Treeway has obtained Phase I results showing its oral ALS treatment could be more effective than its injectable counterpart in targeting oxidative stress, which contributes to neuronal death in the disease.

Treeway, a Dutch biotech, has obtained Phase I results comparing its oral ALS treatment, TW001, to an injectable form of the drug called edaravone. Results showed that, when the treatment is given orally, a higher proportion of the drug enters a patient’s circulation and can take effect than when the treatment is injected.

ALS, or amyotrophic lateral sclerosis, is the most common type of motor neuron disease and leads to a loss of motor control. There is no cure for the disease but treatments can improve a patient’s quality of life. Edaravone, for example, may slow nerve damage associated with ALS symptoms. Treeway’s treatment targets oxidative stress, which occurs when oxygen-containing molecules called free radicals damage the body, and plays a role in the progression of the disease.

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The intravenous form of edaravone was approved to treat ALS in 2015 in Japan and received a US approval last year. Because previous efficacy data on the repurposed drug is already available, Treeway is preparing a Phase III study for TW001, the oral form of edaravone.

Oral ALS Treatment Shows Promise Over Its Injectable Form

In recent years, ALS research has made progress towards improving the lives of patients. For example, Oxford BioDynamics is developing a diagnostic test for ALS that looks at epigenetics — modifications to the DNA structure that determine whether certain genes are active or inactive. In Spain, researchers are using stem cells to stop progressive muscle paralysis in the disease, which may eventually lead to new treatment options.

While Treeway’s oral drug TW001 may be able to slow the pathogenesis of the disease, the treatment is in a very early stage of its development. We will have to see how the treatment will progress in Phase III.


Images by TuckerBlade, Jinning Li/Shutterstock

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