Outside Markets, page-2

Here is what keeps my eyes on the prize...

#1 Orphan Drug Pricing "In 2014, the average orphan drug cost per patient per year was $137,782(US)
I am not sure how accurate this source is but multiple sources seem to agree that Orphan Drugs can easily go for $100,000 USD a year.

#2 Potential Sales
Using a flat rate of $100,000 USD, each application of Trofinetide could produce over a billion dollars of sales a year at peak market share penetration.

How?

For instance, for FragileX alone the CDC estimates that 1 in 5000 Males have FXS. This is a more conservative number than other sources which state that it is more common, eg 1 in 4000.

It isn't hard to see how a even a small % of potential patients getting access to Trofinetide could mean big dollars even  in a single drug designation. I encourage you to do the sums based on potential peak sales, I have gotten anywhere from 1.5 to 1.9B for FXS in my model, which appears somewhere in the ballpark as to what BP have estimated.

My Bull case assumes 1 in 5000 males under 18 globally (190,000 total suffers) with 10% getting the treatment, spits out 1.9B of sales at peak. Although FragileX life expectancy is normal and Females can get the condition, albeit with less dramatic symptoms.  

The potential outcome for multi designations in Trofinetide can multiple this effect and drastically changes the sums.

#3 Interest in Orphan Drugs and CNS/Autism Spectrum conditions
No need to go into detail here, this has been well documented on this forum.

#4 Favourable FX Movements
Although this is minor, the more the AUD slips against the USD, the cheaper Neuren will be to a potential partner or take over candidate.