Biotech Strategy Blog

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

Posts tagged ‘New Product Development Strategy’

BIO-2011-Interational-Convention-Washington-DC

I am excited to be attending, for the first time, the Biotechnology Industry Organization (BIO) international convention that takes place in Washington DC in just over a week’s time from Monday June 27 to Thursday, June 30th.

This meeting has something for everyone interested in the biotechnology industry whether it be deal making, partnering, licensing, drug discovery or personalized medicine. There are 16 specialized tracks where industry experts provide insight and best practices.

In addition, there are numerous networking and social events plus an exhibit hall that showcases the world’s biotech regions and how they are promoting innovation.

At meetings where there are parallel sessions, I apply “the law of two feet” (thanks to Podcamp for this) that says if you are not getting what you want from the session, it’s OK to walk out and go to another one.

My top 10 sessions at BIO reflect my personal interests in innovation, science and new product development:

Tuesday June 28

  • How will we afford Personalized Medicines?
  • The Biomarkers Consortium: Facilitating the Development and Qualification of Biological Markers
  • Personalized Oncology: The emergence of Personalized Medicine Strategies in Oncology Clinical Development and Deal Making
  • Navigating the New Law on Licensing Biosimilars

Wednesday June 29

  • Lessons from a Mature Public-Private Partnership. The Alzheimer’s Disease Neuroimaging Initiative
  • Emerging Markets. The Future of Growth for Biologics?
  • The Role of Imaging Biomarkers in Early Phase CNS Drug Development
  • The Promise of MicroRNA-based Therapeutics in Cancer

Thursday Jun 30

  • After the Fall. Venture Capital and the Biotech Funding Landscape
  • Regulatory Issues for Tissue Engineered Products

If you have plans to be at BIO 2011 do say hello after one of the sessions or receptions. You can reach me at the meeting via twitter (@3NT).  See you in DC!

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Nanotechnology is leading to innovation in drug delivery, and new ways to treat diseases.

In an April 3, 2011 online article in Nature Chemistry, researchers from the IBM Almaden Research Center, Institute of Bioengineering and Nanotechnology in Singapore and Zhejiang University in China publish groundbreaking data on how biodegradable nanoparticles could be used to treat infectious diseases such as methillicin-resistant Staphylococcus aureus (MRSA).

The research shows how nanoparticles can selectively disrupt microbial cell membranes, walls and inhibit the growth of gram-positive bacteria, MRSA and fungi.

What makes this research exciting, is that the nanoparticles did not cause haemolysis or break-up of red blood cells.  The authors note that nanoparticles for the treatment of infectious diseases could be “synthesized in large quantities and at low cost” and are therefore “promising as anti-microbial drugs.”

The global market for infectious diseases was $90.4 billion in 2009 and is projected to reach $138 billion in 2014.  In the United States there are now more deaths from MRSA than there are from AIDS (18,650 MRSA deaths in 2005 compared to 16,000 for AIDS according to a paper in JAMA).

With more than 94,000 MRSA infections a year in the United States, and the increasing resistance of MRSA to existing anti-microbial therapies, treatment of infectious diseases is a major public health concern. Hospital acquired MRSA infections particularly target the elderly and those vulnerable through weakened immune systems.

Innovations in nanotechnology and drug delivery, such as the one published by Nederberg, Zhang, Tan & Xu in Nature Chemistry, open the door to potential new anti-microbial therapies.  It will be interesting to see how this research is commercialized and translated into new products and treatments.

ResearchBlogging.orgNederberg, F., Zhang, Y., Tan, J., Xu, K., Wang, H., Yang, C., Gao, S., Guo, X., Fukushima, K., Li, L., Hedrick, J., & Yang, Y. (2011). Biodegradable nanostructures with selective lysis of microbial membranes Nature Chemistry DOI: 10.1038/nchem.1012

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.

Innovation in drug delivery presents opportunities for biotechnology companies, and is an area I expect we will see major leaps forward through nanotechnology.

Nanotechnology is the application of science and engineering to materials that are between 1 and 100 nanometers (nm) in size.  The Environment Protection Agency (EPA) defines nanotechnology as “the creation and use of structures, devices, and systems that have novel properties and functions because of their small size.”

1nm is one-billionth of a meter.  To put this in context, 1nm is one seven-thousandth of the width of a red blood cell or one eighty-thousandth of the width of a human hair. These are unimaginably small materials that are engineered to operate at the molecular and atomic level.

What’s more, there are now more than 1000+ consumer products on the market that utilize nanotechnology from the titanium particles in sunscreens to the silver contained in advanced first aid strips/plasters.  Nanotechnology will impact more than $2.5 trillion of manufactured goods by 2015.

Lux Research predicts that by 2014, 16% of manufactured goods in healthcare and life sciences will include nanomaterials.

To date, the United States leads the way in the fast evolving field of nanotechnology.  Between 2001 and 2010, the U.S. Government invested $12.4 billion in nanoscale science, engineering and technology through the U.S. National Nanotechnology Initiative (NNI).

The National Cancer Institute’s “NCI Alliance for Nanotechnology in Cancer” has an excellent website that outlines the potential impact of nanotechnology.

Some of the promising new cancer diagnostics and therapies based on nanotechnology include:

  • Positron Emission Tomography (PET) imaging agents that can be used to assess the responsiveness of tumors to chemotherapy
  • Chemically engineered adenovirus nanoparticle that stimulates the immune system. This is in phase 1 trials for chronic lymphocytic leukemia (CLL).
  • Cyclodextrin-based nanoparticle that encapsulates a small-interfering RNA (siRNA) agent that shuts down a key enzyme in cancer cells
  • CRLX101, a cyclodextrin-based polymer conjugated to camptothecin is in clinical trials with solid tumor patients
  • A nanoparticle based magnetic resonance imaging (MRI) contrast agent that binds to αvβ3-intregrin, a protein found on newly developed blood vessels associated with tumor development. This is in early clinical trials
  • Technology for the detection of cancer biomarkers such as prostate specific antigen (PSA)
  • Use of carbon nanotubes to improve colorectal cancer imaging.

Emerging companies such as Bind Biosciences are focusing on targeting cancer, inflammatory, cardiovascular diseases and infectious diseases with therapeutic nanoparticles.  Their lead product BIND-014 is currently in phase 1 development.

Innovations in nanotechnology will continue to present new product opportunities for biotechnology, pharmaceutical, medical imaging and diagnostics companies, and should be on everyone’s radar.

 

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The Lancet yesterday published news of the world’s first tissue engineered implant of a urethra (the tube that carries urine out of the body from the bladder).

This research by Atlantida Raya-Rivera and colleagues at the Wake Forest Institute for Regenerative Medicine and Metropolitan Autonomous University in Mexico is another step towards when we may be able to regenerate a wide range of body parts. This would solve many of the donor shortages for livers and kidneys that exist today.

In their paper, Raya-Rivera describe how they took a tissue biopsy from five Mexican boys and by then seeding these cells on a scaffold, grew new urethras. These were subsequently transplanted into the boys (aged 10-14) between 2004-2007.  The results show that the tissue engineered urethras remained functional for up to 6 years, appeared normal within 3 months of implantation and allowed a urine median end maximum urinary flow rate between 16-28 mL/s.  To put this in context, the average urine flow rate of males aged 8-13 is 12mL/s, suggesting that the tissue engineered urethras functioned well.

For those who suffer from complex urethral problems as a result of disease, infection or congenital defects, this research offers the prospect of a new treatment option.  More research is required with a larger sample size to validate the findings, and to confirm that no strictures are seen long-term after reconstruction.

Ongoing research at the Wake Forest Institute of Regenerative Medicine (WFIRM) into engineering human livers, kidneys, pancreatic beta cells and heart valves suggests that, if successful, regenerative medicine will have a major impact on the treatment of future diseases.  I can imagine a world for people with diabetes where new pancreatic insulin producing cells could be engineered and implanted.

The potential to replace non-functional or diseased organs and tissues with a replacement tissue engineered new one (like replacing a car part) will have a tremendous impact on the pharmaceutical and biotechnology industry. Blockbuster drug franchises could disappear overnight.  Regenerative medicine is an exciting area to watch over the next few years.

References

This post was chosen as an Editor's Selection for ResearchBlogging.orgRaya-Rivera, A., Esquiliano, D., Yoo, J., Lopez-Bayghen, E., Soker, S., & Atala, A. (2011). Tissue-engineered autologous urethras for patients who need reconstruction: an observational study The Lancet DOI: 10.1016/S0140-6736(10)62354-9

BBC health reported this past week on the news that Professor Dr. Joachim Boldt, the former head of anaesthesia at the Klinikum Ludwigishafen hospital in Germany had published 102 papers in leading academic journals without first having obtained ethics committee (EC) or institutitional review board (IRB)  approval for the research.

These studies on patients undergoing surgery or intensive care led to the development of new guidelines for managing the administration of colloids for fluid replacement during surgery. Questions are now being asked about the validity of these scientific findings and whether any fabrication of research data took place.

When Dr Boldt submitted research for publication he indicated that EC/IRB approval had been obtained, a claim that was not checked by any of the journals.  It now appears that EC/IRB approval had not been obtained. Last week the editors of 16 leading publications formally retracted the papers they had published from Dr Boldt.

The news that an anaethetist failed to obtain informed consent or EC/IRB approval for clinical research comes as no surprise to me.

I conducted anaesthesia breathing system research in the early 1990’s in a joint industry/academic partnership program. My research was published in the British Journal of Anaesthesia, who also published several studies from Dr Boldt.

At that time, many of the anaethestists I worked with questioned the need for patient informed consent for research with a new breathing system. They argued the patient would have to use one anyway during the operation, and even if this was a research study it was not necessary. Requiring formal signed informed consent for the clinical trials I did was a novel experience for some of the anaethetists I worked with.

Indeed, if the editors of the journals were to look closely at european clinical research related to medical devices published prior to EC Directives and standards such as EN540, ISO14155 coming into effect, they might find that many researchers did not obtain IRB/EC approval or informed consent for that work.

I think the journal editors are right to condemn the lack of research integrity that took place with Dr Boldt.  All of us in the pharmaceutical, biotechnology and medical device industries who undertake clinical trials to bring new products to market rely on the goodwill of patients to participate in the medical research process.

The news that Dr Boldt did not respect the rights of individuals, and failed to follow the Declaration of Helsinki, the fundamental “Ethical Principles for Medical Research Involving Human Subjects”, published by the World Medical Association, undermines the integrity of the clinical research process for all of us.

Moving forwards, I would suggest that the editors of journals require authors to submit a copy of the EC/IRB approval letter/notification with their manuscripts.  Any form that just requires you to tick a box or sign off to show compliance is open to potential abuse by a very small minority.

All EC/IRB approvals have to be in writing, so this step would not be an onerous burden and would provide some confidence of valid ethics approval, and in the process support the integrity of the clinical trials that we all rely upon.

If you would like to follow this issue in more detail, Ivan Oransky, on his excellent Retraction Watch Blog has been writing about this story since last October.

I was recently in San Francisco so thought I would continue my theme of writing about biotechnology regions that I visit around the United States.

Growing up in England, I remember listening to the radio broadcasts of the late Alistair Cooke, who from 1946 to 2004 shared his “Letter from America“; the longest running radio programme ever produced.  In the pre-internet era his mixture of anecdotes, insights and reflections reminds me of modern day blogs.

San Francisco remains a favorite city of mine. Fueled by access to venture capital and proximity to major research universities such as Stanford, University of California at Davis, Berkelely & San Francisco, start-up companies continue to thrive in the Bay area. BayBio, Northern California’s Life Science Association runs many excellent events. Their annual conference in April is focused on “Powering Global Innovation.”

The anchor tenant in the San Francisco biotech mall remains Genentech, and no other company in the area has had the same growth trajectory.  What catapults a company forward is a combination of a breakthrough product and ability to capture its value. The licensing deals and acquisitions we see today in the biotechnology industry, to some degree limit the ability of emerging biotech companies to repeat Genentech’s model. Risk sharing, partnering and the desire of venture capitalists for an early return on investment, all limit the ability of a biotech company to make it to the major leagues. In the end, even Genentech ended up being acquired by Roche.

What’s the future in San Francisco? It remains a high cost place to live but with a pool of talent in the entrepreneurial culture of the West Coast. There is also the uncertainty about the California economy and the cost of doing business, which is most likely set to increase.  In some way, my impression is that San Francisco has not quite taken off as a biotechnology city in the same way that Boston and Cambridge has. Feel free to comment if you disagree or have an opinion otherwise.

Following on from yesterday’s news that Gilead had acquired Calistoga and CAL-101, another company that is exploring the interface between cancer and inflammation is Paris based AB Science.

Pharma Strategy Blog has an excellent interview with the CEO, Alain Moussy.  AB Science is an emerging French biopharmaceutical company, and I previously wrote about its IPO.

The company has adopted a unique market entry strategy of obtaining approval first in animal health for their tyrosine kinase inhibitor, masitinib.  In 2008, AB Science gained European approval for canine mast cell tumors and in December 2010 FDA approval.

The company recently announced that on February 8, 2011 it had its first US sale of masitinib to vets.

Masitinib is in fact a multi-kinase inhibitor that inhibits wild type and mutant forms of stem cell factor receptor (c-KIT, SCFR), platelet-derived growth factor (PDGFR), fibroblast growth factor 3 (FGFR3) and to a lesser degree, focal adhesion kinase (FAK).

Sally Church on the Pharma Strategy Blog has written about how AB Science’s strategy makes sense – if you look at Pfizer, they obtain more revenue from animal health than they do from oncology.  AB Sciences’ Masivet® in Europe, Kinavet® in the United States competes against Pfizer animal health’s tyrosine kinase inhibitor, Palladia® (toceranib), which also targets mast cell cancer in dogs.

Not only does this growth strategy generate revenue for an early-stage company like AB Science, it also allows the company to build a sales and marketing infrastructure in the United States and Europe while waiting for the results of pivotal phase 3 studies in humans.

The phase 2 clinical trial data for masitinib in combination with gemcitabine in pancreatic cancer were impressive (28% survival at 18 months).  The phase 3 clinical trial results are expected this year.  The clintrials.gov listing shows the date for the estimated primary completion date (Overall Survival) as November 2010 with study completion in November 2011.  Obviously the exact timing depends on how fast subjects were accrued, but I would be surprised if we didn’t see some data presented at ASCO or ESMO, especially if positive.

In terms of targeting inflammation, masitinib is in phase III development for mastocytosis, rheumatoid arthritis (RA) and asthma.  AB Science announced on January 27, 2011 the first patient recruited into their phase 3 study in severe asthma.

The company’s new product development strategy is way ahead of many of its competitors in identifying the links between cancer and inflammation, and choosing to target market opportunities in both areas.

AB Science is an exciting company to watch, and I expect that we will see important new data come out at major scientific meetings this year.

In an acquisition that highlights the importance of cancer and inflammation, Gilead Sciences today announced the acquisition of Seattle based Calistoga Pharmaceuticals for $375M.

Calistoga’s pipeline is focused on the development of PI3 kinase inhibitors for cancer and inflammation. Sally Church on Pharma Strategy Blog has written extensively about “The potential of the PI3K pathway inhibitors in lung cancer”, and discussed Calistoga’s CAL-101 compound and its development for hematological malignancies in her report on “What’s hot at ASH in 2010”.

I encourage you to read (if you already don’t) Sally’s excellent Pharma Strategy Blog for further information on the science and mechanism of action of the PI3K pathway (way beyond my pay grade) and her view on CAL-101’s potential.

Sally will also be at the timely AACR meeting on targeting PI3K/mTOR signaling in cancer that is being held in San Francisco later this week.

What makes CAL-101 interesting to me is its potential in targeting inflammatory mediators. CAL-101 is a first in class PI3K delta specific inhibitor; the delta isoform of phosphoinositide-3 kinase (PI3K) is expressed in leukocytes involved with a variety of inflammatory, autoimmune and hematological cancers. Increasingly I think we will see companies investigating the cross-talk between inflammation and other diseases.

In addition to the upfront payment of $375M, there are potential milestone payments of $225M.  The deal is set to close in the second quarter of 2011.

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