Last year I blogged a couple times about Dr. Christopher "the renegade" Centeno from Regenerative Sciences, Inc (aka "Regenexx"). He's in Colorado collecting, growing, and re-administering stem cells for now a growing list of orthopedic-related conditions.
As previously discussed on this blog, FDA CBER's Mary Malarkey sent a warning letter in July 2008 to Dr. Christopher J. Centeno, Medical Director of Regenerative Sciences, Inc (RSI), doing business as Regenexx. The company responded on its website that they do not fall under FDA jurisdiction. One of the reasons cited is that their entire procedure occurs within the state ergo no interstate commerce ergo no federal jurisdiction.
The good doctor (aka @stemcelldoc on Twitter) is not only rapidly expanding the indications for which he'll pump you up with stem cells, he is really ramping up his marketing. He is actively promoting his services on the internet (blogging, a Twitter stream, etc) encouraging people to come from all over god's green earth to visit Colorado and get shot up with a dose of their own stem cells.
Now I know that archaic legal paradigms weren't designed for this new-fangled world but according to a good ole fashioned sniff test many would say that smells a lot like interstate commerce to them.
This is not a critique of the Regenexx therapy, Dr. Centeno, or the company. I'm not saying the Regenexx therapy doesn't work. I'm betting it does. I'm not saying I wouldn't be tempted to go if I had joint problems. I likely would. I'm not saying what they're doing is wrong or even illegal - sometimes it takes a gutsy cowboy/girl or two to change the rules for good. BUT if the FDA lets this happen in Colorado it seems to me a slippery slope to letting all autologous cell therapies go unregulated. And I don't see that happening any time soon.
Rumor has it Regenexx is planning to open a new clinic in Florida. There's the slope in clear sight.
On other fronts, there have been reports of the Regenexx therapy being reimbursed by at least one insurer. Aside from the FDA, this is quickly gaining all the hallmarks of a legitimate medical procedure. As of November, they had performed 250 procedures.
In September last year Dr. Centeno posted on an online discussion board:, "To set the record straight, we have been working with regulatory council for 3 years on our needle based, surgical procedure. We have responded to the FDA unititled [sp]letter and we believe we have answered all of their questions. This is a surgical procedure and not a new biologic drug. We have been communicating with the FDA and they have had no response, so we assume that we have answered their questions."
The FDA may not have any more questions but unfortunately I doubt that ends the issue.
Interstate commerce aside, Regenexx appears to be manufacturing a biological therapeutic. While they appear to have taken references to cell expansion or manipulation off their website, according to a recent blogger allegedly quoting from a letter from the company, the stem cells are collected, expanded (at minimum) and reinjected. The letter apparently stated: "The procedure involves drawing our patient’s blood and bone marrow, growing their stem cells in our laboratory for approximately 3 weeks and then injecting the stem cells into the affected joint".
If this is true, it seems to me this is not just a doctor doing his doctor thing and the big, bad FDA should let a doc be a doc. This would be a doctor manufacturing a biologic without any of the manufacturing controls created by regulatory agencies and industry to keep such things safe - safe, for instance, from contamination.
If there are no such controls in place god only knows what kind of back-alley operations might come to town selling stem cells from basement labs. It is not far-fetched. If this is allowed to continue, other docs will get in this business. Some will decide they just want to run the clinic not grow the cells. They outsource that to company X which is being run by some half-baked science dropout who decided this is probly better money than making meth so let's start a stem cell grow-op! Ok, that's the extreme but you see my point.
I've got a lot of sympathy for people who push against the rules. I believe Dr. Centeno and his company are doing what they believe is right and I'm sure they are doing everything they can to ensure their product is safe and efficacious. I also believe that in years to come we may well be comfortable enough with these types of products that this may well be as commonplace as Dr. Centeno would have it but I don't think it will be in his lifetime. At least not in the US. I kinda hope I'm wrong.
We can't simply let the market decide which therapies will succeed. We need a minimum level of safety assurance and oversight by an agency like the FDA and without any controls how long is it before someone perhaps not as diligent or trained as Dr. Centeno makes a product that does more harm than good?
I think the FDA is building its case and a showdown is on it's way to Denver-town.
Tuesday, February 3, 2009
Monday, February 2, 2009
How many cell therapy products are in development? Simple question, no?
I have touched before on the nomenclature issues related how we 'define' this industry. Is it cell therapy, regenerative medicine, etc? To some it might seem 'merely semantic' but we all know how difficult it is to communicate something that is really not well-defined. To the extent we need to enhance the profile and understanding of this sector in the world outside it, the better we define the industry the easier it is to communicate. Similarly troubling is the lack of good data regarding the size of the industry. How many companies and/or products? What are the revenues? A broader consensus on how to define the industry can only help us in our dialogue with investors, partners, policy-makers, regulators, etc.
There are somewhere between 225 and 325 companies globally with at least one cell therapy on the market or in development (clinical and pre-clinical). On that much there is a fairly general consensus.
But that allows for a fairly wide margin of error and confidence in the data only gets shakier from there. There is little consensus on the companies that are on that list, the types of products they have, or the status of those products. If one draws the circle broader to include regenerative medicine products, tools, and reagents, the number of companies reaches >650 thus exaggerating the lack of clarity.
In previous posts here I've talked about the number of cell therapy products I believe are currently the subject of industry-sponsored clinical trials globally (and this post will address the topic in more detail). I've also addressed the fact that there is a lot of misconception in the industry regarding particularly the number of products in late-stage, industry-sponsored trials. In large part this is because there is a real lack of complete and reliable sources for this kind of data.
One of the oft-cited sources for this kind of information is www.clinicaltrials.gov. I thought I'd put it to the test. Here are the results of my admittedly rudimentary analysis:
If I type in cell therapy (no quotes) as the only search term, I get 14,022 trials in the search results. When I refine the search by selecting only those funded by 'industry', this narrows it down to 3,817. If I refine it further to include the term 'cell' in the 'intervention' field, this narrows it down to 1,827. A % of these do not involve cell therapies.
If I type in "cell therapy" (with quotes) as the search term, I get 132 trials. Almost all these I would define as cell therapy. When I refine this search to include only those funded by 'industry', I get 32.
In another search I looked for currently open studies, employing 'biologic' as the 'intervention', selected 'industry' as the funding source and searched for the phrase "cell therapy" (with quotes). This produced 6 studies all of which are cell therapy studies.
My next search was exactly the same but with "stem cell" (with quotes) as the search phrase. This produced a list of 19 studies including many of the previous nine but also at least 2 studies I would not define as employing a cell-based therapy but rather another type of biologic.
Using all the same search parameters except typing in the words 'cell' and 'therapy' (without quotation marks) in the search field produces 144 results but a high percentage are not what I would define as employing a cell-based therapy but rather other types of biologics (e.g., Rituximab, Campath). Using the search word "cells" produces 169 results but again many are trials of non cell-based biologics.
To test the database using a specific, I typed in 'Amorcyte' into the sponsor search field. No results. I typed it into the 'search term' field and got one trial listing Emory University as the sponsor.
The data is all over the map and rife with problems associated with the lack of auditing control over the integrity of the data going in. If this is, as many believe, the best single public source of clinical trial data for cell therapies, it is at the very least inadequate to get an overall picture of the true number of trials in the industry. If there are better ways to conduct the search, I'm open to critique.
Industry and market reports are another often relied upon source for this kind of data. I have reviewed several of the leading cell therapy industry/market reports currently available. They rarely - if ever - purport to contain exhaustive company or product lists and the lists they do contain are rife with inaccurate and incomplete information in terms of the companies they list or do not list, the types of therapies the listed companies are said to be pursuing, and the indications to which those therapies are intended to apply.
I certainly do not claim 100% accuracy in my database but I claim it be be as exhaustive as any at least in terms of the list of companies (though likely not in its fields of information such as financial data, clinical trial details, etc). Certainly, the database has its weaknesses (e.g., companies from Asia about which I find it notoriously difficult at times to find detailed information). Having said that, here's what I have.
I am currently tracking >700 companies which I define as stakeholders in the cell therapy sector. This includes ~250 therapeutic companies [i] with ~340 cell-based therapeutic products in the market or in some stage of clinical or pre-clinical development [ii]. These products can be roughly broken down into the following stages:
78 Pre-clinical
77 Phase I
89 Phase II
33 Phase III
67 Commercial (previously discussed in this blog <click here>)
Let me pause here momentarily to say that in an upcoming blog I will break down my 'commercial' number with more transparency. I've been criticized for saying there are so many 'products' on the market when a % of them are unapproved products being sold in unregulated markets. This, in some people's minds, is the equivalent of including crack cocaine in an estimate of the size of the pharmaceutical sector. First let me say I love that critique. I understand. I've never said these are good products, approved products, profitable products... or anything about the products. I believe the critique is a little rough because these products are perfectly legal in the countries in which they are sold. You may think high-priced facial creams claiming to make one's skin feel younger are a fraud but you wouldn't discount them from an analysis of the size of the cosmetics industry.
My only criterion for this number has been whether or not a product is a cell therapy that can be bought legally somewhere in the world. Having said that, I will clarify my list and make it more transparent so readers can make their own assessment. Even after I categorize my list and you limit it down to approved products in regulated markets, it's still two or three-fold what most people think there is.
Note that the numbers for categories other than 'commercial' only include 'products' which are in development in jurisdictions which do have an approval process for such trials and/or products.
I'm also working now on categorizing the products in my database by autologous vs allogeneic, strictly cell-based therapies vs. some combination with another intervention (e.g., drug, device, gene-modification, etc), and by indication (e.g, oncology, diabetes, cardiovascular, neuro, immuno, etc). Stay tuned for those results.
I asked Proteus Venture Partners if they would provide me with a snapshot of their dataset to compare against mine. Proteus is putting together a $200-300 million fund focusing exclusively on 'regenerative medicine' products (as they define them). They have been working now for 3+ years with some of the brightest minds in the sector, to define the industry and put together their own proprietary database intended to drive their investment decisions. They break the industry into 5 sub-categories: tissue engineering, cell therapy, aesthetic medicine, supporting tools/devices, regenerative compounds.
Proteus tracks regenerative medicine which is - as they define it - broader then my 'cell therapy' data set. They also track clinical trials versus my 'products'. However this should be almost the same as my 'products' because when a company is testing the same product for two different indications, I count that as two products.
Proteus has been kind enough to share with me - and permit me to share with you - their numbers. They are tracking ~260 companies with ~430 clinical trials (and commercial products) in the 'tissue engineering' and 'cell therapy' categories'. These trials are broken down as follows:
195 Pre-clinical
77 Phase I
23 Phase II
15 Phase III
124 Commercial
Unfortunately you will note significant differences between my numbers and theirs. A number of potential reasons exist why this is likely the case.
As a VC, Proteus is particularly interested in early-stage technologies (pre-clinical) in which it can participate in the first or early stages of funding. They are - I suspect - decreasingly interested as trials/products become later-stage where there is less opportunity for them to participate in value building. My interest is more in those technologies already in clinical trial and I am increasingly interested the closer the trial/product comes to market. Note our complete agreement on the number of Phase I trials/products where our interests are likely the most closely aligned. For this reason, one might be tempted to trust the Proteus numbers on the early end of the scale and my numbers more on the late-stages.
Proteus also includes some academic-sponsored trials in their pre-clinical number while I restrict my database to only those which are industry-sponsored. This may explain a large bulk of the difference in the pre-clinical number.
On the other end of the spectrum, about 80% of the 'commercial' products Proteus reports are 'tissue engineering' products; the remaining 20% are cell therapy products (~25 products). A number of the tissue engineering products, while regenerative medicine products, would not be included in my 'cell therapy' products because they do not involve the use of cells ex vivo as part of the therapeutic product (e.g., a non-cellular scaffold). If one assumes this is true of 1/2 these products then this would mean our numbers of commercial products are about the same.
That leaves us with the significant difference in the phase II and III numbers. One would expect the Proteus numbers to be higher than mine because they track a wider range of products than I track -- but they are lower. As I suggested above, perhaps phase II and III products are of less interest to Proteus so their numbers in these categories are not as comprehensive as mine. Interestingly, however, we agree on roughly the same number of companies overall. Perhaps my data does not capture multiple trials for the same products as accurately as Proteus' data.
While there are some unanswered questions in comparing the data sets, at the very least the numbers give a range which is likely the best studied and most accurate, data-driven set of numbers that are currently available.
Over the course of the past few weeks I have provided analysis on the number of cell therapy products already commercially available (and how that differs from common perception), the number of phase III/pivotal cell therapy trials currently underway (and how that differs from common perception) and here I have discussed the number of products currently in development.
Coming soon will be a commentary on some of the trends I see coming down the pipe for cell therapy. This will be much more of an op-ed type piece. The 'popcorn report' for cell therapy, if you will. More opinion, less data.
Getting back on the data track I will - as promised above - provide more insight into what types of products are on my 'commercial' list. I also hope to bring you sometime later this Quarter a snapshot of how my data on the therapeutics products in development and in the market breaks down by category (autologous vs allogeneic; strictly cell-based therapies vs. some combination with another intervention (e.g., drug, device, gene-modification, etc); and indication (e.g, oncology, diabetes, cardiovascular, neuro, immuno, etc). Stay tuned for those results.
I know that my data, perspective, and analysis is not without its critics but I hope at least it's of use in spawning a discussion where to-date there has been little data and much misinformation.
--Lee
p.s. Happy birthday nephew Eli! :)
[i] Other estimates typically range from 225 to 300 therapeutic companies. Sources: author’s database; Cell Therapy Pages (Connexon Communications); Proteus Venture Partners; Bionest Partners. Cell therapies and tissue engineering. February 2007; Burger SR. 2004. Cell and Gene Therapy - Challenges and Strategies for an Emerging Industry. Cell and Gene Therapy 5:9-14.
[ii] “Pre-clinical development” is defined to product in development prior to initiation of a phase I trial but not including products in the early research phase.
There are somewhere between 225 and 325 companies globally with at least one cell therapy on the market or in development (clinical and pre-clinical). On that much there is a fairly general consensus.
But that allows for a fairly wide margin of error and confidence in the data only gets shakier from there. There is little consensus on the companies that are on that list, the types of products they have, or the status of those products. If one draws the circle broader to include regenerative medicine products, tools, and reagents, the number of companies reaches >650 thus exaggerating the lack of clarity.
In previous posts here I've talked about the number of cell therapy products I believe are currently the subject of industry-sponsored clinical trials globally (and this post will address the topic in more detail). I've also addressed the fact that there is a lot of misconception in the industry regarding particularly the number of products in late-stage, industry-sponsored trials. In large part this is because there is a real lack of complete and reliable sources for this kind of data.
One of the oft-cited sources for this kind of information is www.clinicaltrials.gov. I thought I'd put it to the test. Here are the results of my admittedly rudimentary analysis:
If I type in cell therapy (no quotes) as the only search term, I get 14,022 trials in the search results. When I refine the search by selecting only those funded by 'industry', this narrows it down to 3,817. If I refine it further to include the term 'cell' in the 'intervention' field, this narrows it down to 1,827. A % of these do not involve cell therapies.
If I type in "cell therapy" (with quotes) as the search term, I get 132 trials. Almost all these I would define as cell therapy. When I refine this search to include only those funded by 'industry', I get 32.
In another search I looked for currently open studies, employing 'biologic' as the 'intervention', selected 'industry' as the funding source and searched for the phrase "cell therapy" (with quotes). This produced 6 studies all of which are cell therapy studies.
My next search was exactly the same but with "stem cell" (with quotes) as the search phrase. This produced a list of 19 studies including many of the previous nine but also at least 2 studies I would not define as employing a cell-based therapy but rather another type of biologic.
Using all the same search parameters except typing in the words 'cell' and 'therapy' (without quotation marks) in the search field produces 144 results but a high percentage are not what I would define as employing a cell-based therapy but rather other types of biologics (e.g., Rituximab, Campath). Using the search word "cells" produces 169 results but again many are trials of non cell-based biologics.
To test the database using a specific, I typed in 'Amorcyte' into the sponsor search field. No results. I typed it into the 'search term' field and got one trial listing Emory University as the sponsor.
The data is all over the map and rife with problems associated with the lack of auditing control over the integrity of the data going in. If this is, as many believe, the best single public source of clinical trial data for cell therapies, it is at the very least inadequate to get an overall picture of the true number of trials in the industry. If there are better ways to conduct the search, I'm open to critique.
Industry and market reports are another often relied upon source for this kind of data. I have reviewed several of the leading cell therapy industry/market reports currently available. They rarely - if ever - purport to contain exhaustive company or product lists and the lists they do contain are rife with inaccurate and incomplete information in terms of the companies they list or do not list, the types of therapies the listed companies are said to be pursuing, and the indications to which those therapies are intended to apply.
I certainly do not claim 100% accuracy in my database but I claim it be be as exhaustive as any at least in terms of the list of companies (though likely not in its fields of information such as financial data, clinical trial details, etc). Certainly, the database has its weaknesses (e.g., companies from Asia about which I find it notoriously difficult at times to find detailed information). Having said that, here's what I have.
I am currently tracking >700 companies which I define as stakeholders in the cell therapy sector. This includes ~250 therapeutic companies [i] with ~340 cell-based therapeutic products in the market or in some stage of clinical or pre-clinical development [ii]. These products can be roughly broken down into the following stages:
78 Pre-clinical
77 Phase I
89 Phase II
33 Phase III
67 Commercial (previously discussed in this blog <click here>)
Let me pause here momentarily to say that in an upcoming blog I will break down my 'commercial' number with more transparency. I've been criticized for saying there are so many 'products' on the market when a % of them are unapproved products being sold in unregulated markets. This, in some people's minds, is the equivalent of including crack cocaine in an estimate of the size of the pharmaceutical sector. First let me say I love that critique. I understand. I've never said these are good products, approved products, profitable products... or anything about the products. I believe the critique is a little rough because these products are perfectly legal in the countries in which they are sold. You may think high-priced facial creams claiming to make one's skin feel younger are a fraud but you wouldn't discount them from an analysis of the size of the cosmetics industry.
My only criterion for this number has been whether or not a product is a cell therapy that can be bought legally somewhere in the world. Having said that, I will clarify my list and make it more transparent so readers can make their own assessment. Even after I categorize my list and you limit it down to approved products in regulated markets, it's still two or three-fold what most people think there is.
Note that the numbers for categories other than 'commercial' only include 'products' which are in development in jurisdictions which do have an approval process for such trials and/or products.
I'm also working now on categorizing the products in my database by autologous vs allogeneic, strictly cell-based therapies vs. some combination with another intervention (e.g., drug, device, gene-modification, etc), and by indication (e.g, oncology, diabetes, cardiovascular, neuro, immuno, etc). Stay tuned for those results.
I asked Proteus Venture Partners if they would provide me with a snapshot of their dataset to compare against mine. Proteus is putting together a $200-300 million fund focusing exclusively on 'regenerative medicine' products (as they define them). They have been working now for 3+ years with some of the brightest minds in the sector, to define the industry and put together their own proprietary database intended to drive their investment decisions. They break the industry into 5 sub-categories: tissue engineering, cell therapy, aesthetic medicine, supporting tools/devices, regenerative compounds.
Proteus tracks regenerative medicine which is - as they define it - broader then my 'cell therapy' data set. They also track clinical trials versus my 'products'. However this should be almost the same as my 'products' because when a company is testing the same product for two different indications, I count that as two products.
Proteus has been kind enough to share with me - and permit me to share with you - their numbers. They are tracking ~260 companies with ~430 clinical trials (and commercial products) in the 'tissue engineering' and 'cell therapy' categories'. These trials are broken down as follows:
195 Pre-clinical
77 Phase I
23 Phase II
15 Phase III
124 Commercial
Unfortunately you will note significant differences between my numbers and theirs. A number of potential reasons exist why this is likely the case.
As a VC, Proteus is particularly interested in early-stage technologies (pre-clinical) in which it can participate in the first or early stages of funding. They are - I suspect - decreasingly interested as trials/products become later-stage where there is less opportunity for them to participate in value building. My interest is more in those technologies already in clinical trial and I am increasingly interested the closer the trial/product comes to market. Note our complete agreement on the number of Phase I trials/products where our interests are likely the most closely aligned. For this reason, one might be tempted to trust the Proteus numbers on the early end of the scale and my numbers more on the late-stages.
Proteus also includes some academic-sponsored trials in their pre-clinical number while I restrict my database to only those which are industry-sponsored. This may explain a large bulk of the difference in the pre-clinical number.
On the other end of the spectrum, about 80% of the 'commercial' products Proteus reports are 'tissue engineering' products; the remaining 20% are cell therapy products (~25 products). A number of the tissue engineering products, while regenerative medicine products, would not be included in my 'cell therapy' products because they do not involve the use of cells ex vivo as part of the therapeutic product (e.g., a non-cellular scaffold). If one assumes this is true of 1/2 these products then this would mean our numbers of commercial products are about the same.
That leaves us with the significant difference in the phase II and III numbers. One would expect the Proteus numbers to be higher than mine because they track a wider range of products than I track -- but they are lower. As I suggested above, perhaps phase II and III products are of less interest to Proteus so their numbers in these categories are not as comprehensive as mine. Interestingly, however, we agree on roughly the same number of companies overall. Perhaps my data does not capture multiple trials for the same products as accurately as Proteus' data.
While there are some unanswered questions in comparing the data sets, at the very least the numbers give a range which is likely the best studied and most accurate, data-driven set of numbers that are currently available.
Over the course of the past few weeks I have provided analysis on the number of cell therapy products already commercially available (and how that differs from common perception), the number of phase III/pivotal cell therapy trials currently underway (and how that differs from common perception) and here I have discussed the number of products currently in development.
Coming soon will be a commentary on some of the trends I see coming down the pipe for cell therapy. This will be much more of an op-ed type piece. The 'popcorn report' for cell therapy, if you will. More opinion, less data.
Getting back on the data track I will - as promised above - provide more insight into what types of products are on my 'commercial' list. I also hope to bring you sometime later this Quarter a snapshot of how my data on the therapeutics products in development and in the market breaks down by category (autologous vs allogeneic; strictly cell-based therapies vs. some combination with another intervention (e.g., drug, device, gene-modification, etc); and indication (e.g, oncology, diabetes, cardiovascular, neuro, immuno, etc). Stay tuned for those results.
I know that my data, perspective, and analysis is not without its critics but I hope at least it's of use in spawning a discussion where to-date there has been little data and much misinformation.
--Lee
p.s. Happy birthday nephew Eli! :)
[i] Other estimates typically range from 225 to 300 therapeutic companies. Sources: author’s database; Cell Therapy Pages (Connexon Communications); Proteus Venture Partners; Bionest Partners. Cell therapies and tissue engineering. February 2007; Burger SR. 2004. Cell and Gene Therapy - Challenges and Strategies for an Emerging Industry. Cell and Gene Therapy 5:9-14.
[ii] “Pre-clinical development” is defined to product in development prior to initiation of a phase I trial but not including products in the early research phase.
Friday, January 23, 2009
Cell Therapy Industry HiLites 2009-01-23
I hope to see many of you next week at the Phacilitate Cell & Gene Therapy Forum in Washington, DC. I suspect we are all today much more keenly anticipating Jane Lebkowski's talk (Geron) than we were last week.
I will be giving a short summary of the cell therapy sector in a lunchtime symposium on Tuesday sponsored by Progenitor Cell Therapy. I'll also be there with the founders of BioBusiness.TV interviewing executives, delegates, and doing some face time of my own in front of the camera all in support of an effort to launch a cell therapy channel on the new up-and-coming biotech business video site. We're looking for a sponsor for this cell therapy content - if you're interested, let me know - I think it's great value!
As a result of my being away all next week, I will more than likely skip the Industry HiLites posting next Friday. I'll try not to lose any news in the interim between reports.
While it's tempting to lead off with the news of Suzanne Somers' appearance this week for NeoStem (NYSE Alternext US: NBS) on CNBC's "Conversations with Michael Eisner", I suppose rightly so the big news of the week (year? decade?) is clearly Geron's news of FDA approval for its embryonic stem cell-derived therapy (GRNOPC1) for thoracic spinal cord injury patients. I dedicated a brief post last night to the news I helped break based on an early leak by the Morris Daily Herald. I also chewed up a fair amount of Twitter bandwidth this morning doing 'live tweeting' during Geron's webcast. (see http://twitter.com/celltherapy). At midnight (EST) Thursday night, Geron updated their website with everything they're willing to share about their product, trial, manufacturing, testing, etc. It's actually quite informative >> click here.
Earlier in the week in-PharmaTechnologist.com discussed Lonza's implentation of a plan to spend"$26m to expand capacity at its facility in Walkersville, Maryland, "as regulatory progress made with Osiris’ Prochymal looks set to kick start the era of cell therapies". This will add capacity by Q1 2010 to meet anticipated demand by its customer Osiris for production of Prochymal should it be approved.
Financially, the Company intends to raise further funding for its programes over the course of this year, and its convertible loan facility from certain existing investors will provide cash resources sufficient to finance the Company's operations into the third quarter of this year. (see articles in the Financial Times and NatureNews for more details).
Angel Biotechnology Holdings plc (AIM: ABH) issued a press release congratulating ReNeuron on receiving this approval because Angel is, of course, ReNeuron's manufacturing partner.
BioBusiness.tv has posted a great interview of Osiris CEO, Randy Mills by Bill Kridel of Ferghana Partners Group. Randy dares to use the word "blockbuster" in conjunction with his expectations for Prochymal. He also reports that Osteocel has now treated 30,000 patients since its launch in July 2005.
Nuvasis, Inc (NASDAQ: NUVA) is projecting $28M in Osteocell sales in 2009. While that's only 8% of its overall revenue outlook of $350M, it's not insignificant. Add that to over 250,000 patients treated to-date by Appligraf (Organogenesis) for total revenue in 2008 alone of ~$70M, ~13,000 patients to-date for Carticel (Genzyme), and the fact Advanced Biohealing's facility is able to produce 250,000 units of Dermagraft per year....and we just might have the beginnings of a viable sector here.
Mesoblast (ASX:MSB) received approval from the Australian regulators to proceed with a randomized, placebo-controlled phase II trial of their allogeneic stem cell product, RepliCart, for knee osteoarthritis after acute traumatic injury. StemCellDigest immediately commented on the potential patent questions vis-a-vis Osiris.
TAP launched its automated 'CellCelector' at SelectBio's Stem Cells World Congress. The device consists of an inverted Olympus microscope, robotic arm and liquid handling station integrated with image acquisition and analysis software. The system allows researchers to set parameters for cell or colony types they want (including size, proximity to other colonies or roundness). The picking tool on the robotic arm gently picks and dispenses cells into a microplate well in just 30 seconds. CellCelector can fit into any standard laminar flow hood and can be fitted with an autoclave compatible metal tool for scraping adherent cells or a disposable glass capillary for picking single cells.
StemCellDigest.net summarized The Money Raising Sagas Of Advanced Cell Technology Inc.
Amidst circulating rumors and clear evidence of financial distress, Opexa Therapeutics, Inc. (NASDAQ:OPXA) - developing patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis (MS) and diabetes - provided an update on corporate activities.
Looks like a PerkinElmer has a strategy congealing around its ViaCord/ViaCell acquisition. After announcing a collaboration with MD Anderson last week, this week they announced that its ViaCord Research Institute, which focuses on supporting science, technology and medical treatments using cord blood stem cells, will support the University of Massachusetts Medical School (UMMS) in its research efforts into the potential use of umbilical cord blood-derived stem cells in treating type 1 diabetes.
According to a recent report, while more than 10,000 Australian parents have paid between $3000 and $5700 to have their child's cord blood collected, frozen and stored in the seven years since private collection companies began operating in Australia, no samples have been used for medical treatment.
This one is more the news behind the news. Novartis has recently posted a position for a Regenerative Medicine Ophthalmology Research Investigator PhD/MD. The description? "The candidate will lead a research team dedicated to developing regenerative medicine therapeutics for ophthalmic disease. The team will collaborate with other technology groups within the Novartis Institutes for Biomedical Research (NIBR) to identify novel chemical and biologic modulators of adult stem cells, validate the therapeutic potential of these discoveries, and develop these assets into therapies targeting blinding eye diseases with a high unmet need." So...while it would appear they're stuck on finding non-cell therapy regenerative compounds, they're playing with stem cells. That's a good thing.
With all the other noise around Bioheart (NasdaqCM: BHRT) last week, I missed some important potential good news. Bioheart, Inc. announced last wek that they have filed with appropriate agencies in Switzerland for reimbursement approval for its MyoCell(r) myogenic cell therapy for heart failure. The initial reimbursement application filings are targeting the sickest Class III and IV heart failure patients who have not responded well to drugs and do not qualify for a bi-ventricular pacemaker. Bioheart is applying for a reimbursement level matching exactly that charged to provide bi-ventricular pacemakers for this same patient population.
So how can companies like Bioheart file for 'reimbursement' before the therapy is approved? I asked this question of Eric Faulkner of RTI Health Solutions. He provided this answer. "This is not a filing for ‘full reimbursement’ but is a filing for reimbursement subsidy for their clinical trials in certain target markets. Usually these are competitive and not every thing gets funded, but if approved the manufacturer likely covers some costs (e.g., cells, catheters, etc.) and the hospital would be covered by the govt. (e.g., some of the inpatient facility and provider costs). This can be a way to defray costs of conducting the trial leading up to regulatory market clearance. CMS and some limited # of US commercial payers have similar provisions in areas of high unmet need." I learned something.
Linda Powers took advantage of all the hype and interest around Geron's news to remind the world through an article by John Sterling in Genetic Engineering News that her $140 million Toucan Capital venture capital fund "holds the largest number of stem cell, regenerative medicine, and living cell companies in the world [16 companies]". She also stated "not enough attention is being paid to the biomanufacture of stem cells and other living cells" like is done by her portfolio company Cognate Bioservices.
Lord knows looking at my stock portfolio, I'm no stock analyst - not even a good stock picker. With that in mind (i.e. this ain't no stock recommendation or even general negativity about Dendreon), below is a little analysis on Dendreon this week by David Gaffen on Wall Street Journal's MarketBeat blog that I share with you more for what it tells me about the trial, the data, and analysts' thinking than for what it might mean for the stock:
It’s been a rough week for Dendreon, a biotechnology stock with a fervent group of followers that wait with baited breath for every bit of news about its Provenge treatment for prostate cancer. The stock was hit hard earlier in the week after news that data from company’s ongoing trial for its treatment will be released in April, earlier than expected, because the trial has surpassed the threshold for number of deaths to incur a final analysis. The problem, as some Wall Street analysts see it, is that the company amended its trial late in the process to include sicker patients. The drug needed to show a 22% reduction in the risk of death to be successful, and it was at 20% earlier in the trial. “We firmly believe that the death events occurring after the May 2008 cutoff date consist of a greater percentage of patients that were enrolled later in the trial (i.e., sicker patients), which would make it even harder for any drug to offer interim analysis,” write analysts at Brean Murray Carret & Co. “Therefore, the 20% difference reported at interim is actually further from the 22% goal required at final analysis than it may seem at first glance.” Some are remaining optimistic, however, including David Miller, who writes on Minyanville.com that “long-suffering Dendreon bulls (like me) will be rooting for those extra 2 points of patient benefit.” This stock has been a volatile one — and if the 22% threshold is reached, expect wild action. Shares hit a 52-week low of $3.42 Tuesday.
Finally for you lab-heads and quality-geeks out there... ISCT is offering a webinar February 18, 2009, entitled (Part 1): Validation and Qualification of Equipment and Reagents.
And that wraps up a momentous week for cell therapy. I love the smell of stem cells in the morning...
Safe travels.
I will be giving a short summary of the cell therapy sector in a lunchtime symposium on Tuesday sponsored by Progenitor Cell Therapy. I'll also be there with the founders of BioBusiness.TV interviewing executives, delegates, and doing some face time of my own in front of the camera all in support of an effort to launch a cell therapy channel on the new up-and-coming biotech business video site. We're looking for a sponsor for this cell therapy content - if you're interested, let me know - I think it's great value!
As a result of my being away all next week, I will more than likely skip the Industry HiLites posting next Friday. I'll try not to lose any news in the interim between reports.
While it's tempting to lead off with the news of Suzanne Somers' appearance this week for NeoStem (NYSE Alternext US: NBS) on CNBC's "Conversations with Michael Eisner", I suppose rightly so the big news of the week (year? decade?) is clearly Geron's news of FDA approval for its embryonic stem cell-derived therapy (GRNOPC1) for thoracic spinal cord injury patients. I dedicated a brief post last night to the news I helped break based on an early leak by the Morris Daily Herald. I also chewed up a fair amount of Twitter bandwidth this morning doing 'live tweeting' during Geron's webcast. (see http://twitter.com/celltherapy). At midnight (EST) Thursday night, Geron updated their website with everything they're willing to share about their product, trial, manufacturing, testing, etc. It's actually quite informative >> click here.
Earlier in the week in-PharmaTechnologist.com discussed Lonza's implentation of a plan to spend"$26m to expand capacity at its facility in Walkersville, Maryland, "as regulatory progress made with Osiris’ Prochymal looks set to kick start the era of cell therapies". This will add capacity by Q1 2010 to meet anticipated demand by its customer Osiris for production of Prochymal should it be approved.
On the fundraising front, Belgian biotech Cardio3 BioSciences has rounded up €13.7 million from venture groups and grants. Cardio3 raised €7.2 million in a Series B and an additional €6.5 million in cash advances from the Walloon Region General Directorate for Economy, Employment & Research. Much of the new money will be used to fund a 240-patient clinical trial of C-Cure, its lead program. C-Cure is designed to allow the differentiation of a patient's own cells into 'cardiopoietic' cells which grow into new heart cells and repair heart muscle.
Additionally, International Stem Cell Corporation (OTCBB:ISCO) has received the second $1 million tranche of an anticipated private equity financing of up to $5 million to be funded over the next several months. As we speculated might be the case a few weeks ago, the investors were the same as the last round: X-Master, A. Semechkin, and R. Semechkin. According to a recent SEC filing X-Master is a New Hampshire corporation which provides software consulting and computer hardware services and which holds real estate and other assets and investments. The SEC filing also reported that the source of funds for the acquisition of the shares in the previous rounds was primarily from the proceeds of loans made to X-Master by Hartcom Impex, Ltd., a British Virgin Islands corporation and from the working capital of X-Master. Not your garden-variety financing by any means.
After much delay, ReNeuron has received permission from UK authorities to launch a phase I trial for stroke patients using their neural stem cell product ReN001. The ReN001 cells will be administered by direct injection into the affected region of the brain in a straightforward surgical procedure.
Financially, the Company intends to raise further funding for its programes over the course of this year, and its convertible loan facility from certain existing investors will provide cash resources sufficient to finance the Company's operations into the third quarter of this year. (see articles in the Financial Times and NatureNews for more details).
Angel Biotechnology Holdings plc (AIM: ABH) issued a press release congratulating ReNeuron on receiving this approval because Angel is, of course, ReNeuron's manufacturing partner.
BioBusiness.tv has posted a great interview of Osiris CEO, Randy Mills by Bill Kridel of Ferghana Partners Group. Randy dares to use the word "blockbuster" in conjunction with his expectations for Prochymal. He also reports that Osteocel has now treated 30,000 patients since its launch in July 2005.
Nuvasis, Inc (NASDAQ: NUVA) is projecting $28M in Osteocell sales in 2009. While that's only 8% of its overall revenue outlook of $350M, it's not insignificant. Add that to over 250,000 patients treated to-date by Appligraf (Organogenesis) for total revenue in 2008 alone of ~$70M, ~13,000 patients to-date for Carticel (Genzyme), and the fact Advanced Biohealing's facility is able to produce 250,000 units of Dermagraft per year....and we just might have the beginnings of a viable sector here.
Mesoblast (ASX:MSB) received approval from the Australian regulators to proceed with a randomized, placebo-controlled phase II trial of their allogeneic stem cell product, RepliCart, for knee osteoarthritis after acute traumatic injury. StemCellDigest immediately commented on the potential patent questions vis-a-vis Osiris.
TAP launched its automated 'CellCelector' at SelectBio's Stem Cells World Congress. The device consists of an inverted Olympus microscope, robotic arm and liquid handling station integrated with image acquisition and analysis software. The system allows researchers to set parameters for cell or colony types they want (including size, proximity to other colonies or roundness). The picking tool on the robotic arm gently picks and dispenses cells into a microplate well in just 30 seconds. CellCelector can fit into any standard laminar flow hood and can be fitted with an autoclave compatible metal tool for scraping adherent cells or a disposable glass capillary for picking single cells.
StemCellDigest.net summarized The Money Raising Sagas Of Advanced Cell Technology Inc.
Amidst circulating rumors and clear evidence of financial distress, Opexa Therapeutics, Inc. (NASDAQ:OPXA) - developing patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis (MS) and diabetes - provided an update on corporate activities.
Looks like a PerkinElmer has a strategy congealing around its ViaCord/ViaCell acquisition. After announcing a collaboration with MD Anderson last week, this week they announced that its ViaCord Research Institute, which focuses on supporting science, technology and medical treatments using cord blood stem cells, will support the University of Massachusetts Medical School (UMMS) in its research efforts into the potential use of umbilical cord blood-derived stem cells in treating type 1 diabetes.
According to a recent report, while more than 10,000 Australian parents have paid between $3000 and $5700 to have their child's cord blood collected, frozen and stored in the seven years since private collection companies began operating in Australia, no samples have been used for medical treatment.
This one is more the news behind the news. Novartis has recently posted a position for a Regenerative Medicine Ophthalmology Research Investigator PhD/MD. The description? "The candidate will lead a research team dedicated to developing regenerative medicine therapeutics for ophthalmic disease. The team will collaborate with other technology groups within the Novartis Institutes for Biomedical Research (NIBR) to identify novel chemical and biologic modulators of adult stem cells, validate the therapeutic potential of these discoveries, and develop these assets into therapies targeting blinding eye diseases with a high unmet need." So...while it would appear they're stuck on finding non-cell therapy regenerative compounds, they're playing with stem cells. That's a good thing.
With all the other noise around Bioheart (NasdaqCM: BHRT) last week, I missed some important potential good news. Bioheart, Inc. announced last wek that they have filed with appropriate agencies in Switzerland for reimbursement approval for its MyoCell(r) myogenic cell therapy for heart failure. The initial reimbursement application filings are targeting the sickest Class III and IV heart failure patients who have not responded well to drugs and do not qualify for a bi-ventricular pacemaker. Bioheart is applying for a reimbursement level matching exactly that charged to provide bi-ventricular pacemakers for this same patient population.
So how can companies like Bioheart file for 'reimbursement' before the therapy is approved? I asked this question of Eric Faulkner of RTI Health Solutions. He provided this answer. "This is not a filing for ‘full reimbursement’ but is a filing for reimbursement subsidy for their clinical trials in certain target markets. Usually these are competitive and not every thing gets funded, but if approved the manufacturer likely covers some costs (e.g., cells, catheters, etc.) and the hospital would be covered by the govt. (e.g., some of the inpatient facility and provider costs). This can be a way to defray costs of conducting the trial leading up to regulatory market clearance. CMS and some limited # of US commercial payers have similar provisions in areas of high unmet need." I learned something.
Linda Powers took advantage of all the hype and interest around Geron's news to remind the world through an article by John Sterling in Genetic Engineering News that her $140 million Toucan Capital venture capital fund "holds the largest number of stem cell, regenerative medicine, and living cell companies in the world [16 companies]". She also stated "not enough attention is being paid to the biomanufacture of stem cells and other living cells" like is done by her portfolio company Cognate Bioservices.
Lord knows looking at my stock portfolio, I'm no stock analyst - not even a good stock picker. With that in mind (i.e. this ain't no stock recommendation or even general negativity about Dendreon), below is a little analysis on Dendreon this week by David Gaffen on Wall Street Journal's MarketBeat blog that I share with you more for what it tells me about the trial, the data, and analysts' thinking than for what it might mean for the stock:
It’s been a rough week for Dendreon, a biotechnology stock with a fervent group of followers that wait with baited breath for every bit of news about its Provenge treatment for prostate cancer. The stock was hit hard earlier in the week after news that data from company’s ongoing trial for its treatment will be released in April, earlier than expected, because the trial has surpassed the threshold for number of deaths to incur a final analysis. The problem, as some Wall Street analysts see it, is that the company amended its trial late in the process to include sicker patients. The drug needed to show a 22% reduction in the risk of death to be successful, and it was at 20% earlier in the trial. “We firmly believe that the death events occurring after the May 2008 cutoff date consist of a greater percentage of patients that were enrolled later in the trial (i.e., sicker patients), which would make it even harder for any drug to offer interim analysis,” write analysts at Brean Murray Carret & Co. “Therefore, the 20% difference reported at interim is actually further from the 22% goal required at final analysis than it may seem at first glance.” Some are remaining optimistic, however, including David Miller, who writes on Minyanville.com that “long-suffering Dendreon bulls (like me) will be rooting for those extra 2 points of patient benefit.” This stock has been a volatile one — and if the 22% threshold is reached, expect wild action. Shares hit a 52-week low of $3.42 Tuesday.
Finally for you lab-heads and quality-geeks out there... ISCT is offering a webinar February 18, 2009, entitled (Part 1): Validation and Qualification of Equipment and Reagents.
And that wraps up a momentous week for cell therapy. I love the smell of stem cells in the morning...
Safe travels.
Thursday, January 22, 2009
Geron obtains FDA approval for phase I human embryonic stem cell study
According to reports filed tonight, the FDA has approved Geron's clinical trial of embryonic stem cells for spinal cord injury.
Why the Morris Daily Herald gets the scoop on this Associate Press story is a mystery to me but according to an article filed this evening in the Morris Daily Herald, picking up the feed from Associated Press science writer Malcolm Ritter, Geron (Nasdaq: GERN) received approval to proceed with its trial "this week. "
When I started writing this the news had not yet come from Geron. Then their site went down. Now it's up on Geron's home page. It's official!
There are people who question Geron's decision to go to the clinic in a spinal cord injury model and others who have have critiqued Geron's business model or corporate style but there is no question that today is not only Geron's day but - if this is true - it is a red letter day in the inevitable progress of cell therapy, stem cell research, and patients with spinal cord injury.
"'It's a milestone and it's a breakthrough for the field'.. said Ed Baetge, chief scientific officer of Novocell Inc."
This marks a new day for embryonic stem cell research and the imminent lifting of the restrictions on federal financing of embryonic research will be icing on this cake.
It will be most interesting to see now the effect on the fortunes of other stem cell companies - embryonic and adult - cell therapy companies generally and, in particular, the stocks of those which are publicly traded.
_____________________
Here is a copy of the full article from the Morris Daily Herald:
THURSDAY JANUARY 22, 2009
Last modified: Thursday, January 22, 2009 6:03 PM CST
US approves 1st stem cell study for spinal injury
Why the Morris Daily Herald gets the scoop on this Associate Press story is a mystery to me but according to an article filed this evening in the Morris Daily Herald, picking up the feed from Associated Press science writer Malcolm Ritter, Geron (Nasdaq: GERN) received approval to proceed with its trial "this week. "
"'The company gained federal permission this week to inject eight to 10 patients with cells derived from embryonic cells,' said Dr. Thomas Okarma, president and CEO of Geron Corp."The timing of this is ludicrously delicious. The FDA just happened to wait to approve the trial until Obama is sworn into office? And/or did Geron just happen to wait to release the news until the new Administration came in? I can't believe there was political pressure or influence but the timing of this is admittedly freaky.
When I started writing this the news had not yet come from Geron. Then their site went down. Now it's up on Geron's home page. It's official!
There are people who question Geron's decision to go to the clinic in a spinal cord injury model and others who have have critiqued Geron's business model or corporate style but there is no question that today is not only Geron's day but - if this is true - it is a red letter day in the inevitable progress of cell therapy, stem cell research, and patients with spinal cord injury.
"'It's a milestone and it's a breakthrough for the field'.. said Ed Baetge, chief scientific officer of Novocell Inc."
This marks a new day for embryonic stem cell research and the imminent lifting of the restrictions on federal financing of embryonic research will be icing on this cake.
It will be most interesting to see now the effect on the fortunes of other stem cell companies - embryonic and adult - cell therapy companies generally and, in particular, the stocks of those which are publicly traded.
_____________________
Here is a copy of the full article from the Morris Daily Herald:
THURSDAY JANUARY 22, 2009
Last modified: Thursday, January 22, 2009 6:03 PM CST
US approves 1st stem cell study for spinal injury
AP Science Writer
NEW YORK (AP) — A U.S. biotech company says it plans to start this summer the world's first study of a treatment based on human embryonic stem cells — a long-awaited project aimed at spinal cord injury.
The company gained federal permission this week to inject eight to 10 patients with cells derived from embryonic cells, said Dr. Thomas Okarma, president and CEO of Geron Corp. of Menlo Park, Calif.
The patients will be paraplegics, who can use their arms but can't walk. They will receive a single injection within two weeks of their injury.
The study is aimed at testing the safety of the procedure, but doctors will also look for signs of improvement like return of sensation or movement in the legs, Okarma said.
Whatever its outcome, the study will mark a new chapter in the contentious history of embryonic stem cell research in the United States — a field where debate spilled out of the laboratory long ago and into national politics.
While some overseas doctors claim to use human embryonic stem cells in their clinics, stem cell experts said they knew of no previous human studies that use such cells.
"It's a milestone and it's a breakthrough for the field" because Geron passed the safety hurdles for getting federal clearance to launch the study, said Ed Baetge, chief scientific officer of Novocell Inc. His company hopes to begin a similar human study for treating diabetes in a few years.
In addition, said spinal cord injury researcher Dr. Wise Young of Rutgers University, "a lot of hope of the spinal cord injury community is riding on this trial."
Embryonic stem cells can develop into any cell of the body, and scientists have long hoped to harness them for creating replacement tissues to treat a variety of diseases. But research has been controversial because embryos must be destroyed to obtain them.
President Barack Obama has promised to relax the Bush administration's restrictions on federal financing for such research. But Obama's ascent to the White House had nothing to do with the U.S. Food and Drug Administration's granting permission for the new study, Okarma said in a telephone interview Thursday.
In fact, the company says, the project involves stem cells that were eligible for federal funding under Bush, although no federal money was used to develop the experimental treatment or to pay for the human study.
Other human cells, called adult stem cells, have been tested before in people to treat heart problems, for example.
In the Geron study, the injections will be made in the spine at the site of damage. The work will be done in four to seven medical centers around the country, Okarma said.
Animal studies suggest that once injected, the cells will mature and repair what is essentially a lack of insulation around damaged nerves, and also pump out substances that nerves need to function and grow.
Apart from assessing safety, investigators will hope to see some signs of improvement in the patient, Okarma said. The idea is "not to make somebody ... get up and dance the next day," he said, but rather to provide some level of ability that can be improved by physical therapy.
Each patient will receive a low dose of anti-rejection drugs for about two months, because after that time the medications shouldn't be needed, Okarma said. The study will follow each patient for at least a year.
Okarma said he can't estimate how much such a therapy would cost if it proves effective, but that "this is not going to be a $500,000 price tag. It will be remarkably affordable ... in the context of the value it provides."
Evan Snyder, a stem cell researcher at the Burnham Institute for Medical Research in La Jolla, Calif., said scientists in the field will focus chiefly on the study's results about safety.
"The one hope that everybody has is that nothing bad happens," he said.
Geron Corp. has spent at least $100 million on human embryonic stem cell research. Founded in 1992, it does not have any therapies on the market.
However, the company is considered the world's leading embryonic stem cell developer thanks to its claims on several key stem cell technologies. Geron helped finance researchers at the University of Wisconsin who first isolated human embryonic stem cells in 1998. The company has retained exclusive rights on several of those cell types.
Friday, January 16, 2009
Cell Therapy Industry HiLites 2009-01-16
Ok, seriously, this week's HiLites blog almost killed me. Hope you find it useful. This was the week for cell therapy I thought last week would be. I'm going to try and recover now...
Dendreon's (NASDAQ: DNDN) Mitch Gold announced at this week's JP Morgan conference (click here to hear the presentation) that the IMPACT study of 500 men with terminal prostate cancer will offer a final answer by April on whether its experimental immune-boosting drug helps patients live longer. When the company went to the FDA with its BLA for PROVENGE in late 2006 and - despite the advisory committee's 13-4 vote recommending approval (March 2007) - the FDA voted against approving it at that point, it was this trial the FDA said they would prefer to see the data from before deciding. There is, as always, a much longer checkered history of ever-changing FDA reviewers, missed clinical endpoints, retrospective data analysis to find a patient subset for which the therapy worked, etc, etc all of which are partial drivers I'm sure for deciding to wait for the data from this second phase III study --- but it all comes down to this. In a mid-point preliminary review of the data, the endpoints were not met - but they were almost met. Mitch Gold is, of course, confident. As was the case in early 2007 there will be much speculation and the stock will be traded wildly and the cell therapy industry will wait with bated breath - though perhaps not quite as bated as 2 years ago when it seemed more rode on the decision than it does now.
The company has built its own manufacturing facility on (perhaps in due course only for) the US East Coast (New Jersey). They still own the worldwide rights to market the drug. They intend to go to the US market with a ~120-person sales force and plan to seek a commercialization partner for marketing outside the U.S.
In other late-stage cell therapy news, Osiris had a successful pre-BLA meeting with the FDA in which they reportedly reached agreement on the timing and content of their anticipated BLA submission. This will be the first US marketing application for a stem cell product making it much-anticipated, globally monitored, and precedent-setting. The entire industry has been waiting for this moment for several years (the last cell therapy approved being 10 years ago!) and will be watching this in much the same way as we were all watching Dendreon's BLA submission a couple year's ago.
Reported key results of the pre-BLA meeting were:
• FDA agreed with the proposed content and structure of the BLA for Prochymal for the treatment of GvHD.
• The primary endpoint of the Phase III trial that will be reviewed by FDA for approval was confirmed to be GvHD Complete Response (CR). A CR is complete remission of the disease.
• FDA indicated that the statistical analysis plan was appropriate.
• The overall safety database (Integrated Summary of Safety) of Prochymal is sufficient in scope for the indication. No additional clinical data beyond what was presented is anticipated to be necessary for the BLA.
• Agreement was reached on BLA requirements for Chemistry, Manufacturing and Controls (CMC) data.
• The FDA concurred with the proposed product stability and process validation plan for submission and provided guidance on how to present the data for ease of review.
• Agreement was reached on the timing and content of the BLA in rolling submission format. The rolling submission is an FDA provision available to drug candidates that have received Fast Track designation, which allows for completed sections of a BLA to be submitted on an ongoing basis. It can facilitate the process by allowing FDA to complete review of sections as soon as they are available. It is anticipated that the nonclinical sections, such as toxicology, will be the first submitted for review.
• The BLA will be in the electronic Common Technical Document (eCTD) format. The eCTD format facilitates the review of the BLA and allows parallel submission of the dossier in other territories.
• At the time of submission, Osiris will also submit the request for Priority Review of the Prochymal BLA. Investigational drugs with Fast Track designation are eligible for consideration for Priority Review, which provides for an accelerated six month application review by FDA
In other news not to be overshadowed by the news about the pre-BLA meeting, Osiris received FDA clearance to broaden the Prochymal Expanded Access Program (EAP) to patients of all ages suffering from life-threatening GvHD. In May of 2008, FDA approved the first Prochymal EAP for the treatment of pediatric GvHD patients. Congress and the FDA created the expanded access program to facilitate the availability of promising new drugs to desperately ill patients before general marketing begins.
If you're interested, click here for an interview of Osiris CEO, Randal Mills, done by PharmaExec.com this week.
Invitrogen launched a xenogeneic-free media for human embryonic stem cell culture.
After now at least 4 years of GE Healthcare's campaign to build a 9-figure cell therapy division, a rocky relationship with Thermogenesis, and the splashy acquisition of Wave Biotech, GE has made another entree into the sector with its announcement of a partnership with Cytori (NASDAQ: CYTX) to commercialize Cytori’s Celution® System in select European countries - exclusive for 18 mos in select countries. While GE is admittedly not lighting any fires with the pace in which they are building this division it is presumably (hopefully) strategic, synergistic, supportive of its overall business and...please let this be true...something Jeff Immelt will continue to wholeheartedly support despite the perpetual and perpetual re-orgs within the company.
Celprogen claims to have successfully de-differentiated adult somatic cells (primary skin fibroblast, keratinocytes, cardiac, liver and neuronal cells) to their embryonic pluripotent cells (iPCs) using a proprietary non-viral method involving their de-differentation media and matrix system. The iPCs were then reportedly differentiated into cardiac, neuronal and hepatic tissue with Celprogen’s Stem Cell Differentiation system.
The PerkinElmer ViaCord Research Institute and M. D. Anderson Cancer Center have agreed to extend collaboration on a study of cord blood stem expansion systems in adult transplantation. The study seeks to discover methods of treating more adults using cord blood stem cells using 'co-culture' expansion technology. Based on the results of this trial, ViaCord, PerkinElmer's cord blood banking business, and M.D. Anderson will determine the feasibility of using Unrestricted Somatic Stem Cells (USSCs) derived from cord blood in the co-culture of cord blood units for transplantation. USSCs, proprietary to ViaCord (patent pending #09/985,335), are a type of stem cell found in umbilical cord blood, which have the ability to differentiate into many cell types, including endothelial cells, fat, bone, cartilage and neuronal cells.
ERYtech Pharma, French biotech company developing a pipeline of products involving the encapsulation of therapeutic molecules inside red blood cells for a variety of orphan indications, has signed an agreement with Penn Jersey Region of American Red Cross for the "GMP" clinical batches production of ERYtech Pharma’s products in the United States.
I'll seque momentarily to the regretably inevitable 'bad' news ...
On the verge of filing a BLA, Isolagen (Amex: ILE) is forced to downsize its operations to minimize burn-rate and postpone bankruptcy while exploring its 'options'. On November 6, 2008, the Company disclosed in an SEC Form 10-Q that there existed substantial doubt about its ability to continue as a going concern, and that its ability to continue as a going concern was contingent, among other things, upon its ability to secure additional adequate financing or capital prior to January 15, 2009 under its current operating plan and condition. The company announced it was pursuing dual paths, including pursuing potential financing alternatives as well as continuing potential strategic partnership discussion. As of this week neither event has happened according to an SEC filing.
The Company has now deviated from its normal operating plan and is focusing its remaining cash resources on specific clinical programs, namely efforts to complete its Biologics License Application related to the Company’s Phase III Nasolabial Fold study, and continuing its IT-R-008 Phase II/III Acne Scar trial. All other clinical programs have been suspended in order to preserve the Company’s remaining cash resources. The company believes it can survive for awhile but not sure how long. Its largest debt is a $90M liability which can be called by one of the noteholders as early as November.
Arbios Systems, Inc. (OTC: ABOS) has filed for chapter 11 bankruptcy protection while they continue to seek bids for the sale of the Company and/or its assets - namely the SEPET™ Liver Assist Device (a blood purification therapy designed for use with a standard blood dialysis system). The company shuttered its operation in August to focus "day-to-day operations exclusively on obtaining financing or consummating a strategic transaction'. In October the company sold all its rights and interest in its bioartificial liver system, HepatAssist, to HepaLife Technologies, Inc. (OTCBB: HPLF). This was really the cell therapy part of the company. The HepatAssist™ Cell-Based Liver Support System - a hollow fiber cartridge housing living porcine liver cells - purportedly functions as a bioartificial liver that not only detoxifies the blood, but, uniquely, provides whole liver function - intended to treat acute and acute-on-chronic liver failure patients.
This is a buyers market and the reality is many potential investors or purchasers are not motivated to buy a going concern for $x if they can scoop up the assets and/or business at a significant discount under distress. Regretable for those companies that have created and nurtured good technologies but allowed their business to fail; good news for those with cash; perhaps also good news - say some - for a much-needed reorg of an industry with a much more disciplined commercial focus.
Last month we talked a little about Bioheart (Nasdaq:BHRT) and their IPO less than a year ago raising just under $6 million. Now the stem cell company Bioheart has alerted the SEC that it missed a scheduled $180,000 loan payment and is seeking new money to keep the company going.
The Miami Herald reports that the Sunrise, FL-based developer has warned investors that it faces some tough choices including potential layoffs, bankruptcy and closing the business. But, reports Jim Carroll from Fierce Biotech reports, "the company's CEO, Howard Leonhardt, says the lender--BlueCrest Venture Finance Master Fund--has agreed to provide an extra 90 days for the company to make the payment. Bioheart, he adds, had to deal with an unexpected delay in receiving a $2.5 million loan and he insisted that the company is not in danger of closing."
According to Leonhardt, ''The last 24 months in a row we've paid on time and it was an unforeseen event. We're not thinking of bankruptcy at all; it's not even close to consideration -- maybe laying off employees.'' But all is not as serene as a pic of the debonair Leonhardt, accompanying the Herald story, would seem to suggest. A group of board directors and key executives have departed the company in the last two weeks, including CFO William Kline.
Nonetheless, there is reason to be optimistic despite the alarm bells. Bioheart - which is not just a one-trick cell therapy pony - recently announced the approval or release of four new products. In October, it launched in the United States and Europe a heart failure monitor that patients can use at home. Several major sales contracts are pending and the company has set a $20 million goal for 2009, Leonhardt said. It has also won FDA approval for the Bioheart Monebo CardioBelt, which heart patients wear around their waist, as well as commercial approval for two other technologies in Europe. Also, while I still have unanswered questions about who is supplying who with what, they also recently signed a cell supply agreement with Life Technologies to sell myoblast cell-based research products to life science researchers. By March, Leonhardt said, Bioheart should be on sound footing cash-wise and would try to fund operations solely with revenue and profits. Until then, however, ''we have to bring some more capital in now to bridge the gap,'' Leonhardt said.
On now to more optimistic fronts...
In a recent interview, CIRM President, Alan Trounson, said he (CIRM) is lobbying for the creation of an 'investment' fund - sponsored by government and health insurers - to drive commercialization of early regenerative medicines. He fears that without such a 'new paradigm', the lack of funding on the scale typically provided by the pharmaceutical industry, could well be a roadblock in regenerative medicines being brought to market. Trounson predicts within 10 years we'll have new drugs based on stem cell discoveries and clinical products being tested for spinal repair, retinal repair, cardiac function repair, multiple sclerosis, etc.
BioE recieved FDA 510(K) clearance for its cord blood processing system. PrepaCyte-CB is available immediately to public and private banks separating potentially therapeutic cells and stem cells from cord blood.
Pluristem Therapeutics (NasdaqCM: PSTI; DAX: PJT) filed an IND with the FDA to initiate a two-center Phase I clinical trial in the U.S. utilizing PLX-PAD - placental adherent stromal cells (ASCs) expanded in the Company's proprietary PluriX TM 3D bioreactor - for critical limb ischemia. The company filed a IMPD in Europe for a similar trial there.
The Swedish investment company Karolinska Development AB has made what it calls a "significant investment" in EvoStem Finland Oy - a company that develops stem cell and other tissue engineering based treatments for veterinary use (and intends to go human at some point). In 2007 EvoStem Finland Oy introduced to the Scandinavian market its first product TendoStem(R) - a stem cell based treatment service for tendon and ligament injuries in horses.
Kiadis Pharma has recently come out with a bunch of news (some of which I'm a little late reporting). Most recently they Dr. Maarten Egeler, MD PhD as Chief Medical Officer and in December they reported the appointment of Mark Skaletsky to its Supervisory Board and, most significantly, announced they have received two orphan drug designations for Reviroc™ from the FDA - one for diffuse large B-cell lymphoma and the other one for the treatment of follicular lymphoma. Reviroc™ is under development for the elimination of cancer cells from an autologous graft in bone marrow transplantations for end-stage blood cancer patients.
Skaletscky, for those of you who don't know him, is very well credentialed. He served as president and CEO of GelTex Pharmaceuticals, Inc. (acquired by Genzyme for more than $1 billion in December 2000), was chairman and CEO of Enzytech, Inc., and president and Chief Operating Officer of Biogen, Inc. He is also a former chairman of the board of directors of the Biotechnology Industry Organization (BIO). Currently he is chairman, CEO and president of Fenway Pharmaceuticals, Inc.
Let me digress momentarily with a little history and speculation...
In 2001, Theratechnologies (TSX:TH) reorganized its business activities through the transfer of its ex vivo photodynamic cell therapy program to its newly created subsidiary, Celmed BioSciences, the acquisition of NeuroGeneration and NeuroGenomics, two California corporations which were developing technologies for the treatment of neurodegenerative diseases, and investments totaling $60 million by Societe generale de financement du Quebec (SGF) and the Solidarity Fund QFL. Michel F. Levesque, founder of NeuroGeneration became Vice President, Medical Affairs with Celmed BioSciences.
In 2006 Kiadis B.V and Celmed BioSciences Inc. merged the two oncology-focused companies. At that time, CelMed Biosciences was a wholly-owned subsidiary of Theratechnoloies (another Montreal company). The new combined company was named Kiadis Pharma B.V. Investors include Alta Partners, LSP, and DFJ Esprit. At or around that time, Levesque took back NeurGeneration which he is now attempting to re-launch from his practice in Hollywood, CA.
Kiadis still describes itself as a "Dutch-Canadian enterprise" which is "endorsed by our investors, academic partners and a cross-Atlantic management team". There is much cross-pollination between Montreal and Amerstdam. For instance, Pierre Caudrelier, was until this week, CMO at Theratechnologies, was previously CMO at Kiadis Pharm, VP of Clinical Research at Celmed, and VP of Clinical Research and General Manager the the Canadian subsidiary of IDM (another company that had cell therapy products with US-EU connections). Research collaborations also exist with Italy, Germany and the US (including NIH's John Barrett).
Here comes the complete and utter speculation. On the one hand we have an unnamed fairly large multi-national company (not GE) looking for acquisitions in the cell therapy space. Links have been established with personnel at Theratechnologies - a company which I believe no longer has any cell-based therapy research. On the other hand, totally unrelated to this, I am led to believe that Kiadis is looking for a partner with money. Other than some personnel and history, there would not appear to be anything still linking Theratechnologies and Kiadis. However, just to make things a little curious Theratechnologies announced this week that Dr. Pierre Caudrelier was leaving his position as chief medical officer to pursue other career opportunities. Despite the complete lack of any evidence other than this tenuous string of bits and bytes, something tickles my senses here. I'm betting there is more to this puzzle than I have yet been able to put together (or am willing to say out loud at this point). Stay tuned.
On the regulatory front...
FDA CBER has announced their Cellular, Tissue and Gene Therapies Advisory Committee 2009 meeting schedule: May 14-15 and November 5-6
For those of you using genetically engineered pigs for to grow cells, tissues, or organs that can be transplanted into humans with a reduced risk of immune rejection, you will know the FDA this week issued its Final Guidance on Regulation of Genetically Engineered Animals. For the rest of you, I can't think of better night time reading...
The FDA issued a Draft Guidance for Industry entitled: "Current Good Tissue Practice (CGTP) and Additional Requirements for Manufacturers of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps)" [pdf version]. Although you can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit written or electronic comments on the draft guidance by April 16, 2009.
Let me mop up with a few final lighter entries.
Rurther to my blog on the subject back in October, BioTechniques just released interesting results on a survey of the extent to which scientists are using social media tools and which one for what. See more details in the comments section of my original blog post of social media use.
If you're planning to be at the Phacilitate Cell & Gene Therapy Forum in Washington, D.C. January 26-28 (and you should plan to be there if you have any interest in this field), here's a couple things to watch for:
- DCi Biotech consultant, Dawn Driscoll is moderating a lunchtime session on Tuesday the 27th hosted by Progenitor Cell Therapy in which yours truly, Robert Preti, and RTI Health Solutions, Eric Faulkner will be speaking. The session is entitled"Select Issues in Commercializing Cell Therapies" and will include a talk by Faulkner on positioning cell therapies for reimbursement.
For more details, click here.
- I will be there with the founders of BioBusiness.TV who will be there to capture some interviews from cell therapy industry executives for a cell therapy channel we are opening for the site.
- Anyone interested in potentially incorporating in vivo cell tracking/imaging in their cell therapy clinical trial, come find me.
Ok...one more thing just for kicks. For a chuckle and then a shake of the head in dismay check out the Niche blog on ESC-derivative facials! The hype is really getting out of control.
____
That's it. That's all I've got for you. That, my friends, is how the cell therapy industry hit me me this week.
--Lee
p.s. Quick question: Do you mind/like this rather rambling format or should I get more organized somehow or another? Comment below...
Dendreon's (NASDAQ: DNDN) Mitch Gold announced at this week's JP Morgan conference (click here to hear the presentation) that the IMPACT study of 500 men with terminal prostate cancer will offer a final answer by April on whether its experimental immune-boosting drug helps patients live longer. When the company went to the FDA with its BLA for PROVENGE in late 2006 and - despite the advisory committee's 13-4 vote recommending approval (March 2007) - the FDA voted against approving it at that point, it was this trial the FDA said they would prefer to see the data from before deciding. There is, as always, a much longer checkered history of ever-changing FDA reviewers, missed clinical endpoints, retrospective data analysis to find a patient subset for which the therapy worked, etc, etc all of which are partial drivers I'm sure for deciding to wait for the data from this second phase III study --- but it all comes down to this. In a mid-point preliminary review of the data, the endpoints were not met - but they were almost met. Mitch Gold is, of course, confident. As was the case in early 2007 there will be much speculation and the stock will be traded wildly and the cell therapy industry will wait with bated breath - though perhaps not quite as bated as 2 years ago when it seemed more rode on the decision than it does now.
The company has built its own manufacturing facility on (perhaps in due course only for) the US East Coast (New Jersey). They still own the worldwide rights to market the drug. They intend to go to the US market with a ~120-person sales force and plan to seek a commercialization partner for marketing outside the U.S.
In other late-stage cell therapy news, Osiris had a successful pre-BLA meeting with the FDA in which they reportedly reached agreement on the timing and content of their anticipated BLA submission. This will be the first US marketing application for a stem cell product making it much-anticipated, globally monitored, and precedent-setting. The entire industry has been waiting for this moment for several years (the last cell therapy approved being 10 years ago!) and will be watching this in much the same way as we were all watching Dendreon's BLA submission a couple year's ago.
Reported key results of the pre-BLA meeting were:
• FDA agreed with the proposed content and structure of the BLA for Prochymal for the treatment of GvHD.
• The primary endpoint of the Phase III trial that will be reviewed by FDA for approval was confirmed to be GvHD Complete Response (CR). A CR is complete remission of the disease.
• FDA indicated that the statistical analysis plan was appropriate.
• The overall safety database (Integrated Summary of Safety) of Prochymal is sufficient in scope for the indication. No additional clinical data beyond what was presented is anticipated to be necessary for the BLA.
• Agreement was reached on BLA requirements for Chemistry, Manufacturing and Controls (CMC) data.
• The FDA concurred with the proposed product stability and process validation plan for submission and provided guidance on how to present the data for ease of review.
• Agreement was reached on the timing and content of the BLA in rolling submission format. The rolling submission is an FDA provision available to drug candidates that have received Fast Track designation, which allows for completed sections of a BLA to be submitted on an ongoing basis. It can facilitate the process by allowing FDA to complete review of sections as soon as they are available. It is anticipated that the nonclinical sections, such as toxicology, will be the first submitted for review.
• The BLA will be in the electronic Common Technical Document (eCTD) format. The eCTD format facilitates the review of the BLA and allows parallel submission of the dossier in other territories.
• At the time of submission, Osiris will also submit the request for Priority Review of the Prochymal BLA. Investigational drugs with Fast Track designation are eligible for consideration for Priority Review, which provides for an accelerated six month application review by FDA
In other news not to be overshadowed by the news about the pre-BLA meeting, Osiris received FDA clearance to broaden the Prochymal Expanded Access Program (EAP) to patients of all ages suffering from life-threatening GvHD. In May of 2008, FDA approved the first Prochymal EAP for the treatment of pediatric GvHD patients. Congress and the FDA created the expanded access program to facilitate the availability of promising new drugs to desperately ill patients before general marketing begins.
If you're interested, click here for an interview of Osiris CEO, Randal Mills, done by PharmaExec.com this week.
Invitrogen launched a xenogeneic-free media for human embryonic stem cell culture.
After now at least 4 years of GE Healthcare's campaign to build a 9-figure cell therapy division, a rocky relationship with Thermogenesis, and the splashy acquisition of Wave Biotech, GE has made another entree into the sector with its announcement of a partnership with Cytori (NASDAQ: CYTX) to commercialize Cytori’s Celution® System in select European countries - exclusive for 18 mos in select countries. While GE is admittedly not lighting any fires with the pace in which they are building this division it is presumably (hopefully) strategic, synergistic, supportive of its overall business and...please let this be true...something Jeff Immelt will continue to wholeheartedly support despite the perpetual and perpetual re-orgs within the company.
Celprogen claims to have successfully de-differentiated adult somatic cells (primary skin fibroblast, keratinocytes, cardiac, liver and neuronal cells) to their embryonic pluripotent cells (iPCs) using a proprietary non-viral method involving their de-differentation media and matrix system. The iPCs were then reportedly differentiated into cardiac, neuronal and hepatic tissue with Celprogen’s Stem Cell Differentiation system.
The PerkinElmer ViaCord Research Institute and M. D. Anderson Cancer Center have agreed to extend collaboration on a study of cord blood stem expansion systems in adult transplantation. The study seeks to discover methods of treating more adults using cord blood stem cells using 'co-culture' expansion technology. Based on the results of this trial, ViaCord, PerkinElmer's cord blood banking business, and M.D. Anderson will determine the feasibility of using Unrestricted Somatic Stem Cells (USSCs) derived from cord blood in the co-culture of cord blood units for transplantation. USSCs, proprietary to ViaCord (patent pending #09/985,335), are a type of stem cell found in umbilical cord blood, which have the ability to differentiate into many cell types, including endothelial cells, fat, bone, cartilage and neuronal cells.
ERYtech Pharma, French biotech company developing a pipeline of products involving the encapsulation of therapeutic molecules inside red blood cells for a variety of orphan indications, has signed an agreement with Penn Jersey Region of American Red Cross for the "GMP" clinical batches production of ERYtech Pharma’s products in the United States.
I'll seque momentarily to the regretably inevitable 'bad' news ...
On the verge of filing a BLA, Isolagen (Amex: ILE) is forced to downsize its operations to minimize burn-rate and postpone bankruptcy while exploring its 'options'. On November 6, 2008, the Company disclosed in an SEC Form 10-Q that there existed substantial doubt about its ability to continue as a going concern, and that its ability to continue as a going concern was contingent, among other things, upon its ability to secure additional adequate financing or capital prior to January 15, 2009 under its current operating plan and condition. The company announced it was pursuing dual paths, including pursuing potential financing alternatives as well as continuing potential strategic partnership discussion. As of this week neither event has happened according to an SEC filing.
The Company has now deviated from its normal operating plan and is focusing its remaining cash resources on specific clinical programs, namely efforts to complete its Biologics License Application related to the Company’s Phase III Nasolabial Fold study, and continuing its IT-R-008 Phase II/III Acne Scar trial. All other clinical programs have been suspended in order to preserve the Company’s remaining cash resources. The company believes it can survive for awhile but not sure how long. Its largest debt is a $90M liability which can be called by one of the noteholders as early as November.
Arbios Systems, Inc. (OTC: ABOS) has filed for chapter 11 bankruptcy protection while they continue to seek bids for the sale of the Company and/or its assets - namely the SEPET™ Liver Assist Device (a blood purification therapy designed for use with a standard blood dialysis system). The company shuttered its operation in August to focus "day-to-day operations exclusively on obtaining financing or consummating a strategic transaction'. In October the company sold all its rights and interest in its bioartificial liver system, HepatAssist, to HepaLife Technologies, Inc. (OTCBB: HPLF). This was really the cell therapy part of the company. The HepatAssist™ Cell-Based Liver Support System - a hollow fiber cartridge housing living porcine liver cells - purportedly functions as a bioartificial liver that not only detoxifies the blood, but, uniquely, provides whole liver function - intended to treat acute and acute-on-chronic liver failure patients.
This is a buyers market and the reality is many potential investors or purchasers are not motivated to buy a going concern for $x if they can scoop up the assets and/or business at a significant discount under distress. Regretable for those companies that have created and nurtured good technologies but allowed their business to fail; good news for those with cash; perhaps also good news - say some - for a much-needed reorg of an industry with a much more disciplined commercial focus.
Last month we talked a little about Bioheart (Nasdaq:BHRT) and their IPO less than a year ago raising just under $6 million. Now the stem cell company Bioheart has alerted the SEC that it missed a scheduled $180,000 loan payment and is seeking new money to keep the company going.
The Miami Herald reports that the Sunrise, FL-based developer has warned investors that it faces some tough choices including potential layoffs, bankruptcy and closing the business. But, reports Jim Carroll from Fierce Biotech reports, "the company's CEO, Howard Leonhardt, says the lender--BlueCrest Venture Finance Master Fund--has agreed to provide an extra 90 days for the company to make the payment. Bioheart, he adds, had to deal with an unexpected delay in receiving a $2.5 million loan and he insisted that the company is not in danger of closing."
According to Leonhardt, ''The last 24 months in a row we've paid on time and it was an unforeseen event. We're not thinking of bankruptcy at all; it's not even close to consideration -- maybe laying off employees.'' But all is not as serene as a pic of the debonair Leonhardt, accompanying the Herald story, would seem to suggest. A group of board directors and key executives have departed the company in the last two weeks, including CFO William Kline.
Nonetheless, there is reason to be optimistic despite the alarm bells. Bioheart - which is not just a one-trick cell therapy pony - recently announced the approval or release of four new products. In October, it launched in the United States and Europe a heart failure monitor that patients can use at home. Several major sales contracts are pending and the company has set a $20 million goal for 2009, Leonhardt said. It has also won FDA approval for the Bioheart Monebo CardioBelt, which heart patients wear around their waist, as well as commercial approval for two other technologies in Europe. Also, while I still have unanswered questions about who is supplying who with what, they also recently signed a cell supply agreement with Life Technologies to sell myoblast cell-based research products to life science researchers. By March, Leonhardt said, Bioheart should be on sound footing cash-wise and would try to fund operations solely with revenue and profits. Until then, however, ''we have to bring some more capital in now to bridge the gap,'' Leonhardt said.
On now to more optimistic fronts...
In a recent interview, CIRM President, Alan Trounson, said he (CIRM) is lobbying for the creation of an 'investment' fund - sponsored by government and health insurers - to drive commercialization of early regenerative medicines. He fears that without such a 'new paradigm', the lack of funding on the scale typically provided by the pharmaceutical industry, could well be a roadblock in regenerative medicines being brought to market. Trounson predicts within 10 years we'll have new drugs based on stem cell discoveries and clinical products being tested for spinal repair, retinal repair, cardiac function repair, multiple sclerosis, etc.
BioE recieved FDA 510(K) clearance for its cord blood processing system. PrepaCyte-CB is available immediately to public and private banks separating potentially therapeutic cells and stem cells from cord blood.
Pluristem Therapeutics (NasdaqCM: PSTI; DAX: PJT) filed an IND with the FDA to initiate a two-center Phase I clinical trial in the U.S. utilizing PLX-PAD - placental adherent stromal cells (ASCs) expanded in the Company's proprietary PluriX TM 3D bioreactor - for critical limb ischemia. The company filed a IMPD in Europe for a similar trial there.
The Swedish investment company Karolinska Development AB has made what it calls a "significant investment" in EvoStem Finland Oy - a company that develops stem cell and other tissue engineering based treatments for veterinary use (and intends to go human at some point). In 2007 EvoStem Finland Oy introduced to the Scandinavian market its first product TendoStem(R) - a stem cell based treatment service for tendon and ligament injuries in horses.
Kiadis Pharma has recently come out with a bunch of news (some of which I'm a little late reporting). Most recently they Dr. Maarten Egeler, MD PhD as Chief Medical Officer and in December they reported the appointment of Mark Skaletsky to its Supervisory Board and, most significantly, announced they have received two orphan drug designations for Reviroc™ from the FDA - one for diffuse large B-cell lymphoma and the other one for the treatment of follicular lymphoma. Reviroc™ is under development for the elimination of cancer cells from an autologous graft in bone marrow transplantations for end-stage blood cancer patients.
Skaletscky, for those of you who don't know him, is very well credentialed. He served as president and CEO of GelTex Pharmaceuticals, Inc. (acquired by Genzyme for more than $1 billion in December 2000), was chairman and CEO of Enzytech, Inc., and president and Chief Operating Officer of Biogen, Inc. He is also a former chairman of the board of directors of the Biotechnology Industry Organization (BIO). Currently he is chairman, CEO and president of Fenway Pharmaceuticals, Inc.
Let me digress momentarily with a little history and speculation...
In 2001, Theratechnologies (TSX:TH) reorganized its business activities through the transfer of its ex vivo photodynamic cell therapy program to its newly created subsidiary, Celmed BioSciences, the acquisition of NeuroGeneration and NeuroGenomics, two California corporations which were developing technologies for the treatment of neurodegenerative diseases, and investments totaling $60 million by Societe generale de financement du Quebec (SGF) and the Solidarity Fund QFL. Michel F. Levesque, founder of NeuroGeneration became Vice President, Medical Affairs with Celmed BioSciences.
In 2006 Kiadis B.V and Celmed BioSciences Inc. merged the two oncology-focused companies. At that time, CelMed Biosciences was a wholly-owned subsidiary of Theratechnoloies (another Montreal company). The new combined company was named Kiadis Pharma B.V. Investors include Alta Partners, LSP, and DFJ Esprit. At or around that time, Levesque took back NeurGeneration which he is now attempting to re-launch from his practice in Hollywood, CA.
Kiadis still describes itself as a "Dutch-Canadian enterprise" which is "endorsed by our investors, academic partners and a cross-Atlantic management team". There is much cross-pollination between Montreal and Amerstdam. For instance, Pierre Caudrelier, was until this week, CMO at Theratechnologies, was previously CMO at Kiadis Pharm, VP of Clinical Research at Celmed, and VP of Clinical Research and General Manager the the Canadian subsidiary of IDM (another company that had cell therapy products with US-EU connections). Research collaborations also exist with Italy, Germany and the US (including NIH's John Barrett).
Here comes the complete and utter speculation. On the one hand we have an unnamed fairly large multi-national company (not GE) looking for acquisitions in the cell therapy space. Links have been established with personnel at Theratechnologies - a company which I believe no longer has any cell-based therapy research. On the other hand, totally unrelated to this, I am led to believe that Kiadis is looking for a partner with money. Other than some personnel and history, there would not appear to be anything still linking Theratechnologies and Kiadis. However, just to make things a little curious Theratechnologies announced this week that Dr. Pierre Caudrelier was leaving his position as chief medical officer to pursue other career opportunities. Despite the complete lack of any evidence other than this tenuous string of bits and bytes, something tickles my senses here. I'm betting there is more to this puzzle than I have yet been able to put together (or am willing to say out loud at this point). Stay tuned.
On the regulatory front...
FDA CBER has announced their Cellular, Tissue and Gene Therapies Advisory Committee 2009 meeting schedule: May 14-15 and November 5-6
For those of you using genetically engineered pigs for to grow cells, tissues, or organs that can be transplanted into humans with a reduced risk of immune rejection, you will know the FDA this week issued its Final Guidance on Regulation of Genetically Engineered Animals. For the rest of you, I can't think of better night time reading...
The FDA issued a Draft Guidance for Industry entitled: "Current Good Tissue Practice (CGTP) and Additional Requirements for Manufacturers of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps)" [pdf version]. Although you can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit written or electronic comments on the draft guidance by April 16, 2009.
Let me mop up with a few final lighter entries.
Rurther to my blog on the subject back in October, BioTechniques just released interesting results on a survey of the extent to which scientists are using social media tools and which one for what. See more details in the comments section of my original blog post of social media use.
If you're planning to be at the Phacilitate Cell & Gene Therapy Forum in Washington, D.C. January 26-28 (and you should plan to be there if you have any interest in this field), here's a couple things to watch for:
- DCi Biotech consultant, Dawn Driscoll is moderating a lunchtime session on Tuesday the 27th hosted by Progenitor Cell Therapy in which yours truly, Robert Preti, and RTI Health Solutions, Eric Faulkner will be speaking. The session is entitled"Select Issues in Commercializing Cell Therapies" and will include a talk by Faulkner on positioning cell therapies for reimbursement.
For more details, click here.
- I will be there with the founders of BioBusiness.TV who will be there to capture some interviews from cell therapy industry executives for a cell therapy channel we are opening for the site.
- Anyone interested in potentially incorporating in vivo cell tracking/imaging in their cell therapy clinical trial, come find me.
Ok...one more thing just for kicks. For a chuckle and then a shake of the head in dismay check out the Niche blog on ESC-derivative facials! The hype is really getting out of control.
____
That's it. That's all I've got for you. That, my friends, is how the cell therapy industry hit me me this week.
--Lee
p.s. Quick question: Do you mind/like this rather rambling format or should I get more organized somehow or another? Comment below...
Friday, January 9, 2009
Cell Therapy Industry HiLites 2009-01-09
Ok so there was no deluge of cell therapy news this week despite my fears (hopes?). The only flood was the river (nee creek) behind my house. Wow, water can rise fast! Anyway...that's not cell therapy, THIS is cell therapy:
Osiris Therapeutics (NASDAQ:OSIR) received a $5 million milestone payment for reaching the first production threshold of the Osteocel supply agreement with NuVasive. In July of 2008, Osiris announced the sale of the Osteocel business to NuVasive for an initial payment of $35 million, followed by up to $50 million in additional milestones. Osiris expects to achieve the remaining $45 million in milestone payments from NuVasive, including $17.5 million for further delivery of product, $12.5 million upon transfer of certain manufacturing assets, and a $15 million payment upon NuVasive’s achievement of $35 million in cumulative Osteocel sales.
After announcing last week that they had raised $1M of an anticipated $5M round of financing, International Stem Cell Corporation (OTCBB:ISCO) issued a press release to 'discuss' the recent funding. The additional funds are to be raised in four $1M tranches in February, March, June and September.
They need the additional funds to meet their goals for 2009, namely
(1) eliminating $1 million of outstanding secured debt;
(2) supporting first quarter pre-clinical trials; and
(3) providing marketing and expansion capital to position the Company`s "Lifeline" subsidiary - which makes and sells specialty cells and growth media - to the point where it will be generating profit (within two years) that they can then plow back into the company's clinical research.
I'm no corporate finance expert but this is what I read from the SEC filing behind the press releases. Andrei Semechkin, Rouslan Semechkin, and X-Master, Inc were the investors putting up the $1M to-date. By way of a "material and essential inducement for the Investors to enter into the Preferred Stock Purchase Agreement, the Company agreed to employ Rouslan Semechkin and Andrei Semechkin in accordance with the terms" of the employment agreements attached to the stock purchase agreement. Andrei is going to be paid at least $180,000 per year for the next 5 years as the company's Chief Business Officer and Rouslan is also on salary.
So, provided ISCO stays around, Andrei and Rouslan will get their $1M back over 5 years in addition to whatever benefits they may glean from their shares. This certainly seems like a nice, low - albeit not entirely without - risk arrangement. On the other hand, this could be seen as them putting their own money in first where they are asking others to follow or they may buck up more money on the subsequent tranches. However this plays out, it is another testament to the fact that in biotech, survivability is so-oft closely if not directly tied to financing creativity!
You likely won't find a press release about this one but J&J is quietly shuttering at least two of their cell therapy programs. Their autologous HIV cell therapy program and their Treg program are both on the out-licensing block after corporate decided to shut down the programs - the former after reportedly 'good' phase II data and the latter even before it went clinical. J&J is not announcing what's behind the decisions though unofficial reports are that it is not a lack of belief in cell therapies overall. Speculation is that it is primarily HQ needing to conserve spending on longer-term programs. Whatever is behind the decisions, it's a little disappointing because for a couple of years now J&J has been quietly been the largest cell therapy company in the world if one added up all their cell therapy internal programs and external investments. They may well still deserve this title but they're a little smaller this month then they were before.
So...since it was a little slow on the industry front, I'll finish with three non-business related items I found interesting this week:
WIRED has published its much-read list of top scientific breakthroughs of the past year and not only do life sciences figure prominently in the top-10 but two of them are cell therapy related. On the list: A new approach to reprogramming stem cells to avoid a tendency to become cancerous; using a bone marrow transplant from an HIV-resistant donor in Germany to eradicate the virus, pointing to gene editing as a possible cure; and using a patient's stem cells to grow a new trachea. The new trachea was number two on the hit parade and earned kudos as the greatest single breakthrough in life sciences.
Adding fuel to the 'cancer stem cell' debate, Canadian researcher Mick Bhatia, and his team, have published a letter abstract in Nature Biotechnology, on a series of tests they claim can distinguish between cancer stem cells and the good kind. Thus enabling, inter alia, the better targeting of cancer stem cells.
Cell Transplantation has become an "open access" journal from the 1st January 2009, starting with volume 18, making it available on the World Wide Web without subscription to researchers and clinicians as well the public and members of the media.
Finally, Dawn Driscoll (DCi Biotech) and I are putting on a 1/2-day course entitled Commercial Considerations for Cell-Based Therapies Feb. 24 at the Moscone Center in San Francisco immediately prior to CHI's Stem Cell Congress (part of their Molecular Medicine Tri-Conference). Let me know if you're interested.
That's cell therapy the way I saw it in this first whole week of 2009...
Osiris Therapeutics (NASDAQ:OSIR) received a $5 million milestone payment for reaching the first production threshold of the Osteocel supply agreement with NuVasive. In July of 2008, Osiris announced the sale of the Osteocel business to NuVasive for an initial payment of $35 million, followed by up to $50 million in additional milestones. Osiris expects to achieve the remaining $45 million in milestone payments from NuVasive, including $17.5 million for further delivery of product, $12.5 million upon transfer of certain manufacturing assets, and a $15 million payment upon NuVasive’s achievement of $35 million in cumulative Osteocel sales.
After announcing last week that they had raised $1M of an anticipated $5M round of financing, International Stem Cell Corporation (OTCBB:ISCO) issued a press release to 'discuss' the recent funding. The additional funds are to be raised in four $1M tranches in February, March, June and September.
They need the additional funds to meet their goals for 2009, namely
(1) eliminating $1 million of outstanding secured debt;
(2) supporting first quarter pre-clinical trials; and
(3) providing marketing and expansion capital to position the Company`s "Lifeline" subsidiary - which makes and sells specialty cells and growth media - to the point where it will be generating profit (within two years) that they can then plow back into the company's clinical research.
I'm no corporate finance expert but this is what I read from the SEC filing behind the press releases. Andrei Semechkin, Rouslan Semechkin, and X-Master, Inc were the investors putting up the $1M to-date. By way of a "material and essential inducement for the Investors to enter into the Preferred Stock Purchase Agreement, the Company agreed to employ Rouslan Semechkin and Andrei Semechkin in accordance with the terms" of the employment agreements attached to the stock purchase agreement. Andrei is going to be paid at least $180,000 per year for the next 5 years as the company's Chief Business Officer and Rouslan is also on salary.
So, provided ISCO stays around, Andrei and Rouslan will get their $1M back over 5 years in addition to whatever benefits they may glean from their shares. This certainly seems like a nice, low - albeit not entirely without - risk arrangement. On the other hand, this could be seen as them putting their own money in first where they are asking others to follow or they may buck up more money on the subsequent tranches. However this plays out, it is another testament to the fact that in biotech, survivability is so-oft closely if not directly tied to financing creativity!
You likely won't find a press release about this one but J&J is quietly shuttering at least two of their cell therapy programs. Their autologous HIV cell therapy program and their Treg program are both on the out-licensing block after corporate decided to shut down the programs - the former after reportedly 'good' phase II data and the latter even before it went clinical. J&J is not announcing what's behind the decisions though unofficial reports are that it is not a lack of belief in cell therapies overall. Speculation is that it is primarily HQ needing to conserve spending on longer-term programs. Whatever is behind the decisions, it's a little disappointing because for a couple of years now J&J has been quietly been the largest cell therapy company in the world if one added up all their cell therapy internal programs and external investments. They may well still deserve this title but they're a little smaller this month then they were before.
So...since it was a little slow on the industry front, I'll finish with three non-business related items I found interesting this week:
WIRED has published its much-read list of top scientific breakthroughs of the past year and not only do life sciences figure prominently in the top-10 but two of them are cell therapy related. On the list: A new approach to reprogramming stem cells to avoid a tendency to become cancerous; using a bone marrow transplant from an HIV-resistant donor in Germany to eradicate the virus, pointing to gene editing as a possible cure; and using a patient's stem cells to grow a new trachea. The new trachea was number two on the hit parade and earned kudos as the greatest single breakthrough in life sciences.
Adding fuel to the 'cancer stem cell' debate, Canadian researcher Mick Bhatia, and his team, have published a letter abstract in Nature Biotechnology, on a series of tests they claim can distinguish between cancer stem cells and the good kind. Thus enabling, inter alia, the better targeting of cancer stem cells.
Cell Transplantation has become an "open access" journal from the 1st January 2009, starting with volume 18, making it available on the World Wide Web without subscription to researchers and clinicians as well the public and members of the media.
Finally, Dawn Driscoll (DCi Biotech) and I are putting on a 1/2-day course entitled Commercial Considerations for Cell-Based Therapies Feb. 24 at the Moscone Center in San Francisco immediately prior to CHI's Stem Cell Congress (part of their Molecular Medicine Tri-Conference). Let me know if you're interested.
That's cell therapy the way I saw it in this first whole week of 2009...
Saturday, January 3, 2009
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