Patient-friendly therapeutic for the treatment of Amyotrophic Lateral Sclerosis

Development of TW001 for the treatment of Amyotrophic Lateral Sclerosis

In collaboration with the University Medical Centre Utrecht and the Leiden Academic Centre for Drug Research, Treeway is developing a patient-friendly oral formulation of edaravone to treat Amyotrophic Lateral Sclerosis (ALS). ALS is a fatal neurodegenerative disorder characterized by the progressive loss of motor neurons in the brain and spinal cord. Worldwide, approximately 140,000 people are diagnosed with ALS each year. Life expectancy after diagnosis varies greatly between patients but is usually between 2 to 5 years. New therapeutics are in high demand as the current treatment only prolongs the patient's lifespan by 2 to 3 months.

In June 2015, Radicut® was approved in Japan for the treatment of ALS. 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® however, is administered via injection into a vein (intravenous) which is believed to have many limitations. Intravenous administration is invasive and patients can’t take the drug at home.

The consortium 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 can take their medication at home. Common problems for ALS patients, such as opening bottles and difficulty swallowing, will be considered during development of TW001.

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. TW001 has the potential to significantly slow down ALS disease progression and to improve the patient’s quality of life.

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Treeway is developing a patient-friendly therapeutic for the treatment of Amyotrophic Lateral Sclerosis. A devastating disease for which there is currently limited treatment and no cure.
Technology Readiness Level (TRL)
Time period
36 months