Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Cancer Immunotherapy

Posts tagged ‘New Product Development’

The Boston Globe today reported that Blueprint Medicines had received $40M in Series A venture funding.

The VC funding from Third Rock Ventures to the Boston/Cambridge based company is reported to be the largest early-stage funding for a New England life sciences start-up.

Many thanks to @rndubois for his tweets about this that drew it to my attention. You can read more about the financing in Blueprint’s press release.

What makes this exciting news?  First it adds to the growing reputation of Boston/Cambridge as a hot-spot for cancer research.  Blueprint Medicines will be focused on translational medicine and the development of new kinase inhibitors for the treatment of cancer.

Secondly, it confirms what is taught at business school, that investors back management expertise and their belief in the entrepreneurs ability to execute.  In the case of Blueprint Medicines the scientific co-founders are Dr Nicholas Lyndon and Dr Brian Druker, who were instrumental in the development of imatinib (Gleevec/Glivec), a tyrosine kinase inhibitor that revolutionized the treatment of chronic myeloid leukemia (CML).

Blueprint Medicines is a company to watch for the future and Biotech Strategy Blog wishes it well in the quest for personalized medicine and more effective cancer treatments.

The launch of the company in Boston/Cambridge adds to my view that Boston is emerging as the premier biotech region on the East Coast for start-ups interested in oncology and translational medicine.

Today at the European Association of Urology (EAU) annual meeting in Vienna, the big news was that 2010 was a “Grand Cru” year for new treatments for advanced prostate cancer.  Not only that, but sanofi-aventis announced that they had received European marketing approval for cabazitaxel (Jevtana®) in metastatic hormone resistance prostate cancer mHRPC.

The fact that there are now several new treatments available (or expected to be available in the not too distant future) is good news for patients and physicians.

What is interesting about prostate cancer is that it in terms of incidence it is comparable to breast cancer, yet seems to end up with far fewer resources and publicity.  Prostate cancer is to men, what breast cancer is to women.

The EAU 2011 Congress website has a variety of podcasts and webcasts of presentations, and I encourage anyone interested in the latest developments to check out the wealth of information they offer.  In particular, the presentation by Professor Johann De Bono from the Royal Marsden in the high risk prostate cancer plenary session today was one of my highlights of the meeting.

The take home message I obtained from EAU in Vienna is the excitement of new treatment options for castration resistant prostate cancer (CRPC) such as cabazitaxel, sipuleucel-T and abiraterone.  The challenge may well be to work out how best to use these new therapies, ie in what sequence and what potential combinations may evolve in the future.

However, as Professor Bertrand Tombal from Louvain in Belgium declared, 2010 was a Grand Cru for new prostate cancer treatments.  That is good news indeed.



Nanotechnology is set to have a major impact on drug development and new products for the diagnosis and treatment of cancer.  Research from UCSF and Northwestern University published earlier this year in “Science Translational Medicine” shows this potential.

Edward Chow and colleagues describe how binding the cancer chemotherapy doxorubicin (DOX) to carbon nanoparticles 2-8nm in diameter in the form of a diamond, “nanodiamond” (ND), improved drug efficacy and overcame drug resistance.  Although this pre-clinical animal research has not yet been confirmed in humans, it raises the possibility of more efficient chemotherapies and the hope of increased survival rates as a result.

The conclusion from this research is that nanodiamonds may be a viable drug delivery platform for small molecules, proteins and nucleic acids. This technology could have an application in wide range of diseases.

Why is nanoparticle-mediated drug delivery more effective? The paper suggests one reason is that the nanodiamond-doxorubicin complex (NDX) allows for a more gradual release of DOX, allowing for increased tumor retention and increased circulation time.

It’s important to note that the NDX complex does not specifically target the drug efflux pumps, such as MDR1 and ABCG2 transporter proteins, responsible for chemoresistance. Instead the NDX complex appears to overcome drug resistance passively by the way DOX is released from the nanodiamond.

This research shows that taking old drugs and combining them with new drug delivery technology may offer therapeutic benefits.  The authors conclude that this research, “serves as a promising foundation for continued NDX development and potential clinical application.”

If successful in humans, it will translate into new product development and market opportunities for emerging biotechnology and biopharmaceutical companies.


ResearchBlogging.orgChow, E., Zhang, X., Chen, M., Lam, R., Robinson, E., Huang, H., Schaffer, D., Osawa, E., Goga, A., & Ho, D. (2011). Nanodiamond Therapeutic Delivery Agents Mediate Enhanced Chemoresistant Tumor Treatment Science Translational Medicine, 3 (73), 73-73 DOI: 10.1126/scitranslmed.3001713

I would like to thank Victor Pikov, a neurophysiologist and biomedical engineer at Huntington Medical Research Institutes (HMRI) for drawing my attention to his NeurotechZone Blog that has a really fascinating post on the manufacturing of the next generation of artificial retina, the Argus™ 111, by the Lawrence Livermore National Laboratory (LLNL).

If you have an interest in this area, then Victor is also co-chair of 3rd International Conference on Neuroprosthetic Devices (ICNPD-2011) to be held in Sydney from November 25-26, 2011. Further information can be found on NeuroTechZone.

Second Sight Medical Products recently obtained a CE mark and European Market Approval for the Argus™ II system that incorporates 60 electrodes into the retinal prosthesis.

However the next generation of artificial retina, the Argus™ III is already in development.  It has 200 electrodes – a quantum leap forwards.  It’s hard not believe that an array that is four times as densely packed with sensors, will not provide improved vision.

Second Sight will no doubt be planning clinical trials for Argus™ III and it sounds like it will provide a further leap forward in the technology to restore some sense of vision to patients who have lost their sight through age-related macular degeneration (AMD) or retinitis pigmentosa (RP).

I have taken the liberty of embedding below, the excellent YouTube video that Lawrence Livermore National Laboratory (LLNL) have produced about their manufacturing of the Argus™ III artificial retina. It is well worth watching!


I wrote last week about Second Sight’s European Marketing Approval for the Argus II “artificial retina”.  What this news also stands for is the success of collaboration as a route to innovation.

The Artificial Retina Project (“Restoring Sight through Science”) through which Argus II was developed is a collaborative effort between six United States Department of Energy (DOE) research institutions, 4 universities and private industry.

Each offers unique scientific knowledge and specialist expertise, without which it is unlikely the project (that is continuing with the development of a more advanced Argus III artificial retina) would have been successful.

I’ve listed the collaborators below and as recorded on the DOE website, what they bring to the Artificial Retina Project.

DOE National Labs:

  • Argonne National Laboratory – Performs packaging and hermetic-seal research to protect the prosthetic device from the salty eye environment, using their R&D 100 award-winning ultrananocrystalline diamond technology.
  • Lawrence Livermore National Laboratory (LLNL) – Uses microfabrication technology to develop thin, flexible neural electrode arrays that conform to the retina’s curved shape. LLNL also uses advanced packaging technology and system-level integration to interconnect the electronics package and the thin-film electrode array.
  • Oak Ridge National Laboratory – Measures the effect of increasing the number of electrodes on the quality of the electrical signals used to stimulate the surviving neural cells in the retina.
  • Sandia National Laboratories – Develops microelectromechanical (MEMS) devices and high-voltage subsystems for advanced implant designs. These include microtools, electronics packaging, and application-specific integrated circuits (ASICs) to allow high-density interconnects and electrode arrays.
  • Brookhaven National Laboratory – Performs neuroscience imaging studies of the Model 1 retinal prosthesis.


  • Doheny Eye Institute at the University of Southern California – Provides medical direction and performs preclinical and clinical testing of the electrode array implants. Leads the Artificial Retina Project.
  • University of California, Santa Cruz – Performs bidirectional telemetry for wireless communication and chip design for stimulating the electrode array.
  • North Carolina State University – Performs electromagnetic and thermal modeling of the device to help determine how much energy can be used to stimulate the remaining nondiseased cells.
  • California Institute of Technology – Performs real-time image processing of miniature camera output and provides optimization of visual perception.

In October 2004, Second Sight Medical Products and the DOE signed a Co-Operative Research and Development Agreement (CRADA) in which the above institutions agreed to share intellectual property and royalties from their research, with Second Sight chosen to be the commercial partner.  As part of the CRADA, Second Sight obtained a limited, exclusive license to the inventions developed during the DOE Retinal Prosthesis Project.

You can find more information about the history of this fascinating project on the Artificial Retina Project website, that also has links to several patient stories from around the world.

The Artificial Retina Project is a case study on the success of collaboration.  Whether such an ambitious project that was funded by the US Government would ever have taken place in the private sector is the question that comes to my mind?  Would a private company have been able to harness the intellectual power of 10 research institutions in this way?

If not, then do governments have a role to play in biomedical innovation by drawing partners together so that advances in basic research can be applied to new products, whether they be new drugs or novel devices?

And if you agree that governments do have a role to play what should be the extent of government funding?  In the case of artificial retina, the DOE has funded this since 1999, with its contribution rising from $500K to $7M per year. Those numbers may also be direct costs, and not reflect the cost of investments in buildings, research facilities etc.

I’d be interested in any thoughts you would like to share on this.

Following on from my blog post last week that discussed the use of iPads and other tablet computers in clinical trials, MIM Software have just received FDA 510(k) clearance to market their iPhone and iPad medical imaging app in the United States. This is the first such approval by the FDA, and the app will be sold in Apple’s itunes store.

This new mobile radiology application will allow physicians to review medical images on their iPhone and iPad.  The FDA in their press release indicate that it is not intended to replace full work stations, but to provide the ability to view images and make diagnoses when a workstation is not readily available.

The FDA reviewed luminance, image resolution quality, and results from demonstration studies with radiologists that showed that images could be safely interpreted for diagnostic purposes under appropriate lighting conditions.

What is more, using software from MIM, the images can be further analyzed and distance measurements made.

The ability to have wireless access to medical images will be particularly useful to physicians working remotely, in emergency situations and in clinical trial networks where the central imaging review facility may not be local.

As the screen resolution of iPad’s and other tablet computers increases, perhaps we will see advanced visualization software available on the iPad?  It is certainly an area where innovation is taking place, and one that I think will impact clinical research in the biotechnology industry before too long.

The December 17, 2010 issue of “Science” has the catchy of title of “Insights of the Decade”, one of which is an article by Jennifer Couzin-Frankel, “Inflammation Bares a Dark Side”, that describes the ubiquitous role of inflammation. She concludes that:

“Mediating inflammation in chronic diseases is a new frontier, its success is still uncertain.”

Inflammation has been shown to play an important role in multiple chronic illnesses such as cancer, and in type 2 diabetes it promotes insulin resistance and the death of pancreatic beta cells.  In 2007, Marc Donath and colleagues published a landmark study in the New England Journal of Medicine where he used the drug anakinra, in patients with type 2 diabetes, to block interleukin-1 (IL-1), a cytokine that mediates the inflammatory response. The conclusion of the paper was that:

“The blockade of interleukin-1 with anakinra improved glycemia and beta-cell secretory function and reduced markers of systemic inflammation.”

The finding that diabetes patients whose inflammatory response was blocked did better, has led several companies to work on drug development in this area.

One of these is the biotechnology company, Xoma, whose stocked jumped 200% in the week before Christmas.  Although there was no press release or announcement of any company news, it looks like investors decided to take a gamble that the phase 2 trial results for Xoma 052 in type 2 diabetes will be positive.  As often happens, the wisdom of the crowd, led to others joining the share buying frenzy.

Source: Google Finance. Xoma had previously announced on November 4, 2010 (emphasis added) that:

Enrollment completed in Phase 2a trial of XOMA 052 in patients with Type 2 diabetes:

This randomized, placebo-controlled trial, in which 74 patients were enrolled, is designed to evaluate extended biologic activity and safety of XOMA 052. Outcomes will include diabetes measures such as hemoglobin A1c, or HbA1c, and fasting blood glucose, or FBG, and C-reactive protein, or hsCRP, a biomarker of inflammation associated with cardiovascular risk. Interim results from the first three months of treatment in this six month trial are expected to be announced in the first half of January 2011.

Enrollment completed in Phase 2b trial of XOMA 052 in patients with Type 2 diabetes: This randomized, placebo-controlled dose-ranging trial enrolled 420 patients and is designed to further evaluate the safety and efficacy of XOMA 052 dosed once monthly compared to placebo. The results will include data on measurements of HbA1c, FBG and hsCRP. Top line results are expected to be announced in the first quarter of 2011.

Xoma 052 is a high affinity monoclonal antibody that targets the inhibition of IL-1 beta.  Its ultra-high affinity allows for monthly dosing and lower dose levels which supports patient compliance in chronic diseases. Positive phase 2 results for Xoma 052 in Behcet’s Uveitis was presented in November to the American College of Rheumatology.

According to the November 2010 Xoma Corporate Presentation, the overall market size for diabetes is $22B, of which the IL-1 share is $7B, raising the possibility that Xoma 052 could be a blockbuster if shown to be safe and effective.

Source: Xoma November 2010 Corporate Presentation

Looking at the above, perhaps the rush to buy Xoma stock before the holidays, was perhaps not as much of a gamble as one might think. Xoma 052 is certainly a product to watch this year.


As reported today by BBC News,  a NJ based specialist pharmaceutical company, Antares Pharma has developed a gel based contraceptive, Nesterone® that is as effective as taking a pill, acccording to phase 2 clinical trial data presented at the annual meeting of the American Society of Reproductive Medicine (ASRM) in Denver.

According to researchers, the contraceptive delivered by the gel is as effective as that delivered by a traditional pill, but doesn’t show many of the adverse events and side effects such as weight gain & acne.  As always with promising phase 2 data, there is no guarantee that the phase 3 trial will show the same results, but this is definitely an innovative product to watch.

Nesterone® is being jointly developed with the Population Council (a non-profit organization). According to the lastest Antares Pharma investor presentation, they are looking for partnership opportunities for this product.  Based on the positive phase 2 data and a market worth $1B,  I am sure they will now have some business development interest.

Also, the company has a number of other injectable and transdermal gel products in development and collaborations with Teva, Uman Pharma and BioSante Pharmaceuticals. It will be interesting to watch how this small, emerging company and its products develop. A company to watch.

Human eye cross-sectional view. Courtesy NIH N...Image via Wikipedia

VEGF Trap-Eye is a formulation of VEGF Trap (aflibercept) and is an anti-angiogenic agent that can be injected into the eye to stop the proliferation of blood vessels. Regeneron (REGN) are co-developing it with Bayer (BAY) and it is currently in clinical trials for the treatment of wet Age-Related Macular Degeneration (AMD), Diabetic Macular Edema (DME) and Central Retinal Vein Occlusion (CRVO).

Phase II DME clinical trial results presented at the
Angiogenesis 2010 meeting in Miami showed the primary endpoint of a
statistically significant increase in visual acuity over 24 weeks compared to
the standard of care (laser treatment) was met.

There are high levels of vascular endothelial growth factor (VEGF) associated with DME, so the news that VEGF Trap-Eye has biological activity in this disease is positive.What makes this data promising is the fact that DME is the leading cause of blindness in adults under 50 and there are 370,000 Americans with clinically significant DME with 95,000 new cases a year.

The ability to treat DME by an eye injection, rather than use an expensive laser will make it easier to treat the disease. It will be interesting to see what how the cost of treatment with VEGF Trap-Eye compares to laser therapy procedures, should the agent make it to market.

The recent pricing issue faced by Genentech with its VEGF inhibitors Lucentis
(eye indications) and Avastin (oncologic indications) are also relevant because
Regeneron are developing VEGF-Trap in cancer with it’s partner sanofi-aventis
(a client).

For VEGF Trap-Eye, Regeneron retains all U.S. marketing
rights, while Bayer has rights to market ex-US in return for a 50/50 profit
share with Regeneron.The results so far look promising and aflibercept looks like an interesting agent well worth watching as the development moves forward.

Reblog this post [with Zemanta]

Welcome to the biotech strategy blog which provides commentary and insight on current news and emerging trends in biotechnology.

As a strategy and marketing consultant with a background in clinical development I am interested in how biotechnology companies grow, manage alliances, partner with CROs and bring new products to market. I hope that you will find posts on this blog to be informative and interesting.

error: Content is protected !!