Treeway announces positive data from two separate Phase I TW001 clinical trials

Successful dose and confirmatory study solid platform for phase II/III trial in ALS patients.

TW001 was tested in both single and multiple dose phase l studies in healthy volunteers and in ALS patients. In the two Phase 1 clinical studies TW001 was shown to be safe and well tolerated and adequate exposure levels with the oral formulation were detected. Based on these trial data, the company is now aiming to start a pivotal phase 2/3 study in 2016. Earlier, Treeway obtained orphan drug designation for edaravone of ALS from both the FDA and EMA.

Inez de Greef, CEO of Treeway comments on the trial outcome: “The positive results of our Phase I program are an important milestone for the development of TW001 for ALS. This outcome is 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."

TW001, Treeway’s lead compound, is an oral formulation of edaravone. Edaravone is a well-established free radical scavenger, which 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.

Treeway has developed an oral formulation (TW001) to overcome the problems associated with intravenous administration. The reformulated drug allows for chronic daily use and its route of administration (oral) is patient friendly.

Currently, edaravone is marketed in Japan by Mitsubishi Tanabe as an intravenous formulation (Radicut) for the indication stroke and ALS. The intravenous formulation of edaravone is administered to ALS patients via 1-hour infusions in a hospital setting. Drug holiday cycles are part of the treatment regimen (patients are treated 10 out of 28 days).