Micromedic and Florida U. Scientists Take on Jaw Bone Rot

Under its agreement with the university, Micromedic has committed to finance the R&D and protection of intellectual property.

Micromedic Technologies is seeing the first fruits of a strategic collaboration agreement signed two months ago with the University of Florida. Scientists at the university are working on a blood-test kit which the Israeli company would commercialize - the kit could revolutionize life for people taking a family of bisphosphonate-type drugs.

The drugs, used to treat osteoporosis and certain types of cancers, can have a devastating side effect in about 10% to 15% of patients: bone rot (osteonecrosis) of the jaw, or BONJ. Worse, the bone can deteriorate years after taking the drug and at present there is no way to predict who will develop the problem, says Micromedic chief executive David Solomon.

The University of Florida is a world leader in pharmacogenomics, the branch of pharmacology studying the influence of genetic variation on drug response in patients. The school and Micromedic agreed to co-develop new pharmacogenomic tests, with Micromedic commercializing the products. The news is the first application of this strategic agreement.

"Today pharmacology has a problem: Most drugs used to treat serious diseases such as diabetes and cancer have horrible side effects, and/or don't work on many people - as many as 50%," Solomon says. "You can't tell in advance on whom it will work."

Treatment is therefore reduced to trial and error - "one size fits all," he explains. And that means a person could be taking a powerful drug that does him no good and causes horrible side effects.

The trend today is personalized medicine: creating the right drug for the right person through genetic profiling. Diagnostics through generic profiling gives drug companies a way to develop drugs with the greatest efficiency. And drug companies are the perfect partners for scientists in pharmacogenomics because the tighter focus could reduce costs, Solomon says.

A lot of people receive osteoporosis medications, about 150 million people in the United States alone, according to Micromedic. Similar medications are also given to cancer patients with bone problems. When a patient develops BONJ, therapy must cease, Solomon explains: there's no choice. But the truly pernicious aspect of the dilemma is that the problem's life cycle can be years and years.

Micromedic's immediate target audience is cancer patients receiving large doses of bisphosphonate-type drugs, especially if administered intravenously. But all people taking this family of osteoporosis drugs are exposed, though the amounts are smaller and the time to develop BONJ (if it develops) is slower.

"Experts call it a time bomb for a certain percent of the population," says Solomon.

He hopes that a genetic testing kit, which involves just a blood test, can identify cancer patients who might develop bone rot of the jaw.

The United States Food and Drug Administration hates the situation, says Solomon. There is a risk that drugs will be yanked, which is bad for the drug companies and bad for the people who can use the drugs without side effects. "The situation is unacceptable," he says.

Scientists at the University of Florida, led by Professor Joseph Katz of the Department of Oral & Maxillofacial Surgery, believe they have identified the genes that make people susceptible to BONJ. "We have initial proof of concept and now want to validate the story. It could be a revolution," says Solomon.

Micromedic's role is to translate research into products, he explains. "It's something between a holding company to a synergetic holding company. We have five subsidiaries; each has a collaboration with a world institute." Its business model is a license agreement. Solomon says Micromedic boasts world experts with global reputations on the team: "We're a link in a very necessary food chain: We make the product and sell to the gorillas."

Assuming the scientists are right about the genes they discovered, when may people start hoping for succor? Or at least an indication that they had better eschew bisphosphonate-type drugs such as Zometa and Alendronate? (Sales of drugs of this sort have reached $5 billion a year. The problem is not a narrow one.) Possibly within a couple of years, says Solomon.

The development and approval process for biotechnological products is very different from that of drugs, the CEO elaborates. "Drugs have a very specific profile: From each 5,000 molecules only one may reach the market. The time to market is very long," he says. Also the risks are high: The cliche is that it costs a billion dollars to develop a new drug.

Here the numbers are very different. The time to market is typically shorter and the chance of success is greater. In vitro tests are much simpler, he says.

At this phase the Floridan scientists have presented with preliminary proof of concept, no more. Proof will take two stages: internal validation at the University of Florida and external validation through multi-center clinical trials, which means - blood tests. But it shouldn't take long compared with clinical trials of drugs, he says. His very tentative timetable is between one to two years to apply to the FDA for approval. Even here, time is shorter. FDA approval may take a matter of months, assuming it gets all the data it needs, Solomon adds.

Under its agreement with the university, Micromedic has committed to finance the R&D and protection of intellectual property. In exchange for licensing, the Israeli company will pay the university 8% royalties of net sales of the product or resultant uses.

Meanwhile, during the last 12 months, shares of Micromedic have tumbled 45%, reducing the company's market capitalization to NIS 23 million.