TL,
Please justify market not being large enough. Find it hard to believe that if cognitive improvement is proven (of course this remains to be seen), all sufferers with adequate financial/social supports to have access to the medication, wouldn't start taking it (particularly given very favourable safety profile). Someone with more financial nouse than me should be able to crunch the numbers:
From a relatively recent publication, estimated prevalence in UK 1 in 8065 - those are high numbers. Say 70% take it, multiply by estimated cost (no idea, probably >10000$/yr/patient), minus expenses including likely partnering deal, determine reasonable P/E ratio etc = no bloody idea but more than 100m MC!!!
Worth a read:
http://www.hdsa.org/images/content/1/9/19276/hd%20insights%20volume%201.pdf
Interesting reading Dr Tabrizi's outlook on promising biomarkers in HD:
"INSIGHTS: You presented that a number of different biological markers that you're helping develop track with HD. Can you tell us which of these biomarkers you view as the most promising?
TABRIZI: The markers that I've been most impressed with are the structural imaging markers – caudate volume, striatal volume, and whole brain atrophy. These are the markers that I think are the most promising and the most robust at this stage.
However, a number of cognitive and quantitative motor markers are also important. We are developing these markers with our TRACK-HD team. The interesting part about the imaging is that we need to understand more about what these structural changes mean biologically. We need to understand what the pathological substrate is and the functional relevance of these imaging changes. That's something through our ongoing work we're seeking to do – to look at neural compensatory networks and functional plasticity.
INSIGHTS: You talked a little bit about your initial skepticism of structural imaging and how that's been converted.
TABRIZI: My initial skepticism was that whether imaging would demonstrate convincing change, whether it would be robust enough, and whether you would be able to remove inter-scan variability to get good scan quality. Now I understand a great deal more about how the technologies. We have advanced ways of studying the brain, particularly with 3T MRI; we can very accurately and carefully map regions of the brain with very high sensitivity and low measurement error. Imaging gives us insight into regional changes. I've become converted in that imaging does appear to correlate very closely to functional and cognitive decline."
Ring a bell with REACH2HD exploratory sub study of neuroimaging????
DYOR/don't listen etc.
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TL, Please justify market not being large enough. Find it hard...
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