One of the most closely watched developments in the quest for new multiple sclerosis treatments is the experimental drug alemtuzumab (Lemtrada) from Cambridge, MA-based Genzyme, a unit of French drug giant Sanofi (NYSE: [[ticker:SNY]]). Last November, Genzyme revealed that in a late-stage trial, the drug reduced relapse rates and the risk of long-term disability, and today, at the annual meeting of the American Academy of Neurology, the company shed more light on that data.
Genzyme reported that 65 percent of alemtuzumab patients in the trial were relapse-free at two years, versus 47 percent of patients who were treated with a form of interferon sold under the brand name Rebif by EMD Serono and Pfizer (NYSE: [[ticker:PFE]]). Also at that time, 29 percent of patients treated with Genzyme’s drug saw their level of disability improve, while only 13 percent of patients in the comparator arm experienced an improvement in disability. “We found this quite striking,” says Michael Panzara, therapeutic area head for multiple sclerosis, immune diseases, and neurology for Genzyme. “What this means is that patients receiving alemtuzumab were twice as likely to experience an improvement or reversal of disability.”
Alemtuzumab is an antibody that targets a protein on cell surfaces called CD52. The drug appears to deplete the T and B cells that cause cell damage in MS. Patients in the trial had a form of the disease called relapsing-remitting multiple sclerosis, which causes episodes of weakness, sensory changes, and severe fatigue. Genzyme’s executives believe that in addition to improving the symptoms of the disease, alemtuzumab will offer a significant convenience benefit over other therapies. In the trial, patients received alemtuzumab as a daily IV infusion for five days, but then didn’t receive any medication for a year, at which point they were put on the drug again for three days. But the patients in the comparator group had to take interferon as an injection under the skin three times a week for the entire two-year trial period.
The data released today also included observations from imaging tests that tracked patches of inflammation in the central nervous systems of the MS patients in the trial. Significantly fewer patients taking Genzyme’s drug formed new patches than did patients in the comparator arm. Patients taking alemtuzumab also experienced a lessening of changes in the brain, such as a loss of neurons and the connections between them. “We set a high bar in this program, studying patients with more active disease, and studying our drug against a well-recognized, strong therapy,” says William Sibold, business unit head for MS at Genzyme. Sibold, who joined Genzyme about five months ago from Avanir Pharmaceuticals, previously worked in Biogen Idec’s (NASDAQ: [[ticker:BIIB]]) neurology business. “Having spent a lot of time in MS, I had not expected a product to achieve these results. We see a lot of potential to help patients.”
Patients on Genzyme’s drug did experience more infections and thyroid issues than those taking interferon, but most of the adverse events were mild to moderate, and they were easily manageable, the company said in a statement. “Safety gets a lot of attention in MS,” Panzara says, because patients can be on MS drugs chronically, for years on end. Overall, however, the relatively mild side effects were “reassuring,” he says.
Biogen is also presenting data on one of its highly anticipated MS drugs, dimethyl fumarate (BG-12), at this week’s American Academy of Neurology meeting. The company said in a statement today that the oral drug has been tested in more than 2,600 patients and that in relapsing-remitting MS it “demonstrated efficacy across a variety of clinical and radiological outcome measures.” Biogen submitted the drug to the FDA for approval earlier this year.
Genzyme plans to file for FDA approval of alemtuzumab by the end of this quarter, and it will be reviewed in an expedited manner by the agency, which has granted it “fast track” status. During the meeting, Genzyme is also presenting data on teriflunomide, a daily pill to treat MS, which Genzyme submitted for approval to the FDA last fall. “To be successful in this space, you have to have highly differentiated products,” Sibold says. “I think we’re laying the right foundation with this portfolio of products.”
