Development of a Patient-Friendly Formulation of Edaravone for the Treatment of ALS
Abstract
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by the progressive loss of motor neurons in the brain and spinal cord. New therapeutics are in high demand as the current treatment only prolongs the patient s lifespan by 2 to 3 months. In June 2015, the intravenous formulation of edaravone (Radicut?) for the treatment of ALS was approved in Japan. Edaravone, the active ingredient of Radicut?, is a free radical scavenger that targets oxidative stress, a process known to play an important role in the pathogenesis of ALS. Results from a clinical study with Radicut? showed a positive effect on disease progression. Radicut? is administered via injection into a vein (intravenous), and the treatment schedule consists of 10 days of treatment, over a period of 2 weeks, followed by a 16-day drug holiday, a period in which the patient is not given edaravone. Intravenous administration of edaravone, however, is believed to have many limitations including the invasive route of administration and the treatment holidays. Treeway aims to develop a patient friendly oral formulation of edaravone, which can be taken by mouth, to overcome the problems associated with intravenous administration. TW001 will provide a major contribution to patient care as patients are able to take their medication at home. Common problems for ALS patients, such as opening bottles and difficulty swallowing, will be taken into account during development of the formulation. The patient friendly and easy route of administration allows for TW001 to be taken every day. It is believed that taking TW001 more frequently will increase the effect of the drug on slowing disease progression. It is anticipated that TW001 will be beneficial for all patients as oxidative stress is a factor involved in the disease progression of both familial ALS as well as sporadic ALS. Studies with a concept formulation of TW001 have shown that oral administration of edaravone is possible. As such, the objective of the proposed study is to develop a stable, patient friendly, oral formulation of edaravone for the treatment of ALS. The project will consist of different studies to test the new formulation and will make the formulation ready for a Phase III clinical study. The new formulation is expected to be ready for a Phase III study in the second half of 2017 after which, the clinical study can be started. The Phase III study will be done with the final formulation. Positive results from a Phase III study are crucial for marketing approval of TW001 by the U.S. Food and Drug Administration and by the European Medicines Agency. Approval of TW001 would be an important step in the right direction on the path to develop better therapeutics that can prolong the patient s lifespan and improve quality of life.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Aug 07, 2017
- Source ID
- W81XWH1710090
Entities
People
- Sytske Moolenaar
Organizations
- United States Army