Indications

Phrixus is developing P-188 NF for a number of indications, but with initial emphasis on Duchenne muscular dystrophy, where the medical need is the greatest and the most urgent. For Duchenne muscular dystrophy, the product is Carmeseal-MD, a formulation of P-188 NF for once-a-day subcutaneous administration by the patient or the caregiver. For acute decompensated heart failure, the product is Carmeseal-HF, a formulation of P-188 NF for intravenous administration in the emergency room or the cardiologist’s office.

Duchenne muscular dystrophy

Duchenne’s is the most devastating of the muscular dystrophies. No drug is fully approved for the treatment of Duchenne muscular dystrophy; no drug is approved at all for the treatment of respiratory or cardiac deficits in these boys and young men.

Dusty Brandom

Dusty Brandom

About one out of every 3,500 boys is born with the mutation for Duchenne muscular dystrophy. It affects approximately 20,000 boys and young men in the United States and approximately 20,000 to 30,000 boys and young men in Europe.

While respiratory failure has historically been the main cause of mortality in DMD, the use of steroids and respiratory assist devices has led to a shift: Most young men with DMD today die from heart failure. Such weakness of the heart muscle, called cardiomyopathy, occurs by age 18 in 90 percent of boys and young men with Duchenne muscular dystrophy.

While there has been much progress developing new medicines to extend the time that a small percentage of the boys and young men can walk (those with specific, rare mutations), these medicines are not of benefit to those boys and young men who do not have these specific mutations or who are already non-ambulatory.

Phrixus is developing Carmeseal-MD to treat the disease pathology independent of the genetic defect that causes the disease with initial focus on respiratory and cardiac deficits in non-ambulatory boys, the most underserved subpopulation in DMD.

For more information on Duchenne muscular dystrophy, see Coalition Duchenne and Parent Project Muscular Dystrophy.

Acute Decompensated Heart Failure (ADHF)

ADHF is the most severe form of heart failure in the general, adult population.

There are one million hospitalizations a year from acute decompensated heart failure, which Phrixus intends to target with Carmeseal-HF, once Phrixus has made sufficient progress developing Carmeseal-MD.

An analysis of Medicare data published in 2010 in the New England Journal of Medicine showed that one in five patients discharged from a hospital is re-admitted within 30 days. For heart-failure patients, the rate is about one in four.

Within 60 days of being discharged from hospitals up to 40% of ADHF patients are re-admitted or have died.

Heart failure

Heart failure occurs when the heart is unable to pump enough blood to the body. Fluid then accumulates in the ankles and lungs, leaving patients with shortness of breath, sapped energy and a sensation of drowning.

Heart failure affects five million Americans.

It costs the health care system $37 billion annually, according to the American Heart Association. Hospital care accounts for 80% of that cost.

Heart failure is the most common cause of hospitalization for adults 65 and over. More than 25% of these patients return to the hospital within 30 days.

 

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