Replies to post #2003 on Insmed Inc (INSM)

[Do DD]

From noonansyndrome.org:

Does your child have Noonan syndrome and isn’t growing well?

Approximately 80% of children with Noonan syndrome are shorter than the average child of the same age and sex. About half of adults with Noonan syndrome have short stature. There is currently no FDA approved treatment for growth failure in Noonan syndrome. Although not FDA approved for this use, recombinant human growth hormone (rhGH) is sometimes used to treat short children with Noonan syndrome, however recently published data suggest catch-up growth with rhGH may not be optimal.

Insmed Inc. is initiating a study in Noonan syndrome of a different type of growth-promoting treatment. The rationale for trying this therapy is that it bypasses a problem that may affect responsiveness to growth hormone in Noonan syndrome. The study is being carried out to see if administration of the study drug for one year will improve growth in eligible children with growth failure due to Noonan syndrome.

Your child may qualify for the study if they:

* Have been diagnosed with Noonan syndrome or are suspected of having Noonan syndrome
* Are below a certain height
* Are at least 2 years old and not in puberty yet
* Are not currently being treated with recombinant human growth hormone

If you are interested in finding out more about our clinical study please talk with your physician and share this information with them, or you can contact Insmed Incorporated at [e-mail] or 1-866-464-7539 to obtain information on the nearest participating physician.

Insmed Incorporated is a biopharmaceutical company focused on the discovery and development of drug candidates for the treatment of metabolic diseases and endocrine disorders where there are unmet medical needs. For more information, please visit www.insmed.com.

Let's reason as follows:

- IPlex will work better for Noonan syndrome short stature than Naked IGF-1.
- Incidence for Noonan Syndrome conservatively 1:2500
- Potential group of Noonan Syndrome patients given iPlex treatment (2-12 year = 10 year cohort for US+EU (700MM residents) based on birthrate of 10/1000/yr. And 80% of this population).
= 22400 patients treated maximum/year. Lets assume dosing is identical to Primary IGFD. This would mean an max yearly income from Noonans of $32850*22400=$735MM/yr.
- If we do this same math for Lazards guesstimate of 1500 patients=$50MM/yr.
- This would mean for Orphan drug usage for Noonans a guaranteed income of $350MM in 7 years. It would at a minimum mean a SP of > $3.00 only from Noonan's alone. Given Lazards formula. Let alone the income stemming from other indications.

I'm going to hold at least for some years given this kind of data.

E.

[parentsgratae]

No this is still CW's case; a Magistrate hears and even decides pretrial motions which can be appealed to the primary Judge if a party complains about the ruling; CW would not recuse herself because she had been reversed in a similiar case. Actually it is to INSM's great advantage to have her as the judge; she may be on vacation but is still the Judge of this case; perhaps this is why a Magistrate heard the Motions and arguments. Or she could be on another case; hard to determine the reason. Still likely the Magistrate will assign research to a briefing attorney and take the complex matter under advisement and rule later; Also it is unusual for the Judge to reverse the decision of the Magistrate on pretrial matters. I am sure abharp....has more knowledge of federal practice than I do; my practice was primarily in state court.