Novartis drug effective in advanced MS

A Novartis drug has been shown to reduce risk of disability progression in secondary progressive multiple sclerosis, an advanced form of the disease.

The Swiss pharma said the phase 3 EXPAND study, evaluating the efficacy and safety of oral, once-daily, BAF312 (siponimod) in secondary progressive multiple sclerosis (SPMS), met its primary endpoint of a reduction in the risk of disability progression, compared with placebo.

The EXPAND study represents the largest randomised, controlled study in SPMS to date, Novartis added.

Many patients who progress from the relapsing form of the disease to SPMS will continue with their existing medications, such as Biogen's Tecfidera (dimethyl fumarate).

Once these drugs cease to have an effect, doctors switch patients to another one of the many other drugs approved for relapsing-remitting disease, or to the chemotherapy, mitoxantrone.

Vasant Narasimhan, global head of drug development and chief medical officer for Novartis, said: "SPMS is a particularly disabling form of MS, and there is a need for effective treatment options to help delay disability progression in those living with the condition.”

Top line results of the EXPAND study, including primary and key secondary endpoints, will be presented as a late breaking oral abstract at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) annual conference next month in London.

Novartis will complete full analyses of the data and evaluate next steps in consultation with health authorities.

The EXPAND study is a randomised, double-blinded, placebo-controlled phase 3 study, comparing the efficacy and safety of BAF312 versus placebo in people with SPMS.

It included 1,651 people with SPMS from 31 countries. Patients were randomised to receive either 2mg BAF312 or placebo in a 2:1 ratio respectively.

Primary endpoint was an improvement in the time to three-month confirmed disability progression, as measured by the expanded disability status scale (EDSS), versus placebo.

Secondary endpoints included delay in the time to six-month confirmed disability progression versus placebo, the time to confirmed worsening of at least 20% from baseline in the timed 25-foot walk test (T25FW), T2 lesion volume, annualised relapse rate (ARR), and the safety and tolerability of BAF312 in people with SPMS.

BAF312 (siponimod) is a selective modulator of specific types of the sphingosine-1-phosphate (S1P) receptor.

The S1P receptor is commonly found on the surface of specific cells residing in the central nervous system (CNS), that are responsible for causing CNS damage that drives loss of function in secondary progressive MS (SPMS).

BAF312 enters the brain and by binding to these specific receptors, may prevent the activation of these harmful cells, helping to reduce loss of physical and cognitive function associated with SPMS.

There are three types of multiple sclerosis (MS): relapsing-remitting MS (RRMS), secondary progressive MS (SPMS) and primary progressive MS (PPMS).

The evolution of MS results in an increasing loss of both physical and cognitive function.This has a substantial negative impact on the approximately 2.3 million people worldwide affected by MS.

Novartis already markets MS drug Gilenya (fingolimod), a daily pill indicated for relapsing forms of MS and is also in development for paediatric MS.

Extavia (interferon beta-1b for subcutaneous injection) is approved in the US for the treatment of relapsing forms of MS. In Europe, Extavia is approved to treat people with relapsing remitting MS, secondary progressive MS (SPMS) with active disease and people who have had a single clinical event suggestive of MS.

Novartis is also developing ofatumumab (OMB157), a fully human monoclonal antibody in development for relapsing MS. Ofatumumab targets CD20, and is expected to begin phase 3 pivotal studies in the second half of 2016.

In the US, the Sandoz division of Novartis markets Glatopa (glatiramer) 20mg/mL, the first generic version of Teva's Copaxone (glatiramer) 20mg.