The Richmond, Va., biopharmaceutical company Insmed announced July 27, 2009, that it will not supply its experimental drug Iplex to any new patients with amyotrophic lateral sclerosis (ALS) for the foreseeable future, and that it intends to analyze the available data on Iplex for ALS and type 1 myotonic dystrophy (MMD1, or DM1) before deciding whether to proceed with development of the drug for either disease.
Iplex is a combination of a protein called insulin-like growth factor 1 (IGF1) and IGF binding protein 3. Laboratory studies have suggested it may preserve muscle or nerve tissue under adverse circumstances, including degenerative disease.
MMD1 is a degenerative muscle disease, and ALS is a degenerative disease of the cells in the nervous system that control muscles.
Iplex and MMD1
MDA helped Insmed develop and test Iplex in 69 adults with MMD1. In June, the company announced the drug had failed to improve muscle function, strength or endurance in this disease, although it did improve the ability of cells to respond to insulin in some trial participants. (See Iplex Shows Limited Benefit in MMD1.)
Iplex has never been available to MMD1 patients except through a clinical trial.
Iplex and ALS
In March, Insmed announced it would work with the U.S. Food and Drug Administration (FDA) to develop a clinical trial of Iplex in patients with ALS, in which it has never been formally tested.
At that time, the FDA said it would process individual requests for compassionate use of Iplex by U.S. residents with ALS through March 6, 2009, but that subsequent access to the drug in the United States would only be through a formal trial. (See FDA Allows Formal Testing of Iplex in ALS.)
There is no evidence that the drug is effective against ALS, but it has been available for use by ALS patients in other countries, particularly Italy, and data so far suggest the compound is reasonably safe.
Insmed's July 27 press release said there are approximately 70 patients with ALS who currently receive Iplex, 12 in the United States and the remainder around the rest of the world. The 12 U.S. patients are being treated under special "single-patient Investigational New Drug" applications approved by the FDA.
Insmed said it believes it has enough Iplex to supply these 70 patients for no more than two years.
In the same July 27 press release, Insmed's chairman, Melvin Sharoky, said, "We believe it is in the best interests of patients who are currently receiving Iplex to ensure that our current limited inventory is conserved, in order to maintain their drug supply as long as possible, while allowing the company time to consider its development options."