Fingolimod is an oral agent that is now being researched in three Phase III trials. This is the first of a new class of potential MS medications known as “S1P receptor agonists,” which work by keeping certain immune cells out of circulation. It does not kill immune cells, but rather “locks” them up in the spleen and lymph nodes, which means that if patients stop the medication, their immune system will be back to normal within a week. The recently reported results of one phase III clinical trial of Fingolimod, TRANSFORMS, has shown that Fingolimod is more effective than interferon beta-1a (Avonex), which is currently a standard of care in the treatment of MS.
The study calculated the annualized relapse rate at one year in patients given one of two varying doses of Fingolimod or an injection of interferon beta-1a. Patients given Fingolimod 0.5 mg exhibited a 52% reduction in relapse compared to those given interferon beta-1a. Those given Fingolimod 1.25 mg also showed a significant reduction in relapses (38%) compared to interferon beta-1a. Both doses met their primary outcome measure – a measurement that is required by the FDA to determine the effectiveness of each study drug – and there was no statistically significant difference seen between the two doses, meaning that the higher dose did not seem to provide greater benefit than the lower dose.
The safety profile of the therapy seen in TRANSFORMS was in line with previous clinical studies. The compound was generally well-tolerated and side-effects included fatigue and headache. However, like with all study drugs there are potential risks. Fingolimod does affect other organs, including the heart, liver and eyes. The therapy suppresses the immune system and there are concerns about the development of potentially serious infections and certain types of cancer, such as skin cancer. There were 7 cases of localized skin cancer lesions seen in the TRANSFORMS study, all of which were successfully removed. The benefit to risk factor of Fingolimod is still being researched and further study on the safety profile needs to be done; more detailed information will likely be released sometime this year.
TRANSFORMS is the first of three clinical trials of Fingolimod. The three different studies collectively will involve more than 3,400 patients from around the world. The two other trials, FREEDOMS and FREEDOMS II, which are both two-year placebo-controlled studies, are underway and results are expected later this year. Novartis, the company that manufactures Fingolimod, expects to send the drug to the FDA for approval at the end of 2009. It is currently unavailable outside of clinical trials.