Treeway prepares phase 3 study for TW001 to treat ALS
Phase 1 study suggests that novel oral formulation improves bioavailability while remaining well tolerated.
Treeway, a biotech company developing therapies against amyotrophic lateral sclerosis (ALS), announced the completion of its Phase I trial of lead program TW001, an oral formulation of edaravone. The study compared the bioavailability of TW001 in its novel market formulation to Radicava, an FDA approved, intravenous formulation of edaravone, indicated for the treatment of ALS. The study indicates increased bioavailability of TW001 in a single-dose treatment whilst no safety concerns developed within the subject group, implying the potential of the novel formulation to improve treatment strategies for ALS patients. Based on these results, Treeway has initiated the upscaling program and preparations for a pivotal Phase 3 study. Edaravone is a neuroprotective agent that reduces oxidative stress, a key contributor to neuronal death in ALS.
“It is Treeway’s mission to identify novel treatment routes for ALS patients and we believe that this study indicates that an oral formulation of edavarone may offer patients a better quality-of-life through a simpler and more effective route of administration,” said Ronald van der Geest, Chief Development Officer of Treeway. “Given the nature of this program and based on the positive results we have seen in this comparative study, we can initiate a pivotal Phase 3 study and move rapidly toward bringing TW001 to patients.”
The randomized, cross-over comparative bioavailability trial tested a single oral dose of 140 mg TW001, compared to a 1-hour intravenous infusion of 60 mg edaravone in 18 healthy subjects. In this study, the bioavailability of the novel oral formulation exceeded the bioavailability of the intravenous infusion of Radicava. Within the subject group of the study the treatment was well-tolerated and no safety concerns arose during the conduct of the study. Furthermore, ongoing formulation studies also suggest that the formulation is stable.
Merit Cudkowicz, MD, Chief Neurology Service and Director of the ALS Clinic at the Massachusetts General Hospital, a key advisor to the company, commented:
“The results of this Phase I study are a promising sign for ALS patients, who are in high need of better and more patient-friendly treatments for their disease. I regard this study to be a key milestone for the development of TW001 in ALS and I look forward to supporting the company’s scientific and clinical progress.”