Impressions of stem cell session @ BIO-CEO&Investor Conference
the webcast of the stemcell session is available @ the following link:
corporate-ir.net
has been a pretty interesting discussion in my eyes where cytori in fact represented the clearest business model and commercialization strategy of all competitors:
some of hedricks key announcements there:
- while we can´t be a 100% shure of being regulated by ind or ide we fell very comfortable with filing 510k
-i can´t give a specific commercialization date for our products, but the olympus-cytori jv has put out a 3-year commercialization plan for celution for the us and globaly
-there will be additional data from spain at the end of the year concerning treatment of crohns desease. they are currently doing clincial research in phase 2b with cultured adps cells
-as already known he assured the begin of clinical trials for heart therapy (europe) and reconstructive surgery (japan) during this year but no information about starting it in the first or second half of the year?!
->personally liked their presentation and still think they will start heart trials during the next few weeks.
-would also wish some more information about their approach to file their 510k with the fda as i think this is one of the main obstacles/key investment points in this business story.
(calhound said last year (ubs presentation) that they are going to file a building block of 510k's during 2006. First half? second half? Will this filings already include aspects of different indications like heart failure to allow them to start clinical trials in the us in 2007 or are this applications bound to the hardware profile of celution?
Hope they will offer an overview and rough timetable with sub-milestones of their "masterplan" for clinical trials and commericalization strategy of heart therapy in europe/us during the next presentations or conference calls.
Maybe they do an update/conference call when they start clinical trials for heart(number of patients, clinics taking part, timeline, hurdles) that will allow the common investor to judge and benchmark their developement a little bit better during the next few years.
some general impressions concerning the session:
Although it was virtually the last session at the conference, the Duke of Windsor Room at the Walldorf Astoria in New York was extremely crowded, which was not surprising at all for the moderator Michael Werner, as he expressed, since the session concerned a discussion by the most distinguished experts on a technology, which will “revolutionise healthcare in the future”.
But this is exactly the point, which concerns the average institutional- as well as the retail investor, when will this all happen? In the embryonic space everything is still pre-clinical, the researchers in the adult stem cell space are somewhat further ahead, with certain indications in the clinic now, so how long will the investor be prepared to wait before he sees commercialization of the technology? 5 to 10 years as usual within the pharma business with the development of a drug though the IND process? And how much will the drug or therapy cost after such a long development period. Will it still be affordable?
Those were the key issues, i.e. how is the pathway to commercialization, how do you get past the FDA and how long will this take and how expensive will this be. The R&R analyst expressed this quite nicely and renamed the session, which was called “getting around the embryonic stem cell issue“, to „getting our arms around the potential of cell therapeutics“, so, how, when and how much will it cost.
Personally, as a CYTX investor, it was for me extremely important and pleasant to hear the following statement coming from another individual than a CYTX-management team member, in respect of current standards at the FDA for cell therapies. In fact, the statement came from Bruce Cohen, CEO of Cellerant Therapeutics, who said that presently applied FDA good tissue practice rules, “imply, that if you do not manipulate, culture, change or modify your cells, then you are not a drug. In that case, you are regulated in respect of approval by the PROCESS and the process involves approval on how you make them (whether the cells made are safe) and not on what they do”
As I have written many times in our German discussion board, this confirms my understanding for the FDA device approval process, that you prove the safety of your process to the FDA and leave the efficacy issue to the clinics (surgeons), or indirectly to the patients, as customers of the surgeons, who basically decide to use the approved “FDA safe” device.
This is pretty neat of course, since the FDA has been very much focussed on the so called „mechanisms of action“ of single homogenized substances, i.e. they want to know exactly how your compound works in respect of „safety“ (side effects etc) and „efficacy“. With the CYTX heart cell therapy, for instance, it would be obvious that not all of the injected cells do reach the heart muscle and “home” into the muscle tissue, many do, but as a side effect also improvement take place on the arteries and positive changes occur in the own bodies enzyme system, which all together lead to the desired result of dramatically improved blood ejection fraction of the heart. With other words, “multiple mechanisms of action“ occur, which taken as a whole are extremely complex and even partially unknown, as to the why, but do lead as a therapy to the desired result of saving lives of patients and at least making them feel and live much better, with the therapy as compared to without it.
3 years road to market is CYTX plan- (my personal expectation, 2 years for Europe and the US one year later) which was supported by an answer from Hedrick on a question on FDA „Interaction“, in which he said that, „ the FDA wants to help the patients and we (the USA) do not want to be left behind in the development of these therapies“
I think, this all sounds extremely positive, since on the cost side the colleagues from the industry were not committing themselves to numbers, but were talking about 50-75.000$ per therapy, without any hesitation with the exception of course that Hedrick feels that the cost of the CYTX therapy would be very modest and possible with a 5-10.000$ tag, which, I feel, makes it affordable for medical insurances worldwide.
We will have to wait and see. Its clear that in the sessions conclusion, the timeline to market was brought back from 5 to 10 years, to 3 to 6 years, depending on the business model.
I think, nothing in life is absolutely sure, except the fact that we all will die someday, but from a chance-risk perspective, it feels good to be part of the team which is saying it will head to the market the soonest and will collect accordingly. |
