Anticipated short-term cell therapy industry clinical milestones

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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)

• 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. 

GamidaCell (www.gamidacell.com)

• 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.

Innovacell (www.innovacell.com)

• 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.

Miltenyi Biotec (www.miltenyibiotec.com)

• 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.

NovaRx (www.novarx.com)

• 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.

NuVasive (www.nuvasive.com)

• 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.

Sanofi's Genzyme (www.genzyme.com)

• 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.

Hope that's helpful and gives you a sense some of the late-stage things to watch for in the coming weeks and months.  

--Lee

The cost of clinical trial data bias/loss, FDA's new job and the need for bold leadership.

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The scandal of clinical trial data loss is eroding the fundamentals of evidence-based research and clinical medicine.

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:

• Nature News Blog
• Fierce Biotech
• PharmaLot

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

Two lessons I learned this week.

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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.

I always read WIRED magazine.  Aside from GEN it's the only magazine I read.  Just reading something outside of cell therapy or biotech often infuses me with an idea that otherwise would have never occurred to me like the need for a cell therapy X Prize or cellular aggregates as microcarriers or tissue-engineered memory and processing devices or even just the conviction to better represent cell therapy to the broader world out there of scientists, engineers, journalists, policy-makers, or perhaps people with too much money looking to be inspired and wanting to make a difference.

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.