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Optimer, Following Pfizer’s Playbook, Has Big Plans for Antibiotic

Xconomy San Diego — 

Pfizer was where Pedro Lichtinger learned about pharmaceutical marketing from people who did some amazing things. For starters, New York-based Pfizer (NYSE: PFE) turned an old-school antifungal medicine into a $1.6 billion cash cow. Now as the CEO of San Diego-based Optimer Pharmaceuticals (NASDAQ: OPTR) Lichtinger is borrowing some ideas from that experience, looking to make the most of his company’s new antibiotic.

Optimer won FDA approval last May for its first marketed product, a twice-daily pill called fidaxomicin (Dificid). That drug is designed to fight a bug called C.difficile that causes diarrhea so severe it can kill patients, especially frail elderly people. It is a common problem in hospitals. The company got off to a respectable start, generating about $24 million in gross sales in its first six months. But the market for treating this pathogen, after it has been diagnosed in a hospital lab, isn’t huge. Optimer can expect to generate about $153 million in U.S. sales in 2015, according to analyst Eun Yang of Jefferies & Co.

So to get the biggest possible bang out of this new molecule, Optimer is thinking about not just treating “C.diff,” but preventing it. Like Pfizer did with fluconazole (Diflucan), Optimer sees a long-range future in which physicians will prescribe the product as part of a standard regimen as a preventive medicine for patients who are at high risk of getting C.diff and who are likely to face a lot of suffering and high-cost hospital interventions if they get the bug. The initial plan is to start with a clinical trial to prove the Optimer’s drug can help prevent that problematic result in patients undergoing bone-marrow transplants.

If this preventive strategy works, then Optimer’s new medicine could be used by up to 20,000 patients a year who undergo such transplants. Given that the drug is currently priced at $2,800 for a typical 10-day course, and it is likely to go up over time, so it could possibly add another $230 million to $380 million in annual sales by 2020, Lichtinger says.

“I came from Pfizer where this concept was applied to Diflucan, where years ago, it was the first major antifungal applied for prophylactic use,” Lichtinger says. “It’s still used today as a generic. I saw that drug go from a relatively small drug into a $1.6 billion drug as a result of this prophylactic approach.”

He was quick to add that he’s not forecasting Optimer’s drug will approach that rarefied sales figure, but he does add there is a wide variety of other patient groups that could benefit from getting preventive C.diff treatment, such as vulnerable patients undergoing heart or liver transplants, certain cancer patients, or those on ventilators in hospital intensive care units. “The opportunity is certainly even bigger than with the primary C.diff indication,” Lichtinger said during an interview earlier this month at the JP Morgan Healthcare Conference in San Francisco.

Bone marrow transplants seem like an obvious place to start on the expansion plan. Patients who get these transplants as treatments for blood diseases go through a period when they are quite vulnerable to infections, as they are going through what amounts to a re-boot of their immune systems. As a preventive measure, most of these patients already get an antifungal medication, and a quinolone antibiotic to ward off bacterial invaders, and some even get an antiviral preventive, Lichtinger says. If patients getting bone marrow transplants end up getting infected with C.diff, then the death rate rises to one out of 20 patients, and treatment of the infection ends up costing about $132,000 per patient, according to Optimer’s market research.

So combining the high cost of infection with a physician specialty that’s well-versed in prophylactic therapy makes this the ideal test case for the new strategy, Lichtinger says. His bet is that physicians will also be receptive to this idea because Optimer’s antibiotic is only approved to fight “C.diff” bacteria, and is a so-called “narrow spectrum” antibiotic as opposed to a “broad-spectrum” antibiotic that kills a wide range of good and bad bacteria. Overprescribing of those powerful broad-spectrum antibiotics, like vancomycin, can lead to the development of resistance to the most powerful antibiotics over time, and hospitals don’t want to contribute to that already significant problem, Lichtinger says.

Of course, Optimer will have to gather proof for this concept before physicians start incorporating the new medicine into the standard of care. The company is getting ready to start a 350-patient clinical trial of bone marrow transplant patients, which should start enrolling people by the end of this year. Results should be in from that trial in the first half of 2014, and the new use of the Optimer drug could be approved by the FDA by the first half of 2015, Lichtinger says.

The company has become so committed to the expansion plan for Dificid that it said earlier this month that it is eliminating its tiny basic research function, a move that led the company to cut the jobs of seven workers. Who knows what those people and their collaborators may have come up with to fill Optimer’s pipeline, but Yang, who rates the company a “Buy,” applauded the new plan to make the most of Dificid. “We do not view Optimer’s discontinuing its discovery programs as negative; rather, we view its better alignment of its R&D spend in clinical programs as positive,” Yang wrote in a note to clients Jan. 9.