Biotech Strategy Blog

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

Posts tagged ‘Clinical Trials’

Paris: If cancer immunotherapy is a revolution in how cancer is treated, then Dr Jérôme Galon is a revolutionary.

In addition to being a Research Director at INSERM in Paris, Dr Galon is one of the co-founders of Marseille based HalioDx, an immuno-oncology diagnostics company that is commercializing the research from his laboratory.

Last month, while in Paris, I had the great pleasure to talk with him in his office at the Centre de Recherche des Cordeliers (CRC) on the left bank of Paris.

At the time of the French revolution in 1789, it was the gathering place of the “Club des Cordeliers,” for famous revolutionaries such as Danton, Marat, and Camille Desmoulins. Dr Galon told me it was where the Declaration des droits de l’homme et du citoyen de 1789 (Declaration of the Rights of Man and Citizen) was signed.

As such, it’s a very appropriate place to find a cancer immunotherapy revolutionary…

To learn more insights on what he had to say about a variety of topics in cancer immunotherapy, subscribers can log-in or you can gain access to BSB Premium Content.

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The February 2011 issue of Nature Reviews Drug Discovery has an interesting review by Kawai, Mödder and colleagues on “Emerging therapeutic opportunities for skeletal restoration.”

Some of the new products they discuss include:

  1. Parathyroid Hormone-Related protein (PTHRP)
  2. Cathepsin K Inhibitors: odanacatib
  3. Wnt-ß-catenin pathway targets: sclerostin, DKK1 antagonists, lithium.

The market opportunity for osteoporosis remains significant, affecting 44 million people in the United States over the age of 50, resulting in healthcare costs in excess of $15 billion a year; numbers that are set to increase with the ageing population of baby boomers.  The low bone mineral density (BMD) associated with osteoporosis results in increased risk of hip fracture, from which the mortality rate is 20-30% in the first year.

The current competitive landscape for osteoporosis includes antiresorptive agents such as the bisphosponates (alendronate, risedronate, ibandronate, zoledronic acid) that inhibit bone resorption.  These compounds reduce fracture-risk by 20-30%, but long-term safety issues remain a concern.  High doses of zoledronic acid (Zometa) has been linked to osteonecrosis of the jaw (see previous blog post).

Amgen’s new monoclonal antibody, denosumab, binds to RANK-L, thereby inhibiting its action, with the result that osteoclasts (the cells responsible for bone resorption) cannot form, function or survive.  The result of this mechanism of action is a reduction in bone loss and bone destruction.

Like zoledronic acid, denosumab also has a risk of osteonecrosis of the jaw developing.  However, one additional long-term safety issue for denosumab is the fact it suppresses TRAIL (tumor necrosis factor-related apoptosis-inducing ligand) that is not only produced by osteoblasts (the cells responsible for bone formation), but also by immune cells.  This raises the possibility of skin and immune adverse events, which were seen in the clinical trial data.

Kawai & Mödder in their review article conclude that:

“There is still a need for therapies that reduce fracture risk beyond the level achievable with bone-resorbing agents, particularly as virtually all of the currently available drugs do not eliminate the possibility of future fractures.”

However in addition to having a market opportunity and scientific rationale, any biotechnology company looking at osteoporosis as part of their marketing strategy, must face up to the increasing ethical concerns over placebo-controlled clinical trials.  This topic was highlighted last year in the New England Journal of Medicine.

In the future there is likely to be increased pressure not to recruit subjects at high-risk of osteoporosis (T score less than -2.5) into placebo-controlled trials, thus increasing the costs, number of patients and time to bring new products to market.  In addition, the regulatory barriers to entry are becoming higher, given that regulatory agencies require a reduction in fractures over 3 years to establish the efficacy of a new drug.  This ultimately results in the need for large, expensive, and long phase III clinical trials.

In forthcoming posts, I will discuss the opportunities for market entry by new osteoporosis drugs targeting the Wnt- ß-catenin pathway, Cathepsin K inhibitors and Parathyroid hormone-related protein.

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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.

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