Treeway, a biotech company developing therapies against amyotrophic lateral sclerosis (ALS), today announced the completion of two successful phase I trials with its lead compound TW001, a more patient-friendly oral formulation of the Japanese stroke and ALS therapy edaravone.

Founded by two Dutch ALS patients, Rotterdam, Netherlands-based Treeway seeks to speed up ALS research in collaboration with academic and research centers around the globe.

TW001 was tested in both single- and multiple-dose phase l studies in healthy volunteers and in ALS patients. In the two studies, TW001 was shown to be safe and well-tolerated, and adequate exposure levels with the oral formulation were detected. Based on those trial data, the company is now aiming to start a pivotal phase II/III study in 2016. Earlier, Treeway obtained Orphan Drug designation for edaravone of ALS from both the FDA and EMA.

Treeway CEO Inez de Greef said of the trial outcome, “The positive results of our phase I program are an important milestone for the development of TW001 for ALS. This outcome not only represents a significant achievement for our company, but first offers important new patient-friendly aspects to the treatment of ALS. We look forward to sustaining this momentum in the coming months, as preparations for the pivotal phase II/III clinical study are now ongoing.”

Edaravone is a well-established free radical scavenger that targets oxidative stress, a process that plays an important role in ALS. Intravenously administered edaravone has been shown to slow down disease progression in ALS patients in previous Japanese clinical trials.

Currently, edaravone is marketed in Japan by Mitsubishi Tanabe Pharma as an intravenous formulation (Radicut) for the indication stroke and ALS. The intravenous formulation of edaravone is administered to ALS patients via one-hour infusions in a hospital setting. Drug-holiday cycles are part of the treatment regimen; patients are treated 10 out of 28 days.