Dr. Fonstein: Cleveland BioLabs is five years old. The company started as a spinoff of the Cleveland Clinic and it is now over two years since we went public on NASDAQ. The company develops two types of drugs. One type is tissue protectors that protect healthy tissues in our body from a variety of stresses, including radiation and chemotherapy. Obviously, this can be further divided into additional indications. One is when we experience radiation as a result of nuclear accident or deliberate attack, which could be called a defense application. Another is where our body experiences radiation or chemotherapy as part of anti-cancer treatment, where such a drug could increase tolerance to treatment and therefore improve regimens. It is also worth noting that these tissue-protecting drugs are selective in protecting healthy tissues and not tumor cells. The other type of drugs we are developing are pure anti-cancer drugs. The foundation that connects both types of drugs is our proprietary understanding of how to control the regulated cell death process, called apoptosis, which allows us to treat cancer cells and healthy cells differently. Our lead anti-cancer compound just completed a Phase II trial in hormone refractory prostate cancer and our anti-radiation drug is about to enter human safety trials. In the first quarter of 2010, we expect to get results of the first arm of our extended safety trial for radiation protection, which may sound early, but this is the stage of only human trial required for a defense application. This means that we would expect to have a sellable drug in approximately two years.
TWST: Would you comment on the pathway for the radiation drug as far as the non-
medical application is concerned?
Dr. Fonstein: This application follows the FDA's animal efficacy rule that was
established in 2002 as a pathway to develop drugs whose efficacy would be
unethical to test in humans. Really, it's hard to imagine that the FDA would
condone irradiating humans using whole-body radiation just to show that you can
save even the vast majority of them. This approval pathway consists of three
critical elements. The first is demonstrated efficacy in two animal species,
with the most advanced species in this case being non-human primates. In this
area we have extensive proof of principle.
The next major component is safety in humans. Obviously to get there, you need
to show safety in animals first, which we have done, and we are about to start
human trials. In animal studies, our drug showed a tremendous therapeutic window
between active dose and toxic dose, which was almost 1,000 times in rodents. The
third component is the study of biomarkers in human volunteers, because we need
to show that what is happening in humans is relevant to what protects the
animals tested in efficacy trials. The fact that we well understand the
mechanism of action behind the high efficacy of our drug plays a critical role
here. In fact, I should mention that our extensive knowledge about our drug's
mechanisms of action led to the recent publication of a feature article in
Science magazine, which is an amazing achievement.
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