Insmed Inc (INSM)

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March '09 Italian ALS results - 20 to 25% efficacy

keep in mind this was a very mixed patient group,
with a lot of patients allready very far deteriorated

also the number of patient with 100 or so is not that big,
but it gives some rationale to wait and see what the tiny number of USA pals patients might or might not see.
those are just a handfull of patients and they are monitored/registered on paper and with video as well as part of their IND acces to iplex.

Iplex still has apoptotic properties with the igf1bp3 component of the drug, and still proven to induce muscular, bone and neurological growth.Also shown to have better safety profile then free igf1.
A potential to ease some suffering in ALS would greatly add value to current value of iplex though.

FDA Position on Allowing Patients with ALS Access to Iplex under an IND fpor those whom applied before march 6th. As well as a lottery based trial form in future IND applications to ensure some form of further data collection.


Saved file:
http://home.xmsnet.nl/privatebanking/INSM/20090310_FDA%20Position%20on%20Allowing%20Patients%20with%20ALS%20Access%20to%20Iplex%20under%20an%20IND.pdf


(old link no longer active: http://www.fda.gov/cder/drug/infopage/mecasermin_rinfabate/FDA_statement.htm )

Based on this data:

Iplex in ALS patients - RESULTS from italian Expaned Acces Program

Saved file:
http://home.xmsnet.nl/privatebanking/INSM/20090310_FDA_Italian_eap_Iplex_experience.pdf

(old link no longer active:
http://www.fda.gov/cder/drug/infopage/mecasermin_rinfabate/Iplex_experience.pdf )


In the patients summarized above, the ALSFRS-R decline from pre-treatment through last available ALSFRS-R value is 0.38 points per month. In 25% of these patients, on-treatment ALSFRS-R values either did not change over time or slightly increased.
To date, 54 patients have been treated for at least 6 months and have both pre-treatment and 6-month ALSFRS-R values. In this cohort, pre-treatment ALSFRS-R is 30.0 ± 7.9, 6-month ALSFRS-R is 25.7 ± 9.6, and the ALSFRS-R decline from pre-treatment through the last available ALSFRS-R value is 0.68 points per month. In 20% of these patients, on-treatment ALSFRS-R values did not change over time or slightly increased.


at 12 months(!!)
In the patients summarized above, the ALSFRS-R decline from pre-treatment through last available ALSFRS-R value is 0.38 points per month. In 25% of these patients, on-treatment ALSFRS-R values either did not change over time or slightly increased.