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.  

Conference

The 13th Annual Digital Pharma East

Digital Pharma East returns to the Pennsylvania Convention Center September 17–20, bringing together over 1000 attendees from biotech and pharma, to better understand how to present business plans, justify budget and innovation, and de-risk proposals getting shut down — essentially, understand how they can return to the office and become champions for their internal digital needs. Join us and save 15% on standard rates when you register with Discount Code DPE19Fierce.

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

Suggested Articles

Bristol-Myers Squibb is out to expand the pool of colorectal cancer patients it can treat with Opdivo—and it’s bringing in Bayer to help.

When AstraZeneca sold its Avlon, England plant in 2016, it thought its liabilities were over. Former employees said the drugmaker broke its promises.

Pharmacy chain Rite Aid has partnered with Google to have their drug recycling stations easily searchable on Google Maps.