IPLEX STATUS AND AVAILABILITY MAY 19, 2009

Many inquiries arrive daily about Iplex, the status of its availability and distribution, when the FDA/Insmed clinical test study of Iplex will begin, if the medication is available, how to purchase it and perhaps most importantly, what are the results of its usage so far.  With that in mind, the following information is being provided. It is, to the best of our knowledge, accurate as of May 19, 2009.

A total of 13 US ALS patients are presently receiving Iplex through individual or small group INDs as approved by FDA on March 10, 2009. Of these, seven are part of the Dr. Michael Schafer/ALS WORLDWIDE small group IND and three others are informally overseen by Schafer/ALSWW as well. The remaining six are overseen by each of six individual physicians. Most of these 13 patients began Iplex usage at initial low-dosage levels during the period April 15-30, 2009 and have since been titrated to their full dosage levels. Weekly email reports, monthly telephone reports and weekly video filming are being maintained on the ALSWW 10 patients being supervised.

Several European and Australian patients are now securing Iplex through IDIS Ltd. of Weybridge, UK, the distributor of Iplex for countries other than the US and Italy. Some US patients are presently seeking Iplex from IDIS through shipment to others outside of the US and may then be trans-shipping to the US. The IDIS current estimated quotation is $374 US per vial and patients must use 2-4 vials daily (weight-dependent) for a monthly cost of approximately $22,500 to $45,000 US. As an experimental and trans-shipped drug, no insurance coverage is available through this method. Email IDIS: internationalsales@idispharma.com

Insmed and US FDA announced a planned Iplex clinical test study for those with ALS in March 2009. At the present time, it appears the intended study has not yet been designed and no patients have been or are being recruited. Possible test study configurations include a lottery-type selection of participants, partial or complete payment by the patient test participants, partial or complete subsidy by Insmed and other variations thereof. The design of clinical test studies to the point of active recruitment usually takes at a minimum between six and 12 months. For further information: iplex_als@insmed.com

In the absence of Iplex, several patients remain on Increlex (free IGF-1) from Tercica, Inc. of Brisbane, CA. Some of them are still on the Patient Assistance Program established by Tercica at the request of ALS WORLDWIDE in February 2008. Others have secured Increlex through medical insurance coverage in whole or in part. Results of Increlex usage have varied widely with some discontinuation due either to adverse side effects or Iplex availability.

The Italian Iplex Expanded Access Program, initiated in January 2007 after successful litigation by ALS patients versus the Italian Ministry of Health, is ongoing. Suggestions of its discontinuation in March 2009 appear to have been erroneous. Patient results have not yet been published, apparently due to some ambiguity in the reporting by Italian physicians and the lack of definitive usage guidelines. Anecdotal reports, however, have been generally positive.

As we know more about Iplex changes in status and/or availability, the information will be provided through our website, www.alsworldwide.org and through email to our Iplex database and Friends of ALS WORLDWIDE lists. Please feel free to inquire further at any time.