The amyotrophic lateral sclerosis (ALS) investigational drug edaravone will be the focus of four presentations by MT Pharma America in Boston.
The presentations, which include data on edaravone and an analysis evaluating clinically relevant symptoms of ALS, are taking place at the 69th Annual Meeting of the American Academy of Neurology (AAN) through April 28.
“The clinical data being presented at AAN continues to deepen our understanding of edaravone’s role in helping slow the decline of physical function,” Joseph M. Palumbo, MD, vice president and head of medical science and translational research, Mitsubishi Tanabe Pharma Development America, said in a press release.
The randomized, placebo-controlled Phase 3 clinical study (NCT01492686) evaluated the effectiveness and safety of edaravone (60 mg, administrated intravenously) once a day in 137 patients with ALS, based on the changes in the revised ALS functional rating scale (ALSFRS-R) scores after 24 weeks of treatment.
Data reported in April 2016 showed the study met its primary efficacy endpoint, meaning that after 24 weeks of treatment, patients had a statistically significant mean change in the ALSFRS-R, compared to placebo-treated patients. In terms of safety, treatment-related adverse effects occurred at similar rates to placebo.
Edaravone is an intravenous potent antioxidant drug in development to relieve the effects of oxidative stress, a possible factor for the onset and progression of ALS.
ALS is a neurodegenerative disease that attacks nerve cells in the brain and the spinal cord responsible for controlling voluntary muscles, affecting a patients’ ability to move, speak, eat, or breathe.
Oxidative stress, which is commonly found in test results of ALS patients, results from an imbalance between the production of free radicals and the body’s ability to counteract or detoxify from their harmful effects.
In 2015, edaravone was approved for use as an ALS treatment in Japan and South Korea. In May 2015, edaravone was granted orphan drug status by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), but it is not yet approved for use in either the U.S. or in Europe.
However, in September 2016, the FDA accepted a new drug application (NDA) for edaravone, with a decision expected by June 16, 2017, based on the U.S. Prescription Drug User Fee Act. If the application is approved, edaravone will be marketed under the brand name Radicava through MT Pharma America.
During the AAN meeting, the company will also present results from an analysis that examined a U.S. claims database of more than 170 million patients and found potential predictors in patients who eventually were diagnosed with ALS.