We've sent out a couple tweets from @celltherapyblog this week that we have now tracked over $1 billion having been brought in by companies in the cell therapy and cell-based regenerative medicine space this year to-date.
We promise to post a breakdown of this stat updating our most recent post on the subject back in July but we want to wait until the month ends and we see a number of pending financings close.
We're closing the week with a Fibrocell Sciences ($FCSC) PIPE that was supposed to close Oct 11, an Immunocellular Therapeutics ($IMUC) secondary offering that supposed to close Oct 23, and an Athersys ($ATHX) secondary offering that aims to close by Halloween.
Watch for more details the first full week of November.
Friday, October 26, 2012
Thursday, October 18, 2012
My cell therapy index portfolio
So there are certainly analysts, traders, and brokers who cover the public segment of the cell therapy industry with much more expertise and experience than I. Certainly among the top of this crop is Henry W. McCusker of Scimitar Equity (@henryw09 on Twitter) who is preparing to launch a major new online initiative that I'm very excited about covering the entire regenerative medicine sector focused primarily on the public companies in the sector.
I tend to focus more attention on the activity of private companies in the sector that tend to get a lot less spotlight but as you know I have created a portfolio of public cell therapy companies comprised of the 30 companies listed below (linked to their page on Google Finance).
I've been watching this portfolio since the beginning of the calendar year (January 2012) and have been commenting on its performance as against some of the major indices. (Oct 8 post and August 10 post).
In addition to these occasional reports I will occasionally report on Twitter (@celltherapyblog) on the stocks in this portfolio which are enjoying the largest gains/losses and which have the largest/smallest trading volumes. From time-to-time I will aggregate this content into a blog post.
Tuesday, October 16, 2012
CIRM addresses some tough questions. Is it all just glass towers and basic research?
At an industry conference recently I heard several new grumbles from companies about CIRM's alleged heavy bias toward funding basic, pre-clinical, embryonic stem cell-focused, academic-based research over clinical-stage, adult stem cell-focused, industry-sponsored product trials, testing, and development.
I myself have shared some concern that for an agency with a key goal of bringing new medicines to the next generation, having less than a handful of projects at the clinical stage this far into its mandate and budget was falling short well of its timeline.
I'll also admit to occasionally harboring a similar sentiment to that of former Intel CEO, Andy Grove, who is, of late, a grumpy critic of the slow pace of life science research when he said of CIRM in a great piece by Jeffrey O'Brien in Fortune Magazine, "CIRM? "There are gleaming fucking buildings everywhere. That wasn't necessary." (The great stem cell dilemma. Fortune. Sept 28, 2012)
So...I decided to try to hit these concerns and criticisms head on with my friends at the California Institute for Regenerative Medicine (CIRM).
What follows is an online interview CellTherapyBlog.com (CTB) conducted with the California Institute for Regenerative Medicine (CIRM) the week of October 15, 2012. In the interview that follows, we were particularly interested in addressing the degree to which CIRM is focused - moving forward - on funding clinical-stage research, industry-sponsored trials, and clinical/commercial-focused product development.
CTB: Would you please remind us of CIRM’s mandate?
CIRM: “To support and advance stem cell research and regenerative medicine under the highest ethical and medical standards for the discovery and development of cures, therapies, diagnostics and research technologies to relieve human suffering from chronic disease and injury.”
CTB: What percentage of grants or grant money distributed to-date has gone to companies?
CIRM: For-profit entities have been and currently are eligible for CIRM funding covering stages of research which range from basic biology programs (in which industry has shown little interest) through Phase II clinical trials. Of these programs, 13% have been awarded to companies thus far. Having built 12 state of the art stem cell facilities and having seeded the field with training and other types of grants of similar purpose, CIRM is now focusing on funding translational and clinical programs.
This is where companies' primary interests are and we expect greater company participation in our translation and clinical Request for Application. The translation and clinical awards programs provide for much larger awards as compared to the basic research and the overall amount of later stage funding is significantly larger than the earlier basic research awards. The number of awards made in the translational and clinical development funding rounds is much less than in the basic science area.
CIRM’s Strategic Partnership Funding Program is a cornerstone of our efforts to fund industry. We expect to make awards through this program approximately every six months to assist companies whose financing demands is frequently at shorter intervals than academic institutions. These awards will be made following a robust peer review process ensuring that awards are made to projects that are based on sound scientific data and have a reasonable chance of success.
CTB: How many CIRM-funded projects will be in clinical trial this year? How many anticipated to be in 2013?
CIRM: Four clinical trials that were fostered by CIRM funds are already in clinical trials for cancer and blood disorders. We expect one or more CIRM-funded projects to join that list in the next year. This includes projects that are in clinical trial already for which we have funded and are funding the follow on studies.
CTB: Is CIRM actively seeking applications for clinical-stage projects? from companies?
CIRM: Yes, we have recently held the first round of applications for our Strategic Partnership Awards that are designed specifically to attract applications from industry and include significant leveraged funding from multinational biopharmaceutical companies and/or venture capital. The first of these awards will be announced at an upcoming meeting of our governing board, the Independent Citizens Oversight Committee. Industry also accesses CIRM funding through the Disease Team awards, which include teams comprised of both academic researchers and industry as partners, consultants and advisors.
CTB: In its funding to-date more CIRM funding has gone to pre-clinical over clinical science, embryonic over adult stem cell research, and infrastructure over labor. Is that a fair assessment?
CIRM: No. We have awarded more basic research grants in numbers, but those grants are much smaller in dollars than those in our translational portfolio. That translational portfolio includes 75 projects that have been awarded nearly $600 million, well over half of the research dollars committed.
When CIRM funding was initiated in late 2006, there was a need to build intellectual and facility capacity because doubts about support from federal sources had limited the entry of scientists into the field and there was a need for “safe harbor facilities. “ Research into stem cells was also at an early stage and so it made sense for us to focus on the discovery phase of basic biology and pre-clinical work to enable more effective utilization of the potential that was evident.
Increasingly however we are moving towards clinical science, to enable a proper assessment of the value of cell therapies and related approaches for advancement of human medicine.
Our focus has always included all stem and progenitor cells. Pluripotential stem cells are immortal and develop into all cells of the body, so the potential is large and the available funding outside CIRM has been modest. We have concentrated on human rather than animal model cells because this is where the need has been greatest. Our goal is to fund transformational research with the highest potential benefit to patients, regardless of the stem cell type they utilize.
As for infrastructure, we spent $271 million in major facilities grants to help create new, state-of-the-art safe harbor research facilities in California which are essential for delivering the goals of CIRM. That investment was used to leverage almost $900 million in additional funds from private donors and institutions to help pay for those facilities. Each facility attracted new researchers to the state, employed local construction workers and created expanded research facilities that will now be able to offer long-term employment for the high tech innovators in stem cell research, transformative new medicines for intractable disease and deliver economic benefit for Californians.
CTB: Given the juxtaposition of the relative dearth of CIRM-funded clinical projects to-date and the mandate to support bringing therapies to the clinic, in the last half of its mandate does CIRM intend to emphasize funding of more clinical projects?
CIRM: Yes, our focus in our new Strategic Plan does just that, emphasizing the increased focus on translation and clinical trials. As described above, we are investing strongly in this sector. But we firmly believe that advancement in medicine is dependent on the science that underpins the medical strategies. We will also continue to support high quality basic science that can transform medical opportunities.
CTB: If so, do you anticipate more of those will involve the use of adult cells over embryonic just by virtue of the fact more of these are closer to or already in clinical testing?
CIRM: We are required by our statute to fund in those areas that are under-invested. Otherwise we are agnostic to cell type. We expect a mixture of embryonic (induced pluripotent stem cells as well when they are ready for clinical studies), fetal, adult, cancer stem and progenitor cells, as well as small molecules, biologics and other approaches, evolving from stem cell assays and research. We are most concerned with the ability to produce results for patients.
CTB: I understand CIRM has made efforts over the past couple year to ease the burden or restrictions on companies applying for funds, is that true?
Yes, we have appointed a Vice President with business development responsibilities and are further strengthening this capacity with key staff. We are actively working with industry to develop sustainable partnerships in research, we hold webinars and face to face meetings with the FDA to better equip industry with the tools that can aid in their investigational new drug (IND) submissions . We also assist industry to better understand what they need to do to successfully apply for CIRM funding.
We have also made changes to our intellectual property regulations and loan regulations to make it even more attractive for companies to partner with us in research.
CTB: I have heard it said that CIRM is not interested in funding late-stage trials. Is that outside CIRM’s mandate or is it simply a matter of not having enough money to fund a late-stage trial?
Our focus has been in moving promising research through the "Valley of Death" phase, from the lab through Phase 1 and 2 clinical trials. We are working with major industry and financial institutions to inform them of our developing portfolio with the belief that they will be interested in taking many of these products to the market place. We are probably unable to afford to do these late stage clinical trials alone and feel it is likely that commercial interests will provide the follow on funding.
CTB: If CIRM’s $20M could be matched with another $20M to fund a late-stage trial, would that be appropriate and feasible to entertain?
CIRM: We are always interested in proposals that will enhance our mission. While this hypothetical has not been put to us we would have to assess the proposal on its merits and our available finances.
CTB: For clinical-stage companies outside California, what legitimate ties to California can be put in place to make one eligible for CIRM funding? Is a company required to have a Californian entity or is it enough to have collaborations with a Californian entity or key service providers located within the state such as a California-based manufacturer or clinical sites in California? What about having some staff in California? Other ways?
CIRM: In our RFA’s we have provided guidance as to what entities qualify for CIRM funding. Future requirments are presently under review by our General Counsel. Certainly, companies will need to show genuine steps at the time of application towards relocation of a significant component of their research activities to California in addition to establishing a California operation with California employees. CIRM funding would be largely limited to in-state activities.
My synopsis:
I'm willing to reserve judging CIRM's overall track record of funding of clinical-stage and industry-sponsored research based on what it has done to-date.
My assessment of CIRM's contributions to clinical-stage science and product development will be heavily weighted on what it does from this point forward.
There is a certain rationale at play here that says they had to spend the first part of the mandate building the research infrastructure and scientific underpinnings required to move successful clinical and product development forward in the last half of its mandate. It may not be a rationale you whole-heartedly endorse but it is credible and I, for one, and willing to give CIRM the benefit of the doubt on this one.
Having said that, my expectations for CIRM in the latter part of its mandate are very high with respect to how much they are going to dedicate to clinical-stage, industry-sponsored research.
However, CIRM cannot do this in a vacuum. What is required is for companies to do what they can to work with CIRM. Don't give up on them based on their past record or your past experience. Let's work with CIRM to help them focus their resources on moving some meaningful clinical milestones forward.
____________
I hope this interview helps clarify for readers just how CIRM views its ongoing and future participation in clinical-stage and industry-sponsored regenerative medicine research, testing, and development.
I would be happy to entertain and channel further questions anyone might have about CIRM (excluding those pertaining to specific applications or projects).
Monday, October 8, 2012
Cell therapy portfolio outperforms major indices year-to-date
On August 10 we created a model portfolio in Google Finance of 29 public companies in the cell therapy sector then we compared how that portfolio was doing against the major indices year-to-date (Since 1 January 2012). See that post here. Bottom line: even though we are still in a relatively bullish market, the CT portfolio was doing better. Significantly better.
So how is the sector portfolio doing now that we've been through three quarters?
In case you can't read the image above, the blue line represents the cell therapy portfolio and here are the stats on performance since 1 January 2012:
- Cell Therapy Portfolio: +63.17%
- Dow Jones: +11.17%
- S+P 500: +15.76%
- Nasdaq: +19.46%
The only change I've made to the portfolio of 29 companies listed in our August 10 post is to add Thermogenesis (KOOL). Today its stock is at .968 up from .7 at the beginning of the year.
You do or should know, I'm no financial analyst. I'm not entirely sure what assumptions are behind this 'model portfolio' or precisely what one should take from this snapshot but what is clear to me is that at least from one perspective the sector is treating investors fairly well.
I certainly welcome comments from more sophisticated investors or analysts. In fact, if anyone with that kind of experience or expertise wants to write a guest post on this blog providing a more sophisticated commentary on what this all means, I would very much welcome the contribution.
In the meantime, I hope this helps.
_________________
Post-publication addition:
Carter Gould, Associate Biotech Analyst at Dawson James Securities emailed me to point out that the cell therapy portfolio is simply riding the bull wave of biotech in general and and the more relevant comparison index is the broader biotech (BTK) index which is up 45% YTD. Here is a YahooFinance snapshot of the BTK performance vs the three major indices.
Friday, September 28, 2012
Anticipated short-term cell therapy industry clinical milestones
What follows is an interesting but not exhaustive list of cell therapy industry clinical milestones we anticipate in the next 3-9 months as selected from the list of cell therapy products we are tracking in late-stage or post-commercial development.
There are other commercial milestones we are monitoring as well as other clinical milestones we expect to see related to cell therapy products in earlier stages of the development pipeline that are not included below.
CellCoTec (www.cellcotec.com)
ERYtech Parma (www.erytech.com)
GamidaCell (www.gamidacell.com)
There are other commercial milestones we are monitoring as well as other clinical milestones we expect to see related to cell therapy products in earlier stages of the development pipeline that are not included below.
CellCoTec (www.cellcotec.com)
- Having completed a trial in Europe of their device to enable POC production of an autologous chondrocyte cellular product in/with a biodegradable, load-bearing scaffold for the treatment of articular cartilage defects, they have now submitted their CE market application. The CE mark application is under review and they anticipate a response in October.
- This device and the potential emergence of Sanofi's MACI in the European market next year may have an impact on Tigenix (EBR:TIG) most directly.
ERYtech Parma (www.erytech.com)
- Their 'pivotal' phase 2/3 trial in Europe of lead product, GRASPA, for the treatment of Acute Lymphoblastic Leukemia (ALL) is scheduled for completion 2H 2013.
- Their 'pivotal' phase 2/3 trial in the US, Israel, and Europe of lead product, StemEx, for the treatment of leukemia and lymphoma, in joint development with Teva, completed enrollment in February and is scheduled for completion 2H 2012. They have not been shy about the fact they expect to be in the market in 2013.
- They raised over 8m Euro in April for a phase 3 trial in Europe for their lead product, ICES13, for the treatment of stress-urinary incontinence which was scheduled for a preliminary clinical data readout in Q4 2012 and be ready for market authorization in 2013. Since announcing the capital raise the company has been stone silent and no clinical trial registry has been filed. Status unknown.
- Their phase 3 trial in Germany of CD133+ cells as an adjunct to CABG surgery for myocardial ischemia or coronary artery disease is scheduled for completion in January.
- Their phase 3 trial in US, Europe, and India of their lead product, Lucanix, for the treatment of advanced Non-small Cell Lung Cancer (NSCLC) following front-line chemotherapy is scheduled in clnicaltrials.gov for completion in October but we have learned they expect their next 'interim analysis' in February.
- They have a series of trials scheduled to complete 2H 2012 intended to provide additional clinical data to support its marketing of Osteocel Plus for the treatment of a growing number of orthopedic applications.
- Having completed their phase 3 trial in Europe of MACI for knee repair (symptomatic articular cartilage defects of the femoral condyle including the trochlea), they expect to file their market authorization application (MAA) in 1H 2013.
--Lee
Thursday, September 27, 2012
The cost of clinical trial data bias/loss, FDA's new job and the need for bold leadership.
Before you right this post off as the stuff of conspiracy theories, fear-mongering, and 'alternative world views' consider that this view is shared by the likes of the FDA, the International Committee of Medical Journal Editors, the Cochrane Collaboration, and researchers at institutions like Johns Hopkins School of Medicine.
Here's the underlying premise as succinctly described by author Ben Goldacre:
"Drugs are tested by the people who manufacture them, in poorly designed trials, on hopelessly small numbers of weird, unrepresentative patients, and analysed using techniques that are flawed by design, in such a way that they exaggerate the benefits of treatments. Unsurprisingly, these trials tend to produce results that favour the manufacturer.
When trials throw up results that companies don't like, they are perfectly entitled to hide them from doctors and patients, so we only ever see a distorted picture of any drug's true effects. Regulators see most of the trial data, but only from early on in a drug's life, and even then they don't give this data to doctors or patients, or even to other parts of government. This distorted evidence is then communicated and applied in a distorted fashion."Authors M. Todwin and J. Abramson summarize it thusly:
"Trials with positive results generally are published more frequently than studies that conclude that a new drug poses greater risks or is no more effective than standard therapy or a placebo. Furthermore, some articles may distort trial findings by omitting important data or by modifying prespecified outcome measures. Lack of access to detailed information about clinical trials can undermine the integrity of medical knowledge."Here is a great list of very recent resources that may convince you of the merits of this concern:
- What doctors don't know about the drugs they prescribe (2012: TED video)
- The drugs don't work: a modern medical scandal (2012: The Guardian)
- Clinical Trial Data as a Public Good (2012: JAMA [subscription req'd])
- Registering Clinical Trial Results. The Next Step (2010: JAMA [subscription req'd])
- The Imperative to Share Clinical Study Reports: Recommendations from the Tamiflu Experience (2012: Plos Medicine)
Yesterday, the US Secretary of Health and Human Services announced (in an FR notice) that the FDA was now charged with ensuring all organizations comply with the heretofore enacted but relatively unenforced requirement to submit all relevant clinical trial data to www.clinicaltrials.gov.
For further commentary on this move see the following reports from:
What is abundantly clear to me is that the FDA is left almost powerless - and if not powerless than certainly without sufficient resources - to successfully enforce its new power. This requires collective industry leadership. Bold, industry-initiated standards, infrastructure and old-fashioned peer pressure.
Here's what I wish.
I wish that as a cell therapy industry we - through organizations like ISSCR, ARM, ISCT, etc and leading publishers of some of our leading journals like Regenerative Medicine, Cytotherapy, Cell Stem Cell, Stem Cells, etc - would take a leadership position on an issue like this.
I believe that as a relatively small and nascent sector of the biopharma industry we are more likely capable of collaborating on something important like this than larger, more established [entrenched] and diverse sectors. Of course it requires the political will and cajones.
The payoff from our sector in taking a leadership role on this issue could potentially be enormous in terms of providing our sector with truly transparent and useful data. Perhaps even more important would be the public profile such leadership would provide the sector. Such a move requires bold leadership, pain, and cost but this is the kind of stuff that moves the needle and goes down as critical pivot points in history.
Just my thought for the day...
--Lee
Friday, September 14, 2012
Two lessons I learned this week.
I learned two valuable things this week I thought I'd pass on in a Friday afternoon post. Actually strictly speaking these are likely things I've learned before but needed to re-learn or to be 'reminded' of their importance.
Please pardon a little stroll away from the typically strict focus on cell therapy -- but in a way that's the theme of today's post.
1. Take time each week to read something from outside your specific profession or job focus.
I'm not talking here about the importance of escaping in the evening with a fiction novel (also very important) but rather reading something professional but from well outside your area of focus. Here are my examples.
Similarly, on a flight this week I reached into the seat pocket in front of me and discovered a recent copy of the Journal of the American Medical Association. I read a fascinating article that has me excited about an idea for how we as a cell therapy industry might lead the way in addressing clinical trial and data transparency that would put our sector in a leadership position, lend the industry a much-needed spotlight, and has the potential to facilitate the kind of meta-analysis and data-mining that could only be done through data aggregation. I believe the concept has the potential to be disproportionately significant for a sector defined by so many small, under-powered trials.
The idea may never see the light of day but the point is the source of the inspiration. In order to 'think' outside the box one typically has to 'be' outside the box. Lesson? Spend some time outside your box.
2. It often takes something very small to make a disproportionately significant impact on someone.
I was reminded recently through an exchange of simple kindnesses just how little it sometimes takes to make a big difference in someone's life. For you what might be so easy to give might be of unparalleled value to someone for whom that is so unattainable.
Lesson? When the opportunity knocks for you to give something small or simple, take it. This kind of charity almost always has the potential to be mre impactful than you might ever imagine.
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