Iplex™
Insmed maintains a propriety protein platform, based on the FDA-approved IPLEX™, as a development candidate for the treatment of neuromuscular and metabolic disorders. Insmed's proprietary pipeline using the IPLEX™ protein platform includes a treatment for myotonic muscular dystrophy (MMD), which was given an orphan drug designation by the FDA. In this indication, Insmed has completed a double-blind, placebo controlled phase II study. In addition, Insmed has an expanded access program for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, in areas outside the United States, and finally, Insmed has an early stage research program investigating retinopathy of prematurity (ROP) through a Material Transfer Agreement with Premacure in Sweden.
Myotonic Muscular Dystrophy (MMD) is the most common form of adult muscular dystrophy, affecting approximately 37,000 Americans, and nearly 60,000 people in the European Union. MMD is a genetic disease characterized by endurance loss, muscle wasting, weakness, pain, cognitive impairment and gastro-intestinal dysfunction. There is currently no cure for the disease, and no specific treatment has been developed to satisfactorily reverse or ameliorate the common symptoms associated with the disease.
With partial funding provided by the Muscular Dystrophy Association (MDA), Insmed conducted a randomized, placebo-controlled, double-blind clinical study in 69 patients with MMD to study the safety and tolerability of once-daily, subcutaneous injection of IPLEX™. On June 25, 2009 Insmed reported that the results of the trial indicated that IPLEX™ did not exhibit a statistically significant improvement in the functional measure of endurance by the six-minute walk test, muscle function, muscle strength, or quality of life in any of the tests utilized in this study. Based on the limited number of subjects enrolled with significant impairments in cognitive function, gastrointestinal function or pain, Insmed was unable to reach any conclusions regarding the effects of IPLEX™ on these endpoints.
IPLEX™ did, however, demonstrate improvements in standard measures of insulin sensitivity and reductions in fasting glucose, fasting insulin, cholesterol and triglycerides, which is consistent with the expected metabolic profile of insulin-like growth factor. Administration of IPLEX™ also resulted in anabolic effects of increased body mass index and higher levels of testosterone. The drug was well tolerated in MMD subjects and demonstrated a safety profile consistent with previous studies of IPLEX™.
Based on the metabolic improvements observed in patients treated with IPLEX™ in this trial and discussions with key opinion leaders, the Company intends to apply for a grant from the MDA to facilitate an additional Phase II trial focused solely on a subset of MMD patients with severe insulin resistance who, based on the results of the completed trial, may be more likely to benefit from IPLEX™ treatment. Alternative methods of assessing muscle function will be considered for the proposed trial.
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 patients enrolled in this program.
In February 2009, we announced that Insmed had entered into a definitive agreement to sell all assets related to Insmed's follow-on biologics platform, including the manufacturing facility that had been producing IPLEX™
In March 2009, we announced that Insmed would make IPLEX™ available to treat ALS patients in the United States under single patient INDs. At that time, we also anticipated initiating a clinical study in the United States to study the effects of IPLEX™ on ALS. Also in March 2009, we announced finalization of the sale of our manufacturing facility, ending our internal ability to manufacture any IPLEX™.
In July 2009, we announced that Insmed would cease the supply of IPLEX™ to any new patients and that the company would not initiate further clinical trials with IPLEX™ in the near term. As of this announcement, the company intends to conserve its limited inventory for the treatment of existing patients. The company intends to analyze the ongoing data collected for various indications, including myotonic muscular dystrophy and ALS, and assess the overall IPLEX™ development program, including possible IPLEX™ manufacturing options with third parties and possible future clinical trials. Initiation of the Phase II clinical trial for ALS patients in the U.S. has been postponed while the Company performs this assessment.
