IPLEX®
Under the proprietary IPLEX® protein platform, while supplies last, Insmed maintains an expanded access program for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. Insmed also provides support for an early stage research program investigating retinopathy of prematurity (ROP) through a Material Transfer Agreement with Premacure in Sweden.
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, Insmed 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.
