Diagnostics & Imaging Week

PARIS — Your genetic makeup can be a compass, but it is not a global positioning system able to pinpoint an impending pathology. Nor can it explain why a woman with no genetic imprint for breast cancer, nor even a family history of cancer, can develop the condition.

Still, pharamcogenomics has opened a door in our understanding of the process of disease and led to the promise of personalized medicine.

But pharmaceutical companies have a different view of this emerging new line in healthcare, seeing it more as a fissure in the foundation of their business model.

The threat of “tailored therapy,” as it is called by Big Pharma, is that it erodes the one-size-fits-all approach that has led to monumental successes — and colossal revenues.

Just as personalized music destroyed the power of the hit music culture, tailored therapy threatens the blockbuster drug market by reversing the focus, emphasizing the personal condition and not the mass-market distribution of a molecule.

In other words, precise diagnostics of the target becomes the primary care focus and the therapy is pushed to the back end of the process in a menu of choices.

It could be called a disruptive technology, except that the technologies used to screen a patient’s “omics” (genomics, proteomics or metabolomics) have been in use for years.

Rather, it is the combination of these techniques in a process that better diagnoses a patient’s profile that is revolutionary.

Instead of the empirical method used by doctors, where the patient is told to take a pill and call if anything goes wrong, this approach screens the patients for a better selection among appropriate therapeutics, such as the right antibiotic.

Antibiotic abuse caused by trial-and-error prescribing could be the poster-child case study for the benefits of point-of-care (POC) diagnostics of the target for both enhanced efficacy and cost savings to reimbursement plans.

The widening base of advanced in vitro diagnostic instruments and increasing sophistication of POC assays may eventually make it possible to continually monitor the efficacy of the therapy, allowing for adjustments to dosage, or even to terminate the treatment.

This emerging pharmaco branch of personalized medicine is increasingly called theranostics, the association of a diagnostic test with a therapy to identify patients who will best respond to treatment, to identify patients who may experience harmful side-effects and to monitor the response to a treatment and determining the most effective dosage.

bioMérieux (Marcy l’étoile, France) lays claim to being the first company to establish a formal business unit dedicated to theranostics development, with a dozen research and development staff that set up offices in Cambridge, Massachusetts, in January 2007.

Last September, bioMérieux signed an agreement to develop a theranostic test for breast cancer with the French pharmaceutical firm Ipsen (Paris).

Just last month, Merck & Co. (Whitehouse Station, New Jersey) signed an agreement with bioMérieux to co-develop an immunoassay test for infectious disease using a theranostic approach.

Following the Merck announcement, Stéphane Bancel, chief executive of bioMérieux, said, “Theranostics is a new business unit for us that we really believe in,” and he has created a global business development division based in Cambridge to develop partnerships and distribution agreements.

During a recent company update with analysts, he said the strategic focus is to become a special partner for pharma and biotech companies in developing drugs that use in vitro diagnostics as a companion for clinical evaluations.

Richard Ding, global business unit leader for theranostics, told Diagnostics & Imaging Week that the immediate focus for the group is early-on collaboration with pharma partners to apply testing to a specific therapy.

“The industry has talked about pharmacogenomics for years but there remained a lack of recognition linking pharma therapies with diagnostic testing,” he said. “This has moved upstream and it is revolutionary to use diagnostic tests to guide the clinical trials before coming to market.”

Pharmas are under heavy pressure to produce market successes in 18 months, he said. Diagnostics helps achieve that objective.

An early-on collaboration to develop a companion POC assay or lab work-up can shorten the lag between launch of a drug and the adoption of tests to encourage use of that drug.

Ding cites the example of a launching a new antibiotic. Under the current approach in primary care, the doctor is left to determine, or to guess, when the antibiotic would be most effective. Over time, diagnostic tests are developed and papers published to better target the indications against specific viruses or bacteria, reinforcing its use, or, alternatively, eroding the market.

Diagnostics also can help pharma companies with a reach-back strategy, he said.

Building a test that operates on a widely available diagnostic instrument enables identification patient subgroups that may benefit from a drug in a company’s existing patented product line and sharpen its marketing to specific “omnic” profiles.

In the mid-term, Ding notes there are therapeutics that cannot be applied without diagnostics, and, in some cases, becoming an FDA requirement.

“We are seeing this more and more in cancer,” he said, “but it will take time for this market to materialize.”

Though partnering closely with pharmaceutical companies, Ding said his business model is very different from that of his partners.

Big Pharma is focused on building an IP fortress around a single molecule, he said, where diagnostic testing of a target is rarely exclusive to anyone. Targets to be tested are at best a shared license, an often in the public domain.

“We do not have a blockbuster model to drive business,” he said. “Yet, I can live with a less stringent IP because the incremental costs of adding an assay to an existing platform are less significant as long as that test is going to help continue to distinguish the offer for that testing platform.”

Ding cites the example of bioMérieux’s VIDAS instrument, which processes upwards of 90 assays. “My end-game is always to launch tests with higher and higher medical value and leverage an installed base that is over 45,000 instruments.”

Working with Big Pharma to push more drugs does not sound like a revolution.

“To this point, we take a medicine to see if it will have the promised effect,” Ding said.

“Now, with theranostics we may be able to say which medicine is best, and that is going to redistribute revenues among pharmaceutical companies and change reimbursement plans. That is definitely disruptive.”