Genzyme Reaching For a Slice From Biogen’s Breadbasket, Multiple Sclerosis Drugs

Xconomy Boston — 

If Genzyme gets its way, it will be eating Biogen Idec’s lunch in a few more years. The two Cambridge, MA-based companies, the largest independent biotechs in the state, could be duking it out with competing multiple sclerosis treatments, in a market worth an estimated $8 billion to $9 billion annually by 2011. It would also be the first time the companies have faced off in a direct competition for patients with the same disease.

Genzyme (NASDAQ: GENZ) is recruiting patients in two final-stage clinical trials of Campath, currently approved as a leukemia drug, for patients with multiple sclerosis. Biogen (NASDAQ: BIIB) is already entrenched as the world’s largest maker of drugs for multiple sclerosis, offering both the current market-leader, Avonex, and Tysabri, a newer and more effective drug co-marketed with Ireland-based Elan.

It’s hard for Wall Street to see this far ahead, but Genzyme envisions Campath as a key to driving its future growth, beyond the financial forecasts the company has given analysts through 2011. The company says Campath, which works by knocking out overactive B cells and T cells of the immune system, may be the most effective agent yet against multiple sclerosis, a disease in which immune cells attack the insulating coating on nerve fibers. About 400,000 Americans suffer from the condition, which can rob patients’ of their ability to walk, see, or speak.

In a mid-stage clinical trial, Campath was able to cut the risk of MS flare-ups by 73 percent over a three-year period, while lowering the risk of progressive disability about 70 percent when compared with a standard treatment. “We’ve actually seen improvement in disability in patients, and that may be unprecedented,” says Terry Murdock, Genzyme’s senior vice president for Campath, from his offices in San Antonio, in a telephone interview. Genzyme will finish enrollment in one of the current Phase III trials in the first half of 2009 and aims to have data ready to send to the FDA for approval in 2011, Murdock says.

The MS patient community is following these late-stage trial closely, particularly since Genentech and Biogen’s Rituxan showed early promise, then failed this spring in a study of patients with the toughest form of MS to treat. And the mid-stage study of Campath was indeed promising. It followed 334 patients with the relapsing/remitting form of MS, who randomly got the drug, or a standard interferon medicine, Merck Serono’s Rebif—its findings caught the attention of researchers because MS drugs usually attempt to clear a lower hurdle, by simply out-performing a placebo.

“The numbers would have been impressive when compared with placebo and thus are even more impressive when compared with a currently approved disease-modifying agent,” said John Richert, executive vice president of research and clinical programs for the National MS Society, in an e-mail. “The Phase III data will be very important.”

Like Biogen’s Tysabri, Campath is an engineered antibody, but it hits a different target on immune cells than Tysabri does. Campath was approved for relapsed patients with chronic lymphocytic leukemia in 2001, and Genzyme obtained it as part of its $1 billion acquisition of San Antonio-based Ilex Oncology in 2004.

The drug, like any other, has side effects. The Phase II trial showed six people had serious cases of ITP, a disorder in which the immune system goes haywire, attacking platelets, which are critical for blood clotting. That can make patients dangerously susceptible to bleeding episodes. Patients on Campath can also become vulnerable to infections, according to the drug’s prescribing information.

Genzyme’s current trials are monitoring patients’ blood samples, and performing regular phone calls to keep close watch on any dangerous side effects, Murdock said. The monitoring takes some extra effort, because patients don’t have to come see the doctor regularly to get treatment with the drug. Campath is designed to be given as an intravenous infusion daily for five days, then patients can take a year off, and get annual boosts, Murdock said.

Murdock wouldn’t say how much Genzyme is investing in the Phase III clinical trials, but it’s clearly a lot of cash. The first trial, in 525 patients, looks at patients who are new to MS treatment. The second study, of 1,200 patients, will test the drug among patients who have had prior therapy. “It’s one of the top priorities for the company, and it’s being adequately resourced,” Murdock says.

For its part, Biogen has a lot riding on getting the most mileage possible out of Tysabri. The company has forecasted it will have 100,000 patients taking the drug by the end of 2010, which would generate $2.8 billion annually at current prices. (Avonex, currently Biogen’s biggest-selling product, had $1.87 billion in sales in 2007, almost 60 percent of the company’s total revenue.) But Tysabri still hasn’t entirely stepped out from under the shadow of PML, the rare, fatal brain infection that has been associated with the drug and led it to be pulled from the market in 2005. Biogen brought the drug back to the market in July 2006 under a stringent monitoring program. No new cases of PML have been reported since, the company has said.

Each passing day without a new PML case makes Tysabri stronger, yet Biogen certainly can hear footsteps from its neighborhood rival. Campath’s efficacy numbers appear so far to be better than Tysabri’s, which is shown to reduce the risk of MS flare-ups by two-thirds. Campath and another experimental drug from Basel, Switzerland-based Novartis, FTY-720, “may offer patients greater efficacy, but there is greater risk than first-generation therapies,” said Biogen spokeswoman Shannon Altimari in an e-mail. “The risk-benefit of these therapies will have to be taken into consideration.”

To date, MS patients on Campath haven’t had any reported cases of PML. If that holds, and if the current studies of the drug are as successful as the earlier ones, Genzyme could end up taking a large bite out of Biogen’s biggest moneymaker.