Mitsubishi Tanabe says ALS drug meets PhIII endpoint

Brain imaging

Osaka-based Mitsubishi Tanabe Pharma said a Japan Phase III study of amyotrophic lateral sclerosis (ALS) candidate MCI-186 met its primary efficacy endpoint in data presented at the American Academy of Neurology annual meeting.

Mitsubishi Tanabe said the candidate known as Radicut (edaravone) relieves the effects of oxidative stress in ALS, also known as Lou Gehrig's disease.

The MCI186-19 study met its primary endpoint of mean change in the ALS Functional Rating Scale-Revised (ALSFRS-R) at 24 weeks, with a frequency of adverse events similar to placebo.  

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Japan's Pharmaceuticals and Medical Devices Agency approved the drug for ALS therapy in June of 2015. Prior to this, its use was limited to aid in the recovery of the brain due to damage following a stroke.

The company also holds an Orphan Drug Designation for Radicut from the U.S. FDA and the EMA--but it is not approved for use in either region as yet. 

Lou Gehrig's disease is a rapidly progressive neurological disease in which patients die, on average, within 3 to 5 years from onset of symptoms. ALS attacks the nerve cells responsible for controlling voluntary muscles, such as those in the arms, legs and respiratory tract.

While a hereditary form of the disease, familial ALS, accounts for 5% to 10% of cases, the majority of ALS cases do not have a definitive cause.

A handful of other drugmakers and biotechs are also looking into potential drug targets for the disease, including a 2013 collaboration between Eli Lilly ($LLY) and the nonprofit Project A.L.S .There is no cure for ALS and, so far, only one U.S. FDA-approved drug--Sanofi's ($SNY) Rilutek (riluzole)--currently exists to help slow the progression of the disease. The drug is now available as a generic. 

- here’s the release