ANN ARBOR, MI--(Marketwire - June 21, 2012) - Phrixus Pharmaceuticals, Inc., a clinical-stage,specialty pharmaceutical company focused on innovative therapies for Duchenne muscular dystrophy (DMD) and heart failure, today announced that it has received an SBIR Phase 1 award for $623,000 from the National Institutes of Health (NIH) for its grant application titled "Treatment of Muscular Dystrophy-Associated Dilated Cardiomyopathy with P-188."
"This funding constitutes validation for the potential utility of Carmeseal in DMD. We expect that it will allow us to extend administration of Carmeseal to a new, clinically relevant route of administration, subcutaneous delivery," said Thomas A. Collet, president and CEO.
"Completion of the work funded by this grant will significantly advance our knowledge of how to best turn Carmeseal into a chronic therapy suitable for daily administration," adds Dr. Bruce Markham, Vice President of Research and Chief Scientific Officer.
DMD is the most devastating of the muscular dystrophies. No drug is approved for its treatment. It is a genetic disease that affects about one out of every 3,500 boys. Approximately 20,000 boys and young men live with this disease in the United States. The hallmarks of DMD are skeletal muscle weakness, respiratory distress, and cardiomyopathy. It is a degenerative disease that leads to premature death.
Heart failure occurs when the heart is unable to pump enough blood around the body. It affects five million Americans and costs the health care system $37 billion annually according to the American Heart Association. Acute Decompensated Heart Failure (ADHF) is the most severe form of heart failure. It causes one million hospitalizations each year.
for once I am happy to see some effort being made for our seniors.
Also noticed that the impacts of the drug in increasing the pumping capacity of the heart have already been tested for sickle cell patients.
Assumption towards DMD is that
"Carmeseal, generically known as poloxamer 188 (P-188), has been shown to boost the blood pumping capacity of damaged hearts. When Carmeseal, which appears to act as a molecular bandaid, is infused into the bloodstream, it encounters and binds to microscopic tears in the heart muscle. This may prevent the pathological leak of calcium into the heart cells, which could cause calcium overload and keep the heart from delivering sufficient oxygenation to the vital organs. Carmeseal, which has been shown to be effective in four animal models of DMD and heart failure, is expected to have its effect in patients with DMD irrespective of the genetic defect that causes the disease."
Sincerly hope for the DMD trials to push through as quickly as possible
Interesting. This is right down the road from me.
"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 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."
Phrixus has concurrence with FDA on the following:
First clinical study under the IND in stable heart failure patients – dose escalation design
Agreement for exploratory Duchenne Muscular Dystrophy study under same IND
Nice update. However I just dont understand why these folks are still talking mice. NIH funding already was based on animal data. Sincerely pray that the funding is not utilized in keeping the lab running instead of initiating a trail.
Any direct connect with these folks? I think they can explain better why they are still talking mice and not about when they start rolling in some of the seniors on the trial.