Iplex Potentially Available For ALS
The biopharmaceutical companies Insmed, Genentech and Ipsen/Tercica jointly announced this week that Insmed's drug Iplex may become available to people with ALS (amyotrophic lateral sclerosis) worldwide.

Iplex is approved in the United States for the treatment of children with growth failure due to IGF1 deficiency, but due to a court-ordered settlement agreement in March 2007, it has until now been available to ALS patients only in Italy as part of an expanded access program.

The drug, which has not been rigorously studied in ALS, is being studied, with MDA support, in type 1 myotonic dystrophy. It's a combination of the protein insulin-like growth factor 1 (IGF1) and binding protein 3 and must be administered by subcutaneous (under the skin) injection.

Myotrophin, a different injectable version of IGF1 that doesn't include the binding protein, has just been found ineffective in ALS in a third clinical trial. Myotrophin was developed by the pharmaceutical firm Cephalon. See 'Tie-Breaker' Myotrophin Trial Negative in ALS.

"There is a rationale for this compound in treating ALS, but so far, there has been no demonstration that it will work in this disease," said Sharon Hesterlee, MDA vice president for Translational Research. "Iplex is based on a natural protein that helps maintain muscle fibers under adverse conditions, and MDA is supporting its development for the muscle disease myotonic dystrophy. If the data support additional clinical testing of the drug for ALS, we will consider supporting those studies as well."

In a statement, Insmed said Genentech and Ipsen/Tercica signed a letter of intent on Nov. 8, whereby they consented to amend the court-ordered settlement agreement to permit Insmed to supply Iplex in connection with expanded access ALS programs worldwide on a royalty-free basis.

Availability of the drug will be dependent on satisfying any regulatory requirements in the country from which a request is made.

Patient or physician inquiries regarding Iplex should be directed to coneal@insmed.com.



http://www.als-mda.org/research/news...iplex_als.html