Iplex™
The company's lead product IPLEX™ is being studied as a treatment for several serious medical conditions, including two neuromuscular disorders, Myotonic Muscular Dystrophy (MMD) and ALS (Lou Gehrig's Disease); HIV- Associated Adipose Redistribution Syndrome (HARS); and retinopathy of prematurity (ROP). Cumulatively, these disorders affect more than 100,000 patients in the United States, and a similar number in the European Union.
Myotonic Muscular Dystrophy (MMD) is the most common form of adult muscular dystrophy, affecting 1 in 8,000 individuals in the U.S. Approximately 40,000 Americans are diagnosed with the disease, and close to 60,000 people in the EU also suffer from this life-threatening disorder. Patients with MMD develop progressive muscle wasting and weakness, cardiovascular problems, cognitive disorders and digestive complications. Eventually becoming totally disabled, patients often die from respiratory or cardiac failure. At present, there is no treatment to halt or reverse the progression of MMD.
IPLEX™ is being investigated in a Phase II clinical study at the University of Rochester School of Medicine, with funding provided by the Muscular Dystrophy Association and the National Institutes of Health. This Phase II program is studying the safety and tolerability of once-daily, subcutaneous injection of IPLEX™ in patients with MMD.
We are currently enrolling patients in a Phase II trial for Myotonic Muscular Dystrophy. Click here for more information.
An estimated 80,000 HIV patients in the U.S have HARS, according to published reports. This disorder is marked by abnormal metabolism including both central fat accumulation (visceral adiposity and buffalo hump) and fat loss in the limbs. These features have increased markedly with the advent of highly active antiretroviral therapy (HAART) for HIV. Recent studies performed in subjects on HAART suggest nearly 50% of individuals develop the morphologic features characteristic of this syndrome.
IPLEX™ is also being explored as a possible therapy for the HARS indication. Data is being collected from a Phase II open-label clinical study at the University of California San Francisco. This study is designed to evaluate the safety and efficacy of IPLEX™ treatment with the primary goal of determining the effects of IPLEX™ on visceral fat distribution and glucose and lipid metabolism.
Clinical work is at an earlier stage in the development of IPLEX™ to treat Retinopathy of Prematurity (ROP). This disease, affecting an estimated 14,000 to 16,000 premature infants each year, causes the lack of development of the small blood vessels in the back of the eye and is a major cause of blindness in premature infants. A Phase I clinical study investigating IPLEX™ as a treatment for ROP has been initiated at the University of Gothenburg in Sweden, in collaboration with scientists at the Harvard Medical School in the U.S.
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's Disease is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their death. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. With voluntary muscle action progressively affected, patients in the later stages of the disease may become totally paralyzed.
In January 2007, we announced that the Italian Ministry of Health requested Insmed to make IPLEX™ available to treat Italian patients suffering from ALS and we are currently supplying IPLEX™ to these patients under an expanded access program. In Italy it is estimated that 1,000 new cases of ALS are diagnosed every year.
