dystrophy (DMD) and heart
failure, today announced that it has completed preclinical studies in mdx mice that demonstrate
a beneficial effect of Carmeseal™ on the diaphragm, the skeletal muscle that supports
respiration, after subcutaneous dosing as low as 3 mg/kg per day.
Phrixus evaluated the effect of Carmeseal in mdx mice, the most widely used animal model of
DMD, in a number of dosing regimens ranging from daily to weekly dosing, and at doses
ranging from 3 to 300 mg/kg per day, all administered subcutaneously. Mice were aged to
seven months and then dosed for five months to allow for full development of the phenotype.
The effect of Carmeseal was evaluated by whole body plethsymography (WBP), an established
method to understand the impact of drugs on respiratory function. These studies indicate that
Carmeseal has a maximal effect on tidal volume, an important measure of respiratory
performance, at doses as low as 3 mg/kg dosed once-a-day. This dosing would translate into a
daily dose for each patient of 105 mg based on a 35 kg pediatric patient compared to bolus
intravenous dosing of up to 120 grams per patient in previous trials for sickle cell disease, a
difference of three orders of magnitude.
“These results demonstrate the utility of Carmeseal in respiratory disease, the main cause of
death in boys with DMD, and 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 Thomas A. Collet, president and CEO. “The results
confirm our previous findings in animal models of heart failure, in which we demonstrated
significant efficacy at doses up to two orders of magnitude lower than previously published,”
adds Dr. Bruce Markham, Vice President of Research and Chief Scientific Officer.