Last year one of the biggest events in the stem cell field was biotech Geron punting its embryonic stem cell (ESC) program.
Some folks opposed to ESCs happily pronounced ESC research dead.
Some folks in favor of ESC thought the world was ending a year early.
Others tried to look on the bright side and hoped for a buyer for Geron’s ESC-related assets, but through the grapevine everything I was hearing was that that was not going to happen.
Now that many months have gone by we can look back at Geron’s goodbye and one would hope that we can learn something.
Such seems the impetus for a Nature Biotechnology opinion piece by Christopher Scott and Brady Huggett: Geron’s quixotic fate .
There is much to like about this article and it contains some amazing facts (e.g. Geron’s application to the FDA was 22,000 pages long and was the longest in FDA history), but at the same time the piece seems off key in some ways.
Starting with the title….
I don’t see anything quixotic about the fate of Geron.
In fact, given that more than 9/10 biotech companies fail completely, one might predict that most individual research programs of biotech companies fail with a near, if not quite 100% rate. Thus, Geron’s ESC program had the odds stacked against it from Day 1. Then if you factor in the reality that this was the FDA’s first experience with ESCs, Geron’s odds became even longer as Geron not only had to navigate the challenging regulatory waters of the FDA, but from what I heard they also had to literally teach the FDA all about ESC.
The Scott and Huggett article also has a warning for Advanced Cell Technology (ACT; stock symbol ACTC), which is now currently the only company with active FDA trials on ESC. They write, addressing ACT directly:
your technology may be revolutionary, your team may be dedicated and you may believe. But it does not matter if no one else will stand at your side.
Sounds ominous, doesn’t it? But I think it is awfully gloomy and negative.
I believe that one big advantage that ACT has over Geron is that Geron had to pave the way all by itself. ACT now is enjoying the benefit of Geron having in essence prepped the FDA for how to handle ESC-related biologic products.
Another advantage is that I believe that ACT’s therapy and target disease are more fiscally practical.
I don’t have some magic biotech crystal ball to tell me the future of ACT, but I have not heard anything specific that makes me especially pessimistic.
The odds are long for any biotech, but ACT has shown something that most biotechs fail at:
longevity
To me that means something in biotech.
“But it does not matter if no one else will stand at your side.”
Someone should inform Scott and Huggett of these 4 standing at ACT’s side:
Bascom Palmer (#1 Eye center, US News)
Wills Eye Inst (#3)
Mass Eye/Ear (#4)
UCLA-JSEI (#5)
I think it is much more important what the leading eye experts in the world think than what these two authors think..
Paul thank you for your Blog this week.
ACTC has chosen the eye for its clinical trial work and this is what will in my opinion allow ACT to achieve proof of concept. In a recent discussion with Matt Vincent the Direct of Biz & Dev. he indicated to me that ACT had enough cash on hand to see the Company through Phase I.
The real hero’s of ACT’s success are the Retail Investors that put up the capital for ACT to achieve the milestones.
I sold all of my stock in Geron in 2/2011 when I realized that Geron had NO WAY to prove its GRNP01 could be tied to improvement of the patients. I am still an ACTC shareholder and will increase my position once the stock continues to shake out…
thank you
JTG
http://www.investorstemcell.com
ACT has an active dialogue with the medical establishment in China. If the FDA proves to be a roadblock, I expect that the company will take its IP and go overseas.
Unfortunately the possibility is that whatever the FDA decides is often at the behest of big pharma, at least to some extent. So many biotech / medical device makers are denied their potential by inexplicable or poorly reasoned negative FDA decisions, and I fear ACTC may eventually fall victim to this same type of cronyism. That they (Geron, ACTC) were given the green light for initial trials was a small miracle in itself. It would not surprise me to see ACTC denied at the post, and all their work and patents be snatched up for pennies on the dollar by some big pharma company, who will then either lock it away permanently and vigorously litigate any possible patent infringements, or who will complete rudimentary final-phase tests which will be green-lighted by the FDA so they can make even more billions. The only possible way to avoid this, imho, is for ACTC to enter a joint venture agreement with one of these big pharma companies, however it may be cheaper for big pharma to wait it out and take the buyout route instead… just depends if there is competition between the big pharmas to get their greedy hands on this tech. It all comes down to power, money and greed – that this technology could change medical history for the better and save or better the lives of millions of people is immaterial at this point.
Thanks for the perspective. ACT needs to nail down a joint venture with a partner big enough to finance trials for the next three years. If they can manage to do that, the future looks good….
Geron’s CEO is still taking a lot of flack over halting the HESC programs. He has admitted to one angry investor who was representing hundreds of other investors that he sees the future of HESCs as very bright in the future. Shareholders have been arguing against divestiture of those assets. They would rather Geron restart those programs when they are in a position to restart them. Geron is incorperated in Delaware so any deal for divestiture will likely require shareholders approval since the HESC programs repressented more than 60% of their pipeline and an investment of over $400 million. There is a patent case between Geron and Viacyte that is waiting for the judges ruling. At the heart of the ruling is the science behind creating definitive endo derm and the markers that show definitive endo derm at different times during the differetiation process. There are currently 2 patent cases now between Geron and Viacyte. Here are links to both cases. Patent case number 1 https://acts.uspto.gov/ifiling/PublicView.jsp?identifier=105734&identifier2=null&tabSel=4&action=filecontent&replyTo=PublicView.jsp
Patent case number 2
https://acts.uspto.gov/ifiling/PublicView.jsp?identifier=105827&identifier2=null&tabSel=4&action=filecontent&replyTo=PublicView.jsp
Geron responsive motions 2 and 3 in the second case bear out the arguement.
Taylor