A billion-dollar pharma startup that Silicon Valley has missed
techcrunch.com
techcrunch.com
>You would never know, to read a lot of Ramaswamy’s press coverage (here’s a recent profile from TechCrunch) that the Axovant drug works through a mechanism that has failed twice in Alzheimer’s clinical trials so far.
>You wouldn’t know from such articles, though, that there have been (and are) other companies which have been formed around the idea of taking pharma cast-offs and getting them through the clinic (larger companies already try to monetize what they can in their portfolio by partnering or outlicensing, of course). The Medicines Company is one such dealmaker, acquiring and partnering late-stage compounds and trying to get them approved. Outside of the for-profit business model, there are a number of initiatives trying to repurpose or revive older compounds for new diseases.No one so far has been able to take over the world doing this. There are not, unfortunately, many big piles of such candidates sitting around. Most of the shelved compounds were shelved because (a) they did not work, and/or (b) they showed toxicity. You’re going to have to figure a way around those problems before you go back into humans, and that’s not easy. There are a few drugs that have been dropped for business reasons, or were lost or mishandled during a merger or the like, but I don’t think that there are enough of those to make a Ramaswamy drug empire.
>It was the biggest biotech IPO ever in the U.S., raising $360 million. It has largely held up, too. Axovant’s shares, which opened at $15, currently trade around $13.25.
and further down:
> When Axovant went public, a columnist at FierceBiotech warned that it should “scare the hell” out of investors
On a personal note the worst investment I ever made was a short position where I was right, but six months too early.
he's discussed his stock positions a few times; the takeaway is along the lines of "i don't have any holdings that could be construed as bias as regards where this blog is hosted; otherwise it's my own business" if i remember right.
We are now much more equipt at finding biomarkers, generally through gene sequencing. Drugs that failed, often failed because they were applied to an entire population of a disease (ie multiple sclerosis). Now, we can subset the MS population by different biomarkers and test just that small subset for efficacy.
While testing anyone that happens to have MS would show a massive failure rate, testing 5% of the population that has specific biomarkers, we may be able to show a huge increase in efficacy and thus the drug would be approved for MS patients that fit the exact bio marker profile.
it's hard work: http://blogs.sciencemag.org/pipeline/archives/2013/12/04/can...
nominal biomarkers can be wholly spurious: http://blogs.sciencemag.org/pipeline/archives/2012/04/10/bio...
biomarkers that we believe to be proxies for disease conditions (like LDL) can be targeted to no effect: http://blogs.sciencemag.org/pipeline/archives/2016/12/14/sim...
etc.
Torceptipib is a great example. Phase 2 looked great; phase 3 was stopped due to an increase in deaths.
Tl;dr Alzheimer's is hard to drug, and the approach this company is trying has been tried before.
The plan is simple. You fund a company and spend $5mn acquiring the rights to a drug that might be worth billions (not very likely, if you can get it at that price). And before investing your own money to find out, you sell one fourth of that company for $360mn because "Hey, it could be worth billions!!!!"
This sounds like a pump and dump or money extraction scheme. I imagine it was mostly sold to pensions, etc.
SV BS spreading from software to Biotech/Pharma.
> https://www.google.com/finance?q=NYSE%3AAXON&fstype=ii&ei=2U...
Wait, what? How is this a startup and how does this have anything to do with SV...
TC is really reaching here...
I think the HN moderators need to remove things like this.