Two New Multiple Sclerosis Pills Show Promise in Late-Stage Studies

No less than three late-stage studies show that two experimental pills for multiple sclerosis -- fingolimod and cladribine -- are effective. If the drugs, developed by Novartis (NVS) and Merck Serono respectively, are approved by the FDA, they will be the first oral treatments available for the disease. Multiple sclerosis is a chronic, irreversible illness that attacks the central nervous system and is characterized by symptoms ranging from numbness to blindness and paralysis.About 85% of MS patients have what is called "relapsing-remitting MS," characterized by attacks -- relapses -- of worsening neurological function, followed by periods in which the symptoms abate and the patient partially or completely regains the functions lost during the attack; the new therapies are targeted for those patients.

Although no cure has been found for MS, several strategies are currently in use to modify the disease's course, treat relapses, manage symptoms and improve function and safety. Those treatments involve cumbersome injections and infusions, so an oral therapy would have blockbuster potential for its manufacturer.

Both Merck Serono's drug cladribine and Novartis' fingolimod reduce the risk of relapses and slow the disease's progression. The Novartis drug even beat an established interferon therapy (Biogen Idec (BIIB)'s Avonex) in 12 months of a non-oral treatment, researchers found. However, since both pills also suppress the immune system, patients who took the drugs had higher rates of infections, including herpes and shingles, as well as other adverse events such as cancer.

"A New Horizon for Patients With Relapsing-Remitting Multiple Sclerosis"

Novartis said it applied last month for U.S. and European approval of fingolimod. The drugmaker expects to hear by February whether the U.S. Food and Drug Administration will conduct a priority six-month review of the treatment. If the fast review is granted, Novartis could bring its MS pill to the market by year-end and sales could reach $1 billion annually, Trevor Mundel, head of global drug development at Novartis, told Bloomberg.

Merck Serono's parent company, Germany's Merck KGaA (not to be confused with U.S.-based Merck & Co.), filed its U.S. application for cladribine in September, but the FDA rejected the filing two months later as incomplete. This could delay its entry to U.S. market. Merck is meeting with the FDA to address the regulatory agency's concerns.

The results "provide a new horizon for patients with relapsing-remitting multiple sclerosis and a welcome increase in the range of treatment options," neurologist William Carroll wrote in The New England Journal of Medicine. Both medicines are likely to be at least as effective as the more difficult-to-administer injected therapies currently available, Carroll wrote.

Among the current treatments on the market are: Avonex, which reduces the risk of disability progression, exacerbations, and the number of lesions in the brain; Teva (TEVA)'s Copaxone, which also reduces relapse rate; and Elan (ELN)'s and Biogen Idec's Tysabri.

Side Effects Are a Concern, But They're "Manageable"

Sales of Tysabri, which has been linked to deadly brain infections, surpassed $1 billion for the first time last year, with 48,800 patients having taken the drug. This demonstrates that patients are willing to risk side effects for an effective MS treatment, doctors say.

However, the The Wall Street Journal quotes Moses Rodriguez, a neurologist and director of the MS Center at the Mayo Clinic in Rochester, Minn., as saying he wasn't particularly impressed by the efficacy data and was concerned about the side effects of the new drugs. The National Multiple Sclerosis Society, on the other hand, called the side effects "manageable," though it said they should be closely monitored over the long term.

In the two-year study of Novartis's pill fingolimod on over some 1,200 patients, the relapse rate was 54% and 60% lower in the different dose groups, compared to the placebo group. Also, the time before a first relapse was longer, and proportionately more patients remained free of relapse during the 24-month study period. The time to disability progression, too, was longer than with placebo. The one-year study of some 1,100 patients showed that patients on the Novartis's pill had significantly greater reductions in annualized relapse rates than those in the interferon group.

Similarly, in an approximately 1,300-patient study of Merck's pill, cladribine, there was a 33% to 31% relative reduction in the risk progression of disability in both dose groups as compared with placebo.

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